Journal
EXPERT REVIEW OF PHARMACOECONOMICS & OUTCOMES RESEARCH
Volume 21, Issue 6, Pages 1145-1158Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1080/14737167.2021.1969229
Keywords
Gene therapies; health technology assessment; cost effectiveness analysis; rare diseases
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The paper discusses the impact of differentiated severity and unmet needs of genetic diseases on the economic analysis of GTs.
Introduction: The limited evidence in the clinical trials of gene therapies (GTs) posed substantial challenges for a reliable health technology assessment (HTA). This paper provides insights into the relationship between the background of diseases and the health economics assessment of GTs.Areas covered: The impacts of differentiated severity and unmet needs of genetic diseases, on the economic analysis of GTs, were discussed.Expert opinion: GTs offer a potential cure or significant clinical improvement, while limitations in clinical evidence constitute major obstacles for a robust assessment of clinical effectiveness and economic outcomes. This uncertainty may be balanced by the severity of the targeted condition and the associated unmet needs, thus leading to a relatively higher acceptance for GTs. Overtime, HTA agencies will become more demanding on comprehensive evidence of long-term effectiveness. With a growing number of GTs on the horizon, to what extent the unmet needs of previously devastating diseases will be fulfilled remain unclear. Nonetheless, comparative studies, either with a historical control group or existing treatments, will be necessary to demonstrate the additional benefits associated with GTs.
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