WASHINGTON, D.C. – U.S. Senators Amy Klobuchar (D-MN) and Roger Wicker (R-MS) have introduced bipartisan legislation aimed at providing patients the ability to play a larger role in the Food and Drug Administration’s (FDA) benefit-risk framework for drug approvals. The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act builds on the foundation of the Klobuchar and Wicker’s bipartisan Patient-Focused Impact Assessment Act, which was signed into law in December.
“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.
The BENEFIT Act would amend 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 FDA’s risk-benefit assessment. This action would incorporate information about patient experience developed by groups like patient advocacy organizations or academic institutions during FDA approval, sending an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process.
As co-chair of the Rare Disease Congressional Caucus, Klobuchar has worked to make progress on rare disease research and funding. Congress has made considerable progress in recent years to ensure that the perspective of patients is considered by FDA reviewers evaluating candidate drugs and other medical products. In 2014, Klobuchar and Wicker’s legislation to help improve the lives of patients with muscular dystrophy was signed into law by the President. The Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD CARE) Amendments of 2014 supports medical research and policies to improve treatments and quality of life for muscular dystrophy patients. 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 now 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.