Legislation Would Elevate Patient Experience in Drug Approval Process

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U.S. Senators Roger Wicker, R-Miss., and Amy Klobuchar, D-Minn., today reintroduced the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act. This legislation would provide patients and advocates the ability to play a larger role in the Food and Drug Administration’s (FDA) benefit-risk framework for drug approval.

“Millions of Americans living with rare diseases are waiting for groundbreaking drugs that could change their lives,” Wicker said. “The BENEFIT Act would put patients first – giving them a larger role in the drug approval process as researchers work to find more breakthroughs.”

“For our health care system to better serve patients, it’s crucial that we consider their experience in the Food and Drug Administration’s drug approval process,” Klobuchar said. “Building on the progress of the 21st Century Cures Act, our legislation will help prioritize the needs of patients and ensure they are at the heart of health policy.”

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 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 to ensure that the perspective of patients is considered by FDA reviewers evaluating candidate drugs and other medical products. As a result of updates to the Prescription Drug User Fee Act and 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 patient perspectives.

The BENEFIT Act is also supported by Parent Project Muscular Dystrophy.

“The field of patient engagement has been critical in advancing therapies to treat Duchenne and other devastating diseases. The patient has gone from being treated as the end user to a key partner in drug development who provides valuable insights about what matters most to them, what they want in a therapy and what level of risk they are willing to accept. Four years ago, Senators Wicker and Klobuchar wrote the law that requires FDA to report on how patient engagement data is used in the review of approved therapies and to provide guidance to inform the development of patient engagement tools. The BENEFIT Act will build upon this law by enshrining valuable patient engagement information within the all-important FDA benefit-risk framework. I am so grateful to Senators Wicker and Klobuchar, two champions of our community and of patient-focused drug development.  PPMD looks forward to another of their important bills becoming law,” said PPMD Founder and President Pat Furlong.

Wicker and Klobuchar first introduced the BENEFIT Act during the 115th Congress.

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Release from Senator Roger Wicker.