The US Food and Drug Administration is setting the stage for a broad debate over how much the agency should rely on industry
payments for support, and how much control FDA should have over how user-fee revenues are spent. FDA officials would like
to allot more fee revenues to drug-safety surveillance, an area that continues to draw attention from Congress as well as
the media. Another challenge for the agency is to encourage and regulate new vaccines and drugs to protect against pandemic
disease and bioterrorism. All these initiatives will stress FDA's tight budget for the coming year, which relies increasingly
on user fees to support its expanding responsibilities.
FDA opened the debate about reauthorizing the Prescription Drug User Fee Act (PDUFA 4) at a public meeting this past November.
FDA Acting Commissioner Andrew von Eschenbach termed PDUFA reauthorization critical to FDA's ability to bring new medical
discoveries to patients. FDA Deputy Director Janet Woodcock described how user fees support a broad range of FDA activities
from early discovery through postmarket surveillance. The added resources from user fees over the past decade have improved
the new drug application (NDA) approval process, noted Steven Galson, director of the Center for Drug Evaluation and Research
(CDER). Most NDAs need only one review cycle to gain approval, he pointed out, and the scientific expertise of CDER's staff
has improved noticeably. An FDA white paper explains the agency's case for more appropriated funding and user fee collection
to support its more-complex and costly drug review process (white paper available at
Similar discussions have begun involving reauthorization of user fees for medical device manufacturers, which also expire
in 2007. Officials at FDA's Center for Devices and Radiological Health want to use some fees collected under the Medical Device
User Fee and Modernization Act of 2002 for postmarket surveillance, a proposal gaining momentum following recent safety crises
involving pacemakers and defibrillators.
Although some consumer advocates and their Congressional allies criticize user fees for extending industry control over the
drug approval process, FDA officials, pharmaceutical manufacturers, and patient-disease groups applaud the program's success
in ending drug lag and speeding new therapies to market. At the same time, FDA officials seek more flexibility that will allow
it to use more of the $250 million user-fee revenues to expand postapproval drug safety surveillance, boost scrutiny of direct-to-consumer
advertising, and support collaborative research activities under its Critical Path initiative.
FDA cites a growing workload to justify fee revisions. Although NDAs and biologics license applications (BLAs) remain fairly
flat, manufacturing and efficacy supplements continue to rise, and chemistry and manufacturing controls supplements have doubled
from 1247 in 1993 to 2481 in 2004. In addition, FDA struggled this past year to prepare for more than 2000 industry-requested
meetings and to assess nearly 350 special research protocols under tight deadlines, including requests for FDA to evaluate
new carcinogenicity and stability-testing methods.
No Christmas trees
Manufacturers basically want to renew PDUFA but hold the line on fee increases and the use of fee revenues for activities
unrelated to drug development and market approval. It now costs sponsors nearly $800,000 to file an NDA or BLA, an amount
spurring proposals for more waivers and reduced fees for small companies and orphan drug developers.
Industry's broader goal is to prevent a PDUFA reauthorization bill from becoming a "Christmas tree" loaded with legislation
peripheral to the drug approval process. Measures to spur development of follow-on biologics, to establish new drug safety
oversight arrangements, to mandate completion of postapproval studies, and to boost oversight of direct- to-consumer advertising
are just some of the popular proposals circulating on Capitol Hill.
Manufacturers have no problem, though, with linking PDUFA legislation to a bill reauthorizing incentives for studying drugs
in pediatric populations, which also is up for renewal in 2007. The six-month patent extensions on drugs that add pediatric
information to labels have been a boon for pharmaceutical companies, while also generating important prescribing information
for physicians and patients. Generics makers object that the patent extensions only boost drug costs for everyone. But, doctors
applaud the new pediatric formulations and useful prescribing information generated by the program.