Objective:

To evaluate the cost-effectiveness of gene therapy for sickle cell disease in Uganda using a framework adapted from US economic data specifically tailored to local healthcare dynamics.

Key Findings:

• Uganda's lifetime standard-of-care cost for SCD is estimated at $21,877, significantly lower than over $600,000 in the U.S., highlighting the need for price adjustments.
• Casgevy is identified as the more viable gene therapy option, with its cost-effectiveness ratio falling within acceptable thresholds when adjusted for Uganda, particularly when considering societal benefits.
• Lyfgenia, due to its higher price, is less likely to meet cost-effectiveness thresholds, emphasizing the importance of pricing in therapy selection.

Interpretation:

Affordability of gene therapies in Uganda is contingent on significant price reductions and the consideration of broader societal benefits, such as increased productivity and reduced caregiver burden.

Limitations:

• The study relies on modeling and may not capture all local healthcare dynamics, which can vary widely.
• Broader societal benefits are difficult to quantify and may vary widely, impacting the overall cost-effectiveness assessment.

Conclusion:

The study provides a framework to support equitable access to gene therapy in low-income settings, emphasizing the need for price adjustments and consideration of societal factors.