薬理と治療

Abstract

Objective　The emergence of new modalities, including gene therapies, has raised in the multifaceted value of hemophilia treatment. This study aimed to collect a wide range of evidence on the value of hemophilia treatment in Japan, mainly from the health economic and patient–reported outcome perspectives, through a systematic literature review. Methods　The target databases were PubMed（since 1990）, the Japanese literature databases ICHUSHI Web and CiNii. We developed a search strategy that included keywords related to “hemophilia,”“cost,”and“patient–reported outcome,” and selected evidence from any study design in Japanese hemophilia patients, excluding those related to efficacy and safety outcomes. Results　Forty articles were finally identified; 3 were published in the 1990s, 10 in the 2000s, 16 in the 2010s, and 11 in the 2020s. By outcome, 11 articles assessed economic outcomes including direct medical cost and work productivity, 5 assessed patientreported outcomes including health related quality of life（ HRQOL） and activities of daily living（ ADL）, and 24 assessed other outcomes. Other outcomes included treatment frequency, schooling, employment, and the psychological burden of patients and caregivers. Of the 24 articles with other outcomes, 15 presented only qualitative outcomes. Conclusion　There has been a body of evidence in Japan focusing on the multifaceted value of hemophilia in addition to the traditional outcomes of treatment cost and quality of life. However, many of the articles discussed the value of hemophilia treatment only qualitatively, not quantitatively.（Jpn Pharmacol Ther 2023；51：1649‒65）