By Frances Bishopp

In a move that already has yielded positive comments from the pharmaceutical and biotechnology industries, the FDA has proposed an initiative designed to speed up development of new and supplemental uses of drugs by using all available data to determine the effectiveness of drugs and biological products.

The document, "New Use Initiative — Evidence for Primary and Supplementary Approvals," was released Thursday and is divided into two separate components: "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products," and "FDA Approval of New Cancer Treatment Uses for Marketed Drug and Biological Products."

The first draft guidance document on clinical evidence outlines the general policy. The second 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.

The new 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.

The initiative provides three standards of guidance on the quantity of evidence needed to support effectiveness, including:

* situations in which effectiveness of a new use may be extrapolated entirely from existing efficacy studies;

* situations in which there is other information from adequate and well-controlled studies in other phases of a disease, other conditions of use, different dosage forms or different endpoints;

* and situations in which a single multicenter study may provide evidence that a use is effective.

The new guidelines will streamline the process that brings medicines to patients, Donald McLearn, a spokesman for the FDA, told BioWorld Today.

What this initiative is talking about is how to provide clinical evidence of effectiveness for human drug and biological products. "What we have given people, particularly if you have a product that has already been approved, is possibly the option not to do new clinical trials. You could possibly get data from other sources such as pharmacokinetic studies or literature," McLearn said.

Lisa Raines, vice president of government relations at Genzyme Corp., of Cambridge, Mass., said the FDA has clarified that one well-controlled study is often sufficient to demonstrate safety and efficacy for the purposes of product approval. "There has been a lot of inconsistency within FDA as to whether that was the rule or not. This should be very helpful in clarifying this rule, both for companies and for staff within the agency."

"A well-designed, well-controlled multicenter study appears to be generally sufficient to get approval," Raines said.

The proposed guidance on clinical evidence of effectiveness is applicable to new drug applications, biologic license applications or efficacy supplements on what evidence should be provided to demonstrate effectiveness. The purpose of the guidance, the FDA stated, is to enable sponsors to plan drug development programs that are sufficient to establish effectiveness without being excessive in scope.

One goal of the initiative is to encourage submission of supplemental applications to add new uses to the labeling of approved drugs. "By articulating how it currently views the quantity and quality of evidence necessary to support approval of a new use of a drug, FDA hopes to illustrate that the submission of supplements for new uses not be unduly burdensome," the initiative stated.

The newly proposed policies could make it easier and less expensive for companies to obtain drug approval and, according to the Pharmaceutical Research and Manufacturers of America (PhRMA), are long overdue.

"The industry has long believed that it is necessary for FDA to update the labeling for products used in cancer research. It applauds the initiative to clarify the evidence needed to demonstrate safety for new cancer therapies," PhRMA said on Friday in a prepared statement. "The ability to conduct one sound clinical trial will help get new medicines to patients quicker, which should always be our first and foremost goal."

The guidance provides the general rules which say one study is enough, Raines said. But it also tells you all the things you must do to make sure that study will be enough, because there may be cases where one study is not enough.

"This will make sure that a standard that has been applied by some parts of the agency but not to others, is applied uniformly," Raines concluded. "This is very positive." *