Topics Held a briefing session on the Cost-Effectiveness Evaluation System
On March 24, 2022, a briefing session on the "Cost-Effectiveness Evaluation System" was held in cooperation with the Economic Affairs Division, Medical Affairs Bureau, Ministry of Health, Labour and Welfare, hosted by the Pharmaceutical Manufacturers Association of Japan (PMAJ) and co-hosted by the Pharmaceutical Research and Manufacturers of America (PhRMA) and the European Federation of Pharmaceutical Manufacturers and Associations (EFPIA). The event was held online and attended by 278 people including the speakers.
The briefing was given by Mr. Shunsuke Yamamoto, Specialist in Medical Technology Evaluation Planning and Coordination, Medical Technology Evaluation Promotion Office, Medical Technology Evaluation Division, Health Insurance Bureau, Ministry of Health, Labour and Welfare, on the revision of the cost-effectiveness evaluation system for pharmaceuticals and medical devices to be applied from April 2022, as well as the "Guidelines for Analysis of Cost-Effectiveness Evaluation in the Central Social Insurance Medical Council (3rd ed. Mr. Takashi Fukuda, Director of the Center for Health Economics and Evaluation, National Institute of Health Sciences, and representative of the study group, explained the key points of the review of the "Analytical Guidelines for Cost-Effectiveness Evaluation at the Central Social Insurance Medical Council (Version 3)" (hereinafter referred to as the "Analytical Guidelines").
Overview and Practice of the Cost-Effectiveness Evaluation System
Shunsuke Yamamoto, Specialist Officer for Planning and Coordination of Medical Technology Evaluation, Office for Promotion of Medical Technology Evaluation, Medical Care Division, Insurance Bureau, Ministry of Health, Labour and Welfare
The review of the cost-effectiveness evaluation system for pharmaceuticals, medical devices, and regenerative medicine products in FY2022 was approved by the Central Social Insurance Medical Council (Chuikyo) Specialist Committee on Cost-Effectiveness Evaluation and the General Assembly on January 19, 2022 ( Figure 1 ).
Figure 1 Cost-effectiveness evaluation procedure
The "Review of the standard analysis process and analysis period" ( Figure 2 ) stipulates that the expert organization (ii) shall be held as soon as the public analysis is completed and the results are available, and that if comprehensive evaluation is possible at the time the expert organization (ii) is held, comprehensive evaluation shall be conducted at that time and the expert organization (iii) shall not be held. (iii) may not be held at the time when the expert organization (ii) is held, and furthermore, the expert organization may be held to hear appeals.
Figure 2 Review of standard analysis process and analysis period
In addition, if the company, the National Institute of Health Sciences, and the public analysis team in charge of the item agree to this, the committee may allow the participation of clinical experts, etc. from the first pre-analysis meeting. In addition, if the company, the National Institute of Health Sciences, and the public analysis team in charge of the product in question agree, the participation of clinical experts and others will be allowed from the first pre-analysis consultation.
With regard to the "arrangement of the treatment of the analysis target population," it was decided that when the size of the analysis target population becomes small, a part of the target population can be excluded from the analysis after clarifying the reasons, taking into consideration the number of patients and the nature of the disease, as well as the degree of impact on the overall evaluation, while also seeking the opinions of experts. The "reevaluation process after the completion of the evaluation" is described in the following paragraph.
Regarding the "reevaluation process after the completion of the evaluation," the National Institute of Health Sciences will select candidate items based on the results of evaluation by overseas evaluation organizations and reviews in medical journals, etc., and the expert organization will prepare a proposal on whether the selected items meet the criteria or not, which will be approved at the Chuikyo General Assembly. The above process will be used to select the candidate items. It was also explained that the applicability to the H3 category would be determined based on the above process.
Regarding "handling of additional indications," it was decided that if an indication is added before the analysis framework is determined, the analysis framework will, in principle, be determined including the added indication. If the entire analysis is expected to be delayed significantly, the analysis will be conducted without including the efficacy, and the applicability to the H3 category will be verified again after the determination of the cost-effectiveness evaluation plan.
Regarding "treatment of cost increase," it was explained that the lowest price adjustment coefficient would be used for the case where the cost increases while the effectiveness is the same (cost increase).
