How to write a proposed pediatric study request and amendment

How do you write a proposed pediatric study request?

Explain the pediatric information need and propose studies with defined populations, objectives, design, formulation, endpoints and timing. Distinguish the PPSR from the PREA iPSP and from the Written Request FDA may issue. For an amendment, compare the operative Written Request term with the proposed change and obtain the appropriate written agency response before relying on revised terms.

Before you begin

BPCA proposed pediatric study requests and amendments; the PREA iPSP is a separate document.

What you will prepare: A reviewable proposal or amendment that identifies the intended pediatric information and evidence plan.

Define the pediatric question before listing studies

A PPSR asks FDA to issue a Written Request; the sponsor's proposal is not itself that request. Explain the drug, relevant indications and age groups, current pediatric knowledge, and the clinical information the program could generate. Keep the iPSP and PPSR separate even when they share evidence.

Prepare a proposal table with the pediatric question, study objective, population, design, endpoints, formulation, analysis approach, feasibility and proposed completion/reporting timing. For each row, explain how the study closes an identified information gap. Flag decisions that still depend on adult or nonclinical data instead of manufacturing a finished protocol.

Make the proposal coherent across the product program

Collect current labeling, relevant development plans, available pediatric evidence and all applicable existing Written Requests or amendments. Where the sponsor has multiple relevant products or indications, make the boundaries explicit and involve their regulatory owners. Show what is already being pursued under PREA, what additional pediatric questions are proposed and why the proposed package is useful.

A practical document order is: request and product scope; pediatric need; existing knowledge and uncertainties; proposed studies; formulation and feasibility; timing; relationship to other pediatric obligations; questions for FDA; supporting references. FDA's May 2023 draft regulatory-considerations guidance offers additional PPSR context, but it must not be represented as a final binding checklist.

Amend the actual Written Request when the program changes

For an amendment, identify the Written Request and each prior amendment by date. Provide the current term, proposed change, reason, supporting information and impact on other terms. Ask FDA to amend the request; do not present the sponsor's revised protocol as evidence that the Written Request already changed.

FDA's FAQ advises seeking an amended Written Request when departing from its terms and states that an amended request must be received before the study reports are submitted for the exclusivity determination. Review changes before conducting the affected work whenever possible. A trial-management amendment and a BPCA Written Request amendment serve different purposes.

Worked review: a protocol change leaves the request behind

Fictional editorial exercise: the clinical team changes the primary endpoint after discussing operational problems internally. The PPSR history is available, but the issued Written Request is not in the authoring folder.

Obtain the operative Written Request and amendments, compare the endpoint and identify whether an amendment is needed. If the endpoint already matches, document that evidence. If it differs, the author cannot promise that the resulting report will qualify for exclusivity. Route the discrepancy before using the report package as a determination request.

Make each proposed study answer a defined pediatric question

Begin with the information gap, then explain why the proposed evidence can address it. A list of familiar trial types is not yet a coherent proposal.

Question exercise: the proposal claims it will establish a dose for younger children, but the study table lists only an adolescent efficacy endpoint. Identify the missing exposure or dose-selection question and the evidence needed to address it. Do not invent a design or sample size merely to complete the table. Explain the uncertainty and the planned basis for the eventual proposal.

Keep product scope explicit. FDA’s exclusivity FAQ advises a proposal covering the relevant pediatric indications and appropriate age groups when the applicant has multiple products containing the active moiety. Reconcile the responsible review divisions and product owners. Do not assume the scope of one internal development project is the entire scope of the proposed Written Request.

For an amendment, use a second table: operative term and amendment date; proposed replacement; scientific, medical or regulatory justification; supporting evidence; effect on other terms; and requested agency action. An endpoint change may affect the analysis, sample-size rationale, duration and timing as well as the one sentence that names the endpoint.

The May 2023 draft explains that a meeting discussion does not itself amend the Written Request, and that silence on an inconsistent protocol does not establish agreement. FDA’s existing FAQ also instructs sponsors departing from the terms to seek an amendment before conducting the affected studies and receive the amended request before submitting study reports. Keep the actual issued amendment in the record; a protocol approval within the sponsor’s own organization is a different event.

Use the pediatric plan guide to maintain the separate PREA record. Once reports are ready, use the exclusivity determination guide to trace each operative term to the evidence actually submitted.

Frequently asked questions

Sources and revisions

Requirements, source recommendations and editorial preparation advice have different roles. Review the scope and revision of the source you use.

Related guides