Off-Label Reimbursement Guidelines for Oncology Drugs Need Revisions

Concerns were recently raised regarding 5 reference guides (compendia) that the US Centers for Medicare and Medicaid Services (CMS) utilizes to reimburse drugs used off-label for cancer.

A group of physician-researchers from the University of North Carolina Lineberger Comprehensive Cancer Center called for this process to be overhauled in a study published by the Journal of the American Medical Association. The physicians said there is not substantial evidence for some approved off-label uses of cancer treatments, which could lead to high costs and poor patient outcomes.

“The quality and consistency of these very important compendia resources is not what it should be, given the level of scrutiny that's appropriate for highly toxic and expensive cancer drugs,” said study corresponding author Ethan Basch, MD, MSc. “We could be causing substantial suffering for cancer patients because of the sometimes cavalier use of off-label drugs. A new, more rigorous approach is warranted in order to protect our patients.”

Since off-label use is a common practice in oncology, a more standardized approach to covering off-label use is needed.

“Off-label drugs are used in many different scenarios in cancer care,” said co-author of the study William Wood, MD, MPH. “They can be used if treatment options are limited in a certain situation, or if a clinician wants to try something unconventional, which could be a high quality decision or a poor quality decision. The concern is that people could be getting treatments that are inappropriate and that the risks may be greater than the benefits of these treatments.”

In accordance with federal law, the CMS should reimburse a drug used outside of the FDA indication if 1 of 5 reference guides deem the off-label approach medically appropriate, the study noted. Private insurers also refer to the guides; however, the quality of the evidence provided in the guide is not always as stringent as the FDA’s standards for approved uses.

According to the study, there were limitations in the level, quantity, consistency, and timelessness of evidence.

The physicians also outlined 4 steps to improve the process: create standards for evaluating evidence that supports off-label use; create a single reference guide; determine whether people reviewing the evidence for off-label use have a conflict of interest; and make the reference guide listings free to the public instead of charging a subscription fee.

They also recommend the FDA or public-private partnership should oversee the process, according to the study.

“The FDA has the ability to review evidence using strict standards, and the purview of the FDA over oncology is increasing with the development of the FDA Oncology Center of Excellence,” Dr Basch concluded. “We are advocating for an approach that would be different from regular drug approval, but improving on the current system.”