Parent Project Muscular Dystrophy (PPMD) applauds this week’s reintroduction of legislation called the BENEFIT Act, bipartisan legislation that will strengthen current law requiring the Food and Drug Administration consider the voice of patients and caregivers when reviewing new drug applications.

The Better Empowerment Now to Enhance Framework and Improve Treatments or BENEFIT Act was re-introduced this week by Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN). It is nearly identical to an earlier iteration of the bill that passed the United States Senate by unanimous approval in August 2017. PPMD is working with Congressional champions in the House to introduce a companion bill this year.

The BENEFIT Act amends food and drug law to ensure that invaluable patient perspectives – such as input from patient preference studies, patient reported outcomes, and other types of patient experience data – are included as part of the FDA risk-benefit review of candidate new drugs.

BENEFIT builds upon earlier legislation Senators Wicker and Klobuchar led and championed, originally drafted by PPMD and included in the 21st Century Cures Act called The Patient Focused Impact Assessment Act (The PFIA Act). Thanks to The PFIA Act, the FDA is now required to report the use of patient engagement in reviews of any FDA-approved therapies.

PPMD and the Duchenne community have been leaders in the field of patient engagement over the past decade. This work includes developing the first-ever Duchenne caregiver preference study, four additional studies on both caregiver and patient preferences, and the first-ever patient initiated draft guidance for drug development. The latter was used by FDA to develop draft and final guidance to industry on Duchenne drug development.

Advocates head to  Washington D.C. next week for the 2020 Advocacy Conference to advocate for Congressional support of the legislation as well as funding for agencies that impact Duchenne. PPMD will be issuing action alerts for those who are unable to attend the conference in person.

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