LAUSR

In Japan, the Pharmaceuticals and Medical Devices (PMD) Act established in 2014 included an additional chapter dedicated to frameworks for human cellular and tissue‐based products. To further evaluate the product considered to have “likely to predict efficacy” at the time of receiving “conditional and time‐limited marketing authorization,” a system has been introduced to determine whether the product is eligible for “full marketing authorization” through statistical “confirmation of the efficacy” in a postmarketing surveillance study using a registry. A movement similar to this regulation has been seen among Western nations. For example, in the United States, Regenerative Medicine Advanced Therapy Designation was introduced in 2017 as a provision of the 21st Century Cures Act. This is similar to Japan's conditional and time‐limited marketing authorization, which presumes efficacy of a product based on surrogate endpoints in life‐threatening diseases. It is true that the current study design has limitations, and study designs that are beyond our imagination should be developed in the future.