PPMD Calls for Greater Transparency and Accountability of FDA Patient Engagement Efforts

Parent Project Muscular Dystrophy (PPMD) is urging Congress to build upon gains in recent years to further strengthen and enhance programs to ensure the patient voice is heeded during the drug development process during a Congressional briefing tomorrow, Tuesday, December 9th at noon.


A champion of policies to enhance the voice of the patient within vital Food and Drug Administration (FDA) deliberations, PPMD is advocating for provisions within the 21st Century Cures Initiatives focused on measuring how the agency is or is not using new patient engagement tools. PPMD and other stakeholders will be presenting on the reform proposal during the briefing tomorrow afternoon.


Reform Proposals for 21st Century Cures Initiative

We have seen significant progress over the years in how the FDA pays attention to the voice of the patient. PPMD has been focused relentlessly on this issue and has produced several groundbreaking tools intended to achieve this goal. Now, we must know that this work is bearing fruit and that FDA will actually be using these new tools as they review therapies for Duchenne and beyond.

Dubbed the Patient-Impact Assessment Act, PPMD would like to see a simple and publicly accessible measurement tool developed whereby FDA reviews could note how they did or did not take various patient-focused drug development tools and resources into account in evaluating products and making their decisions. Such an assessment tool would shed light into how Patient Focused Drug Development (PFDD) tools and authorities are or are not being used and would help identify gaps or shortcomings in the space.

In addition to PPMD, leaders from FasterCures and the Pharmaceutical Research and Manufacturers of America (PhRMA) will also be presenting on related PFDD efforts and recommendations.

“Across the whole continuum of discovery, development, and delivery a new focus on the patient is changing culture, practice, and policy. PPMD has pioneered a new model for advancing the science of patient input, one that will help transform the way in which patient perspectives are collected and understood. FasterCures champions a broader partnership of patient, industry, and government organizations to further define the science of patient input and use it to inform decision-making at all levels; the 21st Century Cures Initiative has reinforced the potential for this collaborative approach.”

– Kim McCleary, director of strategic initiatives at FasterCures 

Invite Your Members of Congress to the Briefing

Please urge your members of Congress and their staff to attend this important briefing hosted by PPMD tomorrow, Tuesday, December 9th on the 21st Century Cures Initiative. We welcome all members of the community to attend as well.

Via email: Click here to send the invite to your members (it takes less than a minute!)

Via phone: Take it one step further! Make a call. Call your members offices and tell them to send a representative. Ask to speak with the health legislative assistant. You can find you members offices by clicking here. Be sure to call both Senate offices and your House rep.

Remember, the more constituents the members hear from the better – Ask your family and friends to send the invite as well!

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