PPMD Holds Landmark Meeting with FDA Leaders to Address Trial Design, Endpoint, and Other Issues

May 03, 2012, 12:16 ET from Parent Project Muscular Dystrophy

Larger Community Roundtable on Trial Design Planned

HACKENSACK, N.J., May 3, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) – the leading advocacy organization fighting to end Duchenne muscular dystrophy, the most common form of muscular dystrophy, convened a high-impact meeting with leaders of the Food & Drug Administration's (FDA) drug review center to seek ways to accelerate the review process and to ensure the patient voice is included when key decisions are being made.

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

The session represented a landmark meeting between high-level FDA leaders, and included the leader of the agency's Center for Drug Evaluation and Research as well as the head of its neurology products division.

Called for by PPMD and attended by Founding President and CEO Pat Furlong and several board members and parents of boys afflicted with Duchenne, the meeting led to a fruitful exchange and a path forward to a larger roundtable discussion that would include industry leaders and focus on clinical trial design and the validation and acceptance of surrogate endpoints.

"The recent PPMD-FDA leadership meeting provides a solid foundation upon which we can build to develop and implement the tools and processes necessary to expedite the FDA's review of potential treatments and accelerate the delivery of safe and effective treatments to our patients," Furlong said.

"FDA leaders provided valuable information and perspectives on trial design and surrogate endpoint acceptance, two critically important issues, and expressed a desire to continue the discussion with industry leaders. PPMD looks forward to working with FDA to convene a larger roundtable discussion and other follow-up activities on these issues in the coming months, and will work with all patients and Duchenne stakeholders to ensure the community's voice is well-represented at these events."

In addition to direct engagement with FDA, PPMD has been advocating on Capitol Hill for measures that would help accelerate FDA's process for reviewing therapies for serious and life-threatening conditions and enable the agency to engage external experts, including patients, on rare disease issue.

"When it comes to saving our sons, we must have the active participation of Congress, the FDA, and industry. PPMD will continue to lead this most important work," Furlong said.

About Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy.  Our mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey.  For more information, visit www.ParentProjectMD.org.

SOURCE Parent Project Muscular Dystrophy