Policy Research Institute The Cost-Effectiveness Evaluation System in Japan: Past and Future -Implications from a Comparison of Japanese and English Cost-Effectiveness Assessment Systems

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In recent years, various factors other than efficacy, safety, and treatment cost have come to be considered in the value evaluation of pharmaceutical products. In particular, the cost-effectiveness evaluation system, which has been in effect for four years, has become more flexible, for example, by including the health-related quality of life of family members and other caregivers in the evaluation. On the other hand, as the value of pharmaceuticals and the sensitivity of their recipients diversifies, we thought it necessary to examine what the issues will be in future evaluations, referring to examples of evaluations in other countries that have introduced systems similar to this system ahead of Japan. This paper introduces OPIR Views and ActionsNo.68 "Japanese Cost-Effectiveness Evaluation System: Past and Future - Implications from the Comparison of Japan and the UK" (March 2023), which we studied based on the past study process of the Japanese cost-effectiveness evaluation system and the comparison of evaluation cases between Japan and the UK.

1. Introduction

The multifaceted value of pharmaceuticals has been manifested in various ways, including the emergence of new modalities (hereinafter referred to as "new modalities"). In addition, with the spread of novel coronavirus infection (COVID-19), health and economics have begun to be discussed as issues closer to home for the public. Focusing on pharmaceuticals, including vaccines, there has been growing interest in the impact of productivity losses and other issues associated with the spread of COVID-19 infection. In recent years, in the framework of Japan's cost-effectiveness evaluation system, it has become possible to evaluate the value of pharmaceuticals not only in terms of efficacy, safety, and treatment cost, but also in terms of the impact on work and the degree of reduced burden on family members and medical personnel, etc. It is extremely important to visualize the impact of these multifaceted factors of pharmaceuticals and to gain public acceptance for the system. It is extremely important to visualize the impact of these multifaceted factors and to gain the public's understanding. However, there are still some problems in visualizing the impact of these factors, such as the availability of data collection and the development of indices tailored to the Japanese population.

This paper describes how the cost-effectiveness evaluation system was studied and evaluated in the Health Technology Assessment (HTA) in Japan, and how elements that cannot be captured by the analytical framework and incremental cost-effectiveness ratio (ICER) have been studied and evaluated. The study examined how factors that cannot be captured by the analytical framework and incremental cost-effectiveness ratio (ICER) have been considered and evaluated. Comparisons were made with the National Institute for Health and Care Excellence (NICE) in the U.K., which evaluates medical technologies using similar analytical methods and has accumulated a large number of case studies. We will discuss whether the current cost-effectiveness assessment is sufficient or whether the process required to properly assess the various values of pharmaceuticals, in particular, needs to be expanded.

2. cost-effectiveness evaluation system in Japan

In Japan, the cost-effectiveness evaluation system became operational in April 2019. The Central Social Insurance Medical Council (Chuikyo) has been discussing the results of the implementation of the system, the results of the study by experts, and opinions from related industries, etc ., until the system was put into operation. The current cost-effectiveness evaluation system is in operation as a result of ongoing discussions at the Chuikyo Expert Committee on Cost-Effectiveness Evaluation.

Japan's cost-effectiveness evaluation system is unique compared to those of other countries in that it aims to select eligible products once they are covered by insurance and to adjust prices based on the evaluation results. As a result, medical services under the insurance coverage can be provided promptly without impeding the access of patients in need of treatment (hereinafter referred to as "patient access"). Since the purpose of this system is price adjustment, its analytical method is calculated based on ICER, and the system adopts a mechanism in which price adjustment is made based on whether the cost-effectiveness is superior when compared to the control technology, etc.

The nature of HTA in other countries varies, and as shown in Table 1, there are cases in which HTA is used to determine reimbursement and cases in which HTA is used for price adjustment.

