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Denied!

When a Medicare Part D patient is denied treatment, what are the options?


Denied!

When a Medicare Part D patient is denied treatment, what are the options?


By Allison Evans, assistant managing editor

An elderly patient sees a dermatologist and is diagnosed with pityriasis rubra pilaris (PRP). After the patient fails a number of oral medications and is unresponsive to topical agents and systemic steroids, the dermatologist prescribes adalimumab. Unfortunately, the patient, who is insured through Medicare Part D, is denied coverage because adalimumab was not prescribed for a “medically accepted indication.”

Dirk Elston, MD, past Academy president and current editor of JAAD, and his colleagues experienced this situation first-hand. Unlike dealing with private insurers, in which a physician can get prior approval for the off-label use of drugs, based on clinical argumentation or peer-reviewed literature, coverage determinations for Medicare Part D are limited by two compendia approved by the Centers for Medicare and Medicaid Services (CMS).

This month, Dermatology World explores how the Medicare Part D program’s reliance on two hard-to-access and often outdated compendia to determine coverage for off-label drug use can leave a subsection of Medicare Part D beneficiaries without adequate drug coverage. 

What are the compendia?

The official compendia used by Medicare Part D to make off-label coverage determinations are Micromedex Drugdex and the American Hospital Formulary Service (AHFS) Drug Information. These are comprehensive listings of drugs and biologics that include a summary of the dosing and characteristics of the drug and recommended uses for specific diseases.

Many physicians are not even aware of the compendia until they receive a denial letter, said John Barbieri, MD, MBA, a dermatology research fellow at the University of Pennsylvania and lead author of a 2019 JAMA Dermatology article that evaluates the compendia and Medicare Part D coverage determinations for off-label prescribing in dermatology (https://doi.10.1001/jamadermatol.2018.5052).

How does Medicare use the compendia?

When CMS developed the Medicare Part D program, coverage was a serious concern, said Joerg Albrecht, MD, PhD, a Chicago-based dermatologist and chair of the AADA’s Drug Pricing and Transparency Task Force. “CMS has used the compendia to ensure uniform and generally generous coverage between carriers.”

When can Medicare Part D deny coverage of a treatment?

Under Medicare Part D, a drug must be prescribed for a medically accepted indication, which is defined by CMS as:

• a use that is approved by the FDA, or

• a use that is supported by one or more citations in at least one of two CMS-approved compendia

Under Medicare Part D, a drug must be prescribed for a medically accepted indication, which is defined by CMS as a use that is approved by the Food and Drug Administration (FDA) or a use that is supported by one or more citations in at least one of the two CMS-approved compendia. Non-FDA-approved drugs that are prescribed outside the compendia’s guidelines have to be denied without exception.

According to Dr. Barbieri and his colleagues who authored the JAMA Dermatology study, “while the formularies may be designed with the best of intentions, incomplete or inadequate formulary construction could prevent patients from having access to necessary, evidence-based treatments, leading to worse outcomes for patients.”

Off-label prescribing

For Medicare Part D, if a medication is not FDA-approved to treat an indication, the off-label use of the drug is dictated by Medicare’s approved compendia. About 20% of all prescriptions written in the United States are for off-label therapies — and the practice is more common for patient populations like children and the elderly. Between 17% and 73% of prescriptions for the 10 most common dermatologic diseases are outside their labeled indications (https://doi.org/10.1016/j.jaad.2018.09.016).

With many prescriptions written for off-label indications, especially for rare conditions, it is important that elderly patients have access to treatments that face little to no possibility of garnering FDA approval. Many dermatologic conditions are so rare that they lack unique international classification of disease codes. According to a 2018 commentary in JAAD, the ICD-10 “does not include erosive pustulosis of the scalp or actinic prurigo, but multiple codes exist for attacks by turkeys and macaws” (https://doi.org/10.1016/j.jaad.2018.09.016).

Prior authorization appeal tool

Quickly and easily create a customized prior authorization appeal letter by using the AADA’s letter-generator tool at staging.aad.org/member/practice/drugs/pa-tool.

