Acthar Gel may be cost-effective for treating pulmonary sarcoidosis
Injection therapy seen as 'value-based' vs. standard glucocorticoids: Analysis
Acthar Gel (repository corticotropin injection) may be a cost-effective approach relative to standard-of-care glucocorticoid therapy for adults with advanced active pulmonary sarcoidosis.
That’s according to an analysis done by the therapy’s developer Mallinckrodt Pharmaceuticals, which found that “the initial high cost of Acthar Gel treatment for sarcoidosis is offset by the reduction in disease progression-related medical costs.”
Overall, said George Wan, PhD, vice president, evidence generation and data sciences at Mallinckrodt, the analysis shows Acthar Gel is a “value-based” therapy option.
“The findings of this health economics analysis demonstrate Acthar Gel’s potential as a value-based, cost-effective treatment option … compared with the standards of care for the treatment of appropriate adult patients with advanced [active] sarcoidosis,” Wan said in an email to Sarcoidosis News.
The study, “Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis,” was published in ClinicoEconomics and Outcomes Research.
Acthar Gel more expensive than glucocorticoids
In sarcoidosis, small clumps of inflammatory cells, or granulomas, accumulate in the body’s tissues and cause organ damage. The most common type is pulmonary sarcoidosis, where granulomas accumulate in the lungs.
Oral glucocorticoids such as prednisone are the first-line treatment for sarcoidosis. These potent anti-inflammatory medications mimic the action of cortisol, a naturally-occurring glucocorticoid, to ease sarcoidosis symptoms. However, they are linked to substantial side effects when used for a long period.
Acthar Gel is approved in the U.S. for people with active pulmonary sarcoidosis. Its use is recommended in both the U.S. and Europe — where it is used off-label — for patients for whom standard treatment, including glucocorticoids, was intolerable or not effective.
The injectable medication, which is more expensive than glucocorticoids, contains a mixture of hormones and other proteins released from the brain’s pituitary gland that are thought to have anti-inflammatory effects via multiple mechanisms.
Data from a Mallinckrodt-sponsored Phase 4 clinical trial, called PULSAR (NCT03320070), showed that adding Acthar Gel to standard predinose led to greater benefits than did a placebo in people with active pulmonary sarcoidosis. Also, patients with worse lung function and multi-organ involvement were more likely to respond to the therapy.
“However, no study has examined the economic benefits of Acthar Gel in this patient population,” the researchers wrote.
Conducting research into the clinical impact, associated healthcare costs, and real-world outcomes with Acthar Gel treatment is essential to better support patients and providers managing an advanced symptomatic sarcoidosis diagnosis.
Wan called sarcoidosis “a challenging and rare multisystem disease.”
“Conducting research into the clinical impact, associated healthcare costs, and real-world outcomes with Acthar Gel treatment is essential to better support patients and providers managing an advanced symptomatic sarcoidosis diagnosis,” Wan said.
Further studies need to examine long-term cost-effectiveness
Now, Wan and colleagues at Mallinckrodt led an analysis to evaluate Acthar Gel’s cost-effectiveness relative to standard-of-care glucocorticoids among people with advanced, active pulmonary sarcoidosis.
Clinical data and parameters were largely sourced from the PULSAR study. Cost considerations in the U.S., obtained from published literature, included treatment costs, such as the price of Acthar Gel and other medications used alongside it, as well as direct medical costs (e.g. hospitalizations) and indirect costs (e.g. loss of work ability).
An incremental cost-effectiveness ratio, or ICER, was generated from that information. This ratio essentially reflects the difference in total costs divided by the difference in quality-adjusted life years (QALY), or the number of years living in perfect health, gained with the treatment.
The researchers set an ICER cut-off of $150,000 per QALY assuming that $150,000 would be the maximum additional cost per QALY that people would be willing to pay for Acthar Gel’s benefits. Anything above that would no longer be considered cost-effective.
An ICER was first determined from an U.S. payer’s perspective, which considered treatment and direct medical costs. In this calculation, Acthar Gel was estimated to result in an ICER of $134,796 per QALY over a 2-year period and $39,179 per QALY over three years.
Another ICER was calculated considering the societal perspective, which also included indirect costs like loss of productivity and functioning. Again, Acthar Gel was found to be cost-effective, with ICERs of $117,622 per QALY over two years, and $21,967 per QALY over three years.
While treatment costs were higher for Acthar Gel compared with standard care over a 2-year period ($254,882 versus $0), other estimated direct medical costs, including those related to lung transplants, corticosteroid-related toxicity, pain, depression, and non-lung symptoms, were substantially lower, at $214,166 versus $403,133.
Likewise, indirect costs related to disability, substance use disorders, and caregiving were estimated at $20,649 with Acthar Gel and $29,047 with standard care.
The researchers noted that while Acthar Gel therapy incurs some additional costs, it “results in a gain in QALYs compared to [standard-of-care] over the short term” — specifically, 0.49 QALYs over two years and 0.75 over three years.
One noted limitation of the analysis is that the clinical trial data and published literature may not reflect real-world experiences, and therefore, the results may have been overestimated or underestimated.
While the analysis found Acthar Gel cost-effective in the short term, additional studies are needed “to examine the long-term clinical effectiveness and cost-effectiveness of Acthar Gel for advanced symptomatic sarcoidosis,” the team concluded.
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