Treatment Issues, Vol 11, No 10; October 1997
Derek Link

The most important feature of the new legislation is that it is incorporated into renewal of the Prescription Drug User Fee Act. PDUFA established a system of fees paid by the drug industry to help support FDA activities. The fees amount to $100 million annually for the FDA and have been responsible for many of the agency's performance improvements over the last five years.

Many provisions of the FDA legislation go beyond the renewal of PDUFA and change the power and authority of the agency in several substantial ways. The most important change relates to the dissemination of "off-label" information. The new legislation allows manufacturers to publicize both approved and unapproved uses of their products. (Under certain circumstances, manufacturers are required to submit, or have plans to submit, a supplemental application to the FDA regarding promotion of off-label drug use. However, there is no mechanism in place for the FDA to review these applications and no approval is required.) In contrast, current regulations allow drug manufacturers to promote only those uses that have been certified by the FDA as safe and effective and that appear in the products' officially approved package insert. (Doctors are free to prescribe medicines for off-label uses, but they have to learn of such applications from noncorporate sources.)

Patient advocates fought the off-label promotion proposal the hardest, but to no avail. It was ironic indeed that during debate on this issue, the "Fen-Phen" disaster hit the headlines. Fen-Phen, a popular diet drug combination that was publicized heavily by weight loss "clinics" even though it was never approved by the FDA, was withdrawn from the market after reports of heart valve defects and pulmonary hypertension in many users emerged. Although this was a classic "off-label" scenario, it had no impact on the congressional debates. In fact, the committee debates on this bill criticized the FDA for allowing Fen-Phen to happen, but then went ahead and passed the off-label provisions anyway.

The new legislation also lifts the requirement for maintaining a registry of recipients of high-risk medical devices, such as heart valves. This registry is vital when patients must be contacted about a newly discovered defect. The registry requirement grew out of the experience with the Shilley heart valves, which were used in the early 1980s and developed leaks. To this day, many patients with the valves have not been contacted because, at the time, no registry was mandated. Under the new PDUFA, companies can choose if they want to maintain the registry.

One provision of the renewed PDUFA will have an immediate negative impact on AIDS: Under the new PDUFA, no monies raised through user fees can be used for FDA-sponsored research. Because of this restriction, the Antiviral Research Laboratory in the Division of Antiviral Research, which relied heavily on PDUFA funding, is slated for permanent elimination. This lab, located in the division that reviews most AIDS drugs, has supported many areas of AIDS research. It helped standardize viral load assays, developed new model systems for preclinical testing of therapeutic agents against HIV, HBV, HCV, and Mycobacterium avium, and had a particular focus on the intracellular metabolism of nucleoside analogs.

But the House did enact a provision sought by patient advocates, although the Senate failed to include the measure. The House voted to require a sole manufacturer of a drug for a serious or life-threatening disease to notify the agency six months before it plans to stop making the drug. The Senate has no language in its bill on this provision. Advocates hope to maintain the House language in conference negotiations.

This year's FDA reform debates were of a different tone and character than those seen last year. Advocates worked hard to ensure that the voice of patients was heard in Washington. Although there are many negative features to this year's bill, the renewal of PDUFA and the generally more moderate approach this year, have produced a superior bill to last year's extreme proposals (see Treatment Issues, May 1996, page 7). President Clinton is expected to sign the legislation when it emerges from conference negotiations.

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