Gene therapies for sickle cell disease are already changing lives in high-income countries, but in places like Uganda—where the burden of disease is among the highest in the world—the same treatments can seem economically out of reach.
A new study led by Felipe Montano-Campos, formerly a graduate student in Dr. Ryan Hansen’s group at the University of Washington CHOICE Institute, takes a closer look at whether that assumption holds. The work, published in Gene Therapy, asks a deceptively simple question: can gene therapies ever be cost-effective in a low-income setting where sickle cell disease (SCD) is most common?
SCD is a major public health challenge in Uganda, where about 13.3% of the population carries the sickle cell trait and a substantial number of children are born with the disease each year. For patients, the condition often means lifelong anemia, recurrent pain crises, frequent hospital visits, and progressive organ damage. Existing treatments such as hydroxyurea can reduce complications, but access is inconsistent. Curative options like stem cell transplantation remain rare, largely due to donor availability and infrastructure constraints.
Against that backdrop, gene therapies such as Casgevy and Lyfgenia represent a fundamentally different approach: a potential one-time intervention that targets the underlying cause of disease. Casgevy works by using gene editing to enable patients’ own blood stem cells to produce functional, fetal-like hemoglobin, reducing or eliminating the sickling of red blood cells, while Lyfgenia introduces a modified beta-globin gene to restore functional hemoglobin production. But with prices in the millions of dollars in high-income countries, their relevance for settings like Uganda has been uncertain.
“This work provides some of the first evidence that gene therapies for sickle cell disease could be cost-effective in a setting like Uganda,” said Montano-Campos. “That shifts the conversation away from the assumption that these therapies are simply too expensive for low-income countries.”
A central challenge in evaluating this question is the lack of local economic data. Most cost-effectiveness analyses of gene therapy rely on U.S. healthcare systems, costs, and patient trajectories, which do not reflect the realities of care delivery in resource-limited settings. To address this, the researchers first built a Uganda-specific model of standard SCD care, estimating lifetime costs from outpatient visits, hospitalizations, transfusions, medications, and management of vaso-occlusive crises. This yielded an estimated lifetime cost of standard care of about $21,877 per patient.
They then used this estimate as an anchor to adapt two established U.S.-based economic models of gene therapy. By comparing the lifetime cost of standard care in Uganda and the U.S., they derived scaling factors that allowed them to translate U.S. cost and treatment assumptions into a Ugandan context. This approach made it possible to evaluate gene therapy not under U.S. pricing conditions, but under a framework adjusted to local economic reality.
“What we did was develop a way to bridge that gap,” Montano-Campos said. “We started by estimating what it currently costs to treat sickle cell disease in Uganda over a patient’s lifetime, and then used that as a reference point to adjust U.S.-based models so they better reflect Uganda’s context.”
The results showed that the answer depends strongly on how value is defined. When only direct healthcare costs were considered, gene therapy remained difficult to justify economically. But when broader societal effects were included—such as reduced caregiver burden, improved productivity, fewer hospitalizations, and improved quality of life—the picture changed substantially.
Under those conditions, Casgevy approached commonly used willingness-to-pay thresholds for Uganda based on GDP per capita, suggesting it could be considered cost-effective in some scenarios. Lyfgenia showed similar health benefits but was less favorable economically due to higher modeled costs.
Importantly, the analysis also showed what happens if U.S. list prices are applied without adjustment. In that case, cost-effectiveness ratios increased dramatically, far exceeding any plausible affordability threshold for Uganda. The contrast highlights a key finding of the study: whether gene therapy is “affordable” is not only a question of clinical value, but also of pricing and context.
“An important part of this work is that it helps estimate what a reasonable price might look like in a setting like Uganda,” Montano-Campos said. “That is, a price where the health benefits would justify the cost.”
Beyond sickle cell disease, the authors argue that the framework could be applied more broadly to other emerging high-cost therapies being developed almost exclusively in high-income countries. As of recent years, the vast majority of gene therapy trials remain outside low-income settings, leaving major gaps in evidence for regions where disease burden is often highest.
“The main contribution is really this framework,” Montano-Campos said. “It provides a practical way to evaluate new, high-cost therapies in places where data are limited or nonexistent, and it helps inform decisions about pricing, access, and how to make these innovations more equitable globally.”
The work does not suggest that implementing gene therapy in Uganda is immediately feasible. Significant challenges remain around infrastructure, financing, delivery models, and regulatory capacity. But it does change the framing of the question—from whether these therapies belong in low-income countries at all, to what conditions would be required to make their access possible.
Fred Hutch/University of Washington/Seattle Children’s Cancer Consortium Member Dr. Anirban Basu contributed to this research.
The spotlighted research was funded by National Institutes of Health, the Washington Research Foundation, the Bill and Melinda Gates Foundation, and the Fred Hutchinson Cancer Research Center/University of Washington/Seattle Children’s Cancer Consortium.
Montano-Campos F, Adair JE, Basu A, Kyeyune RB, Bayigga L, Kityo-Mutuluuza C, Hansen R. 2026. Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda's context. Gene Therapy. DOI: 10.1038/s41434-026-00598-1.
Science Spotlight writer Jenny Waters is a postdoctoral research fellow in the Hsieh lab at Fred Hutch. She studies how mRNA translation coordinates bladder cancer transformation and metastasis by post-transcriptionally regulating expression of oncogenic proteins. Outside of the lab, Jenny enjoys spending time with her dogs, convincing her husband to join her on trail runs, and pretending every steep hill is just a "gentle incline."