News Updates
- Benefits of Integrating Patient Engagement with Diversity, Equity, and Inclusion in New Medical Product Development: A Call for Action
- Drug Shortages in the Global South: A Proposed Parallel Tech and Reg Transfer Framework
- Fair Pay for Patient Engagement: Navigating the Evolving Landscape of Remuneration
- Industry’s Window to Express Interest in Africa Continental Product Evaluation Pilot Closes End of February 2024
- Accelerating Adoption of eLabeling in Singapore: One Company’s Journey
Ten-Year CDER Experience: Ensuring Patient Safety Part 2
Ten-Year Experience for the Center for Drug Evaluation and Research, Part 2 - FDA’s Role in Ensuring Patient Safety
Methods: To better define FDA’s role in the review of requests for expanded access, multiple queries of FDA’s Center for Drug Evaluation and Research (CDER) document tracking system were performed. The queries identified reasons for, and outcomes of, expanded access requests for investigational drugs that were either not allowed to proceed or denied over a ten-year time period. An in-depth review of a random sample of single-patient, non-emergency investigational new drug (IND) applications that were allowed to proceed was also conducted.
Results: Overall, 99.3% of the applications for almost 9000 expanded access of an investigational drug were allowed to proceed. There were 62 requests that were either denied (38 emergency INDs) or not allowed to proceed (24 non-emergency INDs). The most common reasons for denying emergency INDs was that the patient was stable on current therapy and that it was not deemed an emergency. The most common reasons for not allowing non-emergency expanded access INDs to proceed were incomplete application, unsafe dosing, demonstrated lack of efficacy for intended use, availability of adequate alternative therapies, and inadequate information provided in the application on which to base a decision. A review of a random sample of 150 single-patient, non-emergency INDs revealed that FDA recommended changes to dosing, safety monitoring, or informed consent in 11%.
Conclusions: FDA plays a significant role in the protection of patients who receive investigational drugs under expanded access. An extremely small percentage of applications received are not allowed to proceed; however, FDA provides significant input based on information that may not be available to treating physicians in order to ensure patient safety under the applications that do proceed.