or the first time in history, Japan’s new Clinical Research Act, enforced in April 2018, legally defines the term “clinical research” as clinical trials designed to establish the efficacy and/or safety of drugs, medical devices, or cellular and tissue-based products.

However, the new Act may add another bureaucratic step for researchers seeking to advance clinical trials.

The definition excludes clinical trials related to the approval of new medical products that are defined and conducted in accordance with the provisions of the Pharmaceuticals and Medical Devices Act (“Chiken”). The new legal definition is not in line with the global consensus on clinical research, which covers a wider area, including observational studies. Therefore, it might become necessary to refer to clinical research defined by this act as “clinical research-in-law” to distinguish it from our conventional understanding of clinical research.

At present, the Clinical Research Act is legally enforced only for so-called “specified clinical research”:

• “Clinical research” involving unapproved or off-label medical products.
• “Clinical research” involving on-label medical products sponsored by their manufactures/distributors.

The “specified clinical research” must be approved by an ethical committee authorized by the Ministry of Health, Labor, and Welfare (MHLW), and the MHLW must be notified before it is initiated.

What the new Act doesn’t do

Although the Clinical Research Act covers trials involving cellular and tissue-based products, clinical studies on the Safety of Regenerative Medicine Act are not regulated by the Act.

The new Act also does not offer a solution to what many consider one of the biggest obstacles to clinical trials involving unapproved or off-label medicinal products, medical devices, or regenerative products in Japan. MHLW prohibits “mixed medical services” that would involve the combined use of insured and uninsured medical services in a series of medical treatments. The use of an uninsured treatment together with an insured treatment is only permitted under “Chiken” (the Pharmaceuticals and Medical Devices Act), or as an “advanced medical service system,” which is covered by the Medical Service Act.

The researchers should apply for “advanced medical service system” designation when they plan “specified clinical research” that involves unapproved or off-label medical products. However, this process starts after the ethical board approval, and takes at least several months and large amount of paperwork as well as two advisory boards reviews.

References available on request.