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薬理と治療
Abstract
Compassionate use(CU) is a government program allowing the public to access unapproved drugs exceptionally to treat patients with life-threatening or seriously disabling diseases. It requires enough balance within three elements: accessibility for patients, safety insurance, and prevention of any barrier to complete clinical trials. Although the Ministry of Health, Labor and Welfare(MHLW)Advisory Committee recommended introducing CU in 2007, Japan still have not set up any program for CU. There has been a lot of discussion, but CU is still not available for the public. The crucial points of debate could be blurred by discussing at the same time on traditional quasi-CU and on the new preceding movement of using unapproved drugs. In this paper, we systematically reviewed the published literature and materials and arranged them based on what is and what is not CU. As the results, the following are considered not CU: 1) access to an off-label use of approved drugs; 2) an expanded access program(EAP) which allows a company to have early access to their promising drugs after their own judgment; 3) emergency use of unapproved drugs in a social crisis; 4) use of unapproved drugs in advanced medical care. Concerning systems relating to CU, we discussed on ‘Treatment IND’ and the ‘rule of rescue’. We also described a trend of CU in the world in 2012, e.g. the consultation on an introduction of ‘an early access to medicines scheme’ in the UK, and tightening CU by the French government regulatory reform after Mediator scandal. We then discussed what Japanese version CU is expected to be from this review. We think the following are important: 1) not to emphasize a patient’s self-responsibility too much; 2) to deal with adverse events appropriately; 3) to consider patient’s paying ability. Learning from the appropriate concept of CU and overseas experience, these problems are likely to be solved. We hope that this paper will contribute to establish a Japanese version of CU.
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