Office for Advanced Medical Care Evaluation and Health Technology Assessment

Introduction

 The Office for Advanced Medical Care Evaluation and Health Technology Assessment (hereinafter called the "Office") was established in November 2013 to perform evaluations of advanced medical treatments using anti-cancer drugs with high unmet medical needs that are commissioned by the Health Policy Bureau of the Ministry of Health, Labour and Welfare (MHLW) and conducted by an external organization.

 The Office sets up and manages the Assessment Committee on Advanced Medical Care in order to provide support for the creation of study protocols and regulatory submission activities. This includes consultation with the regulatory authority for institutions such as core clinical research hospitals that intend to utilize the Advanced Medical Care B Program to conduct clinical studies of anti-cancer drugs designated as potential treatments with high medical needs by the Evaluation Committee on Unapproved or Off-label Drugs with High Medical Needs. The Office also reviews the technical validity and protocols of proposed clinical studies, and reports the results of such reviews to the Advanced Medical Care Meeting.

 Since 131I-MIBG (indicated for pheochromocytoma, neuroblastoma, medullary thyroid cancer, etc.) is listed as one of the anti-cancer treatments to which the Advanced Medical Care B Program can be applied, we have considered its developmental strategy in coordination with clinical experts, the pharmaceutical industry and regulatory authorities.

 At the end of October 2016, we reviewed an interim draft of the protocol of the "Study on Internal Radiation Therapy Using 131I-MIBG in Patients with High-Risk Neuroblastoma" proposed by Kanazawa University Hospital. On December 27, 2016, we attended a meeting prior to the consultation on drug R&D strategy applied by Kanazawa University Hospital. At the third meeting of the Assessment Committee on Advanced Medical Care held on February 20, 2016 (at the National Cancer Center Hospital, organized by the Office), the proposal of the "Study on Internal Radiation Therapy Using 131I-MIBG in Patients with High-Risk Neuroblastoma" submitted by Kanazawa University Hospital was reviewed. Subsequently, it was also reviewed at the 54th Advanced Medical Care Meeting on June 8, 2017.

 This proposal was implemented through the Advanced Medical Care B Program as the "Study on Internal Radiation Therapy Using I-131 3-iodobenzylguanidine (131I-MIBG) in Patients with Intractable Pheochromocytoma" from January 2016 to December 31, 2017.

 In the context of this study, the MHLW solicited companies to develop this treatment. FUJIFILM RI Pharma (currently FUJIFILM Toyama Chemical) took over this study and is now conducting its phase II clinical study in Japan.

 The Office has explored anti-cancer drugs that have been approved in Europe or the United States but not in Japan, so as to identify target drugs to which the Advanced Medical Care B Program can be applied. It has been publishing a list of these drugs on its webpage since June 2014.

 With the advancement of medical technologies, the costs of drugs and medical devices have soared and there has been concern that this affects the financial status of the medical insurance system. Therefore, the Central Social Insurance Medical Council established the Subcommittee on Cost-Benefit Evaluation in May 2012 and experimentally introduced the cost-benefit evaluation system in April 2016. In this system, the costs and benefits of a drug or medical device are evaluated based not only on data provided by the company but also on re-analysis conducted by the re-analysis group from a neutral perspective. The Office also cooperates in preparing for re-analysis by the re-analysis group.

Research achievements

• The Study on Internal Radiation Therapy Using 131I-MIBG in Patients with Intractable Pheochromocytoma, which the Office supported in consultation with the regulatory authority, was conducted at Kanazawa University Hospital through the Advanced Medical Care B Program (February 1, 2016 to January 1, 2018).
• The Study on Internal Radiation Therapy Using 131I-MIBG (Neuroblastoma), which the Office supported in consultation with the regulatory authority, is now being conducted at Kanazawa University Hospital through the Advanced Medical Care B Program (July 1, 2017 -).
• The list of anti-cancer drugs that have been approved in Europe or the United States but not approved in Japan was updated in February, April, July, September, and December 2017.