Patient Experience Data (PED) in 2019-2023 US FDA NME Drug Approvals: Analysis and Recommendations

Area of Science:

• Regulatory Science
• Patient-Centered Drug Development
• Health Policy

Background:

• U.S. Congress mandated FDA consideration of patient perspectives in drug reviews since 2012.
• Patient Experience Data (PED) is crucial for regulatory decision-making.
• This study evaluates the integration and impact of PED in FDA approval processes.

Purpose of the Study:

• To assess the implementation and impact of patient-focused legislation on FDA drug reviews.
• To analyze the inclusion of Patient Experience Data (PED) in FDA approval documents from 2019-2023.
• To build upon previous analyses by comprehensively evaluating sponsor and FDA use of PED.

Main Methods:

• Reviewed 277 drug and biologic approval documents from FDA's CDER and CBER.
• Assessed the submission and reporting of PED in approval documents and product labels.
• Analyzed PED within PED Tables, benefit-risk frameworks, and product labels for each indication.

Main Results:

• 252 out of 277 (91%) approval documents included a PED Table; 179 (65%) were complete.
• PED was incorporated into the benefit-risk framework for 85 (30%) applications.
• PED was included in the product label for 75 (27%) applications.

Conclusions:

• Systematic collection and utilization of PED have been evolving for over a decade.
• Both FDA and sponsors are increasingly incorporating patient perspectives into drug development and decision-making.
• Enhanced transparency is required to clarify how the FDA reviews and utilizes PED for regulatory decisions.