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

Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy)

Pat Furlong to Speak at NINDS Nonprofit Forum & FDA Patient Network

HACKENSACK, N.J., Sept. 9, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) the leading advocacy organization fighting to end Duchenne muscular dystrophy and working to improve the quality of life and health outcomes of all patients with Duchenne has been invited to present at two upcoming stakeholder meetings.

(Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO)

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

On September 10th, Furlong 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, Furlong will present at the plenary session of theNational Institute of Neurological Disorders and Stroke (NINDS)Nonprofit Forum, an annual meeting attended by dozens of leaders of patient advocacy and related organizations. Furlong will speak about PPMD's collaboration with the FDA on the agency's new initiative to engage patients and caregivers. Furlong 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," Furlong said.

"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," Furlong added.

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.

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