Real-World Evidence, Public Participation, and the FDA

Area of Science:

• Pharmaceutical regulation
• Public health policy
• Evidence-based medicine

Background:

• Concerns exist regarding potential weakening of the Food and Drug Administration's (FDA) evidentiary standards.
• Two key events in late 2016, eteplirsen's approval and the 21st Century Cures Act, fueled these concerns.
• The political climate has amplified discussions about the FDA's future regulatory approach.

Purpose of the Study:

• To examine the implications of recent developments on FDA evidentiary standards.
• To explore the role of patient narratives and real-world evidence in FDA decision-making.
• To propose a model for public participation in FDA processes.

Main Methods:

• Analysis of the eteplirsen approval process, including FDA staff and advisory committee recommendations.
• Review of the 21st Century Cures Act provisions concerning real-world evidence.
• Examination of the influence of patient advocacy and industry-friendly policies.

Main Results:

• The approval of eteplirsen, despite internal recommendations, highlights the influence of external factors.
• The 21st Century Cures Act promotes the use of real-world evidence, potentially altering traditional trial data reliance.
• Patient narratives played a significant role in the eteplirsen decision, raising questions about their broader impact.

Conclusions:

• Recent events present critical questions about the future of evidence used by the FDA.
• The integration of real-world evidence and patient perspectives necessitates careful consideration of regulatory standards.
• A structured approach to public participation is crucial for maintaining public trust and ensuring robust FDA decision-making.