New FDA Draft Guidance Aims to Limit Pediatric Exclusivity Extension Awards

New FDA Draft Guidance Aims to Limit Pediatric Exclusivity Extension Awards

According to a recent Draft Guidance, FDA is considering eliminating written requests for pediatric exclusivity under the Best Pharmaceuticals for Children Act (BPCA) when the studies are already required under the Pediatric Research Equity Act (PREA).

If finalized, the Guidance would remove the FDAs current practice of granting pediatric market exclusivity to sponsors who conduct studies already required by PREA even when no additional indications regarding pediatric health benefits are proposed.

FDA published the Draft Guidance on May 17, the first Guidance to combine recommendations on PREA compliance with guidance on pediatric exclusivity qualification under BPCA. The proposed Guidance is designed to replace the existing one covering PREA compliance.

In an accompanying notice in the Federal Register, FDA states that it has combined the discussion of PREA and BPCA to emphasize "the sponsor's need to consider both laws when developing pediatric drugs and biological products."

The PREA BCPA Overlap

Enacted in 2003, PREA (FDCA §505B) requires drug and biologic studies designed to ensure proper pediatric drug labeling. BPCA (FDCA §505A) offers pediatric drug sponsors an extended market exclusivity period of six months to encourage growth in pediatric drug research. This six-month exclusivity extends other regulatory exclusivities and patents when the sponsor conducts a written request study.

The Draft Guidance generally provides industry recommendations on pediatric study compliance, pediatric exclusivity qualification criteria, deferral extensions, and the incentives and protections offered by pediatric exclusivity under the BCPA. However, the combined BPCA and PREA Guidance does include a notable change—FDA's intent to limit the studies that are eligible for pediatric market exclusivity.

Pediatric Label Change Rate Prompts Policy Change

A recent data analysis led FDA to propose the alternate policy. FDA states that 768 products underwent pediatric labeling changes under PREA and/or BPCA between 2002 and 2019. The Guidance breaks down the numbers to show that 63% of the labeling changes solely involved PREA or Pediatric Rule studies, 21% percent solely involved BPCA studies, and 16% of labeling change studies were conducted under both PREA and BPCA studies.

From this data, FDA concludes that PREA requirements have led to an increase in pediatric labeling changes regardless of BPCA incentives. FDA reasons that it should therefore reserve written requests for those sponsors who perform additional pediatric studies beyond those merely required under PREA when the product may produce pediatric health benefits.

Legal requirements and enforcement under PREA necessitate pediatric labeling changes when relevant, as opposed to the voluntary incentive provided by BPCA. Relatively few pediatric labeling changes resulting from BPCA alone are expected. Most BPCA studies are conducted separately from PREA for use in the adult population.

FDA has opted to offer pediatric exclusivity for studies conducted under BPCA, but not for studies conducted under both PREA and BPCA. Although the number of pediatric labeling changes is low for studies conducted under both PREA and BPCA (16%), those labeling changes could represent a significant loss to pediatric medicine. In that regard, the Guidance fails to recognize that, without BPCA exclusivity, the 16% of pediatric labeling changes resulting from both PREA and BPCA studies may never have occurred.

Finalized PREA BPCA Guidance Impact on Industry

If finalized, the guidance would introduce a significant regulatory change from the 2005 PREA Guidance. In the 2005 Guidance, FDA states, "It is the Agency's policy to offer applicants the opportunity to qualify for pediatric exclusivity under section 505A of the Act for studies required and conducted under PREA."

The new Draft Guidance states, “In general, FDA expects that a [written request] that includes studies or planned studies required under PREA will also include additional indications or populations.” For this reason, FDA will not expect to issue a written request for a product when there are no additional indications or population studies beyond the studies required under PREA.

In short, if drug companies conduct the most useful pediatric studies without the added incentive of six-month exclusivity, then FDA should limit the additional exclusivity to studies beyond those otherwise required under PREA.

Under the Draft Guidance, FDA will award six-month extended exclusivity under BPCA when all four of the following conditions are met:

  1. Sponsor conducts pediatric studies beyond those required by PREA,
  2. FDA approves additional studies as likely to produce pediatric health benefits,
  3. Studies are conducted in accordance with an FDA-issued written request or approved proposed pediatric study request, and
  4. FDA makes an exclusivity determination at least nine months before the patent expiration date and/or regulatory exclusivity protection date to which the pediatric exclusivity will attach.

Pharmaceutical companies whose business models incorporate the six-month extended market exclusivity should consider this potential change moving forward. Failure to implement plans to qualify for the pediatric exclusivity extension could result in earlier market entry for competitors and significant financial loss from predictions based on that additional exclusivity period.

FDA accepted comments on the Draft Guidance through July 17, 2023. Docket ID: FDA-2005-D-0460.


Gregory J. Glover MD JD is a patent attorney and non-practicing physician. A noted expert on developments and emerging conflicts in the pharmaceutical industry, Greg is an expert on regulatory IP issues.

Recent articles: