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PPMD Leadership on Patient-Focused Drug Development Nets Invitation to Present at Two Influential Meetings

Parent Project Muscular Dystrophy (PPMD) has been invited to present at two upcoming stakeholder meetings.

The invitations, to PPMD Founding President & CEO Pat Furlong, were extended by leaders in the U.S. government in recognition of PPMD's groundbreaking work to advance the patient and parent perspective in the drug development and review process.

On September 10th, Pat will speak at the Food and Drug Administration's (FDA's) Patient Network about the role patients and patient advocacy organizations must play to educate drug developers and regulators on a condition and its natural history, to offer perspectives on endpoints and trial design and to recruit clinical trial participants.

The following day, Pat will present at the plenary session of the National Institute of Neurological Disorders and Stroke (NINDS) Nonprofit Forum, an annual meeting attended by dozens of leaders of patient advocacy and related organizations. Pat will speak about PPMD's collaboration with the FDA on the agency's new initiative to engage patients and caregivers. Pat will emphasize PPMD's longstanding leadership in patient advocacy and the connection of such efforts to a robust, basic, and translational research agenda.

"I am honored both NINDS and FDA have recognized the significant work done by PPMD to ensure the patient and parent perspective – particularly on critically important topics such as benefit/risk, trial design, and clinical and surrogate endpoints – is listened to.

"Over the past several months, PPMD has commissioned landmark work around the areas of benefit/risk, including a first-ever scientific survey of Duchenne parents and caregivers. We have presented preliminary information from this work to FDA leaders, including Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER). We were very encouraged by their reaction and look forward to ongoing discussions with agency leaders."

-- Pat Furlong, PPMD Founding President & CEO

The survey's findings suggests that the presence of side effects and risks could be compensated for by a treatment that stops progression of muscle weakness. While parents were not willing to accept unlimited risk, they would be willing to accept risk of a serious side effect if the potential benefit were stopping or slowing the progression of the disease. 

"I am delighted that Pat Furlong will be presenting at our upcoming Nonprofit Summit. I've gotten to work with Pat and PPMD closely over the past decade through the Muscular Dystrophy Coordinating Committee and have been very impressed by the organization's ability to communicate the perspective of its patients and parents to FDA, NIH, industry, and other audiences and to promote a comprehensive research plan spanning from laboratory work through to clinical trials. Patient groups will be very interested in hearing about the organization's current activities with the FDA on defining patients' willingness to accept risk, an issue that all patients and caregivers care about."

-- Dr. Story Landis, NINDS Director

To learn more about PPMD's advocacy agenda, visit ParentProjectMD.org.

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