A landscape analysis and discussion of value of gene therapies for sickle cell disease
| Autor: | Dalyna, Quach, Boshen, Jiao, Anirban, Basu, M A, Bender, Jane, Hankins, Scott, Ramsey, Beth, Devine |
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| Rok vydání: | 2022 |
| Předmět: | |
| Zdroj: | Expert Review of Pharmacoeconomics & Outcomes Research. 22:891-911 |
| ISSN: | 1744-8379 1473-7167 |
| DOI: | 10.1080/14737167.2022.2060823 |
| Popis: | Sickle cell disease (SCD) is a rare genetic disease with limited therapeutic options. Gene-based therapies are being investigated in clinical trials to evaluate their curative potential. The expected life-long benefits of one-time administration of genetically corrected stem cells present uncharted challenges in estimating value of these treatments. Our objective is to conduct a landscape analysis of clinical trials and prompt a discussion estimating the value of gene therapy as a therapeutic option for SCD.We searchedGene therapies could represent a major advance in SCD treatment. Although clinical trials are ongoing, reports of serious adverse events have led to pause of these trials, emphasizing the need to prove long-term tolerability. Measured using the methods of health economic evaluation, we anticipate high up-front costs may be offset by potential life-long benefits of these treatments. During development and after treatment approval, attention should be focused on ensuring adequate availability and equitable access to emerging therapies in underserved areas and low-middle-income countries (LMIC). |
| Databáze: | OpenAIRE |
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