Table 1 Cost-effectiveness evaluation criteria and utilization methods in other countries
Source: Prepared by The Office of Pharmaceutical Industry Researchbased on the website of the Pharmaceutical Research and Manufacturers of America (PhRMA)

Using NICE in the U.K. as an example, ICER is calculated based on quality-adjusted life year (QALY) *2 as the basis of analysis. If the calculated ICER is below a defined cost-effectiveness threshold (£20,000 to £30,000 per QALY, with other mitigation measures available), the cost-effectiveness is considered good. The results of this analysis are used to determine reimbursement, and for the period March 2020-December 2021, 52% (510/980) of all cases were Recommended, excluding anticancer drugs, while 14% (139/980) were Not Recommended under the same conditions. In the UK, reimbursement decisions are not based solely on cost-effectiveness results, but also on various considerations to avoid impeding patient access, such as mitigating thresholds due to prolonged life at the end of life, remedies for analyses involving uncertainty, and other factors that are taken into account by stakeholders (e . g., clinical experts and patient experts). Factors that cannot be incorporated into ICER, such as opinions from clinical experts and patient experts, and the magnitude of unmet medical needs, may also be considered in the process of comprehensive evaluation*4.

In Japan, there have been many discussions on comprehensive evaluation, and the history of these discussions is presented in the next section.

3. history of consideration of comprehensive evaluation (appraisals) in Japan's cost-effectiveness evaluation system

At the time of trial introduction, the comprehensive evaluation mainly considered "whether analysis by the distributor or public analysis is more appropriate from a scientific standpoint" and "whether the subject product is one that should be relaxed under the criteria, such as an anticancer drug. In this respect, the evaluation was not a literal "comprehensive" evaluation like NICE, etc., and the scope of the evaluation was considered to be quite limited*5.

In the evaluation of the trial introduction, Table 2 was proposed as the ethical and social factors to be considered in the comprehensive evaluation and the requirements for applicable items.

Table 2 Proposed requirements for items that fall under ethical and social considerations at the time of trial introduction
Note: The initial proposal refers to the proposal made at the 48th CPC Specialist Committee on Cost-Effectiveness Evaluation, and after reorganization,
The original proposal refers to the proposal made at the 48th meeting of the Specialist Committee on Cost-Effectiveness Evaluation of the Central Council of Medical Examiners.
Note: Items included in the ethical and social considerations are indicated as "0" in the table.
Source: Prepared by The Office of Pharmaceutical Industry Researchbased on data from the Chuikyo

However, two items, innovation and treatment for pediatric diseases, were subsequently deleted, and the evaluation of trial introduction was conducted within the framework of considering the four items shown in Table2*6 in the comprehensive evaluation. The four items shown in Table 2*6 were considered in the overall evaluation.

The reasons for the deletion of the items rearranged from the initial proposal are as follows. First, innovation was removed from the items based on the fact that it was already taken into account at the NHI price calculation stage*7. Second, the item for treatment of pediatric diseases was established with the background that it would not deprive pediatric patients, who are not numerous, of treatment opportunities, and, in fact, the cost-effectiveness evaluation committee of the Central Medical Council In fact, in a simulation using a hypothetical case study, the ICER was judged to be 7.5 million yen/QALY, which is not cost-effective, but the final evaluation was "cost-effectiveness evaluation is acceptable," taking into consideration the fact that "Drug A is a therapeutic drug for pediatric specific disease X, which has a poor prognosis. This is a typical overall evaluation from an ethical and social perspective in a comprehensive evaluation. However, since pediatric diseases were excluded from the cost-effectiveness evaluation, the discussion converged in the direction that they did not need to be added to the appraisal items, and they were removed*8.

Of the 13 items (7 pharmaceutical products and 6 medical devices) that were subject to the trial introduction, Table 3 presents the results of the evaluation of pharmaceutical products and whether they were subject to ethical and social considerations*9.