Evaluating the compendia

To assess the magnitude of the problem, Dr. Barbieri and his colleagues evaluated a list of 238 accepted treatments for 22 chronic, noninfectious, nonneoplastic dermatologic conditions that had at least four systemic therapies, including one considered first-line, but many not approved by the FDA. Only 73 treatments (30.7%) were listed in either compendium. There were frequent inconsistencies between the compendia with 53 (22.3%) medications evaluated included in one compendium but not the other. Additionally, the literature used was often based on decades-old sources, with some citations published before the 1980s.

“We were alarmed that over two-thirds of the medications evaluated were not included in the compendia, including half of the medications with the highest evidence grade (double-blind clinical trial),” Dr. Barbieri said. “In addition, these compendia disagreed with each other almost a quarter of the time, which suggests that the approach used to develop them is both incomplete and inconsistent.”

Neither compendium had any treatments listed for autoimmune progesterone dermatitis, eosinophilic fasciitis, lichen myxedematosus, linear IgA bullous dermatosis, or folliculitis decalvans, according to the study authors. “Many of these diseases are rare, but real,” Dr. Barbieri added.

Oncologists once struggled with similar denial issues since many of their treatments are off-label and require prior authorizations, but they worked with CMS to get approval for compendia specifically for cancer treatments. Dermatology, however, is more unusual in the types of diseases treated and the methods by which they are treated, so it’s not as simple as adding a dermatology-specific compendium, Dr. Albrecht explained. “We have very few physicians and a lot of diseases — a number of which are rare diseases. The rarer the diseases, the less likely you are going to have standardized approaches.”

Rheumatology's success: How did they do it?

The American College of Rheumatology (ACR) was one of the organizations that signed on to the American Academy of Dermatology Association’s (AADA) letter. Like dermatologists, rheumatologists also prescribe many off-label treatments and treat numerous diseases without FDA-approved treatments. In October 2016, the ACR submitted its first request to the American Hospital Formulary Service (AHFS) to add a medication for a specific disease state to the compendium.

Like most physicians, Christopher Phillips, MD, a rheumatologist in private practice in Paducah, Kentucky, and member of the ACR’s Insurance Subcommittee, has never actually seen the compendium due to the high cost to access. “It’s this mysterious thing. We’ve never seen it, but we talk about it,” he said.

“We came up with a list of several thorns in our side and focused on one that we thought was highest yield,” said Dr. Phillips. “For us, that was the use of mycophenolate mofetil for the treatment of scleroderma.”

Currently, there is no FDA-approved treatment for scleroderma, although there have been several large studies demonstrating the efficacy of mycophenolate mofetil, Dr. Phillips said. “We put together a document that explained the background and unmet need for treating this disease. We presented synopses of the most relevant studies and developed a proposal that we then submitted to the AHFS.

While the process has been long, the ACR has met with some success. In August 2017, the ACR was notified that the AHFS would be accepting the proposal — mycophenolate mofetil would be added for the treat-

ment of scleroderma to the compendium. Another year passed, and in October 2018, the organization was notified that the process was moving forward. In November 2019, the ACR was informed the update would be published before the end of 2019.

The process of getting the AHFS to agree to add the drug to the compendium was not overly arduous, noted Dr. Phillips, although figuring out how to make the initial submission is a bit circuitous and opaque. “They don’t just say, ‘Hey, if you’ve got something you want to add to the compendium, here’s who we are and how to find us.’”

With one success under their belts, Dr. Phillips and his colleagues submitted a second update to the AHFS: mycophenolate mofetil for the treatment of connective tissue-associated interstitial lung disease (CTILD). “We followed the same process as the first submission — performed a literature review, gathered relevant studies to support this treatment, and put together another proposal,” he said. Dr. Phillips and his colleagues recently learned that mycophenolate mofetil will be added to the compendia for the treatment of CTILD.

The difficult part about choosing what medication/indication to submit is that it does depend on there being some published data, Dr. Phillips said. “You need a condition where there is data beyond just case reports.” For Dr. Phillips and his colleagues, it was about finding a balance in which they could draw from smaller randomized studies, which each may have included only 20 to 30 patients. The bottom line is that there needs to be a certain amount of data to guide each submission, he said.

The appeal and judgement

In early 2018, Dr. Elston submitted an appeal on behalf of his patient with PRP to the Medicare Appeals Council. He and his colleagues submitted a number of studies and scholarly articles demonstrating the efficacy of using adalimumab to treat PRP. The literature showed a marked reduction in symptoms at eight weeks and clinical remission at four months, with sustained clinical response compared to other agents used to treat PRP.

