Permanent Pediatric Research Laws Would Spur In-House Units – J&J Exec

Enacting a permanent authorization for federal pediatric research programs would encourage pharmaceutical companies to create dedicated pediatric research programs within their operations, a Johnson & Johnson executive suggested to the Senate Health, Labor and Pensions Committee March 27.

The committee convened to discuss the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Authorization for both programs is set to expire in September with the end of the federal fiscal year.

The hearing was chaired by Sen. Chris Dodd, D-Conn., the original author of BPCA. Dodd is developing reauthorizing legislation for the program.

The programs have always had sunset clauses which terminate authorization every five years.

Despite the success of the two programs in stimulating pediatric drug development - there has been a 20-fold increase in the number of drugs tested for children in the last decade - J&J Vice President and head of the J&J Pediatric Drug Development Center of Excellence Samuel Maldonado said the uncertainty created by the sunsets discourages companies from developing dedicated pediatric study units.

Eliminating the sunsets "will provide a platform from which those companies that have made investments in pediatric drug development infrastructures can confidently increase those investments, including expansion into new research areas."

By "'infrastructure,' I mean much more than merely brick, mortar and layers of management," Maldonado continued.

"The building of sustainable pediatric research units from company to company and across the board means training people to be better researchers in pediatrics, developing new and better tools for measuring outcomes in pediatric clinical trials and fine-tuning mechanisms of study ... Suffice it to say this requires significant and sustained investment."

Further, in the "absence of a consistent and predictable exclusivity provision, there will remain a considerable and understandable reluctance among companies with countless competing research priorities to dedicate resources to formal pediatric divisions," Maldonado said. "This is especially true as the cost, size, number and complexity of pediatric studies has increased and the absolute value of the pediatric exclusivity has decreased."

A recent survey of 17 companies by Tufts University researchers found that one-third had hired staff to handle pediatric studies, started a research unit or created a pediatric advisory council.

In 2006, all companies surveyed outsourced all work related to their pediatric trials (¹ (Also see "Pediatric Studies Become Costlier; Congress May Make Them Less Profitable" - Pink Sheet, 19 Mar, 2007.), p. 23).

Both BPCA and PREA address FDA authority in the area of pediatric studies; BCPA permits FDA to provide six months additional marketing exclusivity to sponsors who conduct pediatric studies at the agency's request; PREA codifies FDA's authority to require pediatric studies in certain circumstances.

By the end of 2006, FDA submitted 336 written requests for 782 pediatric studies under BPCA, resulting in more than 300 pediatric studies and labeling updates for 119 drugs, according to the Tufts study.

In the seven years prior to BPCA, only 11 studies of marketed drugs were completed.

While Maldonado advocated permanent reauthorization for both PREA and BPCA, both Dodd and Clinton had reservations about the latter.

"I'd be tempted as author of this legislation to see it become permanent, and I heard testimony of those who want that," Dodd said. "I've also watched enough changes be occurring here [to think that] it'd be worthwhile for us to go back periodically and take a look at how we're doing in this area."

Clinton added, "I think there is agreement that the BPCA act is one that a sunset is important for, to make sure that the incentives that Sen. Dodd designed are working the way they should."

- Jamie Hammon