This engaging discussion from Critical Path Institute’s (C-Path) Global Impact Conference (CGIC) 2025, focuses on the future of regulatory science, drug development, and the role of collaboration between public and private sectors to accelerate biomedical innovation. This session, led by Consilium Salmonson & Hemmings Partners and C-Path Board Members Tomas Salmonson and Robert Hemmings, C-Path Senior Scientific Director Smith Heavner, FDA Senior Advisor Marie Bradley, EMA Head of Regulatory Science and Academia Ralf Herold, and Senior Medical Director at Biogen, Angela Paradis, emphasizes the importance of speeding up drug development to benefit patients faster and at lower costs.
The conversation highlighted the need for better tools, frameworks, and guidance to facilitate the appropriate use of external control arms. Key points include the current regulatory uncertainty, the evolving nature of methodological standards, and the necessity of early engagement between sponsors and regulators to improve study designs and regulatory acceptability.
Key Insights
Limitations of RCTs and Need for Alternative Designs: RCTs, while the gold standard for demonstrating drug efficacy, are often infeasible in rare diseases or when long-term outcomes are needed.
Industry’s Need for Clear Regulatory Expectations: Industry representatives highlight that while external control arms are increasingly considered, uncertainty about regulatory acceptance and standards remains a key barrier.
Risk of Bias and Complexity in Using Real World Data: Regulators and statisticians emphasize that real world data sources differ substantially from clinical trial settings, with risks of confounding, selection bias, and differences in outcome ascertainment.
FDA’s Active Role in Guideline Development and Research: The FDA has issued guidance on external control arms and is actively funding research to address key challenges such as measurement error, index date selection, and hybrid trial designs.
EMA’s Emphasis on Collaborative Regulatory Science: EMA’s involvement in regulatory science research, including collaborations with academia, nonprofits, and industry, highlights the importance of precompetitive research to develop methods, standards, and tools for external control arms.
C-Path as a Facilitator for Toolkit Development: C-Path's role as a convener and facilitator is critical in bringing together stakeholders to develop practical toolkits that can standardize and improve the planning, conduct, and regulatory review of externally controlled studies.
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About Critical Path Institute
C-Path is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org.
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