Longer view on drug safety: New power in FDA law

The recent spate of high-profile withdrawals of, and safety alerts for, approved prescription drugs tarnished the Food and Drug Administration's image as a protector of public health. Bipartisan legislation President Bush signed into law Sept. 27 goes a long way toward restoring faith in the agency.

As the Institute of Medicine pointed out in a September 2006 report, FDA approval does not represent a lifetime guarantee of drug safety and efficacy. The clinical trials on which the agency's decisions are based are too short-lived, conducted on too few people in too controlled a setting for such certainty.

Understanding a drug's true risks and benefits requires attention over the medication's entire lifespan. The drug safety problems that received so much attention over the past few years highlighted the lack of such a "life-cycle" approach, as the IOM calls it, in the government's system.

The problem was that the FDA lacked the clear authority to require studies of drugs that presented safety concerns after they were on the market. The agency could ask drugmakers to conduct further studies but could not force them to do so. Hundreds of agreed-upon studies remain pending in perpetuity, according to an article in the Oct. 26, 2006, New England Journal of Medicine.

The Food and Drug Administration Amendments Act of 2007 now gives the agency the authority to require postmarket drug safety studies. The FDA also can require label changes, medication guides or package inserts.

The law does not allow the FDA to order such actions randomly. Evidence of a safety risk first must be present.

In the case of drugs that have known causal relationships to serious adverse events, the agency can require physicians prescribing the medication to have particular training, experience or certification. For these drugs, the FDA could require that patients be enrolled in a drug registry, that patients be subject to monitoring or that the medication be dispensed only in certain settings.

This step is essentially a codification of current government practices. An earlier version of the legislation went too far in allowing FDA oversight of the practice of medicine. It would have endangered patients' access to drugs -- particularly in rural and underserved areas. Sen. Tom Coburn, MD (R, Okla.), working with the AMA, took action to ensure that the law preserves physician discretion in prescribing.

The act makes sure that the FDA will have the necessary information on which to base rulings that postmarket actions are needed. It requires the agency to monitor multiple public and private electronic databases for adverse event patterns.

Of course, for the system to work, it is important that doctors report adverse drug events to the FDA. They can do this by logging onto the agency's MedWatch Web site (link).

The law recognizes the power of information. It makes participation in the National Institute of Health's clinical trial registry mandatory for those who are conducting such studies and expands the information posted to include trial results. Emphasis is placed on making the data easily searchable online.

Some types of information can be dangerous. With that in mind, the law gives the FDA the power to review direct-to-consumer drug advertising before it hits the airwaves. If the agency determines that an ad would be false or misleading because it fails to disclose a serious risk, it can require the manufacturer to include notification in its ad. Violators face civil fines in the hundreds of thousands of dollars.

The law reauthorizes for five years the Prescription Drug User Fee Act -- a major source of FDA funding. It increases fees from pharmaceutical companies by about $87 million a year, bringing total PDUFA funding to about $393 million annually. Some of this extra funding will pay for the agency's new postmarket surveillance activities.

No drug is risk-free. Safety concerns are bound to surface periodically after drugs are allowed in Americans' medicine cabinets. This law gives the FDA some of the tools necessary to step in when such instances occur without unduly infringing on physicians' professional judgment in prescribing.