When Talk is Not So Cheap

Mar 01, 2012

Jill Wechsler
Hearings last month before the House Energy & Commerce health subcommittee provided a forum for the opening salvos in the user fees renewal campaign needed to keep the FDA going. Commissioner Margaret Hamburg made the case for renewing the Prescription Drug User Fee Act (PDUFA V) and legislation to spur pediatric drug labeling; Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), explained the importance of new fees for generic drugs and biosimilars; and Jeffrey Shuren, director of the Center for Devices and Radiological Health (CDRH), outlined a hard-fought agreement on the medical device user fee program (MDUFA).

Marketers, providers, and FDA leaders want to enact a user fee bill by summer, but the legislators indicated they have pet issues to tag onto this must-pass legislation. The most likely addition is a measure enhancing FDA controls over the global drug supply chain, including stiffer import controls, more inspections of foreign operators and (most likely) wider manufacturer notification about looming drug shortages. Both Democrats and Republicans want to boost foreign inspections to level the playing field for U.S. firms, and new user fees for generic drugs provide needed funding for these activities.

More contentious is whether to loosen conflict-of-interest (COI) requirements for advisory committee members that Republicans say are too stringent and can delay new drug approvals. Orphan drug advocates similarly claim that stiffer restrictions on FDA advisors keep out key scientists in specialized research fields. However, commissioner Hamburg said that the current COI policy and waiver caps are not a problem, and that the issue doesn't need a "legislative fix." That was a shift from her testimony last year that the COI rules made it difficult to find sufficiently qualified individuals to serve on advisory panels.

There was debate over proposed changes to the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), which both offer incentives for manufacturers to study the safety and efficacy of drugs for children and are up for five-year renewals as part of the PDUFA process. The programs enjoy strong support from health professionals, researchers, and pharma companies, but a number of reform proposals have surfaced.

Industry opposes any reduction in the extra six months' exclusivity given for doing requested pediatric studies, as well as new penalties for failing to complete requested studies on schedule. But there's support for FDA to be able to levy fines on companies that miss deadlines. The American Academy of Pediatrics (AAP) also wants pharma companies to submit pediatric study plans at the end of Phase II, instead of waiting until application submission, and to include neonates in more studies. Researchers and sponsors agree that PREA requirements for pediatric studies should be made permanent, but the docs support the Democratic position that the exclusivity provisions authorized by the BPCA should continue to be re-evaluated every five years.

In addition to providing more information on therapies for children, PDUFA may contain new proposals to spur development of critical therapies, including antibiotics to combat drug resistance and treatments for rare conditions. There's bipartisan backing for the "Sprint Act" (Spending Reductions through Innovations in Therapies), which would provide $50 million to fund public/private partnerships doing research on critical conditions like Alzheimer's disease. However, FDA and sponsors are leery of measures that prevent the agency from seeking necessary clinical data to approve a new drug, as sought by smaller biotech firms and patient groups clamoring for more certainty in the approval process.

Complaints about the proliferation of Risk Evaluation and Mitigation Strategies (REMS) may be off the table due to FDA efforts to scale back that program. But there may be some sparring over revising FDA's mission statement to highlight its role in creating jobs and economic growth. Hamburg noted at the Energy & Commerce hearing that FDA would have trouble factoring jobs creation into decisions on new product approvals. "It will get very, very complicated and, frankly," she said, "I think it would be quite inappropriate."

lorem ipsum