FDA Publishes Guidance On Priority Review Vouchers For Rare Pediatric Diseases

Sponsors of treatments for rare pediatric diseases have received draft guidance from the Food and Drug Administration (FDA) as it has published its current thinking on the priority review voucher program. The FDA announced in March 2014 that it implemented section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA) and added section 529, which established the priority review voucher program for certain pediatric diseases. Sponsors with accepted applications are entitled to a faster review of another drug and are entitled to sell the voucher to another company. The FDA also maintains a voucher program for tropical diseases, and some lawmakers are attempting to add the Ebola virus to the list.

Rare Pediatric Disease Priority Review Program

Pursuant to section 529 of FDASIA, the FDA conducts a priority review voucher program for rare pediatric diseases. The new guidance covers that program. Section 529 was enacted to encourage new drugs and new biological treatments for certain rare pediatric diseases. The federal agency acknowledges that incentives for industry currently exist but states that the voucher program is an additional incentive and can be used in conjunction with other incentive programs.

FDA Guidance on Vouchers

The FDA states that the new guidance will assist sponsors in assessing their products’ eligibility for the voucher program. The guidance defines “rare pediatric disease” and also discusses the age range that “pediatric” encompasses. The authors wrote that, while pediatric often refers to children between the ages of 0 to 18, the FDA considers patients from birth to 16 years of age to be the appropriate target group. Rare diseases and conditions are those that affect fewer than 200,000 persons in the United States.

Successful Applications

For the FDA to accept the application, sponsors must meet a set of criteria. The investigational agent must not contain any active ingredient that has previously been approved, the application should rely on clinical data derived from the pediatric population, and the application does not seek approval for an adult indication. The guidance further defines the criteria and clarifies the sponsor’s responsibility in regards to competing in other incentive programs. For instance, a sponsor may apply for several programs at the same time, but each must be a separate and distinct proposal.