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<?xml version="1.0" encoding="UTF-8"?><rss version="2.0" xmlns:content="http://purl.org/rss/1.0/modules/content/" xmlns:wfw="http://wellformedweb.org/CommentAPI/" xmlns:dc="http://purl.org/dc/elements/1.1/" xmlns:atom="http://www.w3.org/2005/Atom" xmlns:sy="http://purl.org/rss/1.0/modules/syndication/" xmlns:slash="http://purl.org/rss/1.0/modules/slash/" > <channel> <title>Vial</title> <atom:link href="https://vial.com/feed/" rel="self" type="application/rss+xml" /> <link>https://vial.com/</link> <description></description> <lastBuildDate>Wed, 01 May 2024 19:15:50 +0000</lastBuildDate> <language>en-US</language> <sy:updatePeriod> hourly </sy:updatePeriod> <sy:updateFrequency> 1 </sy:updateFrequency> <generator>https://wordpress.org/?v=6.5.2</generator> <image> <url>https://vial.com/wp-content/uploads/2021/09/cropped-favicon_512-32x32.png</url> <title>Vial</title> <link>https://vial.com/</link> <width>32</width> <height>32</height></image> <item> <title>Castor vs. Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/castor-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/castor-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 01 May 2024 19:09:30 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[#clinicaltrial]]></category> <category><![CDATA[#clinicaltrials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CROs]]></category> <guid isPermaLink="false">https://vial.com/?p=48790</guid> <description><![CDATA[The landscape of medical research is currently in the midst of an exciting transition wherein technology has rapidly become a critical tool for clinical trial management and patient data collection. For example, the frequency of clinical trials using digital health technologies, such as wearables and telemedicine platforms, increased from 0.7% in 2010 to 11.4% in […]]]></description> <content:encoded><![CDATA[<p>The landscape of medical research is currently in the midst of an exciting transition wherein technology has rapidly become a critical tool for <a href="https://vial.com/glossary/clinical-trial/">clinical trial</a> management and patient data collection. For example, the frequency of clinical trials using digital health technologies, such as wearables and telemedicine platforms, increased from 0.7% in 2010 to 11.4% in 2020 to enable continuous remote monitoring of real-world patient health data. Other technologies like electronic data capture (<a href="https://vial.com/glossary/edc/">EDC</a>) are also deeply integrated into modern clinical trials, but these have been around since the 1990s, known then as ‘remote data capture’ (RDC).</p> <p>As far back as 2009, 41% of clinical trials were estimated to be relying on an EDC system; since then, EDC software have evolved into a user-friendly experience with significant electronic interoperability with electronic patient reported outcomes (<a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome/">ePRO</a>), electronic consent (<a href="https://vial.com/glossary/econsent/">eConsent</a>), electronic <a href="https://vial.com/glossary/case-report-form-crf/">case report forms</a> (eCRFs), and electronic source (<a href="https://vial.com/glossary/edc/">eSource</a>) platforms. Among the leading <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organizations (CROs)</a> providing these technologies are Castor, with its established reputation for reliable EDC technology, and Vial CRO, a newer player quickly becoming well-known for its digital innovation. In this article, we deep dive into the products offered by each of these tech-driven CROs and provide insight into their benefits and drawbacks.</p> <p><strong>About Castor</strong></p> <p>Although many experimental drugs hold significant promise for many patients, the harsh reality is that 93% of these painstakingly developed drugs fail during clinical trials and 35% of trials do not see the light of day due to a lack of patient enrollment. Castor recognizes these discouraging numbers and has made it their mission to make research data reusable, thereby saving sponsors their time, effort, and resources. The company has a history of strongly advocating for clinical trials driven by artificial intelligence (AI) using research data captured in a secure, compliant cloud platform to help expedite the drug development process. With this in mind, Castor was founded in 2012 to provide researchers with a platform that could capture and integrate high-quality data from various sources into one compliant platform. Today, with its team of over 100 professionals, the company is relied on by thousands of medical device, biotech, and academic researchers around the globe to accelerate over 1000 studies.</p> <p><strong>Overview of Castor: Castor EDC, eConsent, and ePRO</strong></p> <p><em>EDC</em></p> <p>The company’s flagship product, <a href="https://www.castoredc.com/electronic-data-capture-system/">Castor EDC</a>, is renowned as the top-ranked EDC platform, boasting over 147,000 users and has been utilized in more than 14,000 studies [<a href="https://www.castoredc.com/about-us/">4</a>]. In addition to its well-established reputation for consistently delivering a reliable and efficient platform, the use of Castor EDC also spans over 90 countries having impacted more than 6 million patients. The company offers a comprehensive suite of tools that integrates seamlessly into the Castor EDC platform, namely eConsent and ePRO.</p> <p><em>ePRO</em></p> <p>The <a href="https://www.castoredc.com/epro/">Castor ePRO module</a> enables capturing data right from the patients themselves, helping improve their trial experience by providing secure, accessible questionnaires through email and the Castor Connect mobile application. With this system, sponsors can expect secure data capture directly from participant mobile devices, real-time survey progress tracking, and automated scheduling of questionnaires.</p> <p><em>eConsent</em></p> <p><a href="https://www.castoredc.com/econsent/">Castor’s eConsent platform</a> offers a range of electronic solutions that enhance efficiency from recruitment and screening to data capture and analysis throughout a clinical trial. This platform allows participants to have a seamless onboarding experience with a customized recruitment portal and pre-screening questionnaires. Sites also benefit from the ability to conduct video-based remote consent visits, enabling participants to engage from the comfort of their homes.</p> <p><strong>About Vial CRO</strong></p> <p>Vial CRO is a San Francisco-based company revolutionizing clinical trials by offering faster, more efficient trial results at a significantly lower cost for biotech sponsors. Founded in 2020, Vial was created to serve as a platform to reimagine clinical trials by deploying technology at every step across multiple therapeutic areas, including <a href="https://vial.com/cro/dermatology/">Dermatology</a>, <a href="https://vial.com/cro/gastroenterology/">Gastroenterology</a>, <a href="https://vial.com/cro/oncology/">Oncology</a>, and more. Vial’s state-of-the-art technology platform integrates all aspects of a trial from onboarding, patient enrollment, site communication, to data collection processes, providing a single, streamlined system. The team at Vial is a blend of <a href="https://vial.com/glossary/clinops-clinical-operations/">ClinOps (Clinical Operations)</a> expertise and software product and engineering expertise, who have worked together to develop Vial’s technology platform and raise over $100M. The funds have enabled the company to successfully become a leading global, full-service CRO reshaping the future of clinical trials.</p> <p><strong>Overview of Vial CRO: Vial EDC, eSource, and ePRO</strong></p> <p><em>EDC</em></p> <p><a href="https://vial.com/edc/?">Vial EDC</a> was developed in-house by Vial’s software engineering team to create a custom platform that streamlines study startup and build processes, minimizes vendor dependencies, and allows for flexible data integration. Housed within a cloud-native infrastructure, Vial EDC boasts a user-friendly interface that has been deployed to over 30 <a href="https://vial.com/glossary/clinical-research/">clinical research</a> sites, impacting more than 1500 enrolled participants around the United States (US). It also maintains strict compliance with regulatory guidelines like the US <a href="https://vial.com/glossary/food-and-drug-administration-fda/">Food and Drug Administration’s</a> <a href="https://vial.com/glossary/21-cfr-part-11-2/?utm_source=organic">21 CFR Part 11</a>, <a href="https://vial.com/glossary/health-insurance-portability-and-accountability-act-hipaa-privacy-rule/">HIPAA</a> (Health Insurance Portability and Accountability Act), and GDPR (General Data Protection Regulation). <a href="https://vial.com/blog/articles/comparing-vial-cros-edc-to-industry-giants/?">Read more here</a> about how Vial’s EDC system compares with that of industry giants.</p> <p><em>eSource</em></p> <p><a href="https://vial.com/esource/?">Vial eSource</a> is an innovative tool that replaces paper-based data capture and integrates with Vial EDC, eliminating double data entry, strengthening data compliance and quality, and enabling centralized remote monitoring. It assures data quality through an intelligent, real-time data capture system, built on CDISC standards using SDTM-controlled terminology for quality on-entry. The company’s eSource interface is powered by standardized data types, data validation, and range rules on entry, required fields, and skip logic, among others.</p> <p><em>ePRO</em></p> <p>Lastly, <a href="https://vial.com/epro/">Vial’s ePRO</a> platform provides a consumer-grade, mobile patient experience that is compliant and customizable for patients in the trial. It features a seamless, user-friendly interface easily accessible by either mobile or desktop, as well as built-in data validation, skip logic, and interactive functions for quick and compliant data capture. The Vial ePRO module also allows for real-time monitoring of patients and improved reporting, as well as accurate, real-time data recording and storage with its connected data flow with Vial ED.</p> <p><strong>Castor versus Vial: A Matter of Fit</strong></p> <p>Both companies offer an intuitive technology suite with the capacity to capture and integrate high-quality data from various sources into a single compliant platform, eliminating the need for double data entry. However, with the inclusion of an eConsent module, Castor’s platform may offer a more well-rounded option for sponsors looking for an all-in-one trial management tool, particularly for decentralized clinical trials (DCTs). Despite these advantages, the emphasis on data integration and the advanced nature of Castor’s technology suite might call for a deeper level of technical expertise from research sites. This increases the burden of staff training for the sponsor or CRO in charge of running the clinical trial.</p> <p>On the other hand, Vial has the advantage of providing a more convenient experience for site staff with its unique eSource tool, which addresses a significant pain point for <a href="https://vial.com/glossary/clinical-research-or-study-coordinator-crc/">clinical research coordinators (CRCs)</a> looking to reduce data entry burden after patient visits. Furthermore, because Vial’s EDC is developed in-house, their system minimizes vendor dependencies and allows for more flexible data integration. However, Vial’s relatively recent emergence in the field might be a point of concern for some sponsors, who may prefer working with more established platforms with a longer track record.</p> <p>Choosing between Castor and Vial CRO ultimately boils down to a matter of fit; therefore, sponsors must carefully consider their specific needs and objectives when making this decision. For those seeking a platform with a strong track record and a focus on data integration, Castor might be the ideal choice considering the company’s established reputation, as well as their advocacy for data reuse and AI-driven DCTs. In contrast, sponsors who prioritize a similarly streamlined all-in-one data entry system that requires a more user-friendly approach might find Vial CRO’s platform more suitable. Despite its relatively new entry into the CRO industry, Vial has shown strong potential with its tech-first approach to clinical trials and can hold its own against established companies like Castor.</p> <p><strong>Conclusion</strong></p> <p>In conclusion, both Castor and Vial CRO offer innovative solutions designed to expedite and streamline the clinical trial process. Although each platform comes with its unique strengths and limitations, the choice between the two will largely depend on the specific requirements and objectives of the sponsor. As clinical trials continue to evolve in the face of advancing technology, clinical trial management tools like those offered by these leading CROs will undoubtedly play a crucial role in shaping a new era of drug development.</p> <p><strong>Vial: The CRO for Biotech, Powered by Technology</strong></p> <p><em>Our mission at <a href="https://vial.com/cro">Vial</a> as a next-generation, tech-first CRO is to empower scientists to discover groundbreaking scientific therapeutics that help people live happier, healthier lives. <a href="https://vial.com/contact-us/">Contact a Vial representative today</a> to discover how we can make a difference for your next clinical trial!</em></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/castor-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Syneos vs Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/syneos-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/syneos-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 24 Apr 2024 20:16:08 +0000</pubDate> <category><![CDATA[Articles]]></category> <guid isPermaLink="false">https://vial.com/?p=48780</guid> <description><![CDATA[Introduction In a landscape where many contract research organizations (CROs) are available, Vial is breaking through with its promise to deliver on its vision to empower scientists to cure all human diseases by reimagining clinical trials. The global, full-service Vial CRO stands out on multiple fronts, and we explore how it compares with a large […]]]></description> <content:encoded><![CDATA[<h2 class="wp-block-heading">Introduction</h2> <p>In a landscape where many <a href="https://vial.com/glossary/cro-contract-research-organization">contract research organizations</a> (CROs) are available, <a href="https://vial.com/">Vial</a> is breaking through with its promise to deliver on its vision to empower scientists to cure all human diseases by reimagining <a href="https://vial.com/glossary/clinical-trial">clinical trials</a>. The global, full-service Vial CRO stands out on multiple fronts, and we explore how it compares with a large leading CRO like <a href="https://www.syneoshealth.com/">Syneos Health</a>.</p> <h2 class="wp-block-heading">Therapeutic Area Coverage</h2> <p>Clinical trials are essential for developing new interventions and advancing patient care. The Vial CRO services encompass various therapeutic areas, including <a href="https://vial.com/glossary/oncology">oncology</a>, <a href="https://vial.com/glossary/dermatology">dermatology</a>, <a href="https://vial.com/glossary/ophthalmology">ophthalmology</a>, <a href="https://vial.com/glossary/gastroenterology">gastroenterology</a>, <a href="https://vial.com/glossary/neurology">neurology</a>, <a href="https://vial.com/glossary/cardiology">cardiology</a>, medical devices, rare diseases, and digital therapeutics (DTx). In addition to the areas above (aside from DTx), Syneos Health offers <a href="https://vial.com/glossary/biosimilars">biosimilars</a>, <a href="https://vial.com/glossary/cell-and-gene-therapies">cell and gene therapy</a>, endocrine and metabolic, <a href="https://vial.com/glossary/immunology">immunology</a> and inflammation, infectious diseases, pediatrics, respiratory, and women’s health services. Digital therapeutics is not explicitly listed as a therapeutic area of expertise for Syneos Health.