Table 3 Evaluation results of items subject to trial introduction (7 pharmaceutical products)
Source: Compiled by The Office of Pharmaceutical Industry Researchbased on materials from the Chuikyo

Table 3 shows the evaluation results for the seven pharmaceutical products that were subject to trial introduction. Of the seven drugs, five were found to be "useful from a public health perspective, such as in combating infectious diseases," and two were found to be "a treatment for a serious disease that does not significantly improve QOL but prolongs survival. Of the seven products, five were drugs for the treatment of hepatitis C, and two were anticancer drugs. Although these results do not apply to all drugs and selected items for cost-effectiveness evaluation due to the bias in the targeted diseases, the results of the ICER-based analysis and uniform decision criteria revealed that there were cases in which cost-effectiveness evaluation would be difficult. After the trial introduction, measures were established to raise the ICER criteria from the basic category for anticancer drugs and other drugs for which consideration was deemed necessary in the comprehensive evaluation ( Table 4 ). When adjusting prices, the price adjustment coefficients for usefulness addition, etc. and operating margin are weighted averaged by the ratio of patients in the applicable analysis population, etc., according to the ICER category.

Table 4 Classification of ICERs in cost-effectiveness evaluation in Japan
Source: Compiled by The Office of Pharmaceutical Industry Researchbased on materials from the Chuikyo

The consideration of "additional costs not included in the analysis from the standpoint of public healthcare" in Table 2 was allowed for additional analysis within the "Guidelines for Analysis of Cost-Effectiveness Evaluation in the Central Social Insurance Medical Council" (hereinafter referred to as "Analysis Guidelines"). In addition, it also states that the position of the analysis must be clearly stated and the range of costs must be determined accordingly, and is based on the "public medical care position," which is implemented within the scope of the public medical insurance system regarding costs, comparison and control techniques, target population, and other factors. The position of analysis and the range of costs are shown in Table 5.

Table 5 Position and cost range of analysis in Japan
Source: Prepared by The Office of Pharmaceutical Industry Researchbased on analytical guidelines

As mentioned above, since Japan is based on the public medical insurance system, the costs of medical technologies that are public medical costs, as well as all related medical costs such as hospitalization costs, examination costs, and costs when an adverse event (AE) occurs, are included. NICE has also included the costs of public care, family care, and productivity losses, which were not included in the price adjustment when it was introduced on a trial basis, but are now allowed to be analyzed if they are important to the medical technology, although they are not used in the price adjustment. In order to conduct basic analysis from the "standpoint of public health care and long-term care", NICE described the history of studies on the treatment of public long-term care costs in Japan.

Treatment of public care costs

In the UK, the Guidelines state that the "public health and long-term care position" is taken, while in Japan, the public health care position is used as the basis for the analysis. The Japanese government has stated in the analytical guideline that the public health care position is the basis of the Japanese government's position.

The second edition of the Analytical Guidelines, which was approved by the Chuikyo Assembly in February 2019, states that the treatment of public long-term care costs is not included in the basic analysis, but in additional analysis, public long-term care costs estimated based on domestic findings may be included. Although not used for price adjustment, it was positioned to be used as a reference for future mechanisms through publication of analysis results and accumulation of case studies*11.

In the third edition of the Guidelines for Analysis, which was approved by the Chuikyo Assembly in January 2022, the sentence that "analysis from the standpoint of public medical care and nursing care" may be conducted if the impact on public nursing care costs is important for the technology to be evaluated*12 remains unchanged, but an additional sentence was added: "In the case of analysis from the standpoint of public medical care and nursing care, the actual 12 The addition of the sentence that, in the case of analysis from the "public medical and nursing care perspective," the impact on QOL values for family members and other caregivers and nurses may be taken into account if the data are available.

In addition, with the recent enactment of the Act for Partial Revision of the Health Insurance Act, etc. for the Purpose of Appropriate and Efficient Management of the Medical Insurance System, the Receipt Information/Specified Health Examination Information Database (NDB) and the Long-Term Care Insurance Comprehensive Database (Long-Term Care DB) were revised to allow consolidated analysis of each database, and to promote their use for public interest purposes. We expect that the use of these databases for cost-effectiveness evaluations will increase in the future.

