Gene therapy for sickle cell disease (SCD) could be cost-effective in Uganda – but only if prices are dramatically reduced and broader societal benefits are considered. That’s the conclusion of a new modeling study adapting US economic data to a low-income healthcare setting.
Researchers developed a framework that scales high-income cost-effectiveness models using local treatment costs. Applying this to two approved therapies – Casgevy and Lyfgenia – they found that, while both deliver substantial health gains, affordability hinges on pricing. Uganda’s lifetime standard-of-care cost for SCD was estimated at just $21,877, compared to over $600,000 in the U.S., underscoring the need for major price adjustments.
Casgevy emerged as the more viable option. When costs were scaled to Uganda’s context, its incremental cost-effectiveness ratio fell within internationally accepted thresholds – particularly when factoring in societal benefits such as increased productivity and reduced caregiver burden. Lyfgenia, with a higher list price, was less likely to meet these thresholds.
The study highlights a broader challenge: translating cutting-edge therapies to regions where disease burden is highest but resources are scarce. By offering a practical framework, the authors aim to support policymakers and funders in expanding equitable access to gene therapy worldwide.
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