Closing the Gene Therapy Gap Early: Preparing a High-burden Health System for a Cure it cannot Yet afford

Domaine:

healthcare

Type de record:

paper

Créateur:

Amb

Éditeur:

Hôte:

Background. Two curative therapies for sickle cell disease were approved in the United States in December 2023 (US Food and Drug Administration, 2023), and England began treating eligible patients with one under a managed access arrangement in January 2025 (National Institute for Health and Care Excellence, 2025; NHS England, 2025). Approximately 79% of the world's affected births occur in sub-Saharan Africa (GBD 2021 Sickle Cell Disease Collaborators, 2023). The therapies exist where the disease largely does not, and remain absent where it is concentrated.

The pricing problem, quantified. Modelling of value-based prices across 13 countries produced base-case prices ranging from US$1,881 in Uganda to US$1.56 million in the United States, with scenario estimates ranging from US$676 to US$3.61 million. These differences reflect willingness-to-pay thresholds of US$120–3,228 per disability-adjusted life year averted in low- and middle-income countries versus US$33,016–100,000 in high-income countries (Morgan et al., 2024). A subsequent Ugandan analysis found one therapy potentially cost-effective at a scaled price when societal benefits are included (Montano-Campos et al., 2026).

Argument. This paper argues that price is the visible obstacle, not the binding one. Even at a price of a few thousand dollars, delivery would require capabilities Nigeria does not currently possess: a regulatory pathway for advanced therapy products; apheresis and transplant-grade centres; a patient registry capable of identifying and following candidates; and a health technology assessment function able to make and defend coverage decisions. Each requires years to build and cannot simply be procured when prices fall.

Contribution. The paper distinguishes preparatory capabilities that are justified regardless of whether
curative therapy ever arrives from those that are not, and recommends investment only in the former; specifies
what a credible refusal capability looks like and why it protects rather than restricts patients; and sets out the
equity claim that Nigerian institutions should be advancing now, while the pricing architecture for these
products is still being decided..

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