Longietti bill to create Rare Disease Advisory Council awaits governor’s signature

HARRISBURG, June 29 -- A bill that would create the Rare Disease Advisory Council and task it with specific duties now awaits the governor's signature. House Bill 239, sponsored by Rep. Mark Longietti, D-Mercer and Rep. Marcy Toepel, R-Montgomery, unanimously cleared the House today after being amended in the Senate.
A rare disease is defined as a disease affecting 200,000 or fewer people in the United States. "One in 10 Americans live with a rare disease," Longietti said. “While we are familiar with some rare diseases, such as ALS or Lou Gehrig's disease, Crohn's disease and Cystic Fibrosis, many of the over 7,000 rare diseases are largely unknown by the public. Worse yet, on average it takes over eight years to diagnose a rare disease and there are FDA-approved treatments for only 5 percent of rare diseases.”

Once signed, the legislation will establish the Rare Disease Council, which will act as a partnership between patients and their families, medical providers, insurance groups and other agencies to support the needs of those dealing with these illnesses. In addition to health care professionals and industry and scientific representatives, the council will include both individuals suffering from a rare disease and parents of children with a rare disease.

The council would be tasked with researching and determining the most appropriate method to collect rare disease data; researching and identifying priorities relating to quality and cost effectiveness of access to treatment and other services, and developing policy recommendations on these issues; identifying best practices in other states and nationally for rare disease management, education and diagnosis; and developing strategies to raise public awareness. The council would also prepare and deliver a preliminary report and a comprehensive report of its findings on these issues.

"I am grateful for the hard work of my colleague Representative Toepel in helping to develop this proposal and for the bipartisan support of all of my colleagues in the House and the Senate for their approval of this important legislation," Longietti said.

The bill will take effect immediately upon the governor's signature.