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# ‘Wide Range’ of Rare Pediatric Treatments Developed
- URL: https://www.fdaweb.com/wide-range-of-rare-pediatric-treatments-developed/
- Published: 2024-02-27T12:00:00.000Z
- Updated: 2026-09-14T14:23:55.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156439

An FDA analysis of the first decade of the rare pediatric disease priority review voucher program establishes that a wide range of diseases have seen development, from rare pediatric cancers to rare genetic disorders. “Continued support of product development for children with rare diseases is needed to find treatments for all children with unmet needs,” the study report in the open-source *Orphanet Journal of Rare Diseases* concludes.

The agency researchers looked at the 569 rare pediatric disease designations granted in the 10 years for products targeting 245 unique rare pediatric diseases. The report says 42% of the designations occurred in 2020, presumably because the voucher program was to begin sunsetting on 9/30/2020, and companies needed to receive their designations before that date to maintain eligibility for a future voucher.

The researchers say they draw these conclusions from their review:

- there is a wide range of pediatric diseases for which product development has been occurring;
- drugs and biologics intended for use in the neonatal population represent a surprisingly small proportion of all rare pediatric disease designations;
- gene therapy products are more than one-quarter of all designations; and
- a substantial number of designations are supported by clinical data.

While a wide range of pediatric diseases is represented, most designations were focused on the neurologic, metabolic, and oncologic therapeutic areas.

The researchers note that the program has not been permanently authorized and questions about congressional action on it can have a negative impact on drug developers.