Paying for Cures: The Ethics and Economics of Gene Therapies for Rare Diseases
Rare diseases, most of which have a genetic basis, collectively affect an estimated 300 million individuals worldwide and are associated with significant morbidity and mortality. Although less than 10% of rare diseases currently have an approved therapy, advances in gene and gene-targeted therapies have brought hope of effective—or even curative—treatment. While the potential for cure has prompted justifiable excitement, the high upfront cost of many of these therapies also raises complex ethical and policy issues.
In response to these challenges, health economists have proposed a range of revisions to traditional value assessment frameworks to include elements relevant to gene therapy, such as “scientific spillover” and “value of hope,” in addition to “health outcomes and costs.” These efforts are focused on systematically assessing the benefits and costs of these therapies to provide evidence of their value and, by extension, their price. However, a lack of real-world data on the long-term effectiveness of these therapies necessitates that these assessments are inherently based on unconfirmed assumptions. In response, policymakers and payers are increasingly…
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ELSIhub Collections are essential reading lists on fundamental or emerging topics in ELSI, curated and explained by expert Collection Editors, often paired with ELSI trainees. This series assembles materials from cross-disciplinary literatures to enable quick access to key information.