Drugmakers have urged Congress to act speedily to reauthorise the Prescription Drug User Fee Act (PDUFA) - under which the Food and Drug Administration receives fees from manufacturers for approving their products – before the current law expires on September 30, but patient groups have condemned the agency’s reliance on money from industry.
The reauthorisation bill (PDUFA IV) is one of the most important legislative issues facing Congress this year - important new medicines are now available to patients much more quickly as a direct result of the law, Bruce Burlington, executive vice president for business practices and compliance at Wyeth, told a hearing of the Senate Health, Education, Labor and Pensions (HELP) Committee. “By virtually any measure, PDUFA has been a resounding success,” said Dr Burlington, who served as the FDA’s acting deputy director for medical affairs when PDUFA was enacted in 1992.
Also testifying was FDA Commissioner Andrew von Eschenbach, who told the panel that the user fee legislation has provided the public with access to 1,220 new drugs and biologics. During the PDUFA era, he said, FDA reviewers have approved 76 new medicines for cancer, 178 anti-infectives, including 56 for HIV or hepatitis, 111 medicines for metabolic and endocrine disorders, 115 drugs to treat neurological and psychiatric disorders and 80 treatments for cardiovascular and renal disease.
Moreover, the FDA now reviews drugs “as fast or faster than anywhere in the world,” he said; the median approval time for priority new drug and biologic applications dropped from 14 months in 1993 to just six months in 2006.
However, user fees have not kept up with costs, and the FDA wants to raise them by around $100 million to $393 million in 2008. The agency’s goals for PDUFA IV are to: create a sound financial footing at the agency; enhance its pre-market review process; modernise and transform the post-marketing drug safety system; and establish a new program to review direct-to-consumer television advertising, said the Commissioner.
It is critical that reauthorisation occurs seamlessly, he stressed, as any hesitation or delay “could trigger sudden erosion in our work force, particularly among senior reviewers whose skills are in very high demand. The repercussions of such a loss would be with us for years to come,” he warned the legislators.
However, a leading patient advocate told the hearing that PDUFA is insufficient and “no substitute” for increasing FDA funding. “Congress needs to provide FDA with more authority, increased appropriations and more consistent agency oversight,” said Diane Edquist Dorman, vice president for public policy at the National Organization for Rare Disorders (NORD). She described the agency as “hardworking and well-meaning,” but said it needs to remember that regulated industries are stakeholders, not customers, and that “it is the patients and consumers who may live or die and who are most at risk based on the quality and independence of its decisions.”
NORD is concerned that the FDA’s PDUFA IV recommendations will deter small companies from developing new orphan drugs, while its proposals to use $37.5 million of industry fees to enhance postmarketing safety and an extra $4 million to strengthen the information technology infrastructure for drug reviews are “inadequate,” said Ms Edquist Dorman.
FDA needs $2 billion, plus user fees, for next year
She urged the HELP panel to become “activists” for agency funding. The FDA needs $2 billion from Congress for FY2008, up $450 million on FY2007 and in addition to user fee revenues, in order to restore the agency to its FY2003 funding level and fund the extra responsibilities it has taken on since then, she said.
The user fee program was condemned by Kim Witczak of advocacy group Woodymatters, who said consumers, most legitimate patient groups and the Institute of Medicine are “deeply troubled” by it. The FDA should be funded totally out of the general Treasury and, if user fees are needed, there should be no conditions attached on how that money is spent. In comparison, the conditions within the current PDUFA system are “morale-destroying,” she claimed.
As “the board of the directors of the FDA,” the HELP Committee needs to make sure that the agency “stops wasting precious time and money,” said Ms Witczak. For example, it needs computer resources to be able to detect medicine dangers faster. The “heavily-censored” Breckenridge Institute analysis of the FDA’s efforts to modernise the Adverse Event Reporting System “describes incompetence and waste that is breathtaking,” she added.
However, “if the culture of the FDA became one of openness, there would be fewer future drug disasters, and I gather it would be a much better place for scientists to work,” said Ms Witczak. She called for the results of Phase I clinical trials to be published, which would, among other things, speed drug discovery at lower cost, and also for the details of all FDA decisions to be made public within a month or two of approval “so that the world can see what the issues are and what needs more study.”
She also backed proposals by Senators Charles Grassley (Republican) and Chris Dodd (Democrat) for a separate, independent Office of Drug Safety, whose director should have authority and responsibility to say they believe there are enough safety questions about a drug, pre-or post-approval, for it not to be approved, or that it should be pulled from the market. If the head of the Office of New Drugs disagrees with this decision, the two officials could present their cases to the Commissioner, who should make a decision within a day, she proposed.
Ms Witczak dismissed suggestions that such a process would slow approvals and create a duplicate bureaucracy. There would be no new expense and the two Offices would still work together, but there would be accountability. “It would make a career professional physician-scientists responsible for standing up or safety when he thinks the facts justify it,” she said By Lynne Taylor.
For details of the FDA’s 2008 budget request, visit: http://www.fda.gov/oc/oms/ofm/budget/documentation.htm