Parent Project Muscular Dystrophy (PPMD), an advocacy organization working to end Duchenne muscular dystrophy, 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. 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.

Over the past year, PPMD organized a comprehensive, multi-stakeholder effort to draft and submit to the FDA a first-ever patient advocacy initiated draft guidance for industry developing therapies for Duchenne. The draft guidance was submitted to FDA in late June and a public comment period closed in early October.

In addition to the draft guidance, PPMD has partnered with Johns Hopkins University to produce a scientific survey assessing Duchenne primary caregivers' views on benefit/risk of potential therapies. FDA officials have commended these products and have urged other patient communities to consider taking similar actions.

"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 actually will be using these new tools as they review therapies for Duchenne and beyond," said Pat Furlong, PPMD founding president and CEO.

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," Furlong said.

In addition to PPMD, leaders from FasterCures and the Pharmaceutical Research and Manufacturers of America (PhRMA) also will 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," said Kim McCleary, director of strategic initiatives at FasterCures. “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 21(st) Century Cures Initiative has reinforced the potential for this collaborative approach.”