The Pharmaceutical Research and Manufacturers of America (PhRMA) urged Congress to reauthorize two pieces of legislation relating to pediatric studies and exclusivity, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). PhRMA made the comments in testimony in a public meeting before the Institute of Medicines (IOM), the health arm of the National Academy of Sciences.

“The combination of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) has driven research to create innovative medicines for children younger than 18 and has greatly advanced American children’s medical care,”  said PhRMA Vice-President of Scientific and Regulatory Affairs Sascha Haverfield, in a PhRMA press release. “…PhRMA urges Congress to permanently reauthorize these two important Acts to allow pediatric research to continue to thrive and to create more therapeutic options for children and healthcare providers.”

PhRMA further noted that permanent reauthorization of BPCA and PREA would facilitate FDA in developing and issuing guidances on pediatric drug development. “Pediatric product development would benefit from updated regulatory guidance from the Food and Drug Administration to help both the innovator industry and the Agency’s reviewers achieve a mutual understanding of the requirements under the law,” said PhRMA in its press release. “Because of the current five-year sunset and reauthorization cycle for BPCA and PREA, no such current guidance exists. Each authorization changes the law. If Congress were to permanently reauthorize both Acts, the FDA would be in a better position to publish and maintain up-to-date guidance.”

PhRMA said that 181 medicines have completed the program requirements established under BPCA, and since 2007, 324 pediatric studies have been completed. The association said that 230 drugs are currently in development to meet the special needs of children.

In its testimony before IOM, PhRMA also urged for more timely regulatory review of pediatric drugs. “While existing guidance on BPCA estimates a 120-day review period for response to a Proposed Pediatric Study Request, the experience of companies has been variable across FDA review divisions,” said PhRMA. “Further, there are currently no specific timeframes for FDA action on a proposal for an amendment to a Written Request. Having predictable timelines is greatly important. In extreme cases, time delays can result in a sponsor’s decision not to proceed with a pediatric program.” PhRMA noted that the reauthorization of BPCA in 2007 included a provision that requires that written requests be fulfilled 15 months prior to expiration of product market protection. “This time limitation highlights the need for timely and clear communication between the sponsors and FDA,” said PhRMA.

With regard to PREA, sponsors are required to commit to timelines for completion of PREA requirements prior to approval of the product, noted PhRMA. Although many FDA review divisions respond to PREA proposals early in the development of a medicine, the formal commitment is for the plan to be submitted at the time of the submission of a new drug application or biologics license application. “Once PREA commitments are agreed upon, usually at the time of product approval, there are no timing goals for FDA review divisions to respond to companies on PREA-related topics. Such review time goals are necessary to enhance pediatric drug development,” said PhRMA.

PhRMA also pointed to ongoing cooperation between the US and European Union for harmonization for provisions relating to pediatric drug development and the need to continue such cooperation, including agreement on pediatric plans and timing of pediatric plans.

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Waivers and Deferrals Under the Pediatric Research Equity Act (August 2009)
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