The Food and Drug Administration's Prescription Drug User Fee program (PDUFA) has to be reauthorized by Oct. 1, 2007, and
all the interested parties are fine-tuning their wish lists for "improvements." Although some consumer advocates and their
Congres-sional allies blast user fees for extending industry control over the drug approval process, FDA officials, pharma
companies, and patient disease groups applaud the program's success in ending "drug lag" and speeding new drugs and biotech
therapies to market.
FDA held a meeting in November to open the PDUFA 4 debate. Acting Commissioner Andrew von Eschenbach termed PDUFA reauthorization
"critical" to FDA's ability to make "biomedical innovation a reality." Deputy Commissioner Janet Woodcock described how user
fees have been expanded twice since 1992 to support a broad range of FDA activities, from early discovery to post-market surveillance,
including improvements in agency information systems.
MORE FOR SAFETY
Now FDA wants to use fee revenues to further extend drug safety oversight and to review direct-to-consumer (DTC) advertising
before it goes public. The agency also would like to devote some of its added resources to improving the increasingly complex
drug development process, as part of its Critical Path Initiative. An FDA white paper describes the agency's case for increas-ing
appropriated funds, in addition to user fees, to support the agency's expanding public health responsibilities. [For more
information, see the white paper, available at
FDA cites an expanded workload to justify fee revisions: while new drug applications (NDAs) and biologics license applications
(BLAs) have been fairly flat, efficacy and manufacturing supplements continue to rise. FDA struggled last year to schedule
and plan for more than 2,000 industry-requested meetings and to assess nearly 350 special research protocols, many involving
new carcinogenicity and stability test methods.
FDA has launched similar discussions involving reauthorization of user fees for medical device manufacturers, which also expire
in 2007. Officials at FDA's Center for Devices and Radiological Health also seek to use more of the 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.
NO CHRISTMAS TREES
Pharma and biotech companies want to renew PDUFA but limit fee increases and the use of fee revenues for activities unrelated
to drug development and market approval. Sponsors now pay almost $800,000 to file an NDA or BLA, which spurs proposals for
more waivers and reduced fees for small companies and orphan drug developers.
The broader goal is to prevent a PDUFA reauthorization bill from becoming a "Christ-mas tree" loaded with legislation peripheral
to the drug approval process. Measures to encourage development of follow-on biologics, to establish new drug safety oversight
arrangements, to mandate completion of post-approval studies, and to boost oversight of DTC advertising are just some of the
popular proposals circulating on Capitol Hill.
However, manufacturers do support efforts to link PDUFA reauthorization to legislation that renews incentives for studying
drugs in pediatric populations, which also is up for reauthorization in 2007. The six-month patent extensions have been a
boon for pharma companies, while also generating important pediatric labeling information.
Before talking about more fees, Abbott Senior Vice President Bruce Burlington, representing Pharma-ceutical Research and Manufacturers
of America at the November meeting, urged further discussion about whether current user fee revenues are wisely spent. Industry
experts expressed support for continued development of FDA computer information systems; however, Alison Lawton of the Biotechnology
Industry Organization (BIO) questioned whether FDA's continuous market application pilot programs have been successful.