</p> <h3 class="wp-block-heading">Oncology</h3> <p>Despite significant progress in <a href="https://vial.com/glossary/cancer">cancer</a> research, many patients face limited treatment options. CROs like Vial and Syneos Health are continuously innovating in the oncology space. The <a href="https://vial.com/cro/oncology">Vial Oncology CRO</a> offers sponsors solutions to support their research, including optimized study design, innovative patient recruitment strategies, and advanced data analytics. By partnering with Vial, sponsors conducting oncology clinical trials can accelerate getting innovative therapies to market. Syneos Health offers diverse and measurable experience across cancer treatment, from <a href="https://vial.com/glossary/immuno-oncology">immuno-oncology</a> and targeted therapies to novel and emerging therapies, including cell and gene therapies.</p> <h3 class="wp-block-heading">Dermatology</h3> <p><a href="https://vial.com/glossary/phase-i/">Phase I</a> Units are critical to advancing care and validating treatments. The <a href="https://vial.com/cro/dermatology">Vial Dermatology CRO</a> Phase I unit provides a unified full-service solution to early clinical development. ****Syneos Health has completed more than 200 dermatology trials for indications that include <a href="https://vial.com/glossary/psoriasis">psoriasis</a>, atopic dermatitis, <a href="https://vial.com/glossary/skin-cancer">skin cancer</a>, rare skin disorders, and skincare and medical aesthetic products.</p> <h3 class="wp-block-heading">Ophthalmology</h3> <p>The <a href="https://vial.com/cro/ophthalmology">Vial Ophthalmology CRO</a> understands the nuances of running ophthalmology trials and has formed a team of in-house ophthalmologists with decades of experience across anterior and <a href="https://vial.com/glossary/posterior-segment">posterior segments</a>. Syneos Health’s experience in ophthalmology clinical studies includes drug and device combinations, cell and gene therapies, orphan and rare diseases, inherited retinal diseases, pediatrics, and biosimilars.</p> <h3 class="wp-block-heading">Gastroenterology</h3> <p>Gastrointestinal (GI) diseases are a significant global health burden, increasing healthcare costs and decreasing productivity. The <a href="https://vial.com/cro/gastroenterology">Vial Gastroenterology CRO</a> offers sponsors full-service CRO solutions to support their GI research, from study design optimization to innovative patient recruitment strategies and advanced data analytics. The Syneos Health gastroenterology clinical study experience includes anal fissures, <a href="https://vial.com/glossary/celiac-disease">celiac disease</a>, colorectal disorders, diverticulitis, <a href="https://vial.com/glossary/gastroenteritis">gastroenteritis</a>, gastroesophageal reflux disease (GERD), hepatic cirrhosis/fibrosis, <a href="https://vial.com/glossary/inflammatory-bowel-disease-ibd">inflammatory bowel disease</a> (IBD), and <a href="https://vial.com/glossary/nonalcoholic-fatty-liver-disease-nafld">non-alcoholic fatty liver disease</a> (NAFLD)/<a href="https://vial.com/glossary/non-alcoholic-steatohepatitis-nash">non-alcoholic steatohepatitis</a> (NASH), among others.</p> <h3 class="wp-block-heading">Neurology</h3> <p>The <a href="https://vial.com/cro/neurology">Vial Neurology CRO</a> experienced CRO executive team works closely with Vial’s expert site operations team to continuously review execution strategies, closely monitor patient recruitment efforts, and mitigate key study risks for neurology clinical trials. The Neuroscience therapeutic area of expertise at Syneos Health includes analgesia, neurology, psychiatry, and clinical surveillance and training.</p> <h3 class="wp-block-heading">Cardiology</h3> <p>Cardiovascular diseases are a leading cause of global deaths and represent a significant healthcare burden. The <a href="https://vial.com/cro/cardiology">Vial Cardiology CRO</a> offers sponsors full-service solutions to support their cardiovascular research. Syneos Health has worked across the entire spectrum of cardiovascular indications, including <a href="https://vial.com/glossary/hypertension">hypertension</a>, <a href="https://vial.com/glossary/atrial-fibrillation-afib">atrial fibrillation</a>, <a href="https://vial.com/glossary/heart-failure-hf">heart failure</a>, ischemic heart disease, <a href="https://vial.com/glossary/thrombosis">thrombosis</a>, <a href="https://vial.com/glossary/dyslipidemia">dyslipidemia</a>, <a href="https://vial.com/glossary/diabetes-mellitus">diabetes</a>, and obesity.</p> <h3 class="wp-block-heading">Medical Devices</h3> <p>Medical devices can transform healthcare and improve patient outcomes; however, they require rigorous testing and evaluation to ensure their safety and <a href="https://vial.com/glossary/efficacy">efficacy</a>. With <a href="https://vial.com/cro/medical-device">Vial Medical Device CRO</a>’s best-in-class <a href="https://vial.com/glossary/clinops-clinical-operations">ClinOps</a> experts and tech platform, sponsors can conduct trials faster and more efficiently, leading to quicker regulatory approval and market access. Syneos Health’s medical device and diagnostics (MD&D) services include MD&D-specific capabilities that span the entire nonclinical and clinical development, regulatory, medical affairs, and commercial continuum to strategically accelerate the product development lifecycle.</p> <h3 class="wp-block-heading">Rare Diseases</h3> <p>Rare diseases affect a small proportion of the global population and often have few treatments. Rare diseases can be severe, life-threatening, and disabling, causing significant emotional and financial strain on affected individuals and their families. The <a href="https://vial.com/cro/rare-disease">Vial Rare Disease CRO</a> offers faster, more efficient clinical trials that allow scientists to develop effective interventions to improve patients’ quality of life, slow disease progression, or even cure the disease. Syneos Health helps companies develop rare disease therapies using novel and integrated approaches, including early engagement of key stakeholders and patient perspectives to accelerate products to market.</p> <h3 class="wp-block-heading">Digital Therapeutics</h3> <p>Digital therapeutics (DTx) are a new class of software-based therapeutic interventions for patients with chronic diseases and mental health conditions. The <a href="https://vial.com/cro/digital-therapeutics">Vial Digital Therapeutics CRO</a> offers sponsors a range of clinical trial solutions, including streamlined study design, optimized patient recruitment, and robust data analytics. With Vial’s expertise and support, DTx sponsors can bring their products to market faster and more efficiently, helping patients access the care they need. By harnessing the power of technology to develop and validate DTx, Vial enables scientists to pave the way for a new era of <a href="https://vial.com/glossary/personalized-medicine">personalized</a> and accessible healthcare. DTx is not explicitly listed as a therapeutic area of expertise for Syneos Health.</p> <h2 class="wp-block-heading">Small vs. Large CRO</h2> <p><a href="https://vial.com/blog/articles/the-benefits-of-working-with-a-small-cro-speed-agility-and-working-with-a-true-partner">Small CROs are flexible, adopt the latest tech, and can customize solutions</a> to meet the needs of sponsors, regardless of their size. Small CROs like Vial offer a high degree of specialization in specific disease areas and geographic focus.</p> <p>Smaller CROs also tend to have a much lower turnover of people compared to large CROs like Syneos Health, providing the stability and continuity sponsors need. For sponsors seeking a high-touch relationship, small CROs can offer close contact, good oversight, and a more personal touch.</p> <h2 class="wp-block-heading">Fixed-fee Pricing Structure</h2> <p><a href="https://vial.com/blog/articles/vial-cro-vs-top-5-large-cros">Unlike Vial, Syneos Health does not offer fixed-fee pricing</a>. At Vial, a fixed-fee pricing model applies to all contracts and helps sponsors stay on budget for the entirety of a project by avoiding costly unanticipated change orders. The <a href="https://vial.com/blog/articles/what-is-fixed-fee-pricing-and-how-does-it-benefit-sponsors">benefits of fixed-fee pricing include</a> transparency, <a href="https://vial.com/blog/articles/fixed-fee-pricing-and-the-importance-of-budget-predictability-in-clinical-research">predictability</a>, shared accountability on commitments, aligned incentives between CROs and sponsors, and sponsors having more control over where funds are spent.</p> <h2 class="wp-block-heading">Trial Start-up</h2> <p>Clinical trial start-up is a critical phase that can impact study timelines and costs if delayed. A recent study found that the average contract negotiation duration exceeded 100 days for industry-sponsored or investigator-initiated contracts, adversely impacting clinical trial start-up time and trial costs. An understanding of factors that affect start-up is essential to increase efficiency. With this knowledge, Vial has a streamlined site activation capability and <a href="https://vial.com/onboarding">cloud-based site start-up</a> that enables simple onboarding for sites in as few as 30 days. Cloud-based forms, uploads, signatures, and approvals allow real-time editing and a birds-eye view into site-by-site start-up statuses. <a href="https://vial.com/blog/articles/vial-cro-vs-top-5-large-cros">Syneos Health claims improved cycle times</a> but does not explicitly reference a 30-day site activation offering (or any timeline).</p> <h2 class="wp-block-heading">Technology and Tools</h2> <p>To remain relevant and offer clients a full range of value-added solutions, small CROs like Vial keep current on the latest technologies, techniques, and tools in <a href="https://vial.com/glossary/clinical-research">clinical research</a>. Small CROs with expertise and a focus in niche areas are well positioned to adopt the latest tech, identify opportunities for application, and provide insights on how tech may improve clinical trial performance. Vial offers powerful data capture and review in one connected system, including <a href="https://vial.com/glossary/electronic-source">electronic source</a> (<a href="https://vial.com/esource">eSource</a>), <a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome">electronic patient-reported outcome</a> (<a href="https://vial.com/epro">ePRO</a>), and <a href="https://vial.com/glossary/edc">electronic data capture</a> (<a href="https://vial.com/edc">EDC</a>). <a href="https://vial.com/blog/articles/vial-cro-vs-top-5-large-cros">Syneos Health has multiple tech partners</a>, and their systems are spread out.</p> <h2 class="wp-block-heading">Reimagining Clinical Trials</h2> <p>Vial is a next-generation, tech-first CRO on a mission to disrupt the clinical trials industry to deliver faster, more efficient results at dramatically lower costs for sponsors. Vial is reimagining the nature of clinical trials to be more efficient and successfully deliver a superior CRO experience. By deploying technology at every step, Vial is driving efficiencies in speed and cost savings for innovative biotech companies of all sizes.</p> <p>Visit us <a href="https://vial.com/about-us/">here </a>and <a href="https://vial.com/contact-us">contact us</a> today!</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/syneos-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>First in Human Episode #60 featuring Rachel Haines</title> <link>https://vial.com/blog/podcast/first-in-human-episode-60-featuring-rachel-haines/</link> <comments>https://vial.com/blog/podcast/first-in-human-episode-60-featuring-rachel-haines/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Tue, 23 Apr 2024 15:40:51 +0000</pubDate> <category><![CDATA[Podcast]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[Drug Discovery]]></category> <guid isPermaLink="false">https://vial.com/?p=48776</guid> <description><![CDATA[Amy Del Medico: Hello everyone, I’m Amy Del Medico, VP of Therapeutic Strategy at Vial. I’m here today with Rachel Haynes from Rinri Therapeutics. Rachel, would you like to tell us a bit about yourself and Rinri?  Rachel Haines: Hi, Amy. I am the vice president of clinical development and clinical operations at Rinri Therapeutics. […]]]></description> <content:encoded><![CDATA[<p><strong>Amy Del Medico:</strong> Hello everyone, I’m Amy Del Medico, VP of Therapeutic Strategy at Vial. I’m here today with Rachel Haynes from Rinri Therapeutics. Rachel, would you like to tell us a bit about yourself and Rinri? </p> <p><strong>Rachel Haines:</strong> Hi, Amy. I am the vice president of clinical development and clinical operations at Rinri Therapeutics. We were a biotech that was established in 2018, based on some early data from Professor Marcello Revolta’s lab at the University of Sheffield, which demonstrated we could take otic neural progenitor cells, differentiate and expand them. These cells were capable of becoming mature auditory neurons. In his early work, we demonstrated auditory brainstem response recovery in a gerbil model of auditory neuropathy was possible. Rinri was founded to translate that into patient benefit. </p> <p><strong>Amy Del Medico:</strong> That’s fascinating. Thank you. What makes hearing loss such a significant global issue? How does Rinri aim to address this challenge through its research?</p> <p><strong>Rachel Haines:</strong> With hearing loss, the estimates of how many people are affected globally varies widely. But, it’s situated somewhere, probably conservatively, around 500 million people globally being affected. Any of us, once we get to middle age, notice that hearing does become more difficult, even just in social situations and when there’s lots of competing background noise.</p> <p>We’re not just thinking about hearing loss as something that affects people who are born deaf. But it really affects the majority of us, whether it’s ourselves directly, or people that we care for, or people that we love, throughout the whole life cycle. </p> <p>Not only does it affect so many people, but there’s a lot of long term consequences for hearing loss, for patients individually, but also the macroeconomic issues around hearing loss. Granted, it’s not life-limiting, but the impacts are massive in terms of people’s feeling of belonging, communicating, access to education, accessibility in the workplace, and having those meaningful relationships with people in a society that is, largely, an auditory world which we live in.</p> <p>Of course, for some individuals, the deaf community and sign language is their main mode of communication and we are absolutely not looking to solve a problem for those people for whom hearing loss is not a problem. It is a wonderful culture to be part of. But, there are people who want to be part of the hearing world because that’s the culture they grew up in. We do need to address this issue from those people who are looking for it.</p> <p>We also know, beyond being a significant issue, it’s a condition many people don’t seek treatment for. Currently, we have some great technology with hearing aids and cochlear implants which we have continued to develop in our field. But, these do not restore natural hearing and people are often very reticent to take them up.