Three–Quarters of U.S. Health Plans Reimburse for Off–Label Uses of Prescription Drugs

According to a study recently completed by the Tufts Center for the Study of Drug Development, (CSDD), 75 percent of all health plans in the United States now reimburse patients for off-label prescription drug treatments. 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. The Tufts CSDD study, reported in the March/April Tufts CSDD Impact Report, 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 80% of plans ascribed a role to cost-effectiveness in off-label use reimbursement decisions; almost 20% said cost-effectiveness data play a “very important” role.