Remember that hot June day in 2015 when hundreds of Duchenne community members rallied together at the U.S. Capitol for PPMD’s EndDuchenne rally? That same day, our longtime Congressional champions Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced the Patient Focused Impact Assessment Act (PFIA, S. 1597) — and our 200+ PPMD advocates from across the nation met with their Senators to urge bill support, passage, and ultimate inclusion within the 21st Century Cures package.

Thanks to that amazing momentum and the grassroots outreach that followed over the coming months, PFIA passed the Senate on March 9, 2016 and was later incorporated as a provision within the 21st Century Cures Act — becoming the language for Sec 3001 of the 21 Century Cures bill.

PFIA was about ensuring that the patient-experience data being generated by our community — and all patient communities — is formally incorporated into FDA’s product review. The bill ensured that there would be much-needed transparency around how the FDA is utilizing any data submitted within relevant reviews, and provides a critical feedback loop to the data sources (patient communities, industry, researchers, clinicians, and academics).

New Section Added to FDA Drug Reviews

Yesterday, we learned that a ‘Patient Experience Data’ section has now officially been added to US FDA Drug Reviews — and that implementation has begun through the FDA’s recent review and approval of Genetech’s hemophilia A treatment, Hemlibra. We are thrilled to see this critical level transparency around patient-experience data within product review put in place by FDA.

PPMD was grateful for a broad coalition of more than 60 rare disease organizations who joined PPMD in our efforts around PFIA including the following Duchenne community partners:

• Charley’s Fund
• Coalition Duchenne
• Foundation to Eradicate Duchenne (FED)
• Hope for Javier
• JB’s Keys to DMD
• John Owen’s Adventure
• Little Hercules Foundation
• Muscular Dystrophy Association (MDA)
• Pietro’s Fight
• Suneel’s Light
• Team Saij
• Two Smiles One Hope Foundation

We were also grateful to the EveryLife Foundation, Genetic Alliance, Global Genes, the National Health Council, and NORD for lending their coalition support to our PFIA efforts.

The implementation of this provision reinforces that the Duchenne community’s leadership in both legislative and regulatory initiatives serves ALL patient communities.

Additional Legislation Moving Forward

As a follow up to our efforts around PFIA, PPMD also has been leading a legislative effort called the BENEFIT Act that passed in the Senate (S. 1052) and is now moving to the House. The BENEFIT Act codifies the inclusion of patient experience data within the agency’s benefit/risk framework by amending the Food, Drug, Cosmetic Act (FDCA) to include PFDD and related data – including info developed by a product sponsor or a 3rd party such as an advocacy organization or academic institution – to be considered as a part of the Benefit/Risk Framework. Watch for updates on BENEFIT in the coming weeks as we begin our work in the House of Representatives.

As our community has long understood, transforming our lived experiences into data that regulators can utilize is critical. But we also realized that there was no formal mechanism for incorporating this patient-experience data into the regulatory reviews and decisions that are so critical to us. So — we carved that pathway.

Advocacy matters. Relationships with our elected representatives are critical to ensure that we can continue to ensure that our Duchenne community’s voice is heard in Washington and beyond. Today’s news is a terrific example of the tremendous impact we continue to make together as a unified Duchenne community — please join us for PPMD’s Annual Advocacy Conference March 4-6.

2018 is shaping up to be another banner year on the Hill for PPMD and our Duchenne community!