Office for Advanced Medical Care Evaluation and Health Technology Assessment

Yasuhiro Fujiwara, Kan Yonemori, Seiichiro Yamamoto, Taro Shibata, Aya Kuchiba, Shogo Nomura, Chisato Hamashima, Nobuko Ushirozawa, Hanae Tanaka

Activity of advanced medical care evaluation

Introduction

In November 2013, our office was established by the National Cancer Center (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 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.

Routine activities

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 are currently discussing 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 a target of this system.

Activity of cost-effectiveness analysis

The project for preparations of cost-effectiveness analysis for trastuzumab emtansine was accepted by the National Institute of Public Health and the following issues were performed;

1)A systematic review for additional effectiveness and adverse effects of trastuzumab emtansine was completed.

2)As for the preparation of the cost-effectiveness analysis for trastuzumab emtansine, the model's validity for the cost-effectiveness analysis was assessed. In addition, the parameters were collected and evaluated the adequateness.

3)HTA (health technology assessment) reports from the following institutions were scrutinized; NICE (UK), PBAC (Australia), CADTH (Canada), IQWIG (Germany), and HAS (France).