</p> <p>For whatever reason, there’s lots of stigma attached to them. At present we don’t have any licensed therapeutics for hearing or restoration of hearing. It’s something that Rinri and colleagues of ours in gene therapy have been working really hard at. At Rinri, in particular, we really believe in our approach to it because hearing loss is essentially a cellular problem. What Rinri is doing is offering a cellular solution for the cellular problem. We believe that cell therapy really is the future to treat these bigger problems of hearing loss.</p> <p><strong>Amy Del Medico:</strong> When you think about it, especially as we have an aging population, it would impact most of us in some way or another. It’s surprising that more therapeutics aren’t being developed. So, it’s really interesting to hear what you’re doing.</p> <p><strong>Rachel Haines:</strong> It is really interesting. So, in age-related hearing loss that you’re talking about, that’s what we call presbycusis, and it’s the target of the first product in our pipeline. We know that in the aging hearing model, it is the auditory neurons, which are degrading or dying. That precedes the hair cell loss. </p> <p>In hearing loss, you have two types of cells, which are important, which are the hair ,cells and the auditory neurons. When you are given a hearing aid it amplifies that sound. It makes it louder. If the hair cells are not working as well as they used to, there’s some fatigue there, or some have died off, the hearing aid is going to make that sound louder.</p> <p>That will be helpful, in terms of people with more mild or moderate hearing losses. But then once you have a more severe or profound loss, that’s where something like a cochlear implant might be [00:05:00] offered. The cochlear implant essentially replaces the hair cells and stimulates the auditory nerve directly. At present you can have a hearing aid which amplifies the sounds, or a cochlear implant, which replaces the hair cells.</p> <p>In both of those instances, if the problem is at the neural level, at the auditory nerve level, if you have populations of auditory neurons, which have died off or are dysfunctioning, neither of those are going to help. That’s what you see often, especially in an aging model where people may get a hearing aid, but they’re still really unsatisfied with using it socially because it’s not helping their speech and noise.</p> <p>It’s that neural function which is helping us do that speech and noise processing. And then equally, when you have a cochlear implant, you are replacing the hair cells, but you’re not treating any issue that might stem from the auditory neurons themselves. So it’s part of the solution. These are fantastic technologies, which have been life changing for many, but they don’t replace natural hearing. </p> <p>With that comes a very big discrepancy between outcomes of people who get hearing aids, and cochlear implants. There’s a lot of unexplained variation of how well people do with them. Also, people who do use them, (as I said) oftentimes find it very difficult to use them in situations with speech and noise, competing sound signals, and often report the big challenge of fatigue. It’s really difficult when you are listening with something less than your optimal natural hearing to do what is essentially a very complex task of listening to noise. It is very rare that we are actually not listening within a context of background noise.</p> <p><strong>Amy Del Medico:</strong> Thank you. For those that aren’t familiar, I wondered if you could elaborate on the landscape of hearing research today. Why it might be challenging to design clinical trials specifically for hearing related conditions. </p> <p><strong>Rachel Haines:</strong> The list of challenges is absolutely immense and very daunting, but extremely exciting. Also it’s a great time to be working in hearing. I remember being a postgrad student many years ago, hearing Professor Marcelo Revolta talk about some of his early work and thinking, “Wow, that’s science fiction!”</p> <p>It’s so exciting that it’s here now, and in the last three months, some colleagues of ours from gene therapy companies have been reporting some very early positive data for children with otoferlin deficiency. The gene therapy data is really encouraging that regenerative therapies for hearing are possible. They’re just there right in front of us. </p> <p>Gene therapy is going to be transformative for the patient population that’s being developed for these children with a monogenetic cause to their hearing loss. But, we know most instances of hearing loss are not monogenetic are actually acquired. For example, in the age related hearing loss and It’s not genetic factors that drive that alone. You’re going to have people who are too old to benefit from these gene therapies once they do develop a problem with hearing. That’s why we’re proposing a cellular solution for cellular hearing loss.</p> <p>What are the challenges in doing that? </p> <p>Many. </p> <p>First of all, it’s always hard being the first to do something. Second of all, one of the motivations that got me into clinical development and operations originally, coming from a background as a hearing scientist, was seeing in my field 20 years ago, we weren’t doing trials in hearing.</p> <p>Why weren’t we doing trials in hearing? Because most of our treatments were devices or interventions. The wealth of clinical trial methodology has come from a drug therapeutics background. So we don’t, even as of 2024, in our field, have tons of expertise about conducting trials.</p> <p>I’ve been absolutely privileged to be able, over the course of my career, to design and oversee the running of several hearing trials. But even for me, this is the first time I’m doing a trial, in an ATMP in this field because it hasn’t existed before. That’s absolutely amazing. Because we don’t have many trials, another challenge is outcomes. </p> <p>Even patient-reported outcome measures we may have in our field are designed to really capture more incremental changes that a hearing aid might be able to offer, a cochlear implant might be able to offer, not [00:10:00] some of the life-changing or very sensitive changes that a therapeutic could offer.</p> <p> A big challenge for us, in hearing, in terms of pushing clinical development forward, is we don’t even diagnose by etiology. We give somebody a hearing test, we measure how bad their hearing is, essentially, and decide what solution we’re going to offer them based on the magnitude of their hearing loss.</p> <p>We can’t even tell you whether somebody has hearing loss because of the hair cells or because of the auditory neurons. We don’t know. We don’t have those diagnostics, now. We just have an increasing amount of genetic data which is still a work in progress and then electrophysiological methods that are allowing us slowly to unpick this.</p> <p> Diagnostics is a big challenge for us. Another really simple thing: you’re giving somebody an advanced therapy, In our case we are going into people with profound hearing loss. So they will be getting a cochlear implant to replace the hair cells. They will be getting RinCell 1 at the same time as the cochlear implant to regenerate those auditory neurons.</p> <p><strong>Amy Del Medico:</strong> It’s really interesting what you were saying about diagnostics because it’s equivalent to giving somebody an eye test, isn’t it? And saying you’ve lost this much vision. We’re going to guess what treatment you need. I’d never really thought about it like that in terms of hearing loss. </p> <p>It’s really interesting. You’re obviously at the cutting edge, which is fascinating. I wondered how Rinri’s approach to generating cells for hearing restoration differs from traditional methods? What advantages might it offer in terms of efficacy and safety for patients? </p> <p><strong>Rachel Haines:</strong> When you are giving a hearing aid and amplifying sound, or giving a cochlear implant and electrically stimulating the auditory nerve. You’re not restoring natural hearing. A hearing aid has certain auditory processing limitations and a cochlear implant is essentially creating electric hearing. I don’t know if you’ve ever heard a simulation of a cochlear implant, a vocoded speech, really fascinating. But it’s not restoring normal hearing. </p> <p>What we are saying is, by regenerating auditory cells, we will be able to restore natural hearing. That is our end goal. That’s very different from the traditional methods, which aren’t a complete treatment. In terms of efficacy, we can hope and expect that by regenerating the cells that allow us to hear that cannot regenerate on their own in humans, we will be addressing the main complaints and challenges that patients with hearing loss face, even when they have aiding. It’s that fatigue. It’s the difficulty of being able to understand speech and noise, using communication, in those really social situations, that are so meaningful to us as humans as we connect.</p> <p>In terms of safety, we don’t have any therapeutics to compare it to. We can look at our surgical approach that we have developed at Rinri, and compare that to the standard cochlear implant surgery that has an excellent safety profile. We’ve done some real cutting edge research around developing this brand new surgical access, going through the round window into the internal auditory canal and getting the cells exactly in the place that they need to grow. </p> <p>That’s never been done before. But we’ve shown that this is safe. We’ve used synchrotron and micro ct imaging, as we’re doing that surgery scanning the temporal bones and showing how we are able to go in and not damage any of the internal structures like the facial nerve and the cochlear artery. </p> <p>We don’t feel this is any more risky than regular cochlear implantation surgery. But, we still have so much to learn. That’s really exciting, going into the first in human. It is one of the reasons that for our first in human trial, we’ve taken this approach to a randomized, open label trial. We don’t have safety data to compare it against. When we want to be evaluating the safety of what we’re doing, we want a really good data set of control participants to compare that against.</p> <p><strong>Amy Del Medico:</strong> That’s so interesting and very exciting stage to be at as well. Thank you. I just had one final question just to wrap things up. I wondered what sort of excites you the most about the future of the industry? Are there any sort of visions of the landscape that you think are coming up?</p> <p><strong>Rachel Haines:</strong> As a hearing scientist, by [00:15:00] heart, I’m so excited for all the clinical development work that we’ve done and how we’re going to keep building on that. What we can keep developing, in terms of our surgical access, our diagnostics, and our outcome measures is so fascinating scientifically, but also for how meaningful it is to patients. </p> <p>At the heart of Rinri and what we’ve been doing, is working, collaboratively with patients, families, and key opinion leaders across the globe to design, not just our trials, but that end product to be what people want it to be with the end goal in mind of reconnecting people to the auditory world. </p> <p>The clinical development that we have in the pipeline is just so exciting. I’m looking forward to us making meaningful contributions not just to developing this therapy, but for diagnostics of hearing, and, eventually, expanding our indication, for example, into monotherapies for people with more mild forms of hearing loss. We are on the precipice of something big, and it’s just an immense privilege to be part of it.</p> <p><strong>Amy Del Medico:</strong> Rachel, thank you very much for the discussion today. It was an absolute pleasure and really interesting to learn more about Rinri.</p> <p><strong>Rachel Haines:</strong> Thank you. It’s been great talking to you. I’m really excited to speak again someday about what we’ve learned from our first inhuman trial.</p> <p><strong>Amy Del Medico:</strong> Sounds good. We’d love to have you back.</p> <p><strong>Rachel Haines:</strong> Thank you so much, Amy.</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/podcast/first-in-human-episode-60-featuring-rachel-haines/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Updates in the Ophthalmology Clinical Landscape</title> <link>https://vial.com/blog/articles/updates-in-ophthalmology-clinical-landscape/</link> <comments>https://vial.com/blog/articles/updates-in-ophthalmology-clinical-landscape/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Thu, 18 Apr 2024 18:46:35 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[Clinical Research]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CROs]]></category> <category><![CDATA[Ophthalmology]]></category> <guid isPermaLink="false">https://vial.com/?p=48768</guid> <description><![CDATA[The field of ophthalmology is dedicated to preserving and restoring vision, recognizing its significance to human well-being. Yet, hereditary and age-related retinal diseases present challenges, often leading to progressive vision loss and potential blindness. Among these conditions, age-related macular degeneration (AMD) and diabetic retinopathy are of particular concern, with the global patient population expected to […]]]></description> <content:encoded><![CDATA[<p>The field of <a href="https://vial.com/glossary/ophthalmology/">ophthalmology</a> is dedicated to preserving and restoring vision, recognizing its significance to human well-being. Yet, hereditary and age-related retinal diseases present challenges, often leading to progressive vision loss and potential blindness. Among these conditions, age-related macular degeneration (AMD) and diabetic retinopathy are of particular concern, with the global patient population expected to exceed 400 million annually.</p> <p>The urgency for novel therapeutic approaches makes ophthalmology a rapidly growing domain of <a href="https://vial.com/glossary/clinical-research/">clinical research</a>. Market research indicates significant growth in the ophthalmic <a href="https://vial.com/glossary/clinical-trial/">clinical trials</a> market, driven by increased disease prevalence, demand for ocular treatments, and rising research funding. Specifically, the global ophthalmic clinical trials market, valued at USD 1.5 billion in 2022, anticipates steady growth at a compound annual growth rate (CAGR) of 6.6% from 2023 to 2030. Additionally, the ophthalmic drugs market, valued at USD 33.81 billion in 2022, is also expected to grow with a CAGR of 7.80% from 2023 to 2030.</p> <p>Dr. Stephen McLeod, M.D., CEO of the American Academy of Ophthalmology, discussed the evolving landscape of ophthalmology on a recent episode of <a href="https://www.realworldophthalmology.com/podcast">RWO: The Podcast</a>. He highlighted <a href="https://www.ophthalmologytimes.com/view/navigating-ophthalmology-s-changing-landscape-with-insights-from-stephen-mcleod">three pivotal forces reshaping the field</a>:</p> <ol><li>Advances in vision sciences research: Progress in treating conditions like AMD and dry eye disease, together with the potential of gene therapy for inherited retinal diseases, is enhancing patient care</li> <li>Focus on health equity: Tools like the IRIS registry enable ophthalmology to address health disparities, ensuring equitable vision outcomes for all patient populations</li> <li>Community engagement: Collaboration within the ophthalmic community strengthens advocacy efforts and supports provider wellness, addressing challenges like burnout and stress</li></ol> <p>This article explores recent developments in ophthalmology clinical trials.