Dr. Elston’s appeal was heard by an administrative law judge who rendered a legal judgement in June 2018: The judgement upheld Medicare’s denial. Because adalimumab was not prescribed for what Medicare deems “medically accepted use,” (i.e., it is not FDA-approved to treat PRP or it is not listed as an accepted indication in either Drugdex or the AHFS compendia) the judge ruled that the Medicare Part D sponsor did not need to provide coverage for the drug.

“It can be extremely difficult, or nearly impossible, to get a denied medication covered. This is most limiting when a serious condition requires timely treatment,” said Arash Mostaghimi, MD, MPA, MPH, director of the inpatient dermatology consult service and co-director of the Complex Medical Dermatology Fellowship at Brigham & Women’s Hospital in Boston.

Seeing eye to eye

When insurance doesn’t cover standard of care treatments, who pays the price and what can be done about it? Find out at staging.aad.org/dw/monthly/2019/september/seeing-eye-to-eye.

Patient access to treatment

The AHFS includes one treatment option for PRP: isotretinoin. It does not, however, support the use of methotrexate, nor does it include newer literature from the early 2000s supporting the use of acitretin and TNF-inhibitors. The indication is absent from Drugdex. Similar inconsistencies and deficiencies were noted for other diseases, according to Drs. Barbieri, Albrecht, and Mostaghimi.

Unfortunately for those with rare diseases, like PRP, not only are there no FDA-approved treatments, but the majority of treatment options are absent from both compendia, leaving the patient without access to necessary, evidence-based therapies.

Dr. Mostaghimi concedes that it makes sense that there should be limitations on what clinicians can and can’t prescribe. “However, there should always be a method to address outliers. Even if rules reflect standard practice, patients are infinitely complex and there will be exceptions. But it is the heterogeneity, the opacity, and inability to adjust the core compendia that is the main problem.”

For Dr. Albrecht, the issue of the Medicare compendia first sparked his interest when he tried to prescribe acitretin for a Medicare patient with hidradenitis suppurativa (HS). “It turned out that HS is practically unknown in the compendia. Only one of the two compendia even lists the drug, which has an FDA indication. All other treatments — from rifampin to doxycycline, acitretin to spironolactone and finasteride — would be denied because they are not in either compendium.” As a consequence, he convinced the patient to start adalimumab, which controls his disease poorly and costs more than acitretin.

Drug access and affordability

See what the AADA has been doing to advocate on specific drug access and affordability issues at staging.aad.org/member/advocacy/priorities/drugs.

Accessibility of the compendia

After receiving a denial letter for an off-label treatment from a Medicare Part D sponsor, it is likely that physicians may want to review the compendia to determine their available treatment options. However, gaining access to either compendium, let alone both, may prove to be an elusive treasure hunt. 

Physicians need access to these compendia so that they can easily find alternatives for their patients. “These compendia are incredibly difficult to access and are cost prohibitive,” said Dr. Barbieri. “If you are in a private practice, you probably can’t access them at all. And even if you’re in an academic center like the University of Pennsylvania or the University of Illinois at Chicago, which have very good libraries, you may only be able to access one.”

What can be done?

The CMS regulation may need to be changed more broadly, said Dr. Barbieri, since patients are being denied coverage because of these flaws. The authors of the study agree that there needs to be a pathway to deal with those instances when an indication or treatment is not listed in either compendia, but a patient needs access to a particular evidenced-based treatment.

“For truly uncommon conditions, there may not be enough incentive to adjust the compendia. There needs to be some case-by-case method to address those situations, especially because of the breadth of diagnoses and conditions in dermatology,” Dr. Barbieri said. “Any compendium is bound to fail when it tries to list second- or third-line treatments for rare diseases in a timely manner,” added Dr. Albrecht.

While attempting to solve this problem is administratively complex, explained Dr. Mostaghimi, there are already governmental mechanisms in place for similar situations — like the VA. When a medication is not part of the VA’s formulary, there is a system in place to access it. “Physicians deal with plenty of flawed processes in daily practice; however, while flawed or challenged, there are known, transparent processes for appeal,” he said. With Medicare Part D denials, however, the compendia are baked into the federal code as the only references for drug approvals.