2019 年 36 巻 4 号 p. 539-542
MHLW launched a committee called “the Evaluation Committee on unapproved or Off–labeled Drugs with High Medical Needs” in 2009 in order to address the issue that many unapproved drugs or off–label uses of drugs are approved in the other developed counties but not in Japan. The MHLW's initiative worked well to let academia and industry collaborate to resolve the situation and showed certain progress, although there has been some issue remained. For example, difference in dosage approved among countries still exist in many cases and generally high dose which is approved in other countries is not approved in Japan. Much effort is needed to get approval of off–label use drug and fill the gap between the indication and clinical practice in pediatric setting. Also burden to development unapproved drug need to be appropriately taken care off. As is often the case with the discussion with regulatory bodies, Ministry of Health, Labor and Welfare (MHLW) has tendency to mandate clinical trial for the drug, which seems unnecessary from academia and industry's perspective.
In this article, our experience to get approval of off–label indication of methylprednisolone for the indication of acute exacerbation of Multiple Sclerosis (MS) was introduced. The safety and efficacy of intravenous methylprednisolone pulse therapy for the patients with MS has been well established and it's used as a standard treatment world widely including Japan with the guideline recommendation. However MHLW judged that there is not enough evidence in Japanese patients and requested to conduct a surveillance of drug utilization to assess actual usage of intravenous methylprednisolone pulse therapy for the Japanese patients with MS. The surveillance was led by Japanese Society of Neurology and twenty–eight neurology department of major hospital all over the Japan participated. Based on the result, “Kochi” application was accepted and the result was published by international journal. Eventually it became good example to use real world evidence for regulatory approval, which have been hot topics these days. In the future it would be desirable to establish more efficient way to collect real world data and across hospital disease registry would be one of the options.
