Gene therapy is rapidly revolutionizing treatment paradigms for a number of rare genetic disorders in wealthy regions like the United States and Europe. Among the most promising applications has been in hemoglobinopathies, including sickle cell disease (SCD), which have historically carried a grievous burden for affected individuals. While these transformative interventions have already made vital inroads into patient care in high-income countries, their potential for global health equity demands urgent attention—especially in low- and middle-income countries (LMICs) where hemoglobinopathies are prevalent but curative treatments remain out of reach.
Sickle cell disease, a genetic disorder characterized by malformed, sickle-shaped red blood cells that impede oxygen transport and cause severe complications, is particularly pervasive in sub-Saharan Africa. Uganda, with its significant SCD burden and limited access to curative options, serves as an acute example of this tragic global health divide. Up to this point, SCD management in Uganda and other similarly affected LMICs has centered largely on symptomatic treatment and supportive care due to resource constraints. However, the advent of gene therapies targeting the underlying genetic defects now thrusts the possibility of durable cures within the realm of feasibility—if economic and infrastructural barriers can be overcome.
A pioneering study led by Montano-Campos and colleagues confronts this challenge head-on by assessing the cost-effectiveness of gene therapies for SCD in Uganda. The researchers sought to bridge the gap between existing cost-effectiveness analyses developed for high-income settings and the pressing need for contextualized economic evaluations that reflect the realities of LMIC health systems. By leveraging a novel framework, the study adapts U.S.-based cost-effectiveness evidence for two authorized gene therapies targeting SCD—Lyfgenia™ (lovo-cel) and Casgevy® (exa-cel)—to Uganda’s unique epidemiological and economic context.
Central to the study’s methodology is the use of a three-state Markov model that projects lifetime costs and health outcomes of the current standard of care for Ugandan SCD patients, primarily adolescents and young adults. This model incorporates transitions between health states representing stable disease, complications, and death, calibrated with local data where possible. The team then applied scaling factors to U.S. cost-effectiveness models for the two gene therapies, adjusting for differences in income, healthcare infrastructure, and societal valuation of health benefits to capture Uganda’s economic environment more realistically.
The application of this framework yields compelling insights. When adopting a societal perspective—which includes not only direct healthcare costs but also productivity losses, caregiver burdens, and broader welfare impacts—the cost-effectiveness of Casgevy® in Uganda begins to approach acceptable thresholds based on the country’s gross domestic product per capita. While the upfront costs of gene therapy remain substantial, the projected long-term benefits such as reduced morbidity, enhanced life expectancy, and improved quality of life produce favorable incremental cost-effectiveness ratios (ICERs) when appropriately scaled.
Interestingly, the analysis underscores that cost-effectiveness varies significantly depending on the perspective adopted. From a purely healthcare payer viewpoint, these novel therapies currently appear costly relative to Uganda’s economic means. However, when societal considerations—the full spectrum of economic and social gains resulting from curing SCD—are incorporated, the balance shifts. This finding is crucial, as it affirms the broader value proposition of innovative gene therapies beyond the immediate medical setting, highlighting the profound ripple effects on families and communities.
The researchers emphasize that biologically consistent efficacy of gene therapy across populations underpins these projections, meaning the therapeutic benefits observed in U.S. clinical trials are expected to translate equivalently in Ugandan patients. Although local clinical trial data remain limited, this assumption is supported by the shared genetic underpinnings of SCD worldwide and a growing body of evidence confirming gene therapy’s robust effectiveness.
Importantly, the study advances a reproducible and adaptable modeling framework that other LMICs can employ to evaluate emerging therapies, even when direct clinical trial data or region-specific economic evidence are scarce. This framework represents a strategic tool to empower local policymakers, global health funders, and pharmaceutical manufacturers to make more informed decisions surrounding resource allocation and pricing negotiations, with the ultimate goal of fostering equitable access.
Given the staggering global burden of SCD—estimated to affect millions with high mortality and disability rates—such economic evaluations are not merely academic exercises but moral imperatives. The disproportionate impact on LMICs, where health systems are often overburdened and underfunded, calls for innovative financial and policy mechanisms. These might include tiered pricing models, international subsidies, and public-private partnerships to subsidize gene therapy introduction in resource-limited settings.
The findings by Montano-Campos et al. herald a crucial inflection point in the gene therapy era. They demonstrate that, contrary to conventional wisdom positioning gene therapies as prohibitively expensive for LMICs, strategic adaptation of economic models can reveal scenarios where these treatments are both clinically transformative and economically justifiable. This dual validation paves the way toward dismantling the entrenched disparities that have historically excluded the majority of sickle cell patients globally from the promise of curative interventions.
Crucially, the study also highlights the need for continuous data collection and health system strengthening in LMICs to refine and validate these models. Establishing real-world evidence for gene therapy implementation—including patient outcomes, delivery costs, and system capacity—is essential to support sustained access and adaptive policy development as the field evolves.
Ultimately, this research offers a pragmatic blueprint for global health equity in the genomic medicine revolution. It champions the idea that with rigorous economic evaluation tailored to local realities, alongside collaborative innovation and policy commitment, groundbreaking treatments need not remain the exclusive province of wealthy nations. Instead, gene therapies could become accessible lifelines for millions suffering from debilitating genetic diseases in the world’s most vulnerable regions.
As gene therapy technology matures and manufacturing costs potentially decline, the window of opportunity widens to translate cutting-edge science into widespread health gains. The study’s framework equips stakeholders with critical tools to navigate this evolving landscape responsibly and ethically. It signals a hopeful trajectory wherein medical breakthroughs are not confined by geography or wealth but rather guided by an inclusive vision of global health justice.
In conclusion, the innovative cost-effectiveness assessment applied to Uganda’s SCD context challenges entrenched assumptions about the feasibility of introducing gene therapy in low-resource environments. By synthesizing high-income data with local economic scaling, Montano-Campos and colleagues illuminate a path toward affordable, equitable cures that could reshape the lives of millions worldwide. Their work underscores that precision medicine’s promise is most powerful when paired with precision public health—a strategy that marries technological innovation with contextualized economic insight to achieve maximal social impact.
The study’s implications extend far beyond sickle cell disease or even gene therapy alone. It provides a replicable model for evaluating any emerging health technology in settings where traditional clinical and economic data may be lacking, thereby expanding the reach of advanced medical innovations globally. As the field progresses, building on such interdisciplinary frameworks will be pivotal in ensuring that scientific frontiers translate into tangible health improvements for all populations, including those historically underserved.
With global health systems facing ever-growing demands, the development of economically viable models that promote access to breakthrough treatments in under-resourced regions represents a new frontier. Montano-Campos’ team has taken a bold first step into this territory, showcasing how thoughtful economic science can serve as a catalyst for equitable healthcare transformation on a planetary scale.
Subject of Research: Cost-effectiveness of gene therapy for sickle cell disease in a low-income country setting, with a focus on Uganda.
Article Title: Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda’s context.
Article References:
Montano-Campos, J.F., Adair, J.E., Basu, A. et al. Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda’s context. Gene Ther (2026). https://doi.org/10.1038/s41434-026-00598-1
Image Credits: AI Generated
DOI: 26 February 2026
