It was seven months to the day that Representatives Upton and DeGette first launched this bold initiative called 21st Century Cures. In fact, that hearing that would set in motion months and months of proposals for sweeping changes to research infrastructure and academic systems took place just across the hall from where we were now gathered. PPMD, FasterCures, PhMRA, a father of a 31-year-old man with Duchenne. Powerful partners. Talking about the power of patient engagement from the perspective of all stakeholder vantage points. Presenting testimony in front of a room full of Congressional staffers about how it is simply not possible to effectively move the needle in therapy development without involving patients in every aspect of the process – from beginning to end – and in a measured and meaningful way.


Our PPMD team has spent the last few months on the Hill sharing the language that we have put forward to help take the patient focused drug development (PFDD) effort to the next level. We have also been working closely with all of our coalition partners from other rare disease groups, to coalition organizations, to industry partners.


On Tuesday, December 9, we held a briefing on Capitol Hill. This day was about bringing it all together in front of our congressional champions and showing them that we all believe that therapy development begins and ends with patients. But more, that patients are data. That patient needs and perspectives are quantifiable and meaningful and powerful. And that whether you are a patient advocacy organization (PPMD), a coalition of industry organizations (PhMRA), a coalition/think tank for both advocacy organizations and industry (FasterCures), or a father of a son with Duchenne, we are all here for the exact same reason; that child with Duchenne.


And as Pat said at the beginning of her testimony – better than anyone else ever could have –

“I believe that patient engagement is the 'blockbuster drug of the century' – helping  patients live longer, richer, more meaningful lives.”


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