FDA may speed up drug approvals

WASHINGTON - The FDA proposed a new use initiative to speed up the development of new and supplemental uses of medications.

The proposals would allow all available data to be used in determining how effective new drugs and biological products really are.

"We now have more ways to determine the benefits and side effects of new drugs," said Michael Friedman, M.D., the FDA's lead deputy commissioner.

For example, in some cases, a drug's effectiveness can be determined through 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 multicenter study.

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

Under the FDA's initiative, two new guidelines have been made available for comment.

One, "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products," outlines the general policy. The other proposal, "Food and Drug Administration Approval of New Cancer Treatment Uses for Marketed Drug and Biological Products," clarifies what evidence can be sufficient for supplemental applications for cancer treatments.

"Our proposal does not lower the 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.

The guidelines cite several instances in which the agency has approved new or additional indications on data other than that collected during new multiple trials.

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 the treatment of symptoms and for improving survival.

Another example of precedence with one principle outlined in the new guidelines is beta-interferon, a biologic sold as Betaseron for ameliorating symptoms in multiple sclerosis. The product was approved on the basis of a single multicenter study that showed both a decreased rate of exacerbations and a decrease in disease activity - two entirely different, but logically related, endpoints. The clinical evidence guidelines 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. PR