Justice in the Distribution of Gene Therapies for Rare Diseases

Abstract

Gene therapies for rare diseases combine unprecedented clinical promise with list prices that break the actuarial assumptions underlying conventional health insurance, since a durable, often lifelong benefit is billed as a single upfront cost. This policy analysis surveys the ethical and economic literature on how these therapies are priced, the financing innovations -- outcomes-based contracts, annuity-style installments, and pooled or supranational funds -- proposed to make that pricing tractable, and the deeper equity gap separating health systems that can experiment with these mechanisms from the much larger population of eligible patients in low- and middle-income countries. It examines the evidentiary uncertainty underlying value-based pricing claims at launch, the practical obstacles to implementing outcomes-based and risk-pooling arrangements, and the upstream diagnostic and delivery infrastructure gaps that limit access even where financing is resolved. It then considers competing justice frameworks -- rule-of-rescue intuitions, prioritarian weighting of the worst-off, and fair-innovation arguments -- that inform how health systems reason about coverage decisions for identifiable, severely affected patients. We argue that financing innovation, diagnostic infrastructure, and global differential pricing must be treated as a single connected policy problem, since addressing any one in isolation risks reproducing the same distributive inequities in a new form.