Step Therapy: When Insurance Decides Which Drug You Get First

Sammantha Dorazio was tired, but she didn’t think anything of it.

She was 16, a three-sport high school athlete in Malden, Mass., a Boston suburb, training for a championship while making college plans. Anyone would be tired. Anyone could have muscle soreness. Chest pain even. She probably pulled something doing a flip turn in the pool.

But the soreness didn’t go away. It got worse.

Then, one day, she had trouble breathing. Her parents rushed her to the emergency room.

Eventually diagnosed with juvenile arthritis, Dorazio endured cycles of flare-ups and remissions for several years. But while studying science at Westfield State University in western Massachusetts, her inflammation spiraled out of control.

That’s when she learned about step therapy.

An increasingly common but still controversial cost-saving measure, step therapy requires patients to try less expensive treatments and find them ineffective before health insurance companies will approve more expensive ones, a process that—as Dorazio discovered—can sometimes take months or years.

To insurers, step therapy is a vital tool in combating rising drug prices. And for most patients, first-line drugs—usually generics or drugs in a similar but less-expensive class of medications—are just as effective as their pricier counterparts.

“A lot of doctors agree that a lot of medical care that we prescribe to patients is not necessary,” said Kristine Grow, spokeswoman for the trade organization American Health Insurance Plans, or AHIP.

But critics point to exceptions—cases in which step therapy’s “one-size-fits-all approach” can lead to “therapies that are doomed to be suboptimal,” Sharona Hoffman, a Case Western University law and bioethics professor, wrote in the Food and Drug Law Journal. (Hoffman’s husband had a poor experience with step therapy after being diagnosed with Parkinson’s disease.)

As is typical for patients with inflammatory diseases, Dorazio was first given methotrexate pills, a low-cost immunosuppressant used in chemotherapy since the 1950s. She took them for eight weeks. But she says she was still swollen and in pain, only now she was throwing up almost daily, her hair thinning and falling out.

Dorazio says her parents’ insurer, the nonprofit Harvard Pilgrim Health Care, required her rheumatologist to switch to methotrexate injections for another eight weeks. That didn’t work either.

“I went through this four-month span of just being so sick and puking my brains out,” said Dorazio, now 23.

Dorazio’s rheumatology team told them methotrexate might not work “due to the complexity of Sammantha’s arthritis, but we had to start there before other medications could be approved by insurance,” her mother, Gail Dorazio, said in an email. “I was frustrated that my child would have to take medication with intense side effects for it to possibly not be effective.”

One weekend, she said, those side effects got so bad she had to rush her daughter to the emergency room.

In an email, Harvard Pilgrim spokesman Philip Tracey wrote that, while he can’t comment on specific cases, the company’s protocols are based on “national guidelines, consensus statements, expert opinion, peer-reviewed literature, and other resources. ... Exceptions for coverage outside the standard step therapy guidelines are based on medical necessity.”

Those guidelines required another 16-week series of injections—and joint pain so bad Dorazio couldn’t hold a pen, she says—before Harvard Pilgrim approved the medication her rheumatologist wanted her to have, Actemra.

Dorazio went into remission.

By this point, however, she says she’d developed permanent nerve damage in her right jaw, a result of unchecked inflammation.

“Could those four months [on methotrexate] have been the ones when we saw the permanent nerve damage?” Dorazio asked.

It’s possible, says Dr. Harry Gewanter, a former director of pediatric rheumatology at the Children’s Hospital of Richmond, Va., and board member of the Coalition of State Rheumatology Organizations, which has lobbied for step therapy reform. While he hasn’t assessed Dorazio, he says she’s “probably correct that the delays in treatment have resulted in significant issues.”

“Early, aggressive treatment leads to better outcomes,” Gewanter said. “That runs counter to the [insurance companies’] view that we need to use the least expensive medicines—or, quite frankly, the most profitable medicines for them—first.”

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During the last 15 years, step therapy has become almost universal.

In 2005, 27 percent of employer-sponsored healthcare plans used step therapy. In 2013, 67 percent did. By 2017, 82 percent were, according to a report commissioned by the Pharmaceutical Care Management Association, or PCMA, which represents the pharmacy benefit managers that operate insurance companies’ drug plans.

Medicare Part D, the prescription drug program, uses step therapy, as well. In 2019, the Centers for Medicare and Medicaid Services, or CMS, reversed an Obama-era regulation and allowed Medicare Advantage plans to expand step therapy to Medicare Part B drugs, most often injections or infusions given in an outpatient setting such as a doctor’s office.

For both private insurers and the federal government, drug prices are a pressing concern. From 2013 to 2017, national spending on prescription drugs rose more than 25 percent, to more than $333 billion, according to AHIP. By 2023, that amount is projected to increase to $435 billion, driven largely by the high prices of specialty drugs.

