Abstract
Objective
To describe the temporal trends in the cost and use of adrenocorticotropic hormone (ACTH), oral prednisolone, and vigabatrin, the first-line treatments for infantile epileptic spasms syndrome (IESS).
Methods
Retrospective observational study using the MarketScan Commercial database from 2006 to 2020. We identified patients with IESS diagnosed between birth and 18 months of age who received at least one of the first-line treatments within 60 days of diagnosis. Costs were adjusted for inflation using the Gross Domestic Product Implicit Price Deflator.
Results
1,131 patients received at least one first-line treatment [median (p25-p75) age: 6.3 (4.5-8.3) months, 55% males], of whom 592 patients received ACTH, 363 patients received oral prednisolone, and 355 patients received vigabatrin. After adjusting for inflation, the median average wholesale price of a 14-day course of treatment increased for ACTH from $3,718 in 2006 to $100,457 in 2020, approximately 2,700% (by a factor of 27); while it decreased for oral prednisolone from $169 in 2006 to $89 in 2020, approximately 50% (by a factor of 0.5); and increased for vigabatrin from $1,206 in 2009 (first year with data on vigabatrin used for IESS) to $4,102 in 2020, approximately 340% (by a factor of 3.4). During the first 60 days after diagnosis, inpatient admission days and costs where higher for ACTH than for oral prednisolone and vigabatrin [5.0 (3.0-8.3) days versus 2.0 (0.0-5.0) days versus 2.0 (0.0-6.0) days, p<0.0001; and $32,828 ($14,711-$67,216) versus $16,227 ($0-$35,829) versus $17,844 ($0-$47,642), p<0.0001]. ACTH use decreased from representing 78% of first-line treatments in 2006 to 18% in 2020, p<0.0001. Sensitivity analyses confirmed the robustness of the results.
Significance
The gap between the cost of ACTH and the cost of oral prednisolone or vigabatrin has markedly widened from 2006 to 2020, while the relative proportion of ACTH use has decreased.
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