Wicker, Klobuchar Aim to Increase Patients’ Role in FDA Drug Approval Benefit-Risk Framework
WASHINGTON – U.S. Senators Roger Wicker, R-Miss., and Amy Klobuchar, D-Minn., have introduced legislation aimed at providing patients and advocates the ability to play a larger role in the Food and Drug Administration’s (FDA) benefit-risk framework for drug approval. The “Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act,” builds on the foundation of the Wicker-Klobuchar “Patient-Focused Impact Assessment Act,” which became law in December.
“The unique viewpoint of patients, including those with rare diseases like Duchenne muscular dystrophy, should be taken into account when the FDA assesses the benefits and risks of drug therapies,” Wicker said. “Our ultimate goal with this legislation is to ensure that all voices are heard in the search for breakthroughs or cures.”
“The 21st Century Cures Act marked an important milestone for incorporating the patient experience into the drug approval process. But there’s still more we can do. This bipartisan legislation will strengthen the role of patients, especially those with rare diseases, when the Food and Drug Administration is assessing the benefits and risks of new treatments,” Klobuchar said.
Specifically, the legislation would address a significant requirement gap by amending the “Food, Drug and Cosmetic Act” to ensure that the patient experience, patient-focused drug development (PFDD), and related data be considered as part of the risk-benefit assessment. This would include information developed by a product sponsor or a third party, such as a patient advocacy organization or academic institution. This action would send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data.
Congress has made considerable progress over the past five years to ensure that the perspective of patients is considered by FDA reviewers evaluating candidate drugs and other medical products. As a result of the last “Prescription Drug User Fee Act” updates from 2012 and, more recently, several provisions in the “21st Century Cures Act,” the FDA has a number of programs and policies in place to evaluate the benefits and risks of potential therapies and to gather and assess the patient perspectives.