</p> <h2 class="wp-block-heading">The OPTIMIZE-2 Trial</h2> <p><a href="https://oculis.com/">Oculis</a>, a global biopharmaceutical company committed to preserving and enhancing vision worldwide, is at the forefront of innovation in eye care. One of its groundbreaking developments is OCS-01, a novel once-daily topical formulation of dexamethasone tailored for treating diabetic macular edema (DME) and enhancing outcomes post-cataract surgery.</p> <p>The OPTIMIZE-2 trial (<a href="https://clinicaltrials.gov/study/NCT06128369?cond=Ophthalmology&aggFilters=status:rec&page=3&rank=25">NCT06128369</a>) is currently underway to evaluate the potential of OCS-01 eye drops in managing inflammation and pain following cataract surgery. This <a href="https://vial.com/glossary/phase-iii/">Phase III</a> multi-center clinical trial, initiated on December 18, 2023, aims to enroll approximately 160 participants randomly assigned to receive either OCS-01, a dexamethasone ophthalmic suspension at a concentration of 1.5% (15 mg/mL), or a vehicle once daily. OCS-01 is administered as a single drop in the study eye once daily for 14 days, starting one day post-surgery.</p> <p>Primary outcome measures of OPTIMIZE-2 include evaluating OCS-01 efficacy in controlling inflammation, determined by the absence of anterior chamber cells on day 15 post-surgery, and assessing post-operative pain in cataract surgery subjects.</p> <p>OPTIMIZE-2 is open to individuals aged 18 and above, who are undergoing unilateral cataract surgery involving phacoemulsification and posterior chamber intraocular lens implantation in the study eye. Furthermore, participants must exhibit an anterior chamber cell score of at least 2 at the post-operative visit. Exclusions apply to individuals with known sensitivities or allergies to dexamethasone, corticosteroids, or any components of the study medication, those with monocular vision, and those showing signs of intraocular inflammation or reporting ocular pain above a specific threshold during pre-surgery assessments.</p> <p>The trial, sponsored by <a href="https://oculis.com/?_gl=1%2Aajroil%2A_ga_89PG7HJHSY%2AMTcxMjA2ODE2MC4xLjAuMTcxMjA2ODE2MC4wLjAuMA">Oculis</a>, has an estimated primary completion by July 2024 and overall completion by December 2024.</p> <h2 class="wp-block-heading">OCS-01 Achieves Primary Endpoints in Phase III OPTIMIZE Trial</h2> <p>In August 2023, Oculis <a href="https://investors.oculis.com/news-releases/news-release-details/ocs-01-first-investigational-eye-drop-front-and-back-eye-met">announced</a> positive top-line results from its OPTIMIZE trial with OCS-01 eye drops. This double-blind, placebo-controlled Phase III study included 241 patients randomized to receive once-daily OCS-01 eye drops or a vehicle for 14 days post-cataract surgery.</p> <p>The trial met both primary efficacy endpoints, showing a statistically significant decrease in inflammation absence on day 15 and pain absence on day 4 post-surgery.</p> <p>Furthermore, OCS-01 was well-tolerated, exhibiting a favorable safety profile with fewer ocular treatment emergent adverse events (TEAEs) reported compared to the vehicle group.</p> <p><em>“The results of the Phase 3 OPTIMIZE trial are exciting because once daily OCS-01 showed to be superior and highly potent in reducing inflammation and pain compared to vehicle with a favorable safety profile. This is significant for patients who have undergone cataract surgery, as they currently need to self-administer multiple daily doses of eye drops to alleviate inflammation and pain. The availability of a preservative-free treatment that requires only a once-daily eye drop could greatly benefit a large number of patients who undergo ocular surgeries worldwide,”</em> said Eric Donnenfeld, M.D., co-chair of Oculis’ Scientific Advisory Board.</p> <p>With these promising results, Oculis is advancing OCS-01 towards regulatory submission with the <a href="https://vial.com/glossary/food-and-drug-administration-fda/">U.S. Food and Drug Administration</a> (FDA), aiming to provide patients with an effective and convenient treatment option for post-operative inflammation and pain.</p> <p>Riad Sherif, MD, Chief Executive Officer of Oculis, stated: <em>“I am very pleased with the positive readout of OPTIMIZE. A once daily topical steroid eye drop has shown solid results in reduction of inflammation and pain and offers the potential of a truly simplified dosing regimen. We are on track to advance OCS-01 for inflammation and pain following ocular surgery towards an NDA submission with FDA. We now have positive Phase 3 top line results with OCS-01 preservative-free eye drops in treating front-of-the-eye inflammation and pain following ocular surgery, as well as Stage 1 Phase 3 results for back-of-the-eye diabetic macular edema (DME) from the DIAMOND program, opening for the first time ever new opportunities for topical eye drops to address highly unmet patient needs in both front- and back-of-the-eye indications.”</em></p> <h2 class="wp-block-heading">Expanding Clinical Applications</h2> <p>In addition to the OPTIMIZE and OPTIMIZE-2 trials, Oculis is conducting a Phase III double-masked, randomized, multicenter study to evaluate OCS-01 eye drops’ efficacy and safety in subjects with DME. The DIAMOND-2 trial (<a href="https://classic.clinicaltrials.gov/ct2/show/NCT06172257?term=OCS-01&draw=2&rank=3">NCT06172257</a>), initiated on December 15, 2023, counts with the collaboration of ICON plc.</p> <p>In May 2023, Oculis <a href="https://www.ophthalmologytimes.com/view/oculis-announces-positive-top-line-results-from-phase-3-trial-of-ocs-01-drops-for-diabetic-macular-edema">announced</a> positive top-line outcomes from stage 1 of its Phase III DIAMOND trial. Patients receiving OCS-01 showed a significant increase in mean best-corrected visual acuity scores, sustained up to week 12, compared to the vehicle group. Moreover, OCS-01 treatment resulted in a significant decrease in central subfield thickness, observed at week 6 and persisted through week 12. OCS-01 demonstrated a favorable safety profile, with no unexpected adverse events reported.</p> <p>“A topical agent has never demonstrated a positive result in DME,” commented Dr. Riad Sherif. “Now, OCS-01 has been validated in two different studies with consistent and repeated positive results. We remain focused on advancing with high priority the DIAMOND Phase 3 trial to Stage 2. This important milestone has the potential to bring us one step closer to providing the first treatment in the form of eye drops to patients with DME which is a devastating and blinding disease.”</p> <p>Additionally, a study sponsored by Quan Dong Nguyen, Stanford University, in collaboration with the Global Ophthalmic Research Center and Oculis, is investigating OCS-01’s performance in resolving fluid collection in the eye in patients with uveitis or those who have had eye surgery. The LEOPARD (<a href="https://classic.clinicaltrials.gov/ct2/show/NCT05608837?term=OCS-01&draw=2&rank=5">NCT05608837</a>) trial initiated on November 8, 2022, is estimated to complete by December 2024.</p> <p>Apart from OCS-01, Oculis is developing other drug candidates, including OCS-02, another topical eye drop for addressing dry eye disease and non-infectious anterior uveitis. OCS-05 represents a promising candidate for diseases like acute optic neuritis, as well as several neuro-ophthalmic conditions, with potential disease-modifying effects.</p> <h2 class="wp-block-heading">The SCOTS2 Trial</h2> <p>An ongoing interventional study initiated on January 2016 is evaluating the efficacy of autologous bone marrow derived stem cells (BMSCs) in treating retinal and optic nerve damage or diseases generally considered irreversible. The study, known as SCOTS2 (<a href="https://clinicaltrials.gov/study/NCT03011541?cond=Ophthalmology&aggFilters=status:rec&rank=7">NCT03011541</a>) is sponsored by <a href="https://www.mdstemcells.com/">MD Stem Cells</a>, a consultancy renowned for its expertise in conducting clinical trials and treatments utilizing BMSCs across diverse medical domains, including ophthalmology, <a href="https://vial.com/glossary/neurology/">neurology</a>, <a href="https://vial.com/glossary/alzheimers-disease/">Alzheimer’s disease</a>, other dementias, autism spectrum disorders, spinal cord injury, and anti-aging interventions. These cells demonstrate superior ability to navigate through the body and reach critical areas like the brain, eyes, and spinal cord, thereby enhancing their therapeutic potential.</p> <p>SCOTS2 involves administering BMSCs through various routes (retrobulbar, subtenon, intravitreal, intraocular, subretinal, and intravenous) to patients with conditions such as macular degeneration, retinitis pigmentosa, glaucoma-related optic nerve damage, among others. Patients will undergo comprehensive eye examinations and imaging over a 12-month follow-up period.</p> <p>The primary endpoint of SCOTS2 is the alteration in best-corrected visual acuity from pre-procedure to 12 months post-treatment, while secondary endpoints include changes in visual fields and optical coherence tomography parameters during the same timeframe.</p> <p>The study, projected to conclude by July 2025, anticipates enrolling approximately 500 participants. To be eligible for the study, participants must have objectively documented damage to the retina or optic nerve, either unlikely to improve or showing progressive deterioration, as well as specific visual acuity and field criteria. Those who have undergone previous ophthalmologic surgical treatment and have remained stable for at least three months post-treatment are eligible, as are individuals currently undergoing stable medical therapy for retinal or optic nerve diseases. Additionally, participants must be over the age of 18, medically stable, and must demonstrate the potential for improvement with BMSC treatment with minimal risk of harm from the procedure.</p> <p>Exclusion criteria include individuals unable to undergo adequate ophthalmologic evaluation, non-compliance with follow-up appointments, incapacity to provide informed consent, and those identified to be at significant risk to general health or visual function if they undergo the procedure.</p> <h2 class="wp-block-heading">Improvements in Patients With Dominant Optic Atrophy</h2> <p>In <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6987313/">Stem Cell Investigation</a>, SCOTS reported significant advancements in treating dominant optic atrophy (DOA), also known as Kjer’s optic neuropathy, the most prevalent inherited optic neuropathy.</p> <p>SCOTS employed minimal manipulation techniques to isolate BMSCs from aspirated autologous bone marrow using FDA-cleared medical devices. Six patients diagnosed with DOA received combinations of BMSCs via retrobulbar, subtenons, intravitreal, or subretinal placements, followed by intravenous injections, depending on their assigned study arm. The procedures exhibited no surgical complications. Importantly, of the six patients, five experienced significant visual improvements, with enhancements observed in both eyes in all cases. Among the treated eyes, 83.3% demonstrated gains in visual acuity.</p> <h2 class="wp-block-heading">The NOVA-1 Trial</h2> <p>Aerie Pharmaceuticals, a clinical-stage ophthalmic pharmaceutical company recently acquired by <a href="https://www.alcon.com/alcon-completes-acquisition-aerie">Alcon</a>, has initiated a Phase I/II study to assess the safety, efficacy, and durability of AR-14034 in treating neovascular AMD (nAMD). AR-14034 is a sustained-release (SR) retinal implant containing axitinib, a pan-VEGF inhibitor, formulated within a distinctive bio-erodible polymer blend using Aerie’s PRINT® technology.</p> <p>The NOVA-1 trial (<a href="https://clinicaltrials.gov/study/NCT05769153?cond=Ophthalmology&aggFilters=status:rec&page=3&rank=21">NCT05769153</a>), initiated on December 6, 2023, is structured into two stages:</p> <p>· Stage 1, a dose-escalation, open-label evaluation will be conducted over 48 weeks with approximately 10 subjects divided into two cohorts. Both cohorts will undergo assessments for safety and preliminary treatment effects</p> <p>· Stage 2 consists of a 56-week double-masked, active comparator, randomized, parallel group evaluation comparing AR-14034 SR with aflibercept. Approximately 130 subjects will be enrolled and randomized to different treatment groups. Participants will be assessed for safety and treatment effects over 56 weeks, followed by a 16-week open-label extension phase</p> <p>The primary outcome measure of NOVA-1 is the mean change from baseline in early treatment diabetic retinopathy study best corrected visual acuity at specified visits. Secondary outcome measures include additional assessments of visual acuity and central subfield thickness using spectral domain optical coherence tomography imaging.</p> <p>Key inclusion criteria include having an active choroidal neovascularization lesion secondary to AMD and specific visual acuity requirements. Exclusion criteria include certain ocular conditions, recent anti-VEGF treatment, uncontrolled glaucoma, and pregnancy.</p> <h2 class="wp-block-heading">Promising Findings in Preclinical Models</h2> <p>Recent findings published in <a href="https://iovs.arvojournals.org/article.aspx?articleid=2787318">Investigative Ophthalmology & Visual Science</a> support the potential of AR-14034 SR implant as a long-term treatment option for nAMD and DME, offering hope for improved therapeutic outcomes with fewer intravitreal injections.</p> <p>In the study, conducted on both rabbits and miniature swine, AR-14034 SR implants were administered intravitreally. Control groups consisted of either placebo-injected or naïve animals. The efficacy of the implant was assessed by inducing retinal blood vessel leakage with VEGF165, followed by quantification using fluorescein angiography.</p> <p>In rabbits, all dose levels of AR-14034 SR implant showed significant reductions in retinal vessel leakage compared to controls at 1- and 2-month intervals. Notably, a single dose level demonstrated sustained efficacy up to 12 months. Similarly, in miniature swine, both implant dose levels significantly reduced retinal vessel leakage at day 41.</p> <h2 class="wp-block-heading">Vial, Your Tech-Enabled Ophthalmology Partner</h2> <p>While emerging treatments offer hope, conducting ophthalmology clinical trials can be challenging. Collaboration between sponsors and <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organizations</a> (CROs) specializing in ophthalmology contribute significantly to the success of clinical trials. <a href="https://vial.com/blog/articles/what-is-a-cro/">CROs</a> offer expertise in trial design, ocular safety monitoring, patient population selection, and efficient data management.</p> <p>[Vial](<a href="https://vial.com/?)">https://vial.com/?)</a>, a next-generation, full-service CRO, is evolutionizing clinical trials to deliver faster, better, and more cost-effective results for biotech and biopharma sponsors. Our [ophthalmology CRO](<a href="https://vial.com/cro/ophthalmology/?)">https://vial.com/cro/ophthalmology/?)</a> team has extensive therapeutic knowledge to support all stages of ophthalmology drug development and clinical research.</p> <p><strong>To learn how Vial’s CRO can support your ophthalmology clinical trial, please visit our website or <em><a href="http://www.vial.com/contact-us">get in touch with a Vial representative today</a>!