Simon Dagenais’ Post

NIH and FDA Seek Comment on Draft Glossary of Clinical Research Terms Related to Innovative Clinical Trial Design The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) have released a request for information including a draft glossary of clinical research terms related to innovative clinical trial design, including studies using real world data to generate real world evidence. The draft glossary is intended to facilitate communication within the clinical research community by helping establish a common vocabulary to more uniformly characterize clinical research. Please see the draft glossary for more information. NIH and FDA will accept comments on the draft glossary until June 24, 2024. Questions about this draft glossary can be sent to [email protected]. #davidlim #regulatorydoctor #fda #nih https://lnkd.in/eNMVivPq

Proud to share we are #7 in the nation for research funding based on a new report from American Association of Colleges of Pharmacy (AACP)! Our faculty secured more than $34 million in grants from the Bill & Melinda Gates Foundation, The National Institutes of Health (NIH) and other organizations.   “This impressive amount of grant funding, secured by a relatively small number of faculty, demonstrates the preeminence of our pharmaceutical scientists who are each experts in their respective fields,” said Dean Brookie Best, Pharm.D., M.A.S. “Our diversity of funding sources also shows our ability to attract competitive funding and solve numerous urgent medical challenges through pharmaceutical science.”    Issued annually, the AACP report is a comprehensive list of total extramural-funded research awards received by U.S. colleges or schools of pharmacy based on public databases and direct collaboration with schools to ensure data accuracy. “Our wide variety of funded projects each target a distinct and pressing area of human health,” said Elizabeth Winzeler, Ph.D., associate dean for research and innovation at the pharmacy school. “These projects will help fight malaria, protect the health of breastfeeding infants, uncover marine-based medicines and discover new drug-like compounds.”   Read the press release ➡ bit.ly/4bRQvL3 #Pharmacy #Research #PharmacySchool #AACP #UCSDPharmacy

Clinical research is an essential component of medical advancement, focusing on understanding health and disease to enhance healthcare practices. This exploration encompasses clinical trials, classified into interventional studies and observational studies. Interventional studies, or clinical trials, involve assigning participants interventions to evaluate their effects on health outcomes. These interventions can range from drugs to surgical procedures or preventive care. Clinical trials progress through four phases, ensuring safety and efficacy before widespread implementation. The International Clinical Trials Registry Platform ICTRP , a global initiative by WHO, facilitates comprehensive and accessible information on human clinical trials. ICTRP strives to enhance data accuracy, raise awareness about trial registration, and promote data utilization. This collaborative effort fosters transparency, benefiting not only researchers but also patients, families, and the broader healthcare community. by Yash Dhikale | Zumbar Pote | Santosh Ghuge | Shital B. Thakre | Dipali S. Shegar "Overview of Pharmacovigilance" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-8 | Issue-1 , February 2024, URL: https://lnkd.in/dMTE_tis Paper Url: https://lnkd.in/dvFjUS7C

Traditional clinical research can be slow and complex. Real-world evidence (RWE) has brought a fresh perspective, but its full potential remains untapped. Only 1% of Phase IV trials use RWE, and around 3% focus on drug-specific data. Enter Prospective Real-World Studies (PrwS), a game-changer. 🔷 What Are PrwS? - PrwS intentionally collect new, comprehensive data on top of existing real-world data (RWD). This includes detailed outcomes, patient-reported data, and more. They seamlessly fit into routine clinical practice, requiring minimal additional technology or training. 🔷 The Benefits - PrwS can revolutionize clinical research. For example, the Prospective Clinico-Genomic (PCG) Study achieved remarkable success in patient enrollment, addressing selection bias. 🔷 The Promise Ahead - PrwS studies offer biopharma companies a significant opportunity. By incorporating prospective real-world data, they can execute post-marketing studies efficiently and enhance RCT control arms at a fraction of the cost without compromising data quality. With investments in technology and infrastructure, PrwS studies are poised to reshape clinical research. #ClinicalResearch #RealWorldEvidence #ProspectiveStudies #HealthcareInnovation #Biopharma

Traditional clinical research can be slow and complex. Real-world evidence (RWE) has brought a fresh perspective, but its full potential remains untapped. Only 1% of Phase IV trials use RWE, and around 3% focus on drug-specific data. Enter Prospective Real-World Studies (PrwS), a game-changer. 🔷 What Are PrwS? - PrwS intentionally collect new, comprehensive data on top of existing real-world data (RWD). This includes detailed outcomes, patient-reported data, and more. They seamlessly fit into routine clinical practice, requiring minimal additional technology or training. 🔷 The Benefits - PrwS can revolutionize clinical research. For example, the Prospective Clinico-Genomic (PCG) Study achieved remarkable success in patient enrollment, addressing selection bias. 🔷 The Promise Ahead - PrwS studies offer biopharma companies a significant opportunity. By incorporating prospective real-world data, they can execute post-marketing studies efficiently and enhance RCT control arms at a fraction of the cost without compromising data quality. With investments in technology and infrastructure, PrwS studies are poised to reshape clinical research. #ClinicalResearch #RealWorldEvidence #ProspectiveStudies #HealthcareInnovation #Biopharma

