Under the Pediatric Research Equity Act (PREA) (21 U.S.C. 355c), all new drug applications for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration are required to contain an assessment of the safety and effectiveness of the product for the claimed indication(s) in all relevant pediatric subpopulations unless this requirement in waived, deferred, or inapplicable. This includes products approved via the 505(b)(2) pathway. Given that a drug developer may want to obtain approval for adults first, it is common to request that the requirement for pediatric studies be deferred until after approval of the new drug application.

The European Medicines Agency (EMEA) implemented a new “Pediatric Regulation” in January of 2007. This regulation requires an initial submission of a pediatric investigation plan (PIP) by pharmaceutical companies upon completion of adult PK studies. A PIP is required for acceptance of any marketing authorization application. Unless a waiver or deferral has been granted, all products that have not been authorized in the European Union before 16 January 2007 must include the results of pediatric studies conducted based on a PIP.

The difference in the US and European approaches has the potential to put discussions with EU regulatory agencies about pediatric clinical studies ahead of discussion with the US. For companies pursuing simultaneous drug development in the US and EU, the submission of a PIP is likely to occur before discussions would ordinarily be held with the FDA. The FDA is aware of this issue and recommends that sponsors begin discussions about an acceptable pediatric program with the FDA when they begin discussions with the EMEA. Even if a deferral is being requested for US pediatric studies, it is wise to seek the FDA’s input before finalizing a PIP. Communication is the key to make certain that any pediatric studies meet both agencies’ requirements.