MEDICAL SOCIETIES COULD PETITION FDA FOR APPROVAL OF NEW INDICATIONS UNDER SENATE LABOR CMTE. FDA REFORM PROPOSAL; BILL LANGUAGE TO BE DRAFTED DURING RECESS

Medical societies could petition FDA for approval of clinically-accepted new indications under the Senate Labor & Human Resources Committee's FDA reform concept paper.

A medical society would be required to support a supplemental NDA with documentation showing that "(1) the new use has existed in clinical practice for at least five years; (2) the new use is common among clinicians experienced in the field; or (3) the new use represents standard medical practice," according to the committee document. FDA "would have a limited time to respond to the petition and, if it denied the petition, would have to demonstrate that the new use posed a significant risk to patients."

The blueprint for "The FDA Performance and Accountability Act of 1995" began circulating to industry and members of Congress the week of Aug. 7 and is the first FDA reform plan released by a committee chairman. Sen. Kassebaum's (R-Kan.) committee plans to draft legislative language during the August recess, which began in the Senate Aug. 12. The committee will meet with industry, patient groups and members of Congress during the break to discuss the concept paper. The medical society provision is expected to be revised. The committee plans to hold a hearing and mark up the bill in September.

By essentially eliminating the efficacy requirement for new indications, the supplemental approval provision would amount to a fundamental change in the FDA approval process.

The provision could help make FDA reform more politically palatable by emphasizing consumer access as a theme of the reform effort. The petition procedure also could help keep drug labeling in line with accepted clinical practice, a goal endorsed by physicians, industry and FDA.

However, the Kassebaum proposal appears to have a downside for industry in that it would allow outside groups to take control of the application process; the concept paper does not explicitly identify any role for industry sponsors in the process. The petition procedure could obligate companies to market products for indications they do not wish to pursue. The Kassebaum proposal for expedited review could have a similar downside for sponsors .

The proposal would allow companies greater freedom to disseminate information about off-label uses of products.

The committee proposes to allow companies to disseminate information from "medical texts and compendia; articles from peer-reviewed, scientific publications; presentations and proceedings of medical and scientific meetings; disease management programs and protocols; and complete extracts or published abstracts of such materials." These "materials could not be prepared by or for the product sponsor and would have to prominently disclose that the materials contain information about uses not approved" by FDA, the document stipulates. The sponsor also would have to include the product label or package insert.

The Biotechnology Industry Organization has proposed a broad definition of acceptable sources of off-label use information, including information from peer-reviewed articles, medical textbooks and other "generally recognized" scientific materials distributed to professionals. The Pharmaceutical Research & Manufacturers of America has suggested more narrowly restricting distribution of articles to those peer-reviewed by journals published by board-certifying medical societies. The two industry groups were not able to agree on a compromise off-label use provision and did not address the issue in their joint bill proposal ("The Pink Sheet" Aug. 7, p. 3).

In keeping with the consumer access theme of the proposal, the concept paper would write into law current FDA procedures that provide access to unapproved therapies for "serious and life-threatening illnesses and seriously debilitating conditions," including treatment INDs, parallel track, compassionate use and open label safety studies. The committee also would "ensure that these avenues are being made available consistently across the various centers and divisions."