</em></strong></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/updates-in-ophthalmology-clinical-landscape/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>First in Human Episode #59 featuring Bas van der Baan</title> <link>https://vial.com/blog/podcast/first-in-human-episode-59-featuring-bas-van-der-baan/</link> <comments>https://vial.com/blog/podcast/first-in-human-episode-59-featuring-bas-van-der-baan/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Tue, 16 Apr 2024 07:38:00 +0000</pubDate> <category><![CDATA[Podcast]]></category> <guid isPermaLink="false">https://vial.com/?p=48715</guid> <description><![CDATA[Amy Del Medico: Hello, everybody. I’m Amy del Medico. I’m here today with Bas van der Baan from Lixte. Bas, welcome. Would you like to give a brief introduction? Bas van der Baan: Sure. Thank you very much. And, great to be here. I joined the board in mid 2022. I have a background in […]]]></description> <content:encoded><![CDATA[<p><strong>Amy Del Medico:</strong> Hello, everybody. I’m Amy del Medico. I’m here today with Bas van der Baan from Lixte. Bas, welcome. Would you like to give a brief introduction?</p> <p><strong>Bas van der Baan:</strong> Sure. Thank you very much. And, great to be here. I joined the board in mid 2022. I have a background in biotechnology. I’ve been in this space for the last 20 years. I co-founded two molecular diagnostic companies and two drug development companies. This is the first time I actually took over from a founder. In September last year, I took over the position of CEO of the company. It’s been a great ride so far.</p> <p><strong>Amy Del Medico:</strong> Fantastic. Thank you. So, we’ve got some sort of broader questions as well as some more detailed questions about the IPLP LB100. But first of all, more broadly, in your opinion, what are the biggest challenges and opportunities facing the <a href="https://vial.com/glossary/oncology/?">oncology </a>drug development landscape today? </p> <p><strong>Bas van der Baan:</strong> So I think, for me now, the biggest challenge obviously is the amount of trials happening. It’s becoming more difficult to enroll patients because there’s just a lot of competing trials out there. But on the upside if you’ve got a really interesting biological rationale, like we do, you can trigger the interest of the PIs and make things happen.</p> <p>So, the great thing is that the time between preclinical concepts in going into the clinic has substantially shortened and we clearly make use of that opportunity within Lixte.</p> <p><strong>Amy Del Medico:</strong> What inspired Lixte to focus on developing PP2A inhibitors? What potential advantages does your approach offer compared to traditional cancer therapies?</p> <p><strong>Bas van der Baan:</strong> What triggered Lixte to take this path was also what really interested me to join as a CEO. They weren’t always on this path. As I just said, I co founded two molecular diagnostic companies and one of them was actually a spinoff of the Netherlands Cancer Institute. I’ve always stayed in close contact with that research department. </p> <p>The Netherlands Cancer Institute is an interesting institute because the research and the hospital are really close together. A lot of things that are happening in the research department spill over to the hospital and vice versa. Interesting setup. One of the inventions there those days was about breast cancer recurrence and the need of chemotherapy. I’ve always stayed in touch.</p> <p>One of the founders, Renee Bernard, always stayed an academic and moved on to combination, right? With the concept that, in <a href="https://vial.com/glossary/oncology/?">oncology</a>, it’s probably not going to be a silver bullet that’s going to solve it. What we see in cancer is that, especially with small molecules, they initially work really well, and then some sort of resistance happens.</p> <p>At the NKI, independently, they were doing a lot of synthetic lethality screens. Both with siRNA as well as CRISPR to figure out the mechanisms of resistance. How can we find that back door a cancer cell is using to escape the therapy? Can I close the back door in parallel and combine the front door, so to speak.</p> <p>They found that the PP2A pathway was a really interesting pathway when they were looking at chemotherapy as well as immunotherapy. Independently of Lixte, they said wouldn’t it be great if we can inhibit that PP2A pathway? Let’s have a look if there’s any company around that’s already working on it.</p> <p>They found Lixte. Lixte was very receptive to their proposals of collaboration. That’s how almost a restart of Lixte started, because Lixte originally was focused on monotherapy, on a few non-oncology indications, and the sweet spot of this drug actually is in combination with either chemotherapy or immunotherapy.</p> <p>We really changed the strategy, discontinued some trials outside the <a href="https://vial.com/glossary/oncology/?">oncology </a>space, discontinued the monotherapy approach, and really focused now on the combination with chemotherapy and immunotherapy based on independent research. That’s where we are now.</p> <p><strong>Amy Del Medico:</strong> That’s really interesting. Thank you. You touched on some of this in your answer, but can you elaborate further on the mechanism of action of LB100 and how it advances, or even enhances chemotherapy, and immunotherapy cancer treatment?</p> <p><strong>Bas van der Baan:</strong> Developers are looking at kinases, right? The onswitch, in signaling. The general perception is there’s too much signaling in a cancer cell. We have to shut the signaling down. The difference between a cancer and a lung cancer cell often is that a cancer cell is way more active, and then, using certain pathways to grow and metastasize. </p> <p>This is a phosphatase inhibitor. Essentially, we’re now targeting the off switch Which is very counterintuitive. Not only is there too much signaling, we’re taking away the off switch too. So what’s happening? What we’ve seen now is that protein phosphatase 2A has a lot of targets. The initial concern was also toxicity, right? That was great because before I joined, they’ve done all the tox studies already, on monotherapy. And it wasn’t toxic, right? It was a potent inhibitor of PP2A and it wasn’t toxic as monotherapy. That fear was taken away. </p> <p>Because it has multiple targets, it has multiple downstream effects. One of the effects is that it pushes the cell into cell cycle. That’s the great thing because [00:05:00] chemotherapy needs a cell while it’s dividing, because then, of course, it can create all that damage in the DNA, which leads to apoptosis.</p> <p>On top of that, what we’ve seen is that one of the other downstream targets of PP2A inhibition is that it inhibits DNA repair. Not only does it lead chemotherapy to do its work in creating damage. It also inhibits the potential repair of the damage, which again makes it an even more effective chemotherapy enhancer.</p> <p>On the other hand, in the area of immunotherapy, it gets even more interesting. What we see has three effects. Two effects outside the cancer cell actually on the immune system. It actually enhances the release of but it also enhances the proliferation of T cells. You get more soldiers to actually fight the tumor. But, the effect on the tumor is interesting because it pushes the cell into the cell cycle and it makes the replication a little chaotic.</p> <p>It creates the PP2A, working on the splicing and on the spliceosome creating new peptides or so-called neoantigens. Those are actually presented on the cell surface. The cancer cell becomes more visible for the immune system with PP2A by increasing the cytokines and increasing T-cell proliferation.</p> <p>A lot of actions and they’re all extremely well described in recent literature because our collaboration with the Netherlands Cancer Institute led to two phenomenal publications that came out actually last week. One will come out tomorrow that precisely describes how this biology works and what the effects are of PP2A inhibition in combination with those therapies.</p> <p><strong>Amy Del Medico:</strong> That sounds like a really exciting approach and good to hear that it’s been well documented as well.</p> <p><strong>Bas van der Baan:</strong> Yeah. It’s one of the critical elements, right? If you’re going to spend your time and put your soul into a company, it’s great if it has a big impact, and by enhancing chemo and immunotherapy, most patients today are treated with either one or the other or both.</p> <p>We know the effect can be better. Chemo response rates are 10 to 20%. Immunotherapy is spectacular in MSI high colon cancer, in lung cancer, in melanoma, right? Tumors with a very high mutation burden. But not so much in all those other tumors. So it can really use that boost. I think this concept can have a very big impact. And then secondly, in today’s day and age, you just got to understand the biology to increase your chance of success.</p> <p><strong>Amy Del Medico:</strong> Absolutely. Everyone, as an industry, is talking about precision medicine. I wondered: is Lixte able to tailor the therapies towards individual patients at this stage?</p> <p><strong>Bas van der Baan:</strong> That’s interesting because I got a lot of experience in molecular diagnostics. That would have been another aspect that I would really like to see in drug development. But, in this case, it is actually pretty broad spectrum. It is a general enhancer of chemo and a general enhancer of immunotherapy.</p> <p>One of the trials we’re conducting is at the MD Anderson Cancer Center in Houston. They proactively approached us. They did a study on a failed immunotherapy trial in ovarian cancer. In the unstratified population, immunotherapy didn’t do much, right?</p> <p>To my knowledge, there’s no approval of immunotherapy in ovarian cancer, but I might not be entirely up to date. But in that trial, at least, immunotherapy didn’t do much in ovarian cancer. If they looked at a subset of ovarian cancer called ovarian clear cell carcinoma. They sequenced all the tumors and what they saw is that there’s a small subgroup that did benefit from immunotherapy. It turned out to be the patients that had a deletion in the PP2A gene. Patients with a PP2A mutation actually responded positively to immunotherapy. </p> <p>What triggered that is okay, can we mimic that mutation? Of course, by inhibiting PP2A and that’s how they found Lixte. They brought Lixte and GlaxoSmithKline with their PD-1 inhibitor, Dostarlimab together. That’s one of the trials we’ve currently opened. It’s not precision medicine. Theoretically, you could leave the PP2A mutants out. But it’s such a small source subset, so that’s not what we’re doing. </p> <p>Currently, we enroll all ovarian clear cell cancers, but it is a very nice indication of the concept that, looking back, you can make immune therapy work in PP2A mutant patients. There’s only a few patients that actually have that mutation. With a vast majority, inhibiting PP2A might be the solution to make immunotherapy work. </p> <p><strong>Amy Del Medico:</strong> You’ve mentioned both breast and ovarian cancer. I wondered if there were any plans to expand LB100 syndications beyond what you currently look at, or if there are any other IPs in the pipeline that you’re developing?</p> <p><strong>Bas van der Baan:</strong> We currently have three trials open. One is the ovarian cancer trial that I mentioned. A second one is a sarcoma trial. It’s advanced soft tissue sarcoma. That’s a very difficult [00:10:00] one, right? Chemotherapy has been the standard of care for the last 40 years and many have tried to get better outcomes in that disease. We’re working with the European consortium for sarcoma, called GEIS out of Madrid, to combine LB100 with chemotherapy in advanced soft tissue sarcoma. </p> <p>The third trial that we currently have open is in small cell lung cancer, where the standard of care is chemotherapy with immunotherapy. At least that’s the standard in the U. S. Still those patients have a very poor prognosis, in later stages of the disease. They ‘re combining LB 100 with chemo and immunotherapy to see if we can give it a double boost. Those are the three that are currently in the works. </p> <p>We’re looking and considering doing something in colon cancer, especially MSI low. We know immune therapy is really effective in MSI high colon cancer because of the high mutation burden, but there’s hardly any benefit and no approval of immunotherapy MSI low, advanced, or metastatic colon cancer.</p> <p> That’s one, based on that neo-antigen production featuring the capacity of PP2A inhibition. We think that we can make those cold tumors immune hot. That’s something that we are considering for the near future based on publications.</p> <p><strong>Amy Del Medico:</strong> So a range of quite different indications. LB100, I believe, is a new class of therapy. I just wondered if you’d come across any particular hurdles from a regulatory perspective?</p> <p><strong>Bas van der Baan:</strong> Not yet. So we have our IND approval in the US and our IMPD approval in Europe. We’re conducting trials both in Europe as well as in the US. It’s definitely a first in class. Lixte is unique in their approach. There’s nobody else taking this approach, which can be a little scary, taking such a new route.</p> <p>But also, We’ve got to do new things and controversial things, too. In many cancers we do see a bit of a plateau in survival. To push for that extra survival benefit, we have to do things that we haven’t done before. One of the latest articles is calling it a paradoxical activation, right? Taking the brakes off, but the preclinical data looks spectacular. Now we’re in a very interesting phase having three trials open to see if that preclinical data actually translates to clinical benefit.</p> <p><strong>Amy Del Medico:</strong> Fantastic. Thank you. From a more of a <a href="https://vial.com/blog/articles/what-is-a-cro/?">CRO </a>perspective, we know clinical trials are complex, costly, time-consuming, and probably no more so than in oncology clinical trials. What sort of role do you feel that <a href="https://vial.com/blog/articles/what-is-a-cro/?">CROs </a>will play in Lixte’s development process? </p> <p><strong>Bas van der Baan:</strong> We’re not just in the clinic because we did our safety trials. The three trials were open and investigator initiated. The roles of zeros at this point are more around monitoring and pharmacovigilance. I’d love to go to the next phase where we do the industry sponsored trials. I’d love to work with a good <a href="https://vial.com/blog/articles/what-is-a-cro/?">CRO </a>to get those larger trials going ; deliver the outcome and the quality necessary for the approval process, both in the US and in Europe.</p> <p><strong>Amy Del Medico:</strong> That’s great. Good to hear. Thank you. Final question. Just looking ahead, I wonder what the long term goals are and the visions for Lixte?</p> <p><strong>Bas van der Baan:</strong> So again, a very interesting company. It has one asset. Some people sometimes say you need to have a pipeline. What if your number one asset doesn’t make it? If you look at the approvals, I think currently there are almost 8000 trials open in oncology, right?</p> <p>How many first in class approvals do you have a year? Maybe double digits, but, at best, you have to be realistic. For us, it really works well. It gets our full attention. For us, it’s really about showing some first signals in humans. Therefore we pick those three trials that we’ve currently open to combine LB100 with only chemotherapy in soft tissue sarcoma to combine it with chemo and immunotherapy in small cell lung cancer and with only immunotherapy in ovarian. We’ve picked all the possible combinations of where we think the sweet spot of our drug is.