FDA Signals Softer Rules on Off-Label Promotion

June 10, 2009

The FDA does not allow pharmaceutical manufacturers to promote unapproved uses of their products, saying that evidence of success with additional indications can be given to physicians only if they request it. But the agency is on the verge of amending that rule to make it much easier for pharmaceutical companies to promote such prescribing.

The FDA does not allow pharmaceutical manufacturers to promote unapproved uses of their products, saying that evidence of success with additional indications can be given to physicians only if they request it. But the agency is on the verge of amending that rule to make it much easier for pharmaceutical companies to promote such prescribing.

An FDA internal draft guidance document (which contains nonbinding recommendations) suggests that distribution of published articles showing favorable results from an off-label use is acceptable-without a physician request and without previous FDAapproval-as long as various criteria are met. For example, the article would have to have appeared in a "reputable" peer-reviewed journal, be distributed in its entirety without abridgment or other editing, and be written by persons who do not have strong financial ties to a pharmaceutical company that might influence their results. In addition, the article would have to contain a label calling attention to the fact that the reported use is not FDA-approved and be accompanied by any published articles that show less-than-favorable results. Only formal articles, not Letters to the Editor, would be covered by the proposal.

It is not immediately clear how much opposition to the draft the FDA will encounter. Rep Henry Waxman (D, Calif), chairman of the House Committee on Oversight and Government Reform and a frequent critic of the agency, denounced the move as one that "would open the door to abusive marketing practices that will jeopardize safety" and would make pharmaceutical manufacturers less likely to go through the expense of seeking FDA approval for off-label uses.

The FDA move came as CMS's policy on off-label use is increasingly under attack. A lawsuit challenging the Medicare policy of refusing reimbursement for drugs prescribed for off-label use unless that use is listed in 1 of 4 recognized compendia was filed by the Medicare Rights Center. The practice "conflicts with standard medical practice," claims the suit, brought on behalf of Judith M. Layzer, a Medicare beneficiary who says she has paid $7208 for the fertility drug Cetrotide, which she takes as treatment for ovarian cancer. Before Layzer went on Medicare in January 2006, the court filing says, her monthly out-of-pocket copay for Cetrotide never exceeded $40 under her employer- based health care plan.