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New FDA Guidelines on Pediatric Studies and Potential Effects on Opportunities for Market Exclusivity

Legislation on pediatric studies has existed for more than 20 years in the US, yet additional guidance from the FDA has been relatively scarce. However, two new draft guidelines have now been published, including policy changes that may affect current opportunities for pediatric exclusivity and when data may be extrapolated from adults. How might this affect pediatric drug development?

What has the regulatory framework looked like so far?

To improve the safety and efficacy of drug use and dosage for children, two acts were passed in recent decades to encourage drug companies to document their products for use in children. The first — the Best Pharmaceuticals for Children Act (BPCA) of 2002 — incentivized the inclusion of children in clinical studies by offering market exclusivity, becoming a “carrot” for drug developers. However, it didn’t have the intended effect quickly enough, so Congress later developed a “stick”: the Pediatric Research Equity Act (PREA) of 2003, which gives the FDA the authority to require pediatric studies in certain cases.

PREA affects the majority of pharmaceutical projects, though some products are automatically exempted, and companies can apply for waivers or deferrals for certain product types and indication areas. In all other cases, pediatric studies must be carried out, though studies in children are typically started with some lag compared to studies in adults.

However, for sponsors that voluntarily conduct pediatric studies, the BPCA offers six months extra pediatric market exclusivity. Keep in mind, the six months can be added to any existing market exclusivity and patent periods.

Some requirements must be met to enable pediatric exclusivity. The FDA must have issued a Written Request (WR), something the company can request. The WR describes which studies are to be carried out and within which time frames. The company must then conduct the pediatric studies and submit study reports to the FDA in accordance with the agreed time frames. However, positive results in the studies are not required for exclusivity.

What’s new? Announced changes that may affect market exclusivity

Intended to replace the previous “How to Comply with the Pediatric Research Equity Act” of 2005, the new draft guidelines published May 2023 include:

    • Pediatric Drug Development: Regulatory Considerations — Complying with the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act, DRAFT GUIDANCE, May 2023
    • Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations, DRAFT GUIDANCE, May 2023

Of particular interest is the proposed policy that would change the FDAs issuance of Written Requests.

Until now, even in cases where the product is subject to PREA, i.e., where the company is required to conduct studies in children, the company has been able to request a WR from the FDA. If a WR is received and fulfilled on time and on the agreed upon terms, sponsors have been able to obtain six months of pediatric exclusivity.

According to “Pediatric Drug Development: Regulatory Considerations,” the FDA plans to change its approach, limiting the issuance of WRs to situations where the company conducts pediatric studies beyond what is required under PREA, and which may generate valuable knowledge for use in children. Bottom line: in cases where only the studies required by PREA are performed, developers will no longer have the opportunity to achieve pediatric exclusivity.

It also discusses what is meant by “fairly respond to a WR,” i.e., the requirement that the completed studies must fulfill what the FDA intended in its WR, and how they will make those assessments.

The possibility of extrapolating data from adults

In the “Scientific Considerations” draft guideline, scientific considerations are discussed when planning and conducting studies in children, although it is also pointed out that the general principles do not deviate from those that apply to studies in adults. The guideline addresses the appropriate time to start pediatric development in relation to the clinical program in adults. The FDA also emphasizes in which case(s) it is possible to extrapolate results seen in adults to pediatric patients without conducting separate studies in children.

According to PREA (the stick), the company is required to develop drug formulations that can be administered to all clinically relevant pediatric age groups. This can be quite challenging, depending on the drug’s mode of administration, volumes, tablet sizes, taste, and other aspects. The FDA, therefore, strongly recommends early interaction with them before developing pediatric formulations.

Finally, the FDA here strengthens the expectations for studies in newborn babies (up to 27 days). Carrying out studies in this age group entails very special challenges, which the FDA also notes. For example, if the FDA issues a WR that does not include the newborn age group, the FDA they must include an explanation as to why they do not require it.

Possibility to comment no later than July 17, 2023

As usual, there is the opportunity to comment on the two new draft guidelines. Comments must be received by the FDA no later than July 17, 2023.


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