Former FDA Leaders Call for Policy Overhaul to Foster Medical Innovation
Los Angeles, Saturday, 27 September 2025.
Ex-FDA commissioners argue for regulatory updates to accelerate treatment approval for rare diseases, highlighting the importance of new data tools and modern clinical trial methods.
The Push for Regulatory Changes
At a recent conference hosted by the Schaeffer Center on FDA reform, former FDA commissioners emphasized the urgent need for regulatory changes to keep pace with rapid advancements in medical innovation. They highlighted the potential of new data tools that can expedite the development and market entry of treatments, particularly for rare diseases. The discussions underscored how evolving clinical trial methodologies necessitate updated regulatory frameworks to ensure patient safety while fostering innovation in healthcare delivery [1].
The Role of Real-World Evidence
The 21st Century Cures Act of 2016, which aimed to accelerate medical product development, has set the stage for integrating real-world data (RWD) and real-world evidence (RWE) into regulatory processes. This integration is crucial for developing treatments more efficiently. The Prescription Drug User Fee Act (PDUFA VII) and the Medical Device User Fee Act (MDUFA V) have outlined mandates to enhance the use of RWD/E in regulatory submissions, supporting the FDA’s efforts to modernize its frameworks [5].
Competition and Global Standards
Former FDA Commissioner Scott Gottlieb highlighted the importance of modernizing the regulatory framework to counter rising biotech competition from China, which benefits from lower labor costs and looser patient safety standards. He advocates for streamlining early development phases to provide American developers with a competitive edge. This strategy is crucial as the U.S. aims to maintain its leadership in rare disease treatments and innovation [1].
Future Directions and Industry Impact
The ongoing discussions and planned reforms are expected to transform the healthcare landscape significantly. By leveraging AI and machine learning in drug development, the U.S. can enhance its competitive position globally. Furthermore, the MedTech Conference in October 2025 will provide a platform for industry leaders to explore patient-centered solutions and the latest innovations in healthcare technology, further driving the transformation of healthcare systems [2][4].