The Ministry of Health and Welfare said on the 28th it will improve related regulations to move up the timeline for clinical research and treatment under advanced regenerative medicine. The move follows the second core regulation rationalization strategy meeting held on Oct. 16, and the government reported the improvement plan it prepared to the Policy Committee on Advanced Regenerative Medicine and Advanced Biopharmaceuticals.
Advanced regenerative medicine is a treatment technology that restores or recovers the structure or function of the human body using stem cells, immune cells and the like, and targets serious, rare and intractable diseases. The government said the focus of this reform is to reduce researchers' institutional uncertainty and to promote the transition to treatment.
First, it will make concrete the criteria for "intractable diseases," which have often been criticized as vague. Although a treatment system for advanced regenerative medicine targeting serious, rare and intractable diseases was introduced in Feb. this year, unlike serious or rare diseases, intractable diseases lack a clear definition, raising concerns that researchers have difficulty determining in advance whether a case qualifies for treatment.
To address this, the ministry set criteria that allow case-by-case determinations of treatment need instead of listing disease names. It also revised the related guidelines by presenting examples of cases and interpretations submitted to the existing review committee, and this will take effect in Jan. next year.
The burden of submitting nonclinical test data for medium- and low-risk clinical research will also be eased. Until now, even low-risk studies were required to submit nonclinical data at the same level as high-risk studies, drawing criticism that the burden on researchers was heavy. Going forward, unnecessary data will be excluded depending on cell type (stem, immune, somatic, etc.), and the submission scope will be differentiated.
Where existing research literature can substitute, it will be accepted, and submission may be omitted for nonclinical tests that are impossible or meaningless. The revised guideline will also take effect in Jan. next year.
The use of overseas clinical data will also be expanded. Under the current system, advanced regenerative medicine treatments can be connected only after safety and efficacy are verified through domestic clinical research, which has delayed treatment timing. To address this, the government plans to expand the regulatory sandbox so that, when overseas clinical trials or clinical research results are sufficient, review of treatment plans can proceed based on those results. In Feb. next year, it will announce related projects to recruit medical institutions and cell processing facilities that wish to participate.
The government will also promote government-led clinical research that reflects patient demand. There is demand for traveling abroad, including to Japan, to receive advanced regenerative medicine, but officials judge that treatment is not sufficiently available domestically. The ministry plans in Mar. next year to solicit multicenter clinical research to verify the safety and effectiveness of autologous stem and immune cell culture treatments for diseases with high demand for overseas treatment, such as degenerative arthritis and chronic pain.
In addition, to strengthen review expertise, it plans from next year to reinforce the review system by increasing review personnel and expanding the pool of expert members.
Jeong Kyung-sil, director general for health care policy, said, "Advanced regenerative medicine is a technology that offers new treatment possibilities for serious, rare and intractable diseases," adding, "Through this regulatory improvement, we will energize clinical research and move up implementation of treatments to expand patients' opportunities for care."