The BENEFIT Act would require FDA to include in the risk-benefit assessment framework of a new drug application how patient experience data was considered in the review process. The Duchenne community knows how important information from patients and caregivers is to clinical research and that this information must inform FDA decisions.
Please fill out the action alert below to encourage your members of Congress to cosponsor the BENEFIT Act. More bipartisan support of the legislation will help increase the chances of the bill advancing!
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Cosponsor the BENEFIT Act (H.R.4472/S.373)
Dear [Decision Maker],
While much progress has been made in Patient-Focused Drug Development, some significant gaps remain. One such gap is the lack of any requirement in law today that the FDA includes as part of its risk-benefit framework any patient experience or patient-focused drug development (PFDD) data. This means that the agency's signature tool for evaluating risk-benefit does not have to data from the patient perspective that could be critical to informing the agency's evaluation and, ultimately, the decision on whether or not to approve a product. To address this gap, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) have introduced S.373 the Better Empowerment Now to Enhance Framework and Improve Treatments or the BENEFIT Act. A companion bill has been introduced by Representatives Matsui and Wenstrup in the House (H.R.4472). This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution be considered as part of the risk-benefit assessment. This action will 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. The BENEFIT Act will continue this evolution by filling a sizeable gap by ensuring such data is fully considered as part of the FDA's risk-benefit assessment for any new products. Advance patient engagement by cosponsoring the BENEFIT Act today.
Sincerely,[Your Name] [Your Address] [City, State ZIP][Your Email]