THE CREATING HOPE ACT: THE RARE PEDIATRIC DISEASE PRIORITY REVIEW VOUCHER INCENTIVE PROGRAM
21 U.S.C. 360ff
Kids v Cancer thanks the U.S. Congress and President for reauthorizing the Creating Hope Act pediatric PRV program as part of 21st Century Cures in December 2016.
Under the Creating Hope Act, companies that develop drugs for kids with cancer and other life-threatening diseases receive a voucher from the FDA. With this voucher comes rights to faster FDA review on any future drug. And the voucher is transferable. The last voucher sale was for $350 million and almost $1 billion in vouchers have been sold.
In the 20 years before the enactment of the Creating Hope Act, there had been only two drugs developed to expressly treat a pediatric cancer that were initially FDA approved for kids: Erwinase and Clofarabine.
By enacting the Creating Hope Act pediatric PRV program – the Rare Pediatric Disease Priority Review Voucher Program, 21 U.S.C. 360ff — Congress created a market incentive for the development of drugs for rare pediatric diseases through the establishment of a priority review voucher. Under this program, a sponsor that develops a drug or biologic for a rare pediatric disease and receives FDA approval for that drug or biologic also receives a voucher. That voucher comes with rights to FDA priority review for any other drug or biologic, including a large market adult drug or biologic that would otherwise receive standard review. This would result in the other drug or biologic getting to market many months earlier, creating significant value. The voucher is fully transferable.
The Creating Hope Act voucher is based upon a neglected tropical disease priority review voucher program that was signed into law as a provision of the Food and Drug Administration Amendments Act of 2007.
FDA Draft Guidance
On November 17, 2014, the FDA published a draft guidance on the Creating Hope Act entitled: Rare Pediatric Disease Priority Review Vouchers, Guidance for Industry. (see sidebar for document).
For companies developing pediatric rare disease drugs:
The criteria for a Rare Pediatric Disease Product Application leading to a pediatric voucher are that a drug or biological product:
- Is for the prevention or treatment of a Rare Pediatric Disease;
- Contains no active ingredient that has been previously approved in any other application;
- Is submitted under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act;
- Itself qualifies for priority review;
- Relies on clinical data derived from studies examining a pediatric population and dosages of the drug intended for a pediatric population
The term “rare pediatric disease” means a disease that is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years.
The Creating Hope Act provides for a rare pediatric designation process to create greater certainty earlier in the drug development process that a drug or biologic would qualify for a voucher, should it be approved. The request for a rare pediatric designation can be made at the same time or after a request for an orphan designation is made.
For companies wishing to exercise a voucher:
A sponsor who wishes to exercise a priority review voucher would likely have a large market adult drug or biologic that would otherwise be reviewed by the FDA pursuant to a standard review. By exercising a priority review voucher, the sponsor would then be able to receive a priority review for that drug or biologic. This would get the drug or biologic to market more quickly, perhaps ahead of a similar competing drug or biologic, thereby generating significant value.
The priority review voucher program does not alter the criteria for a FDA approval. Drugs subject to priority review must still conform to all the FDA requirements to be approved.
A sponsor of a drug or biologic application that is the subject of a priority review voucher shall pay a user fee. The amount of the user fee shall be based on the difference between the average cost incurred by the FDA of a priority review and the average cost incurred by the FDA of a standard review. This amount shall be adjusted annually to reflect current costs.
The Creating Hope Act provides that the sponsor must notify FDA of its intent to submit a drug application with a priority review voucher at least 90 days prior to submission. The user fee shall be due upon the notification.
Pursuant to the Creating Hope Act, the FDA may revoke a priority review voucher awarded if the Rare Pediatric Disease Product Application for which such voucher was awarded is not marketed in the United States within a year.