Ethical considerations for patients' access to investigational medical products

2019 Fiscal Year Final Research Report

• Project/Area Number: 17K17665
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Medical engineering assessment; Medical sociology
• Research Institution: National Cancer Center Japan
• Principal Investigator: Nakada Haruka 国立研究開発法人国立がん研究センター, 社会と健康研究センター, 研究員 (10592248)
• Project Period (FY): 2017-04-01 – 2020-03-31
• Keywords: 臨床試験 / 未承認薬 / コンパッショネートユース / 患者申出療養制度 / 研究倫理

Outline of Final Research Achievements

This study focused on the similarities and differences of non-trial access system to investigational medical products in Japan and the U.S. One of the major common issues is that the final decision whether a patient can use the investigational product depends on the pharmaceutical company which develops the product. There is no legal or regulatory obligation for the company. The major difference among the systems is its main purpose; the U.S. system focuses on single patient’s treatment; the Japanese system focuses on collecting data from the patients. In addition, this study indicated that the information on the Japanese system does not disseminate among the patients and families by surveying patient organization websites. It would be useful to collaborate with patient organizations to disseminate the relevant information. Furthermore, publishing the actual cases of patient’s access to the investigational product would be useful for patients and families to understand the system.

Free Research Field

研究倫理

Academic Significance and Societal Importance of the Research Achievements

本研究は、日米の未承認薬利用制度の比較を通じて、未承認薬利用に関わる倫理的・社会的課題を検討した。日本では、がん遺伝子パネル検査や医療AIによる治療法提案など患者が未承認薬利用を検討する機会の増加が予想される中、それに関わる課題が明らかにされたことにより、患者・市民や倫理審査委員会、医療機関などに提供すべき情報の検討材料となる。また、学術的意義として、研究と診療のはざまに生じる倫理的・社会的課題の検討や、倫理審査委員会の役割の検討などに知見を提供できるものと考える。