Rare Pediatric Disease Designation (RPDD)

There are regulatory frameworks in the EU and the US to support the development of drugs for rare pediatric diseases.

In the EU, both RPDD and Orphan Drug Designation (ODD) are primarily governed by the same regulatory framework. The regulatory framework for both is established by Regulation (EC) 141/2000. This ensures a consistent approach to ODD, regardless of whether they target rare diseases in adults or rare pediatric diseases.

The evaluation of the RPDD application is conducted by the Committee for Orphan Medicinal Products (COMP). The COMP evaluates factors such as the rarity of the pediatric disease, the potential pediatric population affected, and the scientific rationale for the product's potential benefit.

Once RPDD is granted, the sponsor becomes eligible for the incentives and benefits provided under the orphan drug framework.

In the US, the Priority Review Voucher (PRV) program has been established by the US Food and Drug Administration (FDA). It is designed to encourage the development of new drugs for neglected tropical diseases (NTDs) and certain rare pediatric diseases.

Under the PRV program, a voucher can be received that allows for expedited review of a future drug application. The main benefit of the voucher is that it shortens the standard FDA review time for an NDA from 10 to 6 months.

Thus, despite differences between the EU and the US, both regions aim to support the development of treatments for rare pediatric diseases.