The Rare Pediatric Disease Priority Review Voucher program focuses on pediatric patients with rare diseases and unmet needs. Its purpose is to stimulate the development of new drugs for rare pediatric diseases by offering additional incentives for obtaining FDA approval of such products.
What is a rare pediatric disease?
A rare pediatric disease is one that is serious or life-threatening in which the serious or life-threatening manifestations primarily affect patients from birth to 18 years, including neonates, infants, children, and adolescents. It must also be a rare disease or condition, as described in the FD&C Act, with a prevalence of fewer than 200,000 people in the United States.
For your drug to obtain the Rare Pediatric Disease Designation (RPDD), it must meet the following criteria:
It must be intended for the prevention or treatment of a rare pediatric disease
Adequate documentation of prevalence data must demonstrate that the intended pediatric disease or condition is rare
Documentation that the disease is a serious and life-threatening manifestation that primarily affects individuals aged from birth to 18 years
Your application must not be for an active ingredient that is already approved for use
There must be supportive data suggesting that your drug may be effective in the rare pediatric disease or condition [7]
Like the ODD, there are some advantages to applying for the RPDD. The rare pediatric disease Priority Review Vouchers (PRV) aims to incentivize drug development for rare pediatric diseases. [12] The PRV can be used to receive a Priority Review designation of a later NDA or BLA for a different product – it can also be sold.
See the figure below. [11]
Under the current statutory sunset provisions, after September 30, 2024, the FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has an RPDD for the drug, and that designation was granted by September 30, 2024. After September 30, 2026, the FDA may not award any rare pediatric disease PRVs.
Priority Review Voucher Process
RPDD application examples
The BGTC has examples of their successful RPDD applications which you can access through the links below:
Ocular
Systemic
Neurological
An example of a template request form for the RPDD as well as another example of an RPDD application from the PaVe-GT program can be found on their website here.
Requests for RPDD are also sent to the OOPD. This office works in collaboration with the Office of Pediatric Therapeutics to review and grant the RPDD. See the previous section How to submit ODD requests for mailing addresses.
A white paper Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Pave-GT Experience that describes in detail the “how to” of creating ODD and RPDD applications was published by the PaVe-GT team in Human Gene Therapy [14]. We encourage you to read the white paper prior to initiating your application preparation using the templates provided.
The ODD (and where applicable the RPDD) are the main programs that you will be considering during the early stage of development. As your program accrues nonclinical data, consider whether criteria have been met for seeking Fast Track. As you collect clinical data and progress along the development continuum, there are additional programs that may apply, and these are provided below for completeness.