Researchers at the University of North Carolina Lineberger Comprehensive Cancer Center are calling for an overhaul of the process that determines which cancer drugs used off-label are reimbursed by federally funded health insurance in the United States.
In a paper published online in the Journal of the American Medical Association, the authors raised concerns that inconsistencies exist among the five reference guides, or compendia, that the Centers for Medicare and Medicaid Services (CMS) uses to determine which drugs it will reimburse for off-label uses in cancer care. They also cited the “weak quality” of evidence used in some cases to green-light some off-label uses in oncology, which they argue could be leading to poor quality of patient care and high costs.
“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 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.”
The researchers report that the off-label use of drugs is widely practiced in oncology care. A 2013 study found that off-label uses accounted for $5 billion in cancer drug costs in 2010.
“Off-label drugs are used in many different scenarios in cancer care,” said co-author William A. 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.”
Federal law specifies that the CMS should pay for a drug that is not used in accordance with the FDA’s label when one of the five reference guides states that the drug’s off-label use is medically appropriate. Private insurers often use the compendia recommendations to inform coverage decisions.
But the authors write that previous research has found that the quality of evidence in the reference guides used is sometimes less rigorous than standards for FDA-approved drug uses. In their own analysis of off-label uses for cancer drugs, Basch and his colleagues found limitations in the level, quantity, consistency, and timeliness of the evidence cited.
The researchers offer four steps for improving the review process: 1) develop methodological standards for evaluating evidence used to back off-label drug recommendations; 2) combine the findings into one “single, rigorously developed resource” rather than five inconsistent reference guides; 3) assess whether those reviewing and determining off-label drug-use policies have potential financial conflicts of interest; and 4) make the compendia listings, which currently are available only by subscription, free to the public.
The authors also recommended that the FDA, or a public–private partnership, should oversee the decision-making process.
“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,” Basch said. “We are advocating for an approach that would be different from regular drug approval, but improving on the current system.”
Source: UNC Lineberger Comprehensive Cancer Center; August 25, 2016.