Medicare Part D Policy on Off-Label Treatments Thwarts Patient Access, Study Says

A recent study examining Medicare Part D reimbursement policies for off-label uses, using dermatology as a focus, found that health coverage decisions have significant shortcomings that could hamper patient access to evidence-based treatments.
Allison Inserro
February 09, 2019
A recent study examining Medicare Part D reimbursement policies for off-label uses, using dermatology as a focus, found that health coverage decisions have significant shortcomings that could hamper patient access to evidence-based treatments.

The assessment of the literature cited by the compendia that Medicare Part D uses to make payment decisions found that there was no clear pattern when it came to determining the level of evidence that was required. Moreover, some indications were supported by a single reference, such as a case report or even personal communication with pharmaceutical companies. Other evidence had multiple studies, but some of the literature cited was decades old, including references to literature published before the 1980s.

Medicare Part D will not pay for therapies that are off label, but the authors, all dermatologists, note that the specialty treats more than 3000 diagnoses, some of which are rare. They often prescribed treatments that are off-label. Yet in Part D, there is no appeals process, and physicians said they have no way of checking for approval in advance.

The JAMA Dermatology study looked at the 2 compendia that Medicare Part D uses to make coverage decisions: the American Hospital Formulary Service (AHFS) Drug Information and the DRUGDEX Information System. A cross-sectional study was conducted from July 1, 2018, through September 30, 2018. Researchers compiled a list of 22 chronic, noninfectious, nonneoplastic diseases with at least 4 systemic therapies for evaluation.

Just 73 of 238 treatments evaluated, or almost 31%, were included in either compendium. By individual disease, 10 of 22, or 45%, had 1 or fewer treatments included in the DRUGDEX and 15 of 22, or 68%, had 1 or fewer treatments included in the AHFS compendium. Discrepancies where medication was included in 1 list but not the other occurred in 53 of the 238 drugs, or 22.3%.

Interestingly, considering that they were studying a government payer, the authors noted that some less expensive medications were excluded from the compendia, whereas more expensive medications were included. For example, none of the 7 off-label treatments evaluated for hidradenitis were included on either list, but adlimumab, which is FDA approved for that condition, was included in DRUGDEX.

Patients who are unable to access cost-effective off-label treatments and are forced to use the costlier medications would have higher costs, as would the government, the authors noted.

“There’s 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,” the study’s lead author, John Barbieri, MD, a dermatology research fellow at the University of Pennsylvania, said in a statement. “This makes it incredibly challenging as a clinician, since we can find ourselves playing a guessing game while our patients wait for treatment.”

Regarding the quality of the evidence used by the compendia, 56% of treatments with grade A evidence (a double-blind study) were included in either one. The number dropped to 10% (3 of 30) for grade B evidence but rose to 12% (5 of 43) for grade C evidence and 13% (8 of 61) for grade D evidence. Results from randomized controlled trials were ignored.

“It was not uncommon for first line therapies with a grade A or B evidence to be missing from the compendia, while second- or third-line therapies with a lower evidence grade were included,” Barbieri said.

The authors suggested a few ways by which the compendia shortcomings could be fixed. They include developing a list specific to dermatology, although they said this could be impossible or impractical given the number of diagnoses in the specialty.

Another option would be to force Medicare Part D to use evidence from the literature presented by clinicians during a prior authorization process to support coverage determination appeals. Alternatively, an expert panel could be developed to review appeals for therapies not included in the compendia.

Barbieri JS, Claire KT, Mostaghimi A, Albrecht J. Evaluation of clinical compendia used for Medicare Part D coverage determinations for off-label prescribing in dermatology [published online January 23, 2019]. JAMA Dermatol. doi: 10.1001/jamadermatol.2018.5052.


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