Rare Pediatric Disease Designation Sunset Clause
In June of 2020, BioPharma Global wrote a blogpost describing the rare pediatric disease designation (RPD) and voucher programs, noting they will sunset on September 30th, 2020. This required all companies applying for a rare pediatric disease priority review voucher (PRV) after this date to have already been granted RPD for a specific product while requesting a rare pediatric disease PRV by September 30th, 2022, at which point the program would end (Click here to read our RPD Sunset Clause Blogpost)1.
Since our original blogpost, congress has passed legislation to extend the RPD and voucher program to sunset on September 30th, 2024. After September 30th, 2024 FDA will no longer grant PRV to those without RPD, and after September 30th, 2026, FDA will stop granting rare pediatric disease PRV (unless this program is further extended).2 BioPharma Global continues to offer strategy assessments and data reviews related to RPDs, submissions of RPD applications, and writing of RPD applications in addition to our other services (BioPharma Global Services).
The Rare Pediatric Disease Designation and Voucher Program
What is a rare pediatric disease?
Rare pediatric diseases are most currently defined by the 2016 Advancing Hope Act:
(A)The disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents [; and]
(B)The disease is a rare disease or condition, within the meaning of section 526 [of the FD&C Act]3-5.
What is the rare pediatric disease designation and voucher program?
The Rare Pediatric Disease Designation (RPD) and Voucher program was established in the federal Food, Drug, and Cosmetic Act in 2012 and is outlined in section 5295. This program was developed to promote the discovery and invention of drugs that treat or prevent rare pediatric diseases by granting priority review vouchers (PRV) to companies whose products concern rare pediatric patient populations3. Rare pediatric disease PRVs grant the redeemer (for a redemption fee) an expedited new drug application (NDA) or biological license application (BLA) review by the FDA in approximately 6 months compared to the standard 10 month time frame6.
What is the process of applying for RPD and subsequent voucher?
The FDA recommends companies looking to request a rare pediatric disease PRV first apply for a RPD for a particular product3. The RPD application may be applied for separately prior to submitting a rare pediatric disease marketing application and a rare pediatric disease PRV request. If a company receives a RPD prior to marketing application, then at the time of marketing application the company will only need to submit their designation letter and the request for the rare pediatric disease PRV along with the marketing application. For companies wishing to have a RPD review within 60 days of submission, the RPD application must be accompanied with an orphan drug designation application or fast track designation application (within two weeks of submission of one another)3. RPD applications may be submitted alone, but there is no time guarantee for review of RPD submissions without orphan drug designation or fast track designation applications. The requirements outlined by the FDA for a drug company to receive approval for a rare pediatric disease product application and subsequent PRV are:
(A) […] a human drug application as defined in section 735(1) of the FD&C Act:
– For prevention or treatment of a rare pediatric disease
– That contains no active ingredient (including any ester or salt of the active ingredient) that has been previously approved in any other application under section 505(b)(1), 505(b)(2), or 505(j) of the FD&C Act or section 351(a) or 351(k) of the PHS Act.
(B)That FDA deems eligible for priority review.
(C) Is submitted under section 505(b)(1) of the FD&C Act or section 351(a) of the Public 181 Health Service Act.
(D) Relies on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population (see Question 4*).
(E) Does not seek approval for an adult indication in the original rare pediatric disease product application (see Question 5*); and
(F) Is approved after the date of enactment of the Advancing Hope Act of 2016 (September 30, 2016)3,5.
*Answers to questions 4 and 5 may be found in the FDA’s draft guidance on Priority Review Vouchers3
These requirements must be met by the time a company applies for marketing approval (through an NDA or BLA) because the rare pediatric disease PRV request is submitted at the same time as an NDA or BLA for a particular product (during initial submission of these documents)3. Currently, rare pediatric disease PRVs can be requested when applying for marketing approval without having previously received a RPD. As stated above, the program has been extended to sunset on September 30th, 2024, and after this date, companies wishing to receive a rare pediatric disease PRV for products will only be eligible to receive a PRV if they have been granted a RPD by this date2.
If a company’s rare pediatric disease drug product has been approved for market, the company will receive a rare pediatric disease PRV along with their approval notification3. PRVs do not need to be redeemed by the company who initially received them and can be transferred for profit an unlimited number of times3. Companies have previously sold their rare pediatric disease PRV for a large profit, for example, between 80 to 110 million US dollars6.
The RPD and priority review program will begin sunsetting in September, 2024. As mentioned, after September 30th, 2024, rare pediatric disease PRVs will only be granted by the FDA if a company has been awarded a RPD for a drug product by September 30th, 2024, and after September 30th, 2026, rare pediatric disease PRVs will no longer be awarded (unless legislation is again passed that extends this program)2.
For more information such as specifics to be included in rare pediatric disease designation request or individual offices to contact please visit the following websites:
- FDA Rare pediatric disease priority review guidance draft: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-pediatric-disease-priority-review-vouchers
- FDA Rare Pediatric Disease Priority Review Website: https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs
- FDA. Rare Pediatric Disease (RPD) Designation and Voucher Programs. https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs. Published 2017. Accessed 6/11/2020.
- FDA. Rare Pediatric Disease (RPD) Designation and Voucher Programs. 2021.
- FDA. Rare Pediatric Disease Priority Review Vouchers Guidance for Industry: Draft Guidance. 2019.
- FDA. Orphan Drug Act. 1983.
- FDA. Federal Food, Drug, and Cosmetic Act. 2012.
- Mezher M, Gaffney A, RAC., Brennan Z. Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers. https://www.raps.org/regulatory-focus/news-articles/2017/12/regulatory-explainer-everything-you-need-to-know-about-fdas-priority-review-vouchers. Published 2020. Accessed.
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