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Recent Federal Efforts to Incentivize Drug Development for Rare Diseases that Impact Children Set to Expire In Less Than a Year, Casey Plan Would Make Incentive Permanent / Casey Unveils New Legislation – Advancing Hope Act – Will Build Off of Successful Pilot Program that has Already Seen New Products for Rare Pediatric Diseases Come to Market / Doctors at Children’s Hospital of Pittsburgh Detail Need for New Treatments for Children with Rare Diseases

Pittsburgh PA- With an important incentive for drug development to treat rare pediatric diseases set to expire in less than a year, U.S. Senator Bob Casey (D-PA), joined by doctors at the Children’s Hospital of Pittsburgh of UPMC, pushed to make the incentive permanent to continue encouraging drug development that could help find cures for severe diseases and conditions that impact a small number of children. Casey’s new legislation, the Advancing Hope Act, introduced with Senator Johnny Isakson of Georgia, will build off of a successful pilot program the Senator successfully passed into law that has incentivized drug companies to research treatments for rare, life-altering diseases that impact pediatric patients and are challenging to develop because so few children are diagnosed. Doctors at the Children’s Hospital of Pittsburgh of UPMC discussed the intense challenges that children and families face when confronted with rare pediatric diseases and the need for new treatments.

“Our nation has an enduring obligation to be there for the children and families who seek treatment here at Children’s Hospital in Pittsburgh and at children’s hospitals across the country,” Senator Casey said. “If this program is allowed to lapse, Congress will have broken faith with these children and their families. We need to provide certainty for drug developers so that they can count on this incentive when deciding to invest the time and money into developing drugs for rare pediatric diseases.”

During Food and Drug Administration Safety and Innovation Act, or FDASIA in 2012, Casey was able to get language included based on his legislation, the Creating Hope Act, to establish a pilot program at FDA to award three “priority review vouchers” (PRVs) to companies who successfully seek approval of a new therapy to treat a rare pediatric condition. These PRVs could be used by the company, or sold to another company, in order to speed the FDA’s review time of a different product in the future.

Incentivizing Drug Development

Priority review vouchers are incentives meant to catalyze the development of new therapies for historically underserved disease areas, such as rare pediatric diseases affecting fewer than 200,000 children in the United States. Due to the difficulty of developing and marketing drugs for diseases affecting only a small number of patients, in 2012, FDASIA created the Rare Pediatric Disease Priority Review Voucher Program, based on Casey’s legislation, the Creating Hope Act. This program built on the existing Tropical Disease Priority Review Voucher Program.

The incentive in this program is a special voucher that allows its owner to have any experimental drug the company owns (i.e. one that would not otherwise qualify for priority review) reviewed by the FDA under its Priority Review pathway, resulting in a review time of 6 months instead of the usual 10 months.

The sunset clause in FDASIA stated after FDA awards its third rare pediatric disease priority review voucher, a one-year countdown clock begins. After the year is up, FDA may no longer award any additional rare pediatric disease priority review vouchers. The vouchers themselves may still be redeemed. On March 17, 2015, FDA issued its third rare pediatric disease priority review voucher. According to statute, FDA's rare pediatric disease priority review voucher program is now slated to end March 17, 2016.

The Advancing Hope Act

The Advancing Hope Act of 2015 will extend and strengthen the current FDA pediatric rare disease priority review voucher program. The legislation will:

  • Clarify the definition of rare pediatric diseases so the devastating treatment gap can be closed for all kids with rare diseases;
  • Extend program eligibility to all pediatric cancers to encourage the development of life-saving innovations for some of the most deadly childhood illnesses;
  • Extend program eligibility to sickle cell disease, which often presents with life-threatening symptoms during childhood;
  • Remove the sunset provision so the program will continue to be available to companies working to advance treatment options for children; and
  • Reduce the administrative burden on FDA by requiring companies intending to seek a PRV to notify the agency at the time of NDA or BLA submission. Overall, these changes will help to enhance this program to spur innovation in rare and neglected diseases that disproportionately impact children.