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How Prior Authorization Can Impact Patients With Rare Disease


The prior authorization process is a hurdle to getting care for individuals with rare diseases and limited options, according to a rheumatologist and a patient interviewed just before Rare Disease Day.

Prior authorization (PA), a common health insurance practice to ensure that treatment is medically necessary, continues to frustrate health care providers and patients—including those who have rare diseases or benefit from treatments that are considered off label.

Monday is Rare Disease Day, an annual event where advocates try to raise awareness about access to diagnosis, treatments, and improve policies, espcially around insurance issues.

A survey released earlier this month by the American Medical Association (AMA) found that the practice can interfere with a patient’s ability to work, according to just over half of the physicians surveyed. The AMA queried 1004 providers (40% primary care physicians, 60% specialists) in December 2021.

The survey found:

  • 93% reported care delays while waiting for approval
  • 82% said PA can lead to treatment abandonment
  • 30% said PA criteria are rarely or never evidence-based
  • 91% said PA has a somewhat or significant negative impact on clinical outcomes
  • 34% reported that PA led to a serious adverse event, including hospitalization (24%) or permanent disability or death (8%)

In an interview with The American Journal of Managed Care® (AJMC®), Christopher Phillips, MD, a rheumatologist in Kentucky, said one of the issues with rheumatological disease, many of which are rare autoimmune conditions, is a lack of FDA-approved treatments.

“So that presents a huge burden, getting treatments approved,” he said, adding some payers “are gracious enough to cover off label indications,” depending on what documentation is submitted with the request.

But processes and policies can change each year, or patients may switch plans, starting the process all over again.

In addition, Phillips said, it can take several weeks to get something approved. “When you kind of merge together the issues of prior authorization with step therapy…it may take several weeks for you only to learn that the treatment is going to be denied, because there's a step therapy protocol and the payer wants the patient to take this other treatment first, which may or may not be a reasonable treatment for that patient, depending upon their medical comorbidities.”

The more rare the disease, Phillips pointed out, the greater the likelihood that the patient will have either a lack of treatment or no way to pay for treatment that specialists know to be effective but has not yet been confirmed in a clinical trial and approved by the FDA, due to the small patient population.

Speeding PA Processes With a “Gold Card”

The AMA as well as other medical organizations, including the American College of Rheumatology, of which Phillips is a member, endorses the idea that providers who have a good track record with their prescribing patterns should be allowed to get a pass from insurers—something these advocates call a “gold card” concept. Specifically, the PA process would be restricted to outlier physicians, and those who have a 90% PA approval rate over a 6-month period would be exempt from the requirements.

Such a law took effect in Texas in 2021 and Phillips is advocating for a bill in Kentucky that would do the same thing.

In an email to AJMC, a college student from Tennessee described how she battled signs and symptoms of a rheumatological disease from age 8 to 17, when she landed in the emergency department and was admitted. Ultimately, Brooklyn Johnson, 19, was diagnosed with both lupus and juvenile dermatomyositis.

On her second hospitalization shortly thereafter, intravenous immune globulin therapy (IVIG) worked, but because the request was submitted with lupus as the main diagnosis and not juvenile dermatomyositis, it was denied. After that was resolved, carriers refused to pay for infusions given in a hospital as opposed to an infusion center or at home.

Brooklyn also said she has a condition called avascular necrosis, in which blood supply to the bone is cut off, destroying bone tissue and resulting in joint failure, if the damage is nearby. A surgery called core decompression—to remove bits of damaged bone in the hopes of forestalling joint replacement—for her knees was initially approved, and then denied, because there is more evidence for its use in hips and not knees.

“This just shows that the battle with insurance never goes away, and is something I will likely deal with for the rest of my life,” Brooklyn wrote.

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