Gene therapy has a complicated history. Some early trials resulted in catastrophes, including subjects' deaths. In 2003, the world's first gene therapy product (GTP) was approved in China. More recently, EU and US regulators have successively approved seven GTPs, including chimeric antigen receptor (CAR) T cells for refractory cancers and an adeno-associated vector, for treating serious genetic disease. In Japan, where there are no approved GTPs, some clinics have provided domestically-unapproved GTPs for cancer patients; however, in some cases, bereaved individuals litigated against such clinics. Meanwhile, the advent of GTPs is becoming controversial because of the unprecedentedly high treatment cost. The present article has three aims: 1) Reconsider the ethical legitimacy of gene therapy and its use for serious conditions. 2) Compare the Japanese, EU and US regulatory situations concerning GTPs and underscore Japan's need for clearer and more up-to-date regulatory guidance. 3) Call for social understanding of GTPs and deliberations regarding the appropriate and acceptable cost, while noting that regulatory approval does not necessarily meet genetic disease patients' needs.
Keywords: cancer; chimeric antigen receptor; cost; ethics; gene therapy; genetic disease; regulation.
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