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In Reply We thank Dr Otulana for his interest and response to our Research Letter.1 We agree that repository corticotropin (Acthar) may have a place in therapy for a small number of patients who cannot tolerate or do not respond to synthetic corticosteroids. While Acthar use in the Medicare program is attributable to a relatively small number of specialists, costs to the program are substantial. In 2015, Medicare spent nearly twice as much approximately $504 million) on Acthar for 3104 patients than it did on lisinopril prescriptions (approximately $253 million) for 7.8 million patients.2
The emergence of very-high-cost pharmaceuticals has sparked considerable debate about the market for pharmaceuticals and pricing decisions. The pharmaceutical industry often argues that high prices and profits are necessary to incentivize risky and expensive drug development activities. Unlike other high-cost pharmaceuticals, Acthar was first commercialized over 65 years ago with no controlled clinical trials to support its use. In the ensuing years, trials demonstrating the efficacy of Acthar for infantile spasms and to treat relapses of multiple sclerosis have been completed. However, the remainder of studies, as summarized in the review cited by Dr Otulana, are of low quality, consisting primarily of case series, small uncontrolled single-arm studies, and several observational studies of health care utilization.3
Dr Otulana asserts that revenue from Acthar, which exceeded $1.2 billion in 2016,4 is funding research to “generate the evidence to fill in the gap.” We identified 47 Acthar-related prospective studies funded by Mallinckrodt registered to clinicaltrials.gov, 21 of which had a single arm with no comparison group. Of the remaining “controlled” studies, the median enrollment was 40 patients. Given the preponderance of small and uncontrolled single-arm studies, we have serious doubts that these studies will provide scientific evidence sufficient to justify use of Acthar at its current price.
We believe that drug prices need to be related in some way to value, which at a minimum, is derived from a solid foundation of clinical evidence. Regardless of the ultimate validity of Mallinckrodt’s Acthar research program, we question an inverted business model that justifies an excessively inflated medication cost based on the need to generate evidence to support the high cost. Moreover, it does not seem likely that definitive evidence will be forthcoming.
Corresponding Author: Daniel M. Hartung, PharmD, MPH, Oregon State University/Oregon Health & Science University College of Pharmacy, Oregon Health & Science University, Collaborative Life Sciences Bldg (CLSB), 2730 SW Moody Ave, CL5CP, Portland, OR 97201-5042 (email@example.com).
Conflict of Interest Disclosures: None reported.
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Hartung DM, Bourdette DN, Cohen DM. Uses of H.P. Acthar Gel in the Clinical Setting—Reply. JAMA Intern Med. 2018;178(4):583–584. doi:10.1001/jamainternmed.2017.8545
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