</p> <p>We’ve picked three different cancer indications of which I think the ovarian one is the most interesting because we have this mutation data that shows that patients with a PP2A actually do respond to immunotherapy. But, as I said before, most patients will receive either chemo or immunotherapy or both. We’re really looking forward to expanding this indication within a much broader scale of cancers in the future. As I gave an indication already, we’re looking at colon cancer at the MSI low colon cancer at the moment. But, there’s a number of other cold tumors that are not so immune responsive that hopefully we can make immune therapy more effective.</p> <p>Bas, thank you very much for sharing your vision and also the science behind it is very interesting.</p> <p>Thank you very much. Thanks a lot. Great questions. Thank you.</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/podcast/first-in-human-episode-59-featuring-bas-van-der-baan/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>IQVIA vs Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/iqvia-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/iqvia-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 10 Apr 2024 16:55:59 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CRO's]]></category> <category><![CDATA[CROs]]></category> <guid isPermaLink="false">https://vial.com/?p=48673</guid> <description><![CDATA[The increasing costs associated with in-house drug development have prompted biotech and pharmaceutical companies to increasingly outsource to contract research organizations (CROs) for assistance. CROs like Vial, IQVIA, PPD, ICON, among others, have distinguished themselves through strategic partnerships and innovative products and services, positioning them as leaders in the industry. However, one size does not […]]]></description> <content:encoded><![CDATA[<p>The increasing costs associated with in-house drug development have prompted biotech and pharmaceutical companies to increasingly outsource to <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organization</a>s (CROs) for assistance. CROs like <a href="https://vial.com/?">Vial</a>, IQVIA, PPD, ICON, among others, have distinguished themselves through strategic partnerships and innovative products and services, positioning them as leaders in the industry.</p> <p>However, one size does not fit all, and size matters when it comes to choosing the right to CRO to partner with. With over 1,100 CROs worldwide as of 2020, choosing the appropriate partner often requires evaluating the advantages and disadvantages of large global CROs versus mid/small ones. This article provides a comparative overview of Vial and IQVIA.<br><br></p> <h2 class="wp-block-heading">A Brief Introduction</h2> <p>Founded in 1982, IQVIA has emerged as one of the largest CROs in the world. IQVIA stands out for its scale and global reach, offering a variety of services across diverse therapeutic areas and regions. The strong commitment to innovation allows IQVIA to provide advanced analytics, technology solutions, and industry-specific expertise in the health information technologies and clinical research domains.</p> <p>IQVIA has a vast collection of healthcare information, including over 1.2 billion comprehensive, longitudinal, non-identified patient records. The company reported a revenue of $11,116 million for the first nine months of 2023, marking a 4.2% increase on a reported basis and an 4.8% increase at constant currency compared to the corresponding period in 2022.</p> <p>Still in its early stages, Vial was established in 2020 and has quickly emerged as a leading CRO. Positioned as a technology-centric CRO, Vial aims to revolutionize <a href="https://vial.com/glossary/clinical-research/">clinical research</a> by delivering faster, superior, and more cost-effective <a href="https://vial.com/glossary/clinical-trial/">clinical trial</a> outcomes. It draws upon 150 years of collective experience and expert scientific advisory board members to deeply understand clinical trial requirements.</p> <p>Vial’s track record includes over 750 completed trials and a network of more than 35 research sites. By mid-2022, Vial had secured over $100 million in venture funding, with General Catalyst leading a $67 million Series B financing round in November 2022.<br></p> <h2 class="wp-block-heading">Therapeutic Area Coverage</h2> <p>IQVIA:</p> <ul><li>Oncology</li> <li>Central nervous system</li> <li>Infectious disease & vaccines</li> <li>Cardiovascular</li> <li>Dermatology</li> <li>Gastrointestinal & hepatology</li> <li>Endocrinology</li> <li>Allergy & respiratory</li> <li>Rheumatology</li> <li>Ophthalmology</li> <li>Nephrology</li> <li>Reproductive health</li> <li>Early clinical development</li> <li>Cell & <a href="https://vial.com/glossary/gene-therapy-2/">gene therapy</a></li> <li>Rare diseases</li> <li>Pediatrics</li> <li><a href="https://vial.com/glossary/biosimilars/">Biosimilars</a></li></ul> <p>Vial:</p> <ul><li><a href="https://vial.com/glossary/oncology/">Oncology</a></li> <li><a href="https://vial.com/glossary/dermatology/">Dermatology</a></li> <li><a href="https://vial.com/glossary/ophthalmology/">Ophthalmology</a></li> <li><a href="https://vial.com/glossary/gastroenterology/">Gastroenterology</a></li> <li><a href="https://vial.com/glossary/central-nervous-system-cns/">Central nervous system</a></li> <li><a href="https://vial.com/glossary/cardiology/">Cardiology</a></li> <li>Medical device</li> <li>Rare disease</li> <li>Digital therapeutics</li></ul> <h2 class="wp-block-heading">Small vs. Large CRO</h2> <p>As a large CRO, IQVIA offers unparalleled global reach and rapid scalability, making it an invaluable partner for conducting clinical trials across multiple continents. Moreover, IQVIA offers strategic partnership opportunities, multi-year contracts, and quick access to project managers and clinical research associates1,3.</p> <p>IQVIA’s core strengths lie in its specialized global IT infrastructure tailored for healthcare, analytics-driven clinical development, a robust ecosystem of real-world solutions, and an expanding suite of proprietary clinical and commercial applications.</p> <p>In contrast, Vial, a small CRO, offers a more personalized and attentive approach that is particularly well-suited for smaller or midsize biotech, pharmaceutical, or medical device companies.</p> <p>Vial’s emphasis on flexibility, customization, proactive problem-solving, and commitment to addressing delays associated with traditional clinical trial management methods make it an appealing choice for sponsors.</p> <h2 class="wp-block-heading">Technology</h2> <p>IQVIA offers clinical trial data management platforms aimed at optimizing efficiency and improving trial outcomes. The majority of IQVIA Technologies’ Orchestrated Clinical Trials are cloud-based solutions meticulously designed to seamlessly integrate and deliver optimal performance when used together. IQVIA’s portfolio includes integrated data flow ecosystems, electronic clinical outcome assessment (eCOA), <a href="https://vial.com/epro/">electronic patient-reported outcome (ePRO)</a> tools, and remote monitoring solutions.</p> <p>Similarly, Vial’s eClinical platform offers comparable tools, but it uniquely integrates <a href="https://vial.com/edc/">Electronic Data Capture (EDC)</a> and real-time electronic source (<a href="https://vial.com/blog/articles/how-esource-can-streamline-your-clinical-trials/?)">eSource</a> data capture to enhance performance and mitigate data loss risks. The system includes robust user permissions, audit trails, and data management features that are essential for comprehensive data review and cleaning.</p> <p>Vial’s EDC enables real-time data capture through integration with external tools such as ePRO or digital wearables. Importantly, unlike IQVIA, Vial’s EDC is entirely developed by an internal software engineering team, reducing vendor dependencies. This in-house development empowers Vial to directly manage the technology and provide extensive support and training for optimal platform use. It also allows for customized data integration to meet specific study needs.</p> <p>Implemented within a cloud-native infrastructure, Vial’s unified EDC ensures a user-friendly experience while strictly adhering to security standards, including regulatory compliance with 21 CFR Part 11, HIPAA, and GDPR. The platform offers intuitive electronic case report forms (eCRFs) and customizable forms, facilitating structured data collection according to sponsor’s requirements.</p> <h2 class="wp-block-heading">Cost/Time Management</h2> <p>When collaborating with large CROs like IQVIA, sponsors often face challenges such as budget inflation due to delays and variable pricing models. Many sponsors have reported issues where initial discussions fail to adequately address trial risks and realities, leading to delays and increased costs.</p> <p>Vial stands out from IQVIA in several key aspects. Vial employs data-driven approaches to assess risks, optimize trial processes, and enhance efficiencies through its in-house technology platform. This approach replaces paper sources, supports productivity-enhancing workflows, and ensures compliance with relevant regulations. Streamlining trial processes, from onboarding and patient enrollment to data collection and site communication, is a priority for Vial.</p> <p>Vial’s EDC system minimizes protocol deviations, ensures data quality with real-time capture systems, and maintains project timelines. Additionally, Vial ensures streamlined site activation within 30 days or less and accelerates patient recruitment through modern recruitment strategies and an ePRO. The [Vial Site Startup App](<a href="https://vial.com/onboarding/?)">https://vial.com/onboarding/?)</a> facilitates rapid site onboarding, offering a promising solution to overcome prolonged site start-up durations.</p> <p>Vial adopts a fixed-fee pricing model devoid of change orders to streamline cost-effectiveness in drug development. This strategy guarantees a predetermined budget for the entire project, regardless of the incurred time and expenses. By prioritizing transparency and collaboration, Vial ensures sponsors achieve cost savings without compromising trial quality.</p> <h2 class="wp-block-heading">Exploring CRO Partnership Options?</h2> <p>When choosing a CRO, it’s essential to prioritize the appropriate skill sets and acknowledge the significant impact that size can have on project outcomes. Vial CRO recognized the need to evolve and is dedicated to reimagining next-generation, technology-enabled clinical trials while ensuring the quality of research remains paramount. Despite its youth, Vial has demonstrated expertise in cutting-edge technology and efficient clinical operations, thanks to its experienced team of ClinOps executives and technology leaders. Partnering with Vial guarantees sponsors a clear and predictable cost framework, aiding in the more effective management of drug development expenses.</p> <p><strong>To learn how Vial’s CRO can support your clinical trial, please visit our website or <em><a href="http://www.vial.com/contact-us">get in touch with a Vial representative today</a>!</em></strong></p> <p><br></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/iqvia-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>The Costs of Working with a CRO</title> <link>https://vial.com/blog/articles/the-costs-of-working-with-a-cro/</link> <comments>https://vial.com/blog/articles/the-costs-of-working-with-a-cro/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 27 Mar 2024 21:21:25 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[#clinicaltrial]]></category> <category><![CDATA[#clinicaltrials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CRO's]]></category> <category><![CDATA[CROs]]></category> <guid isPermaLink="false">https://vial.com/?p=48212</guid> <description><![CDATA[The Costs of Working with a CRO Running clinical trials is an integral part of the pharmaceutical industry, crucial to the development and approval of new drugs and treatments. However, because these trials come with a significant financial burden, sponsor companies are constantly seeking ways to optimize their operations. Traditionally, they can go about this […]]]></description> <content:encoded><![CDATA[<p><strong>The Costs of Working with a CRO</strong></p> <p>Running <a href="https://vial.com/glossary/clinical-trial/">clinical trials</a> is an integral part of the pharmaceutical industry, crucial to the development and approval of new drugs and treatments. However, because these trials come with a significant financial burden, sponsor companies are constantly seeking ways to optimize their operations. Traditionally, they can go about this in one of two common approaches: acquire clinical trial services in-house or outsource the study either partially or fully to a <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organization (CRO)</a>. CROs are external companies that provide contract-based support in the form of various trial services to the pharmaceutical, biotechnology, and medical device industries. These services range from clinical study design and data management to statistical analysis and regulatory support.</p> <p>Efficiency is crucial when conducting clinical trials and CROs are valuable experts in their field that have consistently played a key role in the success of many studies. However, sponsors must also consider the costs associated with hiring such companies to partner with and whether they are more convenient than hiring support in-house. In this article, we delve into the factors influencing costs of working with a CRO, expenses associated with clinical trials, and whether CROs are more cost-effective than sponsors acquiring support themselves.</p> <p><strong>Increasing Demand for Contract Research Organization (CRO) Support</strong></p> <p>CROs are pivotal to the successful execution of clinical trials, offering a tailored resource of expertise, services, and logistical support that can significantly streamline the process for sponsors. According to a report by Grand View Research, the global CRO services market was valued at approximately US$50.55 billion in 2023 and is expected to grow at a compound annual growth rate (CAGR) of 7.0% from 2024 to 2030. In the last few decades alone, outsourcing clinical trials either in part or fully to a CRO has become increasingly common for the following key reasons:</p> <ol><li>Expansion of R&D capabilities to multiple products and multi-phase development pipelines poses challenges with time and cost constraints</li> <li>Domestic costs of internal services are rising</li> <li>Globalization within the <a href="https://vial.com/glossary/clinical-research/">clinical research</a> industry increasing the availability of international low-cost labor providers</li> <li>Greater emergence of smaller biopharma and biotech start-ups that often have not yet established a reputation</li> <li>Technological advancements improving efficiency in collecting and sharing data</li></ol> <p><strong>Factors Influencing the Cost of CROs</strong></p> <p>Given their demand, hiring a CRO is typically accompanied with considerable costs. However, the exact total may be influenced by several factors, such as the size and phase of the trial, study design complexity, and the extent of services contracted by the trial company. Sponsors must also be cognizant of additional costs associated with more rigorous services such as data management and analysis, maintaining regulatory compliance, and providing individual site monitoring. Furthermore, some long-standing CROs like IQVIA and PPD employ a considerably larger number of employees than newer players like Vial. These organizations are more likely to possess broader geographical coverage worldwide, as well as offer a greater variety and depth in their service offerings. Lastly, the industry reputation and track record of the CRO in question can certainly influence how expensive their services are for those looking to partner with them. Based on these factors, and many more, the costs associated with hiring a CRO can vary significantly. Ultimately, study sponsors must thoroughly review and compare different CROs to find one that offers the best balance of expertise, value, and cost-effectiveness.