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Reimbursement procedure for off-label medications may be unreliable


In the U.S., compendia—lists of medications that summarize disease indications—may be outdated and may contain conflicting information, according to a study published online ahead of print in JAMA Dermatology (Jan. 23, 2019). Researchers say that based on these results, new policies on off-label coverage decisions may be needed, particularly in dermatologic practice where approximately 25% of prescription medications for the 10 most common dermatologic conditions are used off-label.

For patients living in the U.S. who have private insurance, physicians may be able to obtain prior approval to obtain a drug “off-label.” But patients covered under the federal government program, Medicare Part D, off-label coverage is dictated by two compendia. Clinicians cannot check for approval in advance and there is no appeals process if the compendia indicates that coverage should be denied.

“There is no real appeal process, so that rejection is the end of the story, and the rejection does not come with a suggestion of an alternative of what therapy might be approved instead,” lead author Dr. John Barbieri, Dermatology Research Fellow at the University of Pennsylvania in Philadelphia said in a press release. “This makes it incredibly challenging as a clinician, since we can find ourselves playing a guessing game while our patients wait for treatment.”

Copyright-free image from pexels.com (CC0).

Dr. Barbieri and his colleagues created a list of accepted therapies for 22 chronic, non-infectious diseases for which off-label prescribing is an important part of treatment. Then, they analyzed both compendia currently used in the U.S. for inclusion of these treatments: the American Hospital Formulary Service (AHFS) Drug Information and the DRUGDEX Information System.

The investigators found that only 73 of the 238 treatments they evaluated (31%) were included in either compendium. Among diseases, 10 of 22 (45%) had one or fewer treatments included in the DRUGDEX compendium, and 15 of 22 (68%) had one or fewer treatments included in the AHFS. For 53 out of 238 treatments (22%), the medication was included in one compendia but not the other.

“The compendia do not even agree with each other, and the literature they used as a basis for inclusion did not follow any discernable pattern and was often based on decades old sources,” Barbieri said.

Furthermore, only 56% (18 out of 32) of treatments supported with Grade A evidence (a double-blind study) were included in either compendium. The number dropped all the way down to 10% (three of 30) for Grade B evidence (a clinical trial ≥20 subjects). In addition, the compendia ignored results from randomized controlled trials while citations from single patient case-reports as well as personal communication with pharmaceutical companies were included.

Researchers suggested that mandating Medicare Part D to consider evidence from literature that clinicians present during the authorization process may be a way to reduce these inconsistencies. They also recommended the creation of an expert panel that will review appeals for therapies not included in the compendia.

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