Reflecting recent rapid growth of off-label prescribing of prescription drugs, 75 percent of all health plans in the United States now reimburse patients for such treatments, according to a study recently completed by the Tufts Center for the Study of Drug Development. Of health plans that reimburse for off-label uses, over half impose conditions, including step therapy, indication restrictions, prior authorization, and quantity limits, and nearly 90% rely on pharmaceutical compendia to inform their off-label use reimbursement decisions, the study found. The findings were based on a Tufts CSDD survey of 179 third-party payers in the U.S. that administer Medicare and Medicaid pharmacy benefits. "Since patients do not pay directly for most off-label uses, third-party payers have become crucial for market access," said Joshua Cohen, a senior research fellow at Tufts CSDD and lead investigator on the study. "While reimbursement decisions with respect to on-label indications are relatively well understood in terms of evidence requirements, off-label use reimbursement until now has been less transparent." The practice of off-label use involves prescribing medicines for a purpose outside the scope of a drug's intended purpose, as indicated on its label. Once a drug is approved in the U.S., the Food and Drug Administration does not regulate its use, and doctors may prescribe the drug for an off-label use. Cohen noted that with off-label prescribing likely to increase, "health plans will need to balance the value of rapid patient access to the best pharmaceutical care possible, sometimes through the off-label use of a drug, with the need for comprehensive data on the benefits and risks of such care." The Tufts CSDD study, reported in the March/April Tufts CSDD Impact Report, released today, found that: Sixty percent of the health plans surveyed require that off-label uses must be referenced in one or more pharmaceutical compendia to be reimbursed. More than three-quarters of health plans ascribed a "very important" role to peer-reviewed literature as a factor in determining off-label use reimbursement decisions. Almost four-fifths of plans ascribed a role to cost-effectiveness in off-label use reimbursement decisions; almost one-fifth said cost-effectiveness data play a "very important" role.