Do Not Get Us Started on Acthar

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Despite this complete lack of evidence and astronomically greater costs, Acthar is being prescribed by a small but dedicated group of clinicians. Recently, a team of researchers and concerned clinicians (three of whom personally experienced high-pressure selling tactics by the drug’s manufacturer) used Medicare and Medicaid data to investigate the frequency of use and overall expense of Acthar. Their study, published in JAMA Network Open, found that in 2015 only 300 providers wrote more than 10 prescriptions for Acthar.⁴ A total of 235 of these providers were rheumatologists, neurologists, or nephrologists. And out of these 235 specialists, 88% received some sort of compensation from the manufacturer. While many received nominal amounts (less than $200), the top 20% each received over $10,000. There was also an association between receiving higher compensation and writing more Acthar prescriptions—and the Acthar prescriptions written by these frequent prescribers accounted for $200 million in Medicare spending during the period that the study examined. This study also found that more than $1.3 billion had been spent on this drug for several thousand Medicare patients from 2011–2015.

Given the marketing of the drug and the increase in the number of small, open-label studies published since 2015, we suspect these numbers are even higher now. We also know from personal experience that Acthar’s manufacturer is actively looking for clinical researchers open to performing more, small, open-label, nonrandomized trials of their drug.

It’s true that maintaining an active academic research agenda often means that physicians need to solicit and accept grant support and consulting work from pharmaceutical companies. However, it is the responsibility of all clinicians to ensure that they are not overly influenced by this funding, and that they can still objectively weigh the risks, benefits, and financial impact of all treatment decisions. Just because a drug can be prescribed, doesn’t mean it should be prescribed.

Instead of running multiple small unblinded and nonrandomized studies, a much better use of funding would be to conduct large analyses of Medicare and other health datasets to compare the risks and benefits of comparable patients treated with Acthar and prednisone. Patients, insurers, prescribers, and the FDA should mandate large-scale, randomized, prednisone-controlled trials of Acthar prior to allowing further prescriptions for rheumatic indications. These are the studies required for any new immunosuppressant, and the high expense of these trials is used to justify the high costs of the drugs. Drug manufacturers should not be able to aggressively market expensive medications without investing in the kinds of trials that could clarify the drug’s risks and benefits.

So, please, don’t get us started on Acthar. And please don’t start your patients on the drug, either—at least not until a large, randomized, high-quality prednisone-controlled trial has been done to justify the astronomical cost of this more than 80-year-old drug.