Earlier this week, Aetion submitted a letter to FDA commenting on the Agency’s recent draft guidance document Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products; Draft Guidance for Industry.
The Draft Guidance reflects FDA’s commitment to fostering high quality analyses of real-world data (RWD) that can provide evidence for regulatory decisions across the product life cycle, including regulatory review of safety and effectiveness in the context of product approval decisions. Aetion shares this commitment and appreciates the substantial effort that FDA is putting into developing resources that will further it.
The Draft Guidance is a critical next step to advancing FDA’s Real-World Evidence (RWE) Framework and the use of high quality RWE in regulatory decision-making. Greater alignment on principles and best practices, and greater public visibility regarding FDA’s expectations, will enable sponsors and other researchers to approach the analysis of RWD with greater confidence that such efforts will lead to the generation of valuable evidence. Further, integrating transparent, auditable, reproducible, and scientifically valid RWE into regulatory decision-making will facilitate more efficient drug development and enhance understanding of product safety and effectiveness.
Aetion appreciated the opportunity to comment on the Draft Guidance. Key points in our comment letter include:
- Aetion shares and appreciates FDA’s commitment to scientific best practice, many principles of which are reflected in Aetion’s recently published “A Structured Process to Identify Fit-for-Purpose Study Design and Data to Generate Valid and Transparent Real-World Evidence for Regulatory Uses.”
- We recommended that FDA do more in both this Draft Guidance and the recent draft guidance on accelerated approval (“Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics”) to clarify the critical role that RWD and RWE are playing in the accelerated approval pathway.
- We asked FDA to include further discussion of summary-level external controls. The Draft Guidance focuses on patient-level external controls, but summary-level external controls have been commonly used in applications approved by the Agency. (See, for example, ZOLGENSMA and peruse Aetion’s Decision Alerts page to learn more.)
Please view our full comments here. Feel free to contact us with any questions regarding these comments or other issues related to RWE policy and development.