Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations
Garrison L, Pezalla E, Towse A, Yang H, Faust E, Wu E, Li N, Sawyer E, Recht M. Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations. Value In Health 2021, 24: 1628-1633. PMID: 34711363, DOI: 10.1016/j.jval.2021.05.008.Peer-Reviewed Original ResearchConceptsGene therapyDurability of benefitAbsence of randomized controlled trialsQuality of life measuresHemophilia gene therapyLong-term outcomesHealthcare system perspectiveShort-term therapySelf-controlled trialRandomized Controlled TrialsLong-term health benefitsLife measuresOne-time treatmentExpert clinical opinionLong-term benefitsTherapyHemophiliaConventional treatmentControlled TrialsChoice of perspectiveClinical opinionLead-InChronic diseasesTreatmentComparative effectiveness
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