The research and development into rare pediatric diseases have garnered more spotlight attention in recent years thanks to a combination of greater public awareness and newly created laws and legislation. Out of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, Section 529 was added to the FD&C Act — establishing the Rare Pediatric Disease Designation (RPDD) program. The implementation of this program was done with the goal in mind of incentivizing the research and development of new drugs and therapies for rare childhood conditions.
Despite the advancement and encouragement caused by these federal laws, rare pediatric disease research and therapy development remain underserved. This leaves an ongoing issue in the United States and Europe in regards to the medical, social, and economic effects stemming from a lack of treatments or successful care options for children.
Roughly 80 percent of rare diseases are genetic in nature and the development of effective treatment options is a result of “limited resources, lack of research, scarce expertise and patients that are few in number and geographically spread.” With 50 to 75 percent of rare diseases beginning in childhood, it is essential to give additional attention to the Rare Pediatric Disease Designation program in order to potentially limit/reduce rare disease cases in all age groups.
Rare Pediatric Disease Designation Requirements
The RPDD program was established with similar intentions to the Orphan Drug Designation (ODD) program. Therefore, commonalities exist between the two in regards to requirements in order to receive approved designation from the FDA.
Qualifying criteria for RPDD includes:
- The drug must be intended for the prevention or treatment of a rare pediatric disease (a life-threatening or serious condition primarily affecting individuals 18 years old and younger)
- Adequate documentation or prevalence data must demonstrate that the intended pediatric disease or condition is rare (the overall patient population for the disease must be 200,000 individuals or less in the US)
- Must not be for an active ingredient that is already approved for use
- There must be supportive data suggesting that the drug may be effective in the rare pediatric disease or condition
However, the FDA does not require the same level of supportive data showing the effectiveness of a pediatric therapy compared to those therapies seeking Orphan Drug Designation.
“In vitro data supporting the mechanism of action of the drug in the disease or in a related disease may suffice for rare pediatric disease designation, whereas that level of data would not generally suffice for orphan-drug designation.”
How to Apply for Rare Pediatric Disease Designation
While the FDA does provide supporting documentation on applying and achieving Rare Pediatric Disease Designation, the process is highly technical and poses potential pitfalls for sponsors and pharmaceutical companies.
BioPharma Global is a leading expert within the FDA and EMA regulatory space. We have helped numerous parties navigate the RPDD process from start to finish, or revise and edit an ongoing submission to meet agency requirements. Through our detailed approach, we have helped a variety of pediatric therapies reach designation approval.
Through collaboration with our clients, we perform background research on the patient population and the most severe manifestations of the disease, provide hands-on guidance through the paperwork/documentation stage, and our team handles the formal filing for review to the Office of Orphan Products Development (OOPD).
Incentives to Apply for Rare Pediatric Disease Designation
Addressing rare pediatric diseases is a health concern that affects more than just those living with the conditions. In an effort to provide potential life-improving or saving care, the RPDD program was established and has received expansion and further clarifications in subsequent federal legislation including the Advancing Hope Act and the 21st Century Cures Act.
To spur further research and development into therapies for rare pediatric diseases, the FDA incentivizes sponsors with Priority Review Vouchers (PRV) to potentially help drug development companies recoup their expenses sooner. Via a PRV, a sponsor is able to have their new therapy moved to the front of the line for review and potential approval for indication use.
Rare Pediatric Disease Designation Sunset Provisions
The voucher program has opened new doors for drug development and given sponsors a shorter runway for potential FDA review and approval. However, this PRV program is set to end, and therefore sponsors should file for Rare Pediatric Disease Designation to ensure eligibility before the termination date.
From the FDA website: “On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended. Under the current statutory sunset provisions, after September 30, 2024, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by September 30, 2024. After September 30, 2026, FDA may not award any rare pediatric disease priority review vouchers.”
