Improvements in FDA’s benefit-risk analysis and communication began with the fifth reauthorization of the Prescription Drug User Fee Act (PDUFA) V of 2013 (benefit-risk plan, communication plan) (PDUFA V Performance Goals and Procedures), became a priority with PDUFA VI of 2017 (PDUFA VI Performance Goals and Procedures), and expanded with the 21st Century Cures Act (Cures Act) of 2016, which calls for a patient-focused drug development process. Implementation of these Acts can improve FDA’s communication of its drug regulatory decision thought process and incorporate patients’ preferences in drug development and regulatory review.
The FDA Commissioner announced an update to the implementation plan Benefit-Risk Assessment in Drug Regulatory Decision-Making on March 30, 2018, which describes steps the FDA has taken to implement the Acts mentioned above to incorporate enhanced patient benefit-risk analysis. Part of the plan includes improvements to the FDA’s Benefit Risk Framework and the proposed release of new Guidance describing how patient preferences can be utilized for benefit-risk assessment by June 2020.
The purpose of the Benefit Risk Framework is:
- Enhanced communication of the reasoning behind FDA’s regulatory decisions and how it relates to patient wellbeing and public health.
- Additionally, stakeholders may better understand FDA’s decision-making process and incorporate this into their drug development plans.
- Allows for patients to more effectively understand and communicate with the FDA.
- Improves FDA’s ability to assess the benefit-risk ratio and incorporate patient preferences.
Reasons to incorporate patient preferences:
- The benefit-risk assessment conducted by the FDA can include both information submitted during clinical trials and patient preference.
- The “clinical context” of the disease condition can be considered, which includes disease severity and if medical needs are met.
- The results of FDA sponsored patient-focused drug development meetings authorized after the 2012 reauthorization of PDUFA determined that:
- Patients are better equipped to provide benefit-risk assessments.
- Patients may experience different risk-benefit profiles for treatments even for the same disease based on other factors such as age, expectations, support systems, emotional health, etc.
- The FDA is developing guidance to allow for the direct collection of patient experience data to guide regulatory decision making. Plan for Issuance of Patient‐Focused Drug Development Guidance from May 2017 describes the FDA’s plan for development of a guidance under the Cures Act.
Join us in “FDA's Patient-Focused Drug Development – Part 2” to learn more about FDA’s Plan for Issuance of Patient‐Focused Drug Development Guidance.
- The Clinical Pathways Team
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