The potential cost-effectiveness of novel cord blood therapies in children with autism spectrum disorder

• Published in: PloS one Vol. 18; no. 4; p. e0282906
• Main Authors: Borre, Ethan D, Myers, Evan, Hamilton Lopez, Marianne, Kurtzberg, Joanne, Shaz, Beth, Troy, Jesse, Sanders Schmidler, Gillian D
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 18-04-2023; Public Library of Science (PLoS)
• Subjects: Adolescent; Adult; Age; Alternative approaches; Autism; Autism Spectrum Disorder - therapy; Behavior; Biology and Life Sciences; Blood; Care and treatment; Child; Child, Preschool; Children; Clinical trials; Communication; Cord blood; Cost analysis; Cost benefit analysis; Costs; Effectiveness; Efficacy; Efficiency; Evaluation; Fetal Blood; Health services; Humans; Impact analysis; Intellectual disabilities; Intervention; Life Expectancy; Life span; Medical care, Cost of; Medicine and Health Sciences; Mortality; Natural history; Parameter sensitivity; Patient outcomes; People and Places; Pervasive developmental disorders; Quality of life; Quality-Adjusted Life Years; Sensitivity analysis; Social Sciences; Socialization; Standard of care; United States; United States > US
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0282906

To model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD).
Markov microsimulation of ASD over the lifespan was used to compare two strategies: 1) standard of care (SOC), including behavioral and educational interventions, and 2) novel cord blood (CB) intervention in addition to SOC. Input data reflecting behavioral outcomes included baseline Vineland Adaptive Behavior Scale (VABS-3), monthly VABS-3 changes, and CB intervention efficacy on adaptive behavior based on a randomized, placebo-controlled trial (DukeACT). Quality-adjusted life-years (QALYs) were correlated to VABS-3. Costs for children with ASD ($15,791, ages 2-17 years) and adults with ASD ($56,559, ages 18+ years), and the CB intervention (range $15,000-45,000) were incorporated. Alternative CB efficacy and costs were explored.
We compared model-projected results to published data on life-expectancy, mean VABS-3 changes, and lifetime costs. Undiscounted lifetime QALYs in the SOC and CB strategies were 40.75 and 40.91. Discounted lifetime costs in the SOC strategy were $1,014,000, and for CB ranged from $1,021,000-$1,058,000 with CB intervention cost ($8,000-$45,000). At $15,000 cost, CB was borderline cost-effective (ICER = $105,000/QALY). In one-way sensitivity analysis, CB cost and efficacy were the most influential parameters on CB ICER. CB intervention was cost-effective at costs<$15,000 and efficacies ≥2.0. Five-year healthcare payer projected budgetary outlays at a $15,000 CB cost were $3.847B.
A modestly effective intervention designed to improve adaptive behavior in autism can be cost-effective under certain circumstances. Intervention cost and efficacy most affected the cost-effectiveness results and should be targeted to increase economic efficiency.