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PA Rare Disease Advisory Council Established

July 13, 2017

Pennsylvania Governor Tom Wolf recently signed Act 14, House Bill 239, introduced by Representative Marcy Toepel (R–Montgomery), that creates a Rare Disease Advisory Council to study the incidence and prevalence of rare diseases in the state. The council is a unique public/private partnership—including government, hospitals and other providers, patients, and researchers—working towards improving the lives of more than 1.2 million Pennsylvanians suffering from a rare disease.

Rare diseases are those affecting a small population and include:

  • Huntington's disease
  • Lou Gehrig's disease
  • Tourette syndrome
  • Crohn's disease
  • Cystic Fibrosis

The National Institutes of Health has identified nearly 7,000 rare diseases that affect one in 10 Americans, or 10 percent. Only about 200 rare diseases have federally approved treatments.

Pennsylvania’s Rare Disease Advisory Council will focus on the needs of Pennsylvanian’s with these devastating diseases and the actions required to meet those needs, including:

  • Researching and deciding the most appropriate way to collect state data
  • Identifying priorities for quality and cost effectiveness of treatment
  • Ensuring access to treatment for affected individuals
  • Identifying best practices statewide and nationally
  • Increasing public awareness
  • Reporting periodically to state officials with accomplishments and status of next steps. Council reports will be available to the public.

Pennsylvania now becomes the sixth state to establish a Rare Disease Advisory Council.

HAP and Pennsylvania hospitals continue to support this and other state and national efforts that improve care outcomes and patient satisfaction toward achieving a healthier Pennsylvania. For more information about the legislation, which is effective immediately, contact HAP’s Stephanie Watkins, vice president, state legislative advocacy.

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