Treatment of productivity loss

The handling of productivity loss was also discussed in the same manner as public care costs. Until the second edition of the Guidelines for Analysis, the treatment of productivity loss was defined as follows: "Productivity loss resulting from the inability to work due to the disease in question is not included in the basic analysis. In additional analyses, the position is to consider a broader range of costs and productivity losses estimated based on domestic knowledge may be included. While not used for price adjustment, the results of the analysis were to be made public and examples accumulated for reference in future mechanisms. The treatment of productivity losses differs in other countries as well. The United Kingdom evaluates medical technology without including productivity loss in the analysis results. To cite one example, in the analysis of the technology for Bylvay (target disease: progressive familial intrahepatic cholestasis), which was evaluated in the NICE Guidance on Technology Assessment for Rare Diseases, the company submitted the cost including productivity loss, but after NICE pointed out that it was out of the standard method, the company recalculated the cost without productivity loss and the productivity loss was excluded from the analysis. However, after NICE pointed out that this was out of the standard method, the company recalculated the costs without the productivity loss, and the final evaluation was conducted.

Based on the history of consideration of these comprehensive evaluations, we investigated whether there were any cases in which a position other than public health care was taken in the cost-effectiveness evaluations conducted under the Analytical Guidelines Version 2, whether there were differences in the range of costs included in the analysis based on the results of the evaluations between Japan and the UK, and whether there was any consideration of comprehensive evaluations.

4. analysis of cost-effectiveness evaluation items that are no longer evaluated in Japan, based on a comparison between Japan and the U.K. 5.

Based on reports posted on the website*14 of the Center for Health Economics and Evaluation Research (CORE2 HEALTH, C2H) of the National Institute of Health Sciences as of December 2022, the item categories covered by this system and their trends over time are shown ( Figure 1 ).

Figure 1 Number of items subject to cost-effectiveness evaluation in Japan
Note: FY2022 is as of December 12
Source: based on reports published on the C2H website (post-institutionalization, 2019.4-2022.12)

Since the institutionalization of the cost-effectiveness evaluation, 4 items were selected as eligible items in FY 2019, 8 items in FY 2020, 15 items in FY 2021, and 10 items in FY 2022 as of December 12, 2022 ( Figure 1 ). The categories of these items are classified as H1 to H5, respectively. Please refer to the Supplement at the end of this document for the detailed classification of these categories. In the similar drug efficacy method, the items are those for which the additional fee for usefulness is calculated, and in the cost accounting method, among those for which the additional fee for usefulness is calculated or disclosed at less than 50%, the items with a peak market size of 10 billion yen or more are classified as H1 and those with a peak market size of 5 billion yen or more as H2. According to the publicly available information on C2H, all of the items designated as H2 have been evaluated.

The total time required for cost-effectiveness evaluation is 9 months for company analysis, 3 months for public analysis or 6 months for re-analysis, and 2 to 3 months for comprehensive evaluation by a specialized organization, which in total takes more than one year from the selection of items for evaluation. In Japan, not all drugs are subject to the cost-effectiveness evaluation system, but drugs and medical devices that are highly innovative and have a large financial impact are subject to the system (with some exceptions, such as for pediatric patients), so the number of cases is limited compared to other countries. The results of the analysis must be taken into consideration.

In this survey, we targeted the items in Figure 1 whose evaluation status had been terminated, and investigated whether there were any differences in the analysis other than "public health care cost position" and the treatment of costs or productivity losses included in the analysis between Japan and the U.K. The survey covered items that were not listed as similar items and for which information on costs and productivity losses was included in the information published by C2H. The survey covered items that were not listed as similar items, had information on costs and productivity losses in the information published by C2H, and for which NICE's evaluation guidance had been published ( Table 6 ).

