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Epilepsy CurrentsThe ACTH Quandary: Balancing Efficacy and Affordability for Infantile Spasms Treatment

The study analyzed the cost and usage trends for adrenocorticotropic hormone (ACTH), oral prednisolone, and vigabatrin from 2006 to 2020 in treating IESS.


As physicians strive to provide the best care for patients with infantile epileptic spasms syndrome (IESS), they must also navigate a complex landscape of rising treatment costs and varying efficacy, focusing on a cost-benefit analysis for these treatments.

Key Points:

  • 1,131 patients received at least one first-line treatment: 592 patients received ACTH, 363 patients received oral prednisolone, and 355 patients received vigabatrin.
  • The median average wholesale price for a 14-day course of ACTH increased by ~2,700% from $3,718 in 2006 to $100,457 in 2020.
  • Conversely, the cost for oral prednisolone decreased by ~50% from $169 in 2006 to $89 in 2020.
  • Vigabatrin’s cost increased by ~340% from $1,206 in 2009 to $4,102 in 2020.
  • ACTH use dropped from 78% of first-line treatments in 2006 to 18% in 2020.

Additional Points:

  • ACTH treatment resulted in higher inpatient admission days and costs compared to oral prednisolone and vigabatrin.
  • The widening cost gap between ACTH and the alternatives coincides with the reduced use of ACTH.
  • The study found no compelling evidence supporting ACTH’s superiority over oral prednisolone in short-term effectiveness (2 weeks).
  • There are barriers to synthetic ACTH production in the US, leading to a limited market competition for animal-derived ACTH (Acthar® Gel).

Conclusion:

  • As the cost of ACTH soars, its usage decreases, leading to a shift toward more affordable treatment alternatives for IESS. However, the comparative long-term outcomes remain uncertain, necessitating robust evidence to support changes in treatment recommendations.

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“Balancing the efficacy and affordability of infantile spasms treatment is challenging because of the high cost of ACTH.”

Gewalin Aungaroon, MD
Department of Neurology
Cincinnati Children’s Hospital Medical Center
University of Cincinnati.
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