</p> <p><strong>The Costs of Running a Clinical Trial</strong></p> <p>According to a report submitted by Sertkaya and colleagues to the United States Department of Health and Human Services, the average overall cost of conducting <a href="https://vial.com/glossary/phase-i/">phase I</a>, <a href="https://vial.com/glossary/phase-ii/">phase II</a>, and <a href="https://vial.com/glossary/phase-iii/">phase III</a> clinical trials is approximately $4 million, $13 million, and $20 million respectively [<a href="https://aspe.hhs.gov/sites/default/files/private/pdf/77166/rpt_erg.pdf">6</a>]. For new drugs approved by the US <a href="https://vial.com/glossary/food-and-drug-administration-fda/">Food and Drug Administration (FDA)</a>, pivotal phase 3 studies cost a median of $41,117 per patient. However, these costs will vary depending on several factors, such as the phase of the trial. For example, early-phase trials enroll a smaller number of patients, typically between 15 to 20, compared with phase 3 trials, which can require several hundred to several thousand participants.</p> <p>Other factors influencing the costs of running a clinical trial include the required sample size, the geographical location of the trial, the number of clinical sites involved, the therapeutic area of the drug, the type of drug being assessed, and the specific tests and procedures required per protocol. The type of sponsorship must also be accounted for because academic institutions sponsoring clinical trials often have access to fewer financial resources compared with commercial sponsors, which typically have larger budgets for their drug development programs. Personnel salaries, site rentals, and equipment maintenance are other necessary expenses associated with clinical trials. Lastly, global trials will also require additional expenses for international site management, translation services, and regulatory consultants to ensure proper compliance in each participating country.</p> <p><strong>Comparing the Cost of CROs to Sourcing Services In-House</strong></p> <p>Although it may initially appear to be more cost-effective for sponsors to acquire clinical trials in-house, there are several factors which influence how these costs compare those of outsourcing to a CRO. For example, recruitment, training, and maintaining a well-trained team come with hidden costs which can add up quickly. Sponsors who choose to source these tasks on their own must also spend a significant portion of their time setting up the trial, from staff training and protocol development to completing regulatory submissions and managing data analysis. However, by outsourcing these out to a CRO, with its established infrastructure and expertise, sponsors not only free up valuable time to devote instead to developing their R&D efforts, but their clinical trial processes are also more streamlined. The following factors are also important to consider to determine which of these options is best for sponsors:</p> <ul><li>CROs typically have lower internal overhead costs (e.g., legal, accounting, executive management, training, quality assurance, finance, and more)</li> <li>A larger number of resources and services requires more time to manage, especially if there are quality issues</li> <li>Filling open positions for in-house experts can take time, potentially delaying trial timelines</li> <li>Outsourcing offers established infrastructures from CROs with experience overcoming challenges common to clinical trials</li></ul> <p>While it may seem that enlisting the services of a CRO is more costly on an hourly basis, sponsors must conduct a comprehensive analysis that accounts for the above-mentioned factors and many more. Given the steadily growing popularity of outsourcing, hiring a CRO may end up being the more cost-effective solution in many case. However, this will depend heavily on the specific circumstances of the trial. Ultimately, conducting an informed review of these cost factors will ensure companies make the best decision for their clinical development and operational needs.</p> <p><strong>Vial: The CRO for Biotech, Powered by Technology</strong></p> <p><em>Our mission at <a href="https://vial.com/cro">Vial</a> as a global next-generation, tech-first CRO is to empower scientists to discover groundbreaking scientific therapeutics that help people live happier, healthier lives. We have built a sponsor friendly, full-service CRO powered by technology that enables us to deliver flexibility, cost-effectiveness, and reliability. <a href="https://vial.com/contact-us/">Contact a Vial representative today</a> to discover how we can make a difference for your next clinical trial</em></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/the-costs-of-working-with-a-cro/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>First in Human Episode #58 featuring Samarendra Mohanty</title> <link>https://vial.com/blog/podcast/first-in-human-episode-58-featuring-samarendra-mohanty/</link> <comments>https://vial.com/blog/podcast/first-in-human-episode-58-featuring-samarendra-mohanty/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Tue, 26 Mar 2024 09:27:00 +0000</pubDate> <category><![CDATA[Podcast]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[Ophthalmology]]></category> <category><![CDATA[Ophthalmology CRO]]></category> <guid isPermaLink="false">https://vial.com/?p=48053</guid> <description><![CDATA[Amy Del Medico: Hello, I’m Amy del Medico. I’m VP of Ophthalmology here at Vial. I’m here talking today with Samar Mohanty from Nanoscope Therapeutics. Hi Samar, welcome. Samarendra Mohanty: Hello, Amy Amy Del Medico: What inspired you to focus on gene therapies for retinal diseases? What led to the founding of Nanoscope? Samarendra Mohanty: […]]]></description> <content:encoded><![CDATA[<p><strong>Amy Del Medico:</strong> Hello, I’m Amy del Medico. I’m VP of Ophthalmology here at Vial. I’m here talking today with Samar Mohanty from Nanoscope Therapeutics. Hi Samar, welcome.</p> <p><strong>Samarendra Mohanty:</strong> Hello, Amy</p> <p><strong>Amy Del Medico:</strong> What inspired you to focus on gene therapies for retinal diseases? What led to the founding of Nanoscope?</p> <p><strong>Samarendra Mohanty:</strong> Since its inception of optogenetics, researchers have been focused on making optogenetics as a toolkit to modulate activities of selected neurons by light activation to understand how the neural circuitry functions. In my career, I’ve had a chance to lead optogenetics programs at multiple institutions wherein I met key contributions such as past demonstrations of near infrared optogenetics stimulation in cells and in animals. </p> <p>But, I found out patients with retinal degenerative disease lose their sight, and light-sensitive retinal cells, namely rods and cones. Since eyes are transparent, it led me to wonder if we could impart high enough light sensitivity to the remaining cells of the retina. Namely, bipolar cells, so that we can desensitize them to ambient light. And thus, we would have sight-restoring potential. </p> <p>This prospect became very compelling and exciting to me after reading on retinal degeneration and realizing there were no available treatments. However, to be restorative to ambient light, in addition to high light sensitivity of the optogenetic molecules or called obscene. They need to have light sensitivity across the visual spectrum that is from blue to the red, and be able to address retinal degeneration patients with highest unmet need, optogenetics provide the best from cell and gene therapy. We repurpose the existing cells as de facto photoreceptors, which remain intact and integrated in the retina, even in the advanced stage of the disease. A significant market opportunity existed for an optogenetic platform that could restore high-quality real life vision. </p> <p>After observing the robust efficacy and safety of MCO in multiple mice models of retinal degeneration, we founded NanoScope for clinical translation of this transformative therapy.</p> <p><strong>Amy Del Medico:</strong> Thank you. I do think this is one of the most exciting areas of ophthalmology. I wondered if you could elaborate a bit more on the science behind the MCOs and how they restore vision?</p> <p><strong>Samarendra Mohanty:</strong> An MCO is an optogenetic biotherapeutic platform comprised of a multi component transgene. A cell-specific promoter enhancer, and a proprietary delivery system. The bioengineering we conducted to develop MCO platforms is unlike anything that has ever been tried in optogenetics and for retinal degenerative patients.</p> <p>The transgene is made up of three distinct light-sensitive molecules fused into a single contiguous protein when expressed. Unlike classical optogenetics, two of these three light-sensitive molecules are not transmembrane. Each of these light-sensitive molecules is highly sensitive to different parts of the visible spectrum. So, that the overall fusion protein is highly sensitive to the broadband ambient light that normal eyes are sensitive to.</p> <p>The components of the MCO platform, the promoter enhancer, and each light-sensitive molecule in the MCO are tunable, making it possible for us to optimize the characteristics, such as kinetics, sensitivity, and target cell type that are tailored to the needs of patients, and underlying pathology for specific indications.</p> <p>I would like to note that while our proprietary AV vector allows efficient manufacturability and tropism for targeted retinal cells via intravitreal delivery, we have a non-viral delivery approach to target central retina, which matches the geographic atrophies.</p> <p><strong>Amy Del Medico:</strong> The non-viral approach that you’ve developed, the laser delivered NOD technology, what are its main advantages, would you say, compared with traditional gene therapy delivery methods?</p> <p><strong>Samarendra Mohanty:</strong> The viral package gene therapy has been shown to target well in ophthalmic conditions, but is known to evoke inflammation primarily due to the viral capsid. While the inflammation can be controlled and tolerated, especially in advanced retinal degeneration patients, we envision a non-viral approach may be better suited for mass indication, such as geographic atrophies.</p> <p>Furthermore, it will be ideal for targeting the atrophic areas of GA with therapeutic genes, leaving the surrounding healthy retina intact. Our non-viral laser delivery approach overcomes the challenges of viral delivery and provides a unique opportunity to efficiently deliver obscene encoding genes to GA lesions via OCT image guidance.[00:05:00] </p> <p>In the nano-enhanced optical delivery or NOD method, the nanoparticles are mixed with plasmids and injected intravitreally. Before low power near infrared irradiation of targeted retina occurs to transiently and locally enhance the light intensity, leading to permeabilization of the cells and uptake of the plasmid.</p> <p>Another key advantage of a non-viral laser -based NOD method is the ability to re-dose precisely. As atrophy has a certain progression rate. An OCT-guided laser beam can paint the new atrophic areas and sensitize those photoreceptor-lacking regions with the therapeutic obscene without being hindered by immune rejection.</p> <p><strong>Amy Del Medico:</strong> Thanks, Mar. Geographic atrophy is certainly a hot topic at the moment, and I know from what you said previously, MCO-010 does target a broad spectrum of retinal diseases. How does this address the genetic variations present in these conditions?</p> <p><strong>Samarendra Mohanty:</strong> Optogenetics as a method has long been considered a gene agnostic approach for restoring vision due to a variety of genetic mutations. In preclinical models and now in clinical studies, we have shown that MCO-010 can restore vision not just in a mutation-independent manner, but in a disease-agnostic manner for all outer retinal degenerative disease.</p> <p>Since advanced retinal desensitization patients have no photoreceptors, we target the next layer of retinal bipolar cells, which are intact. Thus, irrespective of gene mutation and underlying pathology, we can restore light sensitivity and high-quality vision by repurposing the bipolar cells as de facto photoreceptors.</p> <p>We have so far treated 35 patients across three trials and therefore have the largest population of optogenetically treated patients. I want to emphasize here that before there has been no approach like MCO that restored sensitivity to ambient light across the visual spectrum in real-time in these patients across dozens of mutations that cause the retinal dystrophy.</p> <p><strong>Amy Del Medico:</strong> Extremely interesting, Samar. Very exciting, too. I wondered about one aspect of your therapy. That it can be applied in different lighting conditions. Can you describe the mechanism that allows for this?</p> <p><strong>Samarendra Mohanty:</strong> You are right in noting that, historically, the optogenetic field has struggled to realize the therapeutic potential because no one light-sensitive molecule can perform well across different intensities. And colors of light presented to us in our daily living in real-time as they are presented. Thus, prior attempts have used light-intensifying goggles and have struggled to truly benefit patients with regard to restoring high-quality vision.</p> <p>The simple answer to the unique mechanism by which MCOs can work in ambient light. That lies in the structure of the fused protein. Each of the three components are highly sensitive to different colors of light. If you think of ambient light, whether it be sunlight or office lighting. We are generally talking about some type of white light where a mixture of colors is present that we see blended.</p> <p>One is sensitive to any given color, then it is only sensitive to a fraction of the light in most common places, and thus effectively not that sensitive at all. But if a highly sensitive obscene to multiple colors of light that span across the visible spectrum of white light. Such as MCO, then one will not only be highly sensitive to those individual colors, but the ambient light conditions are sufficient for MCO to generate a vision signal.</p> <p>The components of MCO amplify light and light-induced photocurrent by acting in unison. The unique structure of MCO also provides a high dynamic range. It means the treated patient can See in a bright sunny day in an office building and even under starlight in a cloudy moonlight,</p> <p><strong>Amy Del Medico:</strong> It’s very impressive. I imagine developing a therapy like yours has met with some challenges along the way, as any sort of development program does. Are you able to discuss some of the more significant hurdles that nanoscope has overcome in developing the technology?