Earlier today, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) introduced legislation known as the Patient-Focused Impact Assessment Act or PFIA that would have FDA develop a tool to measure how product reviewers are – or are not – using patient-focused drug development tools like guidance documents and benefit/risk surveys that PPMD has been leading the development of in recent years.
Joining Senators Wicker and Klobuchar as original cosponsors of the bill are Senators Susan Collins (R-ME), Michael Bennet (D-CO), Johnny Isakson (R-GA), and Al Franken (D-MN). Additionally, a broad-based group of more than 50 stakeholders including patient advocates, industry, and foundations including The ALS Association (ALSA), the National Organization for Rare Diseases (NORD), the EveryLife Foundation, the Genetic Alliance, and the National Down Syndrome Society (NDSS) have signed a letter endorsing PFIA.
"PPMD has been an innovative leader in the emerging field of patient-focused drug development, organizing and convening the Duchenne community to create tools – like the draft guidance document and with quantifiable benefit/risk preference data – to be used by FDA to evaluate and approve treatments for boys with Duchenne who desperately await them," said Pat Furlong, Founder and President of PPMD.
"Now that PPMD and other stakeholders are proactively developing such tools, we need to know – and we deserve to know – how the FDA uses them at the point of review. PFIA is a natural evolution of patient-focused drug development (PFDD) efforts and will provide essential information to the Duchenne community, as well as thousands of other disease communities with therapies in the development pipeline. We applaud Senators Wicker, Klobuchar, Collins, Bennet, Isakson, and Franken for their leadership and look forward to moving this legislation into law," Furlong added.
Under the legislation, the FDA will develop an assessment asking reviewers how they took into account various PFDD tools such as benefit/risk data, draft or final guidances, patient experience data, patient preference data, and other items in informing their decisions on whether or not to approve a product. The results will be publicly accessible and an annual report will be submitted to relevant Congressional committees.
In addition to the assessment, the legislation directs FDA to issue guidance to further clarify and inform development of PFDD tools created by patient-industry collaborations. This component will help accelerate innovation in PFDD by providing needed direction from FDA.
"We have come a remarkable way in the field of PFDD in just a few years, driven largely by Congress and several provisions contained within the FDA Safety and Innovation Act," said Furlong. "PPMD is deeply committed to continuing to drive innovation in patient-leadership, and enacting PFIA will help us achieve this aim."
The introduction of this legislation coincides with PPMD's 21st Annual Connect Conference being held in Washington, D.C., June 17-21. A Rally to End Duchenne will be held at the U.S. Capitol at 9AM EDT on Friday, June 19 to urge Congress to move forward with this important legislation and the broader 21st Century Cures initiative. For over two decades, PPMD has gathered leaders in the Duchenne space to connect with patients and families affected by this disorder. This year's Connect Conference includes presentations from over 20 industry partners and expects over 550 attendees. PPMD thanks all of its industry and corporate sponsors at this year's conference, in particular elite sponsors BioMarin and PTC Therapeutics.
For more about Parent Project Muscular Dystrophy's advocacy program, please visit their website.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births (about 20,000 new cases worldwide each year). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures.
Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. Young men with Duchenne typically live into their late twenties.
Duchenne can be passed from parent to child, but approximately 35 percent of cases occur because of a random spontaneous mutation. In other words, it can affect anyone. Although there are medical treatments that may help slow its progression, there is currently no cure for Duchenne.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.