USC White Paper Recommends FDA Reforms to Accelerate Innovation and Broaden Patient Access

Rapid advancements in medical science are fueling the development of innovative treatments, including personalized medicine, gene therapies, and advanced diagnostics. To leverage these technological breakthroughs fully, significant reforms are needed within the Food and Drug Administration (FDA). A new white paper issued by the USC Schaeffer Center for Health Policy & Economics outlines strategic recommendations for modernizing FDA policies to foster innovation, streamline drug development, and improve patient access.

The white paper emphasizes the importance of the FDA providing clear and consistent guidance to drug manufacturers, especially regarding emerging technologies like real-world evidence and innovative clinical trial designs. These approaches could reduce the lengthy and costly process currently involved in bringing new medicines to market, ultimately lowering prices and expanding access without compromising safety and efficacy.

A critical focus of the recommendations is on supporting modernized clinical trial methods. The report advocates for increased early engagement between regulators and developers using unconventional trial designs to clarify acceptance criteria and build confidence in novel methods. This includes the integration of real-world data into the FDA’s accelerated approval pathways, which can expedite access to treatments for serious conditions while ensuring post-approval verification.

Additionally, the white paper highlights the potential of artificial intelligence (AI) and machine learning to enhance the efficiency of drug review processes. The FDA’s recent deployment of AI tools is a promising step, but the agency must establish robust principles for responsible use, transparency, and human oversight of AI-driven decisions. Strategic expansion of these technologies can help identify data quality issues and perform complex analyses more effectively.

Furthermore, the report stresses the importance of incorporating patient perspectives into regulatory evaluations. Engaging patient communities can improve trial design, make regulatory decisions more patient-centered, especially for rare diseases affecting millions, and foster greater trust among stakeholders. Formalizing these approaches can lead to more consistent policies and better integration of patient-preference data into coverage and approval processes.

Overall, the white paper calls for a comprehensive overhaul of FDA processes to align with cutting-edge science and technology, ensuring faster, safer, and more accessible medicines for all patients. Implementing these reforms could significantly accelerate medical innovation and better meet the health needs of the population.

Source: https://medicalxpress.com/news/2025-09-white-paper-outlines-fda-reforms.html