Step therapy and prior authorization—a requirement that physicians get approval before prescribing a drug—are meant to contain those costs. According to the PCMA report, together they’ll shave 6.75 percent off insurers’ drug expenditures over the next decade. (A spokesman for the PCMA did not respond to an interview request.)

Without these controls on prescriptions, proponents of step therapy say that many physicians—perhaps wooed by the $30 billion a year the pharmaceutical industry spends on marketing—would prescribe expensive medications when there’s no medical reason to do so.

Hoffman, the bioethics professor, believes the savings are short-lived. If patients who don’t receive their “drug of choice” stop taking their meds because of side effects or concerns about efficacy, that will lead to health complications—and higher costs—down the line, she wrote.  

No matter what, insurance companies focus on short-term expenses, says Wendell Potter, a former communications executive for Humana and Cigna who lobbies to reform the industry. (Potter receives some funding from the same source as No Patient Left Behind.)

He calls step therapy “a delaying tactic.” Because there’s a lot of churn in health insurance coverage, insurance companies “may or may not be on the hook” when a patient needs more expensive treatments.

Scott Bruun, director of the Oregon-based Chronic Disease Coalition, says step therapy offers insurance companies a “heads I win, tails you lose” scenario.

“Either I stay on [the first-line drugs] permanently, and they never have to pay for that expensive treatment,” Bruun said, “or even if I just delayed that treatment three or four months, that’s three or four months that the insurance company doesn’t have to pay for the more expensive treatment.”

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Bruun was diagnosed with rheumatoid arthritis in 2000, when he was in his 30s, a few years before his election to the Oregon legislature.

Like Sammantha Dorazio, he says his insurance company made him go through months of step therapy—beginning with methotrexate and sulfasalazine, “the latest and greatest in 1950s medicine,” he used to joke—before finally approving Enbrel. (He’s now on Humira.)

“Once the damage is done, it’s done permanently,” Bruun said. “What you try to do with RA is just stop it from getting worse. None of these drugs I was taking—they made me feel a little better, but none of them stopped the progressive damage.”

Step therapy’s savings hasn’t been apparent to consumers, whose premiums, prescription co-pays, and deductibles have risen sharply over the last decade. Physicians, meanwhile, have bristled at what they view as insurance companies’ interference in patient care.

So as step therapy and prior authorization became more ubiquitous, a wide swath of physician organizations and patient advocacy groups urged lawmakers to step in, citing anecdotes of patients who suffered because insurance companies denied access to treatments their physician prescribed.

They found sympathetic ears in statehouses all over the country. Twenty-six states have passed legislation to rein in the practice, according to the National Psoriasis Foundation’s tracker, including three in 2020: Louisiana, South Dakota and North Carolina.

In general, the laws specify when exceptions to step therapy should be granted—for example, if the patient has previously tried the first-line drug or is already using a brand medication—and require insurers to respond to exception requests within 72 hours, or 24 hours if the request is urgent.

But state laws don’t cover the millions of Americans who receive insurance through large corporations’ self-funded employer plans. In 2019, Rep. Raul Ruiz, D-Calif., and Sen. Lisa Murkowski, R-Alaska, introduced federal step therapy reform legislation, though neither bill has received a vote.

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Massachusetts state Sen. Jason Lewis says what turned him from a “supporter” of step therapy reform “into a champion” was the work of a Malden constituent, a college student who reached out three years ago to share her experience—Sammantha Dorazio.

“At that point,” Lewis said, “this legislation became my priority.”

On July 30, the Senate unanimously passed a bill requiring insurance companies to grant exceptions to step therapy protocols if a drug is contraindicated, expected to be ineffective, been tried before, not in the patient’s best interest, or the patient is already stable on another medication.

Watching the vote live, Dorazio cried.

“It’s taken time to educate lawmakers to have it kind of rise on the priority list,” Lewis said.

Because her doctors fought to get her on Actemra, Dorazio says, she’s able to go to school full-time. She’s pursuing an accelerated master’s degree as a nurse practitioner and wants to get a doctorate in health policy to help those, like her, with rheumatoid disease.

But right now, there’s still work to be done on Beacon Hill in Boston, as well as in other state capitals around the country, advocates of stricter controls say. Though the Massachusetts Senate passed the step therapy bill unanimously, the state House never scheduled a vote.  

“No parent should have to watch their children go through what Sammantha went through,” Gail Dorazio said. “I cannot tell you how many nights I've cried myself to sleep over this.”

Jeffrey Billman is an award-winning investigative journalist based in North Carolina.‍