Table 6 Comparative results between Japan and the UK in the analysis of the surveyed items (4 items)
Source: prepared by The Office of Pharmaceutical Industry Researchbased on information available on the C2H website and NICE's evaluation guidance

Although the costs incorporated into the analysis for the surveyed items (four items) varied depending on the comparison and contrast techniques used, only the portion of the costs that corresponded to Japanese public health care costs was included in the analysis for both Japan and the UK (for the UK, the results were from NICE), while in the UK, caregiver quality of life was included in the denominator of ICERs for some items. In the U.K., however, the denominator of the ICER includes the caregiver's quality of life. Based on these results, we reviewed the overall evaluation of the four items based on the UK NICE evaluation guidance for the differences between the UK and Japan, and listed the factors that led to their consideration ( Table 7 ).

Table 7: Japan-UK comparison of the position of the analysis of the surveyed items (4 items) and the range of costs included in the analysis
Note: Costs in Table 7 are extracted from those listed in the reports published on the C2H website and in NICE's evaluation guidance.
Note: The costs in Table 7 are extracted from those listed in the reports published in C2H and in NICE's evaluation guidance, so there are some costs that are not used by the comparison and contrast technique.
Note: CRS: Cytokine release syndrome
Source: Prepared by The Office of Pharmaceutical Industry Researchbased on information provided on the C2H website and NICE's evaluation guidance.

Items requiring consideration such as anticancer drugs

Although Cabometix (generic name: cabozantinib malate) was not found in the CABOSUN trial, economic models estimated that it prolonged life by an average of about 6 months compared to standard therapy (sunitinib and pazopanib), and NICE included this as an end-of-life consideration*15 (TA542: untreated renal cell carcinoma). Similar to NICE, a higher standard than usual (7.5 million yen/QALY) is allowed in Japan. This is because this is an item that requires anticancer drugs and other considerations in Japan's comprehensive evaluation process. The results of the cost-effectiveness evaluation showed that for first-line treatment of unresectable or metastatic renal cell carcinoma, sunitinib was used as the comparator technology and the ICER category was between 2 million yen/QALY and 7.5 million yen/QALY. The ICER category was 2 million yen/QALY or more but less than 7.5 million yen/QALY. For second-line or later treatment of unresectable hepatocellular carcinoma that had progressed after cancer chemotherapy, regorafenib was the comparator technology and the ICER category was cost reduction (equivalent efficacy and cost reduction compared to the comparator technology). The price adjustment factors based on ICER categories were all 1.0, weighted by the percentage of patients, resulting in no change in price adjustment.

With a plausible improvement in survival of 3 months, NICE concluded that ENHARTZ (trastuzumab deltasectan) was likely to meet the criteria for end-of-life treatment, but because it exceeded the ICER criteria, it was conditionally recommended using the Cancer Drug Fund (CDF). to be a conditional recommendation (TA704). Similar to NICE, a higher standard than usual (7.5 million yen/QALY) has been approved in Japan for ENHARTZ because it is an anticancer drug. The results of the cost-effectiveness evaluation of Enhertz showed that for third-line or later treatment of HER2-positive inoperable or recurrent breast cancer with prior chemotherapy, trastuzumab plus vinorelbine was the comparative control technology, and the ICER category was between 7.5 million yen/QALY and 11.25 million yen/QALY. For patients with HER2-positive (IHC 3+), curatively unresectable, advanced or recurrent gastric cancer that progressed after cancer chemotherapy and for whom third-line chemotherapy was administered, nivolumab was the comparator technology and the ICER category was between 11.25 million yen/QALY and 15 million yen/QALY, and for patients receiving third-line For HER2-positive (IHC 2+ and ISH+) patients receiving third-line chemotherapy, nivolumab was the comparator technology and the ICER category was cost-incremental (equal efficacy and increased cost compared with the comparator technology). The price adjustment factors based on ICER categories were, in descending order, 0.7, 0.4, and 0.1, resulting in a weighted average price adjustment of 2.15% based on the percentage of patients.

Cabometix and Enhertz received action by raising their thresholds because they were recognized as end-of-life treatments. Similarly, Japan uses a higher-than-normal standard (7.5 million yen/QALY) *16 for anticancer drugs, so these were found to receive similar consideration in Japan and the UK.