</p> <p><strong>Samarendra Mohanty:</strong> From the start, the selection of multiple light-sensitive molecules from nature required significant R&D activities. Further, a significant amount of bioengineering had to be deployed to fuse them together in a manner that allowed each component to not only add but multiply the effect without compromising the kinetics.</p> <p>There is also a limit to the size of the transgene that AAV vector can package. While this is not a concern in classical optogenetics, because the microbial option sizes are within the AAV payload capacity, the MCO fusion protein size [00:10:00] is rather large. So we have to remain within the limit imposed by the AV capacity.</p> <p>Getting sufficient AV yield with an acceptable full-to-empty ratio was a challenge. It required us to utilize a proprietary vector that could handle the large transgene. A lot of virgin ground remains in the regulatory landscape for gene therapy, specifically with ocular gene therapy for patients with severe vision loss.</p> <p>First, MCO is a mutation agnostic gene therapy, and therefore will be the fastest therapy in the gene therapy space for any indication. We have to develop the strategy and relevant functional potency assay for the new mechanism of action. Second, there is no approved therapy for severe vision loss patients, therefore no precedence on the endpoint.</p> <p>Something as simple as a validated test to measure vision and vision improvement in this patient simply did not exist when we started. In this instance, we not only had to develop a novel test to assess these patients, but also validate them and then educate the regulators on their usefulness. Still, there is work to be done to establish clinical meaningfulness.</p> <p>Lastly, retinal degenerative patients are constantly on a decline in vision, and it may take years before the true impact and magnitude of such restorative therapies can be assessed with respect to the control group. Further, there is no natural history for these patients, especially very low vision patients. Therefore, we are working with regulators to define intermediate clinical endpoints so that the clinical outcome assessments measured at earlier time points can reasonably predict the future impact of this restorative therapy.</p> <p><strong>Amy Del Medico:</strong> It sounds like you’re leading the way with novel endpoint development and certainly novel testing and validation. Given the potential life altering effects of your therapies, how is Nanoscope working towards making these treatments accessible to a wider range of patients in the future?</p> <p><strong>Samarendra Mohanty:</strong> Absolutely. We believe these retinal degeneration patients have been waiting long enough. Now IRDs are the leading cause of blindness in the working age population. The patients can’t wait anymore and we have to find expedited pathways to make these transformative therapies available to them on an urgent basis.</p> <p>FDA and specifically CBER leadership has been publicly vocal that gene therapy approvals are a priority in this season and that avenues such as accelerated approval are attractive paths and becoming the norm to get these life changing therapies to patients. So our strategy within IRDs. Is twofold.</p> <p>Firstly, within IRDs, we hope that initial approval for advanced RP patients will be followed by a level expanding approvals to all IRD indications. Secondly, we hope to expand the approval to moderate vision loss patients so that it can benefit a large majority of all legally blind IRD patients. We also see our results in Stargardt macular degeneration as a read through to the benefit that MCO treatment can provide to advanced AMD patients, specifically those suffering from geographic atrophy, we are working towards taking it to the clinic towards the end of this year.</p> <p><strong>Amy Del Medico:</strong> Yeah, there’s no doubt about the level of unmet needs. It’s very exciting for those patients. My final question really is just to do with looking ahead to the future. I wonder what your thoughts are on the state of gene therapy research for retinal disease? How do you see Nanoscope further contributing to advancements in this particular field?</p> <p><strong>Samarendra Mohanty:</strong> Since most rare diseases in the retinal degeneration space have an underlying genetic mutation, gene therapies are going to be an obvious choice for restorative and durable outcomes. I see Nanoscope as pioneering mutation agnostic gene therapy so that the therapy can be democratized to a wider population irrespective of the underlying mutation.</p> <p>That has been known to be associated with the diseases, or there are mutations that are yet to be identified. Our clinical programs have shown that such mutation agnostic therapies are safe, durable and beneficial to patients. I hope that the groundwork we have done in this space will not only make it easier for our pipeline programs to advance at a rapid pace, but also help other retinal gene therapy programs in development.</p> <p>My view is that a historic opportunity for retinal gene therapy is emerging, where technological advances, efficacies, clinical data, and regulatory momentum are coming together in a way we have not seen before. I believe that Nanoscope holds a strong position within this historic opportunity to serve patients with highest unmet [00:15:00] needs globally.</p> <p><strong>Amy Del Medico:</strong> Samar, thank you. That was absolutely fascinating to hear you talk about what you’ve been developing. We appreciate your time.</p> <p><strong>Samarendra Mohanty:</strong> Thank you, Amy. Thank you for the opportunity.</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/podcast/first-in-human-episode-58-featuring-samarendra-mohanty/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Worldwide Clinical Trials vs Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/worldwide-clinical-trials-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/worldwide-clinical-trials-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Mon, 25 Mar 2024 18:04:00 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CRO's]]></category> <category><![CDATA[CROs]]></category> <guid isPermaLink="false">https://vial.com/?p=48056</guid> <description><![CDATA[Introduction Worldwide Clinical Trials vs. Vial. How do they stack up? Worldwide Clinical Trials is a mid-size, full-service global contract research organization (CRO) that works with biotech and pharma to advance new medications. With an international presence in nearly 60 countries, Worldwide is supported by over 3,400 team members. Vial is a tech-first CRO reimagining […]]]></description> <content:encoded><![CDATA[<h2 class="wp-block-heading">Introduction</h2> <p>Worldwide Clinical Trials vs. Vial. How do they stack up? Worldwide Clinical Trials is a mid-size, full-service global <a href="https://vial.com/glossary/cro-contract-research-organization">contract research organization</a> (CRO) that works with biotech and pharma to advance new medications. With an international presence in nearly 60 countries, Worldwide is supported by over 3,400 team members. Vial is a tech-first CRO reimagining <a href="https://vial.com/glossary/clinical-trial">clinical trials</a> to deliver faster, more efficient trial results at dramatically lower costs for biotech sponsors. More than just a CRO, the people of Vial are “doers” – innovators, engineers, and <a href="https://vial.com/glossary/clinops-clinical-operations">ClinOps</a> leaders working together to build a global, full-service CRO powered by intuitive end-to-end technology.</p> <h2 class="wp-block-heading">Therapeutic Area Coverage</h2> <p>Worldwide Clinical Trials (Worldwide) focuses on the following therapeutic areas: neuroscience, <a href="https://vial.com/glossary/oncology">oncology</a>, rare diseases, and cardiometabolic and inflammatory diseases. With neuroscience experience in <a href="https://vial.com/glossary/alzheimers-disease">Alzheimer’s Disease</a> (AD) and dementia, <a href="https://vial.com/glossary/neurology">neurology</a>, pain and addiction, rare and other diseases, and psychiatry, Worldwide has been involved in neuroscience research since the 1970s. Worldwide Dermatology CRO’s dermatology <a href="https://vial.com/glossary/phase-i">Phase I</a> unit provides a unified full-service solution to early clinical development and has run over 500 dermatology clinical trials covering more than 10,000 randomized patients. In the past three years, Worldwide has collaborated on 105 oncology studies supported by a global team of oncology clinical development experts. In rare diseases, Worldwide is experienced in autoimmune and inflammation; disorders of the heart, lungs, and kidneys; eye disorders; musculoskeletal disorders; blood disorders; genetic disorders; endocrine and metabolic disorders; nervous system disorders; and rare cancers. With more than 30 years of experience in cardiovascular research, Worldwide has global trial experience in all phases. Worldwide also offers an extensive group of subject matter experts in rare and common metabolic conditions. Other therapeutic areas include <a href="https://vial.com/glossary/cell-and-gene-therapies">cell and gene therapy</a>.</p> <p>The Vial CRO team’s expertise spans indications in each therapeutic area, with prior experience at both large and small CROs. In common with Worldwide, Vial therapeutic areas include <a href="https://vial.com/cro/oncology">oncology</a>, <a href="https://vial.com/cro/neurology">neurology</a>, <a href="https://vial.com/cro/cardiology">cardiology</a>, and <a href="https://vial.com/cro/rare-disease">rare diseases</a>. In addition, Vial has expertise in <a href="https://vial.com/cro/dermatology">dermatology</a>, <a href="https://vial.com/cro/ophthalmology">ophthalmology</a>, <a href="https://vial.com/cro/gastroenterology">gastroenterology</a>, <a href="https://vial.com/cro/medical-device">medical devices</a>, and <a href="https://vial.com/cro/digital-therapeutics">digital therapeutics</a> (DTx). Vial’s scientific advisory board has experience across all its therapeutic areas and <a href="https://vial.com/glossary/immuno-oncology/">immuno-oncology</a> for the Oncology CRO. Lastly, Vial’s experienced CRO executive team works closely with the expert site operations team to continuously review execution strategies, closely monitor patient recruitment efforts, and mitigate key study risks for all its clinical trials.</p> <h2 class="wp-block-heading">Evolution of the Sponsor-CRO Relationship</h2> <p><a href="https://vial.com/glossary/clinical-research">Clinical research</a> and drug development have transformed in recent years with the emergence of big data analytics, advanced trial technologies, and rapid advances in precision therapies. The need for closer and more strategic partnerships between sponsors and CROs is in line with these changes. Sponsors increasingly depend on CROs for specialized expertise, global reach, and data capabilities and to navigate the evolving landscape of clinical research and drug development. The team at Vial devotes the time to nurturing relationships with physicians, CMOs, project managers, and other key decision-makers, <a href="https://vial.com/blog/articles/importance-of-cro-sponsor-relationships">understanding that these relationships are invaluable for the success of any project</a>.</p> <h2 class="wp-block-heading">Benefits of Working with a Small CRO</h2> <p>A recent survey of emerging, small, mid-size, and large pharmaceutical and biotech companies in the US and Europe by Worldwide examined sponsor perceptions of CROs. The doubts raised by the respondents – sponsors from organizations of all sizes and geographies – appear to cause an anticipated shift in outsourcing away from large CROs. For instance, 70% of respondents worry that potential project team disruptions lead to delays. In contrast, <a href="https://vial.com/blog/articles/the-benefits-of-working-with-a-small-cro-speed-agility-and-working-with-a-true-partner">small CROs like Vial tend to have a much lower turnover than larger CROs</a>, providing the stability and continuity sponsors need.</p> <h3 class="wp-block-heading">Clinical trial complexity</h3> <p>Advances in <a href="https://vial.com/glossary/omics">omics</a>, genetics, <a href="https://vial.com/glossary/personalized-medicine">personalized medicine</a>, and artificial intelligence (AI) result in increasingly complex clinical trials. In turn, how sponsors work with CROs evolves into a new model requiring specialized services that “one-stop-shop” CROs cannot provide. Clinical trial designs are expected to become more complex, generating more data volume and diversity. According to Hardman <em>et al.,</em> rather than pharmacologists or clinicians, algorithms managed by data scientists are more likely to drive drug development.</p> <h3 class="wp-block-heading">Advanced technology</h3> <p>To support the data-demanding analyses needed for <a href="https://vial.com/glossary/precision-medicine">precision medicine</a>, high-throughput-omics technologies enable the retrieval of comprehensive biological data, while advanced data processing capabilities enable the data analytics required. Small, next-generation, technology-first CROs, like <a href="https://vial.com/cro/">Vial</a>, are poised to adopt the latest technologies, identify opportunities for application, and provide insights on how the tech may be deployed to improve clinical trial performance.</p> <h2 class="wp-block-heading">Cost and Time management</h2> <p>The Worldwide survey respondents revealed their preference for more agile CROs with a good balance of cost and value, amongst others. As Vial has invested heavily in technology and managed best-in-class research sites, it has a breadth of experience that matches or exceeds that of sponsors.</p> <h3 class="wp-block-heading">Site activation</h3> <p>Vial has a massively scalable clinical trial infrastructure and a fully automated end-to-end tech platform that powers reimagined trials. The intuitive technology platform integrates trial onboarding, patient <a href="https://vial.com/glossary/enrollment">enrollment</a>, site communication, and data collection into a connected system for efficiency. A boon for both cost and time management, Vial’s onboarding platform seamlessly allows sites to start a trial in 30 days or less. Worldwide does not explicitly state a 30-day site activation offering.</p> <h3 class="wp-block-heading">Fixed fee pricing structure</h3> <p>Worldwide service contracts are subject to change orders, whereas Vial offers a fixed-fee pricing model that applies to all contracts. A <a href="https://vial.com/blog/articles/vial-vs-top-4-small-cros">fixed-fee pricing model helps sponsors stay on budget</a> by avoiding costly unanticipated change orders.</p> <h2 class="wp-block-heading">Reimagining Clinical Trials</h2> <p>Vial’s modern, intuitive technology platform integrates trial onboarding, patient enrollment, site communication, and data collection processes into one connected system for efficiency. Vial is building towards a more efficient future for clinical trials. By deploying technology at every step, we are driving efficiencies in speed and cost savings for innovative biotech companies of all sizes. <a href="https://vial.com/contact-us">Let’s connect</a> to explore how to work together on your next trial.</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/worldwide-clinical-trials-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> </channel></rss> If you would like to create a banner that links to this page (i.e. this validation result), do the following:
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