Learn how the inclusion of real-world data (RWD) and real-world evidence (RWE) from natural history studies can improve your integrated evidence strategy to help expedite regulatory approval and bring your product to market more efficiently to the patients who need it most.
The New Regulatory Landscape: FDA's 2023 Draft Guidance
The U.S. Food and Drug Administration (FDA) continues to evolve its approach to evidence generation, expanding the types of evidence it will accept to support regulatory decisions about drug effectiveness (1). This white paper provides critical insights into the expanded types of confirmatory evidence and how to integrate them into your strategy, helping to expedite regulatory approval and fulfill unmet medical needs.
Integrated Evidence Leadership
Hear from UBC’s Chief Scientific Officer, Annette Stemhagen, DRPH, FISPE, Senior Director, Epidemiology, Irene Cosmatos, and Executive Director, Evidence Development Study Solutions, Judy Lytle, PhD, MBEE, PMP on how recent FDA draft guidance from September 2023 paves the way for opportunity to capitalize on advancements in RWD and RWE to support the evaluation of drug effectiveness.
Your Guide to the Expanded Confirmatory Evidence
Explore insights and perspectives on:
- 2023 FDA Draft Guidance for the industry: Learn about the new guidance that expands on the types of confirmatory evidence the FDA will accept to substantiate a single adequate and well-controlled clinical investigation. The guidance emphasizes that both the quality and quantity of confirmatory evidence are critical considerations (2).
- Confirmatory evidence from natural history: These studies are crucial for informing indication prioritization, clinical development design, and establishing the generalizability of drug efficacy. The FDA has cited natural history studies as a potential source of confirmatory evidence, particularly for rare disease drugs where a second trial is difficult (3).
- Confirmatory evidence from RWD sources: Understand the necessity of using fit-for-purpose data and engaging in early discussions with regulatory bodies when incorporating RWD into your program.
- Related case study examples: Read about the registration of Blincyto® (blinatumomab), where RWE supported product registration by enabling comparative analyses of effectiveness to be conducted in a single-arm clinical trial (4).
Don’t Delay Approval
Partner with UBC experts to plan a valuable evidence generation strategy that results in the real-world evidence needed to help prove drug effectiveness and ensure regulatory compliance.
References
- US FDA. Guidance Document: Demonstrating Substantial Evidence of Effectiveness with One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence. Accessed 12/29/2023. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-one-adequate-and-well-controlled-clinical
- US FDA. Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products. Accessed 04/11/2024. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-human-drug-and-biological-products
- RAPS. FDA officials stress natural history studies in supporting rare disease drug development. Accessed 04/11/2024. https://www.raps.org/news-and-articles/news-articles/2023/10/fda-officials-stress-natural-history-studies-in-su
- Gökbuget N, Kelsh M, Chia V, Advani A, Bassan R, Dombret H, Doubek M, Fielding AK, Giebel S, Haddad V, Hoelzer D, Holland C, Ifrah N, Katz A, Maniar T, Martinelli G, Morgades M, O’Brien S, Ribera JM, Rowe JM, Stein A, Topp M, Wadleigh M, Kantarjian H. Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia. Blood Cancer J. 2016 Sep 23;6(9):e473. doi: 10.1038/bcj.2016.84. PMID: 27662202; PMCID: PMC5056974.