The results of the Japanese cost-effectiveness evaluation of Kymriah (generic name: tisagen lecleucel) are shown in Table 8. The report does not disclose patient percentages due to company secrecy, so detailed analysis results are not available. For the leukemia indication, the price was judged to be reasonable with an ICER category of less than 7.5 million yen/QALY for both target populations, but for the lymphoma indication, the price exceeded this threshold. As a result, a 4.3% price adjustment was made; since the NICE evaluation guidance did not meet the criteria for end-of-life treatment, the evaluation was made at the usual standard (20,000 to 30,000 pounds per QALY). Japan used a higher-than-normal standard (7.5 million yen/QALY) for evaluation, resulting in a difference in overall evaluation between the two countries. Although this is different from the overall evaluation, we would like to add that NICE recommends the use of Kymriah through the CDF, so it is possible to use it clinically in both Japan and the UK.

Table 8 Results of cost-effectiveness evaluation of Kimuria in Japan
Source: Compiled by The Office of Pharmaceutical Industry Researchbased on information on the C2H website and materials from the Chuikyo

Items related to public care costs

The third edition of the Japanese analytical guidelines states that the impact on QOL values on family members and other caregivers and nurses may be taken into account when the analysis is conducted from a "public health and nursing care standpoint. None of the four items included in the analysis took the public care position out of the non-public health care position ( Table 7 ), and although NICE allows the addition of the impact of public health care and care in the basic analysis, there were no differences in costs other than public health care costs among the items in this study, so we included them in the utility values We investigated whether.

The NICE evaluation guidance for Yurtomiris (generic name: labulizumab) included health-related quality of life, not only for patients but also for caregivers. This is due to the fact that the current therapy, eculizumab, is administered at 2-week intervals, whereas Yurtomiris is better administered every 8 weeks, among other factors. Caregiver health quality of life values have been changed to be used in the calculation of ICER since the third edition of the analytical guidelines and are expected to be included in future Japanese cost-effectiveness evaluation analyses.

The NICE evaluation guidance for Cabometix uses the utility values used in the guidance for tivozanib because QOL data were not collected in the CABOSUN trial. We checked the guidance for tivozanib and were able to confirm the statement regarding quality of life for patients, but were unable to confirm it regarding the impact on family members and other factors. Japan's analysis is from a public healthcare standpoint, but is based on data from the METEOR study (comparison of cabozantinib and everolimus), which is based on data from second-line treatment and beyond, and is estimated using Japanese quality of life values, so the impact of family and other factors is not shown.

Items related to productivity loss

Kimuria has an analysis other than the "public health care cost position" and has submitted the results of a company analysis that includes productivity losses ( Table 9 ). In accordance with the Analytical Guidelines, 2nd Edition, the analysis including productivity losses was not used to adjust prices, but the results of the analysis were such that the cost savings described with respect to productivity would be reduced. Kimuria had a significant impact on costs related to productivity losses because treatment allows for almost complete reintegration into society.

Table 9 Results of Kimria's cost-effectiveness analysis of Japanese companies
Note: B-ALL: Relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia; DLBCL: Relapsed or refractory CD19-positive diffuse large B-cell lymphoma
Source: based on unmasked information from reports published on the C2H website (post-institutionalization, 2019.4-2022.12)

It is difficult to say from this case alone whether the impact on Kimuria's productivity has the potential to be applied to other items in the future, and the accumulation of future evaluation cases is awaited. As mentioned above, NICE does not include productivity loss in its evaluation results. How the impact of productivity loss should be used for price adjustment in Japan is an issue to be considered in the future. Various things can be considered, such as whether the ICER category in the overall evaluation needs to be raised as in the case of anticancer drugs, etc., or whether a price adjustment factor that exceeds 1.0 should be used. However, the items selected for cost-effectiveness evaluation are those with significant fiscal impact, and it is necessary to take into account the social situation in Japan when considering price adjustment. In addition, since the impact of productivity loss is an indirect cost, the scope of its inclusion in the analysis, estimation methods, etc. still vary even in other countries. In the future, we will consider cases in which the impact of productivity loss is reflected in the evaluation, referring to recent cases in the Netherlands, South Korea, Sweden, and other countries where analysis including productivity loss has been conducted.