Regarding the "treatment of cases in which the analysis period is exceeded," if the analysis period is exceeded, the reason for the delay should be confirmed in advance, and if the reason is not reasonable, the smallest price adjustment coefficient should be used.
Regarding the "Treatment of patient ratio," the Board decided to use a patient ratio that can be made public in principle, and if it is difficult to make it public, the Board will request an explanation of the reason for the delay.
Regarding the "handling of nursing care costs," it was decided that the study group will continue to conduct research on public nursing care costs, etc., with reference to efforts in other countries, and that the progress of the research will be taken into consideration in the future.
With regard to "strengthening the analysis system in response to the increase in the number of items subject to analysis," it was decided that efforts to enhance the evaluation and analysis system, including the expansion of human resource development programs, will be systematically promoted in order to ensure stable operation of the system in the future.
With regard to "collaboration with the NHI drug price calculation organization," the NHI drug price calculation organization will share in advance the evaluation of the items subject to cost-effectiveness evaluation, including the addition to the usefulness system of the items, with the expert organization for cost-effectiveness evaluation. The results of the cost-effectiveness evaluation of the item, etc., shall be shared with the NHI drug price calculation organization.
Regarding "measures to deal with conflicts of interest," currently, universities, etc. involved in work related to companies are not allowed to be involved in public analysis at all, regardless of their relationship with the product to be analyzed. However, in general, if the relationship with a company is within a certain standard, it is allowed to be involved in public analysis as a public analysis team.
Guidelines for Analysis of Cost-Effectiveness Evaluation at the Central Social Insurance Medical Council (3rd Edition)
Dr. Takashi Fukuda, Director, Health Economics Evaluation Research Center, National Institute of Health Sciences
The analysis work in the trial introduction was based on the "Guidelines for Analysis of Cost-Effectiveness Evaluation in the Central Social Insurance Medical Council (Version 1)" published in the fall of 2015, and based on issues obtained at that time, the "Guidelines for Analysis (Version 2)" was compiled (February 20, 2019, Central Medical Council Approved on February 20, 2019 by the Chuikyo). The "Analytical Guidelines (Version 3)" reflect the contents based on the framework of this report and necessary revisions based on the results of research conducted by the research group. The purpose of developing the Analytical Guidelines is to present the analytical methods to be used by the Chuikyo in conducting cost-effectiveness evaluations of target items, and covers analyses submitted by manufacturers and distributors as well as public analyses.
For the "Analysis Population," the percentage of patients in each population should in principle be based on the most recent clinical status of the patients for whom the technology is being evaluated.
With regard to the "comparison/control technology," it was decided that, in principle, the comparison/control technology to be evaluated should be one that is widely used clinically at the time the technology is introduced as a treatment for the target population and is expected to be replaced by the technology to be evaluated, and one that has a higher therapeutic effect. The principle concept is to select one technology that is widely used in clinical practice and is expected to be substituted by the technology to be evaluated.
With regard to "additional usefulness," it was decided that, in considering cost-effectiveness, the first step is to evaluate whether the data demonstrate the additional usefulness of the technology under evaluation in comparison with the comparator technology.
Regarding the "choice of effectiveness indicator," the EQ-5D-5L is recommended as the first choice when collecting new QOL values in Japan for cost-effectiveness analysis, and in the case of analysis from the "public medical and nursing care perspective," the impact on QOL values on family members and other caregivers and nurses may be taken into account if actual data are available. The impact on QOL values on family members and other caregivers and nurses may be taken into account if actual data are available.
Regarding "Treatment of Public Care Expenses and Productivity Losses," it was stated that if analysis including productivity losses is conducted, it may be conducted as an additional analysis in addition to the basic analysis, but whether productivity losses can be included or not must take into account the possibility of employment due to disease characteristics, etc. He also indicated that the productivity loss should be estimated based on domestic findings.
Questions and Answers
After the lecture, a question-and-answer session was held, during which the speaker carefully answered each of the questions that had been collected by each organization in advance and those that were submitted online by participants.
( Masahiro Kokubu, Team Leader, Cost-Effectiveness Evaluation System, Industrial Policy Committee, Industrial Development Subcommittee)