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
“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
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,
* 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
* 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
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