Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.
Level 5 - mechanism / opinion, no new human data
Decision-analytic simulation model based on claims data and literature inputs (level 5 by design analogy for economic modeling).
PubMed 38252942 · doi:10.7326/M23-1520
What was done
Researchers performed a comparative lifetime cost-effectiveness analysis evaluating gene therapy versus common care for eligible individuals with sickle cell disease in the United States. Two independently developed simulation models (UW-MEASURE and FH-HISCORE) evaluated incremental cost-effectiveness ratios (ICERs) and equity-informed value-based prices (VBPs) using 2008–2016 Centers for Medicare & Medicaid Services claims data and published literature from both US healthcare sector and societal perspectives.
What was found
At an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193,000 and $427,000 per QALY under the healthcare sector perspective, and $126,000 and $281,000 per QALY under the societal perspective. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on equity-informed threshold values and metric choices. Model divergence was driven by differences in target population definitions and inclusion of quality-of-life effects for splenic sequestration, priapism, and acute chest syndrome. Findings were sensitive to myeloablative conditioning costs, caregiver quality of life, and long-term survival impacts.
Why it matters
This study provides evidence-based price benchmarks for emerging curative sickle cell gene therapies, suggesting they are likely cost-effective under societal perspectives if priced under $2 million.
Limits
Short-term gene therapy efficacy regarding vaso-occlusive events was extrapolated from a single study. Long-term survival and durability benefits were simulated rather than empirically measured, and the two models produced substantially different ICER estimates due to divergent structural assumptions and complication modeling.
Cited by
- supports Top adenoviral-vectored COVID-19 vaccines cost as little as $2 per dose, compared to typical gene therapies that cost around $2 million.