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Date: Thursday, March 13, 1997
FOR IMMEDIATE RELEASE
Contact: Food and Drug Administration Donald McLearn:(301)443-1130
Broadcast Media: (301) 827-3434, Consumer Hotline:(800) 532-4440

FDA's initiative gives industry clear guidance on when the agency can determine that a drug is effective for a new use without requiring data from two new clinical trials. For example, in some cases a drug's effectiveness can be extrapolated from existing efficacy data; it can be shown by evidence from a new single trial supported by already existing related clinical data; or it can be documented by adequate evidence from a single multi-center study.

"The science of drug development and clinical evaluation has evolved so significantly that we now have more ways to determine the benefits and side effects of new drugs," said Michael A. Friedman, M.D., FDA's Lead Deputy Commissioner.

"This initiative outlines how we can simplify the approval process while continuing to uphold standards that have earned the public's confidence."

Under FDA's "New Use Initiative -- Evidence for Primary and Supplementary Approvals," two new guidances are being made available for comment.

One of them, "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products" outlines the general policy. The other proposal, "FDA Approval of New Cancer Treatment Uses for Marketed Drug and Biological Products," clarifies what evidence can be sufficient for supplemental applications for cancer treatments, and describes steps the FDA is taking to foster the updating of labeling for products used in cancer treatment.

"Our proposal does not lower FDA's commitment to high effectiveness standards: it identifies situations in which multiple new clinical trials are not needed," said Janet Woodcock, M.D., director of FDA's Center for Drug Evaluation and Research. "In some instances, we can rely on published scientific reports."

"The initiative allows more flexibility in the assessment of biological products," said Kathryn Zoon, M.D., director of FDA's Center for Biologics Evaluation and Research. The guidances cite several instances in which FDA has approved new or additional product uses on data other than that collected during new multiple trials.

For instance, when the course of the disease and the beneficial effects of the drug are sufficiently similar for both adults and children, the agency has allowed the Pediatric Use section of product labeling to include information extrapolated from adult efficacy data. Examples of such pediatric labeling include ibuprofen, a non-steroidal anti-inflammatory drug, and ondansetron, a treatment for chemotherapy-induced nausea.

In the case of enalapril, a drug for heart failure, the agency accepted two different effectiveness findings, each from a different study, one of which showed symptom improvement and the other improved survival. The drug was approved for both treatment of symptoms and improving survival.

Yet another example of a past application of one of the principles outlined in the new guidelines is beta-interferon (Betaseron), a biologic for ameliorating symptoms in multiple sclerosis. The product was approved on the basis of a single multicenter study which showed both a decreased rate of exacerbations and a decrease in disease activity -- two entirely different, but logically related, endpoints. The Clinical Evidence guidances, however, caution that care should be taken when relying on a single clinical trial, and stress that the quality of scientific evidence is as important as its quantity.

The agency will continue to explore how to improve the supplemental application process for sponsors of all approved products with promising but unlabeled uses.