Office for Advanced Medical Care Evaluation and Health Technology Assessment

Yasuhiro Fujiwara, Kan Yonemori, Natsuko Okita, Emi Noguchi, Nobuko Ushirozawa, Seiichiro Yamamoto, Taro Shibata, Aya Kuchiba, Reiko Uokawa

Introduction

 In November 2013, our office was established by the NCC as a secretariat to "evaluate advanced medical treatments involving anti-cancer drugs due to high unmet medical needs", a project commissioned by the Health Policy Bureau of the Ministry of Health, Labour and Welfare (MHLW).

 Our office's mission is to provide support for institutions, including the "core clinical research hospitals", that are going to conduct clinical studies of anti-cancer drugs identified as potential treatments for diseases with high unmet medical needs by the Evaluation Committee on Unapproved or Off-label Drugs with High Medical Needs, within the framework of the Advanced Medical Care B program of the MHLW.

The Team and What We Do

 We assist institutions by 1) preparing their study plans, 2) supporting their application procedures, e.g., facilitating discussions with regulatory authorities, and 3) reviewing the technical adequacy of the applications and the content of the study implementation plans by establishing and operating the Assessment Committee on Advanced Medical Care. We also report the assessment results to the Advanced Medical Care meeting.

 As of now, the anti-cancer drugs expected to be covered by this system include 131I-MIBG (pheochromocytomas, neuroblastoma, medullary thyroid cancer, etc.). We discussed their development strategy in coordination with clinical experts, the pharmaceutical industry, and regulatory authorities.

 We also make a list of unapproved anticancer drugs (i.e., those approved in the United States and/or the European Union, but not in Japan) for the understanding of drugs as candidates for the framework of the Advanced Medical Care B. Such anticancer drugs may also be candidate to the Patient-requested Treatment.

 The Central Social Insurance Medical Council (Chuikyo) established the Special Committee on Cost-Effectiveness Evaluation in May 2012 and started the trial introduction of the Cost-Effectiveness Evaluation in April 2016. In addition to the submission of data by the company, the cost-effectiveness assessment will include reanalysis from a neutral standpoint by the reanalysis group. Our office is also helping the evaluation for the reanalysis group.