Treatment of public care costs in the UK NICE Technology appraisal (TA) guidance

Although there were differences between Japan and the U.K. in terms of the position of analysis, the Analytical Guidelines Version 3 states that analysis from the "public health and long-term care standpoint" may be conducted if the impact on public long-term care costs is important for the technology to be evaluated. Since Table 7 was limited to four target items, we based our discussion on a report examining another set of evaluation guidance from the United Kingdom.

Table 10: Examples of the treatment of public care costs in UK NICE (nervous system diseases)
Source: Hidetoshi Shibahara et al. in "UK NICE technology appraisal (technology appraisal) guidance
Treatment of public care expenditure in UK NICE technology appraisal guidance: a review of neurological diseases" *17 Source: Created by The Office of Pharmaceutical Industry Research

As a result of the study of 30 publicly available NICE technology appraisal (TA) guidance documents in the U.K. shown in Table 10, costs for in-home care services and facility care services covered by the Japanese long-term care insurance were included in 13 cases, and a further 6 cases were considered for inclusion*18. In six cases, it was difficult to identify whether or not each of the direct non-medical expenses included in the costs were within the scope of services covered by Personal Social Services (PSS), so the final analysis excluded direct non-medical expenses such as nursing care costs . The final decision was made based on the results of an analysis that excluded medical costs.

Although this report is limited to neurological diseases, based on the position of the U.K. analysis, 43% (13/30) of the cases included costs related to public long-term care expenses.

5. summary and future prospects

This report examines the operation of Japan's cost-effectiveness evaluation system to date and, through comparison with the UK, considers whether the current cost-effectiveness evaluation is sufficient or whether the process required to properly evaluate the various values of pharmaceutical products needs to be expanded. In the current comprehensive evaluation, the standard values for items that require consideration, such as anticancer drugs, have been raised, and consideration for anticancer drugs seems to be more flexible compared to the lack of standard values such as for end-of-life treatment in the UK. On the other hand, there is a move to include QOL values for family caregivers, etc. in the analysis, but how to evaluate public care costs and productivity loss in the future, which could be an indicator of social factors*18 such as reduction of the burden of caregiving on the patient's family and improvement of labor productivity, etc. Although there is a move to include family caregivers' and others' QOL values in the analysis, it is not clear whether the reduction of the burden on family caregivers (physical and However, how to reflect this in the system is an issue to be considered in the future, such as how to reduce the burden on family caregivers (physical and mental burden, etc.) *5 and services covered by PSS, which are reflected and taken into account in the analysis in the U.K. but have not yet been used for evaluation in Japan, or the problem of obtaining Japanese data, etc. As mentioned in the handling of public care costs, in the cost-effectiveness evaluation system for the purpose of price adjustment, those already incorporated into ICER, as in the U.K., should be incorporated into the analysis, for example, QOL of family caregivers should be included in the analysis, and examples from other countries, including the U.K., should be referred to and incorporated into the analysis, or the comprehensive evaluation Various measures could be considered, such as raising the criteria for ICER categories as requiring consideration like anticancer drugs in the process, or establishing a price adjustment factor that exceeds 1.0. However, in the case of drugs like Kymriah, which have an impact on the avoided loss of productivity that exceeds the cost of medical care, the financial impact is so great that the balance between benefits and burden must also be taken into account, and the extent to which this can be added to the price adjustment framework when the evaluation process is expanded is a new This is a new issue to be considered when expanding the evaluation process.

In addition, the survey covered a limited number of products (four), and the published information in the report was limited. I would like to see the system reviewed and more cost-effectiveness evaluation cases accumulated.

Supplementary information

Table 11 Criteria for selecting items for cost-effectiveness analysis in Japan
Source: Compiled by The Office of Pharmaceutical Industry Researchbased on materials from the Chuikyo

( The Office of Pharmaceutical Industry Research Senior Researcher Yuki Miura )

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