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Expert Panel to Brief Congress on Innovative Models for Advancing Personalized Medicine and Improving High-Quality, Coordinated Care for Rare Diseases

Successes in cystic fibrosis care delivery and drug development serve as a model for policymakers and greater rare disease community

BETHESDA, Md., Feb. 10, 2014 /PRNewswire-USNewswire/ -- The Cystic Fibrosis Foundation will host a briefing on Capitol Hill to highlight how care delivery and drug development models in cystic fibrosis are an example for health policy experts seeking new ways to treat rare diseases and accelerate drug development.

The panel discussion, "Personalized Medicine and Quality, Coordinated Care: The Cystic Fibrosis Model," will focus on:

  • Innovative and proven cystic fibrosis models for care delivery and drug development
  • How these models can influence health care leaders and the greater rare disease community
  • How the FDA advances treatments for CF and other rare diseases


  • Terence R. Flotte, M.D., Dean, Provost and Executive Deputy Chancellor, University of Massachusetts Medical School
  • Carlos Perez, M.D., Director, Cystic Fibrosis Care Center, Geisinger Medical Center
  • Paul Negulescu, Ph.D., Vice President for Research, Vertex Pharmaceuticals
  • Megan Stacy, a 29 year old with cystic fibrosis
  • Moderator: Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation
  • Congressional Sponsors: Senators Edward Markey (D-Mass.) and Kelly A. Ayotte (R-NH); Representatives Tom Marino (R-PA) and James McGovern (D-Mass.)

WHEN: Tuesday, February 11, 2014, 10:00 a.m.11:00 a.m.

WHERE: U.S. Capitol Visitor Center, Congressional Auditorium


  • The Cystic Fibrosis Foundation's efforts to improve care and spur drug development have significantly improved life expectancy  for those with CF. In the 1950s, children with CF didn't live to see elementary school – now many people with cystic fibrosis are living into their 30s, 40s and beyond. 
  • Thanks to the innovative CF drug development model pioneered by the Cystic Fibrosis Foundation, there is a robust pipeline of promising therapies that target the disease from every angle.
  • Each CF patient is cared for by a coordinated team of providers, who specialize in the treatment of the disease via a nationwide network of CF Foundation-accredited care centers. The National Institutes of Health has cited the CF care center network as a model of effective and efficient health care delivery for a chronic disease.
  • The CF Foundation Patient Registry was the first rare disease registry to publish comparative health outcomes for care centers to accelerate quality improvement.

About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. For more information, please visit

SOURCE Cystic Fibrosis Foundation

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