Yesterday it was the start of the FAST hub RARE-NL, which will focus on treatments for rare diseases, and drug repurposing, i.e. developing existing medicines for new indications. I am happy that I was able to contribute to the meeting yesterday by moderating one of the break-out sessions, and present a summary of the drug repurposing break-out sessions together with Saskia N. de Wildt and Saco de Visser. Regarding drug repurposing, it is also my pleasure to highlight our recent paper in Frontiers in Medicine on international initiatives to advance drug repurposing with involvement of regulators. We identified 10 initiatives, from which we could interview candidates from 8 initiatives to obtain insight in how repurposing can be advanced from a regulatory perspective. Interested to learn more? Read the results here: https://lnkd.in/eNKzw72m Congratulations to Eva Spin, who performed this study during her Master's in Pharmacy, and my UU-colleague Aukje Mantel-Teeuwisse. Lastly, great appreciation for the invaluable input from our FDA-colleagues Heather Stone, Maya Younoszai, Smith Heavner, PhD, RN. Future Affordable Sustainable Therapies, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University #drugrepurposing #regulatoryscience

💡 ❓ Insights for Clinical Research in Low/Middle Income Countries A big thank you to Nick Medhurst for his article on "Enabling 'Good' Clinical Research In LMICs" with The Good Clinical Trials Collaborative. 🔦 This piece sheds light on the need for high-quality clinical trials in low- and middle-income countries (LMICs) and the challenges faced in these settings. 🔑 The key takeaway is the importance of adhering to the five principles of Good RCTs to ensure scientifically sound, ethical, collaborative, context-appropriate, and efficiently managed clinical trials. This approach not only enhances trial quality but also ensures equitable access to essential interventions. 🔗 📖 Read the full article here for a deeper understanding: https://lnkd.in/eyXXffP5 #clinicalresearch #LMICs #goodclinicalpractice #quality #globalhealth #biotech #pharma

Moving beyond randomised controlled trials 🔍 The randomised controlled trial (RCT) is widely regarded as the “gold standard” of clinical research. Praised for its scientific rigour and statistical robustness, the RCT facilitates unbiased evaluation of treatment efficacy and safety. RCTs are, however, not always feasible, especially when the target patient population is hard to come by. With 30% of global drug development pipelines targeting rare diseases, the need for novel methods is pivotal. One such method is the external control arm, a virtual alternative to the traditional concurrent control group. This method has become increasingly popular due to advancements in data science & technology and growing regulatory acceptance. In this article, we unpack the promise of external control arms, covering recent #FDA guidance on the design & conduct of externally controlled trials as well as two recent biopharma success stories. 👉 Keep on reading: https://lnkd.in/dyJJ2ht2 #clinicalresearch #clinicaltrials #ECA #RCT 

Our latest blog post explores the indispensable role of Health Economics and Outcomes Research (HEOR) consultancies in guiding pharmaceutical, biotechnology, and medical device companies through the intricate processes of product development, regulatory approval, and market access. As the healthcare industry evolves to become more outcomes-focused and cost-conscious, HEOR consultancies are more critical than ever. Highlights from the Post: - Strategic Development: From the early stages of product development, HEOR consultancies integrate economic and clinical data to build compelling value propositions that resonate with regulators and payers. - Evidence Generation: HEOR experts employ sophisticated methodologies to demonstrate the economic and health benefits of new technologies, supporting key decisions from pricing strategies to reimbursement negotiations. - Stakeholder Engagement: Effective communication with stakeholders, including payers, healthcare providers, and patient groups, is facilitated to align product benefits with market needs and expectations. Learn how HEOR consultancies not only support compliance and facilitate market access but also ensure that new healthcare innovations deliver real value, improving patient outcomes and enhancing system efficiencies. Dive into the full article for a comprehensive look at how HEOR consultancies drive success in the dynamic landscape of global healthcare. https://lnkd.in/eNXDqzQE #HEOR #reimbursement #costeffectiveness #pharmaceuticals #HTA #marketacces #HealthEconomics 

Today, the FDA announced four U01 grant award recipients for demonstration projects to examine the use of real-world data (RWD) to generate real-world evidence (RWE) in regulatory decision making. The awards and grantees are: •            Methods to Improve Efficiency and Robustness of Clinical Trials Using Information from Real-World Data with Hidden Bias – Duke University and North Carolina State University •            Generating Reproducible Real-World Evidence with Multi-Source Data to Capture Unstructured Clinical Endpoints for Chronic Diseases – Harvard-MIT Center for Regulatory Science and Harvard Medical School •            Real-World Data to Generate Real-World Evidence in Regulatory Decision-Making – ECOG-ACRIN Medical Research Foundation •            Development of Novel Methods to Enable Robust Comparison of Real-World Progression Free Survival (rwPFS) and Clinical Trial PFS in Multiple Myeloma  – Janssen Research & Development, LLC. For additional information about the awards, visit the webpage https://lnkd.in/eSTyje_j