Summary

Responsible for regulating the testing and marketing of all human drugs in the United States, the Food and Drug Administration (FDA) must ensure that the public health is protected by carefully assessing the risks and benefits associated with new drugs and making such drugs available to the public as soon as possible. The Federal Food, Drug and Cosmetic Act gives FDA the legal authority and responsibility for regulating and approving new drugs. The Act requires that FDA approve new drug applications (NDA's) within 180 days, or about 6 months after they are filed, or that FDA give an applicant notice of an opportunity for a hearing on the application's deficiencies. Where more time is needed, the Act permits the time to be extended by mutual agreement between FDA and the applicant. The GAO review of the FDA drug approval process consisted of: (1) obtaining the views and concerns of the drug industry, pharmaceutical associations, and other knowledgeable members of academia and FDA officials; (2) comparing drug approval procedures in Canada and eight European countries with those of the United States; (3) analyzing the FDA review process for selected NDA's; (4) analyzing the work load of FDA physicians, chemists, and pharmacologists involved in reviewing NDA's; (5) interviewing FDA reviewers of NDA's for their perceptions of the drug approval process; and (6) reviewing the FDA use of scientific and management information systems in its drug approval activities.

Of the 132 NDA's submitted in 1975, 52 percent had been approved by the end of May 1979. The average approval time for these applications was about 20 months. Many important drugs were approved in some foreign countries in less time than in the United States. Both FDA and the drug industry contribute to the time it takes to approve new drugs. Major factors affecting drug approval time were: (1) imprecise FDA guidelines, subject to varying interpretations; (2) scientific and professional disagreements between FDA and industry; (3) slow or inadequate FDA feedback to industry and a lack of promptness in notifying drug firms of deficiencies in applications; (4) lengthy chemistry and manufacturing control reviews; (5) limited time spent reviewing and uneven work load; and (6) incomplete NDA's and industry's slow rate of resolving deficiencies. Practices and policies in countries that tended to speed up approval included the development of postmarketing surveillance systems in which widespread usage of drugs after marketing indicate the safety and efficacy which is not obtainable through controlled clinical trials; the use of an expert committee to review and approve, or recommend approval of, important new drugs; and acceptance of foreign clinical data to demonstrate the safety and efficacy of a new drug. Legislation has been proposed which would require drug firms to collect and report adverse drug reaction information and provide informal procedures for resolving scientific disagreements between FDA and drug firms.