THE CREATING HOPE ACT: THE RARE PEDIATRIC DISEASE PRIORITY REVIEW VOUCHER INCENTIVE PROGRAM
21 U.S.C. 360ff
In the 20 years before the enactment of the Creating Hope Act, there had been only two initial FDA approvals for drugs to treat any pediatric cancer – Erwinase and Clofarabine.
By enacting the Creating Hope Act – 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.
How to receive a designation and a priority review voucher
The Creating Hope Act provides for a 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.
On November 17, 2014, the FDA published a draft guidance on the Creating Hope Act entitled: Rare Pediatric Disease Priority Review Vouchers, Guidance for Industry.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). There is some lack of clarity with respect to which pediatric cancers are and are not covered by the draft guidance. Sponsors who have questions about the applicability of their drug product or biologic should address the questions to the Office of Orphan Product Development at the FDA. In addition, please inform Kids v Cancer if you have continued questions.
Pursuant to the language of the Creating Hope Act, a sponsor of a drug or biologic may request that the disease for which it is developing the drug or biologic be designated as a “Rare Pediatric Disease.” In addition, the sponsor may request that the drug or biologic be designated as a “Rare Pediatric Disease Product Application.” These designations would lead to a voucher if the drug or biologic is approved
The criteria for a Rare Pediatric Disease are that the disease primarily affects individuals aged from birth to 18 years; and that the disease is rare within the meaning of the Orphan Drug Act. An orphan designation would satisfy the second criteria.
The criteria for a Rare Pediatric Disease Product Application 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; and
- Does not seek approval for an adult indication before or at the same time as the pediatric indication.
How to transfer a priority review voucher
Unlike the neglected tropical disease voucher which is only transferable once, the Creating Hope Act priority review voucher has unlimited transferability and may be exercised for other drugs or biologics that the sponsor may be developing, or for drugs and biologics developed by another sponsor.
Recently, BioMarin sold a pediatric priority review voucher to Sanofi/Regeneron for $67.5M. In addition, Knights Therapeutics sold a tropical disease voucher, which would have the same valuation as a pediatric voucher, for $125M to Gilead.
How to exercise a priority review 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. To date, no vouchers have been exercised.
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.
For more information
Further questions may be directed to Nancy Goodman at email@example.com, 646-361-3590.