Wicker, Klobuchar, Matsui, Wenstrup Reintroduce BENEFIT Act

Legislation Would Elevate Patient Experience in Drug Approval Process

February 27, 2023

WASHINGTON – U.S. Senators Roger Wicker, R-Miss., and Amy Klobuchar, D-Minn., recently reintroduced the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act. Representatives Doris Matsui, D-Calif., and Brad Wenstrup, D.P.M., R-Ohio, have introduced companion legislation in the House of Representatives. The 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.

“During this Rare Disease Month, Congress has an opportunity to change the lives of millions of Americans who are waiting for groundbreaking drugs to be approved,” Wicker said. “Passing 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 living with a rare disease and seeking an approved treatment, we must ensure that the Food and Drug Administration’s drug approval process consistently factors in patient experience data,” said Klobuchar. “Our bipartisan legislation will give patients and their families a clear seat at the table when the FDA reviews potential new treatments. As the co-chair of the Rare Disease Congressional Caucus, I’ll keep working to prioritize the needs of rare disease patients and their loved ones.”

“Access to the best medical treatments is crucial for patients living with rare and ultra-rare diseases, and that is why we must prioritize the work to close the innovation gap for rare disease therapies," said Matsui. “By elevating patient voices and allowing them to share their experiences in the drug development process, we gain key partners in assessing the benefits and risks of new drugs and biologics. I am proud to reintroduce this important bill that will leverage valuable patient-focused drug development data in the FDA's review process.”

“As a physician, I know how important it is to listen to patients and make sure their voices are heard. Their perspectives and needs are key to developing the best new drugs and therapies,” said Wenstrup. “I’m supporting the reintroduction of this bipartisan, patient-focused legislation to make sure that patients and advocates are considered during the FDA’s review process for new innovative breakthroughs and cures.”

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. But while much progress has been made, some significant gaps remain. One gap is the lack of a requirement in law that the FDA include patient experience or patient-focused drug development (PFDD) data as part of its risk-benefit framework.

The BENEFIT Act would amend the Food, Drug and Cosmetic Act to ensure that the patient experience, 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 will enhance transparency and accountability, sending an important signal to all stakeholders that patient experience data will be incorporated into the agency’s review process, encouraging such entities to continue developing and refining scientifically rigorous and meaningful tools and data.

The BENEFIT Act is supported by many organizations, including Parent Project Muscular Dystrophy, EveryLife Foundation for Rare Diseases, Cure SMA, ALS Association, AliveAndKickn, Alpha-1 Foundation, Alport Syndrome Foundation, American Brain Coalition, American Kidney Fund, Ara Parseghian Medical Research Fund, Barth Syndrome Foundation, Beyond Celiac, Coalition Duchenne, Congenital Hyperinsulinism International, CSNK2A1 Foundation, Cure CMD, Cure HHT, Cure Sanfilippo Foundation, CureDuchenne, CureSHANK, Dravet Syndrome Foundation, FND Hope, FORCE: Facing Our Risk of Cancer Empowered, Foundation for Angelman Syndrome Therapeutics (FAST), Foundation for Prader-Willi Research, Genetic Alliance, Hermansky-Pudlak Syndrome Network, Hope For Marian, International Pemphigus Pemphigoid Foundation, International WAGR Syndrome Association, IWSA, Jett Foundation, Kindness Over Muscular Dystrophy, Klippel-Trenaunay (K-T) Support Group, Little Hercules Foundation, Lupus Foundation of America, MLD Foundation, Mucolipidosis Type IV, National Ataxia Foundation, National Health Council, National Kidney Foundation, National MPS Society, National MS Society, NBIA Disorders Association, Organic Acidemia Association, Phelan-McDermid Syndrome Foundation, PXE International, RASopathies Network, RUNX1 Research Program, Ryan’s Quest, Sophie's Neighborhood, Stickler Involved People, Sudden Arrhythmia Death Syndromes (SADS) Foundation, Susan G. Komen, SYNGAP1 Foundation, The Global Foundation for Peroxisomal Disorders, TSC Alliance, United Mitochondrial Disease Foundation, Usher 1F Collaborative, Wiskott Aldrich Foundation, and Zack Heger Foundation.

“Parent Project Muscular Dystrophy (PPMD) commends Senators Wicker and Klobuchar and Representatives Matsui and Wenstrup for their bipartisan leadership to re-introduce of the BENEFIT Act, legislation that will help ensure the patient voice is heard in the FDA's review process,” said Pat Furlong, Founding President and CEO of PPMD. “Now over 5 years after the passage of the 21st Century Cures Act, the FDA, the patient community, and drug developers have spent significant time and effort generating scientifically rigorous, meaningful patient experience data. It is time for FDA to take the next step by providing some official feedback to the community about if and how such data is used when weighing the benefits and risks of a new drug.”

“The BENEFIT Act is critical to ensuring that we fully deliver on the promise of patient focused drug development by integrating patient considerations in the form of patient experience data into FDA’s signature decision making tool at the time of regulatory review. Our national rare disease community is grateful to our BENEFIT Act champions for ensuring that the lived experience of patients remains central in our regulatory processes,” said EveryLife Foundation for Rare Diseases.

“As the national organization that represents adults and children with a neuromuscular disease known as spinal muscular atrophy (SMA), Cure SMA is pleased to support the BENEFIT Act. Patient voice and perspective are critically important throughout the drug development process, and have been instrumental for the recent approvals of powerful new treatments for SMA,” said Kenneth Hobby, President of Cure SMA. “We applaud Senators Wicker and Klobuchar and Representatives Matsui and Wenstrup for their commitment to individuals with SMA and other rare diseases by requiring that the needs and experiences of patients are considered by the FDA during the drug development and evaluation process.”

“The ALS Association applauds Congress for working together to ensure the FDA hears from people living with ALS about their experiences and preferences as part of the FDA’s drug approval process,” said Melanie Lendnal, Senior Vice Presidents of Policy at the ALS Association. “The ALS Association’s ALS Focus survey solicits feedback from people living with ALS and their caregivers on the care and treatments they receive. This information is critically important and must be considered in FDA’S drug approval risk-benefit analysis. People living with ALS do not have time to wait, their experience with new drug therapies should be highly considered by the FDA.”

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

See the full bill text here.