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.
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 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. 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.
"I am delighted that Pat Furlong will be presenting at our upcoming Nonprofit Summit," said NINDS Director Dr. Story Landis. "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."
To learn more about Duchenne and Parent Project Muscular Dystrophy's advocacy agenda, visit ParentProjectMD.org.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.
SOURCE Parent Project Muscular Dystrophy