Regulatory Focus™ > News Articles > FDA Issues Guidance on Pediatric Study Plans, Saying some Biosimilar Products will need to Submit

FDA Issues Guidance on Pediatric Study Plans, Saying some Biosimilar Products will need to Submit

Posted 12 July 2013 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) has released a new draft guidance document regarding the content of pediatric study plans (PSPs) and the process by which industry should submit them to the agency.


"The intent of the PSP is to identify needed pediatric studies early in drug development and begin planning for these studies," FDA explains in its draft guidance document.

PSPs were first introduced in 1997 under the Food and Drug Administration Modernization Act (FDAMA) as a response to alarmingly low rates of pediatric testing, which had in turn resulted in a paucity of information regarding the safe use of pharmaceutical products in children. While a common refrain heard from regulators is that "children are not simply little adults," physicians had little to inform with which to inform their prescribing habits.

PSPs, then, were created as an incentive for pharmaceutical companies to conduct pediatric testing. In return for conducting pediatric testing on new products - a difficult, expensive and ethically treacherous endeavor - FDA would provide a product with additional months of market exclusivity during which time other products could not be marketed for the same condition.

In addition, under the 2003 Pediatric Research Equity Act (PREA), FDA introduced postmarketing requirements for certain drugs to conduct pediatric testing to support indications for which pediatric use was likely.

Under the Food and Drug Administration Safety and Innovation Act (FDASIA), this requirement was further expanded, and manufacturers are now required to submit PSPs to FDA "early in the drug development process."


But what does "early in the drug development process" mean, exactly? FDA is supposed to issue a regulation regarding PSPs (among other related provisions), but the development of that rule is still ongoing.

In the meantime, FDA said it is issuing the draft guidance document, Content of and Process for Submitting Initial Pediatric Study Plans and Amended Pediatric Study Plans, as a way of describing the submission timelines it intends to later require.

FDA explained that a sponsor must submit the initial PSP no later than 60 calendar days after the date of its end-of-phase 2 development meeting with FDA. In the absence of such a meeting, sponsors should submit the PSP "as early as practicable, but before the initiation of any phase 3 studies, or any combined phase 2 and phase 3 study."

If, however, a product will not be approved on the basis of a Phase 3 trial (such as in the case of some accelerated approvals or products intended for some orphan populations), the sponsor is advised to submit the initial PSP no later than 210 calendar days before the intended submission date.


The suggested content of the PSP is also discussed at length in the draft guidance, with FDA adding recommendations to the already-required components of the submissions (outline of the studies intended to be conducted, as well as any requests for a deferral, waiver or partial waiver).

Sponsors should, for example:

  • briefly summarize the disease, methods of diagnosis, available treatments, and the disease prevalence
  • summarize the proposed mechanism of action of the drug and intended therapeutic benefits
  • explain, to the extent possible, how data from adult populations may be extrapolated to pediatric populations
  • summarize planned nonclinical and clinical studies in tabular form, as well as all populations for which the sponsor intends to request a waiver or deferral
  • detail the development of pediatric-specific formulations
  • summarize relevant nonclinical studies that support a drug's use in a pediatric subpopulation or subpopulations
  • outline all planned pediatric studies, including pharmacokinetic studies, clinical effectiveness studies and safety studies
  • include a general timeline for completing all studies

FDA clarified that sponsors can "request to amend an agreed-upon initial PSP at any time," provided that they justify the need for the change and explain all changes to FDA.

One particularly interesting section of the draft guidance pertains to biosimilar products. If such a product has not been found to be interchangeable with the referenced biologic, that product will be deemed to have a "new active ingredient" for the purposes of PREA, FDA said. The sponsor would then be required to submit a PSP.

The draft guidance is open for comment for 60 days, or until 15 September 2013.

Content of and Process for Submitting Initial Pediatric Study Plans and Amended Pediatric Study Plans

Federal Register Notice

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