Congress has lost a stunning opportunity to reform the Food and Drug Administration (FDA). The authorization for the agency's critical "user fees"-approximately $100 million paid annually by regulated industry to help FDA expedite the approval of new medicines-expired on October 1. The need for another five-year reauthorization provided a strong incentive for the Clinton administration to accept meaningful reforms.
Characteristically, however, the Congress settled for a half-baked compromise dictated by FDA itself and by Democratic defenders of expansive federal regulation.
FDA reforms are overdue. Physicians routinely report in surveys that FDA's policies actually threaten the well-being of their patients. In large part this is because the United States drug-approval system is significantly slower, more costly, and more intrusive than that of other industrialized nations. Seventy-three percent of drugs approved by FDA during 1987-93 already had been approved abroad. FDA has constantly sought out new mandates and promulgated new requirements, regardless of the costs to patients and regulated industry. The average time required to take a drug from research lab to patient's bedside has more than doubled since 1964, from 6.5 to 14.8 years. Between 1990 and 1993 alone, FDA pushed the average cost of bringing a new drug to market from $359 million to more than $500 million, by far the highest price tag in the world.
The legislation, which was passed on November 9 and which the White House has promised the president will sign, approaches FDA reform in several inconsequential ways.
First, it changes FDA's mission, adding the obligation for "promptly and efficiently reviewing clinical research" and making decisions "in a timely manner." But it is naïve to think that this symbolism will have any impact on the agency's 30-year tradition of risk-aversion and foot-dragging.
Second, it calls upon FDA to develop a plan by 2000 for clearing the legendary backlog of products awaiting approval. With this provision, Congress has made itself a hostage to an endless series of demands for additional resources the agency will claim are essential for meeting the required goal.
Third, it permits FDA to approve a drug for marketing on the basis of a single clinical trial where previous statutory language referred to "trials," plural. That is largely symbolic. FDA easily could have made a case for approval on the basis of a single, definitive trial under the previous language. But the point is moot: The average number of trials performed to support approval of a new drug is currently more than 40!
Fourth, it codifies many policies that are already in place, adding nothing to the status quo but attempting to convey the impression of a lengthy list of improvements.
The most important provision in the legislation offers drug companies greater latitude in supplying scientifically sound information to doctors about drugs' "off-label" uses (those not yet approved by FDA). Companies currently are prohibited from distributing such critical information.
But even this improvement comes at a high price-substantial additional paperwork involved in convincing FDA that formal applications for approval of the new uses are forthcoming. And FDA's discretion in these matters provides yet another "stick" for regulators to use on drug companies.
A welcome provision permits manufacturers to submit "health care economic information," such as data on a drug's cost-effectiveness, to hospitals and health-maintenance organizations.
The bill contains other minor improvements, such as loosened restrictions on health claims for food products and expanded use of third parties, including academic institutions, to review medical devices.
One sneaky provision actually increases the scope of FDA's regulation by expanding the agency's jurisdiction to activities that occur completely within a single state. For the first time, FDA explicitly will have regulatory authority over such small-scale research by an academic or practicing physician testing an innovative therapy. The bill also creates a new program, to be implemented by the Agency for Health Care Policy and Research and FDA, to investigate "ways to improve the effective use" of drugs and medical devices. Too bad no one informed the Congress that the era of big government is over and that such activities are now more properly performed in the private sector.
What is most worrisome about the legislation is what it fails to include.
Many critical reforms repeatedly recommended by blue-ribbon panels are conspicuously absent. These include reducing the redundancy of regulation of early-stage clinical trials, which could be overseen solely by hospital-based review boards. This pivotal change would reduce significantly the time and costs involved in clinical trials, a particular advantage for smaller companies.
Neither does the bill include a binding reciprocity provision that, for example, would limit FDA review of a new drug to a maximum of, say, 60 days after its approval in the United Kingdom or by the European Medicines Evaluation Agency. FDA would then have to show cause why the drug should not be marketed in the U.S., or it would be approved automatically.
The legislation is disappointing, and conservative congressional staffers are justifiably furious that its contents have been dictated by congressional Democrats and Clinton political appointees at FDA. Much of the blame belongs to House Commerce Committee Chairman Thomas J. Bliley (R-Va.), who represents Virginia's tobacco-producing region and is considered by many observers to be the tobacco industry's most potent lobbyist in Washington. Bliley has trod lightly in approaching FDA reform in return for the agency returning the favor on tobacco regulation.
Following the 105th Congress' failure to accomplish meaningful FDA reform, the costs of drug development will continue to rise, fewer drugs will be developed, and market competition will erode. Patients will suffer higher prices and benefit from fewer breakthrough drugs. Those are the effects of congressional Republicans' pusillanimity.
Henry I. Miller is a senior research fellow at the Hoover Institution and author of Policy Controversy in Biotechnology: An Insider's View (Austin, Texas, R.G. Landes Co. and Academic Press, 1997).