Points of View How medical technology assessment should be utilized in the field of health and medicine Based on the institutionalization of cost-effectiveness evaluation

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Yosuke Nakano, Senior Researcher, National Institute of Biomedical Innovation Policy

1. Introduction

The system of cost-effectiveness evaluation started in earnest in Japan in April 2019. This system covers pharmaceuticals, medical devices, and regenerative medical products. Until now, the value of pharmaceuticals and other products has been evaluated mainly from the perspective of clinical efficacy and safety, as well as medical usefulness, but a new perspective of cost-effectiveness has been incorporated into this evaluation system.

Recently, while there has been discussion of a sustainable social security system, the launch of innovative and expensive drugs such as immune checkpoint inhibitors and CAR-T cell therapy has attracted public attention to the price and cost-effectiveness of drugs in particular. In this article, we will review the current status of cost-effectiveness assessment in Japan and consider the future utilization of health technology assessment (HTA), including cost-effectiveness, in the field of pharmaceuticals and health and medical care.

Overview of the cost-effectiveness evaluation system

The introduction of a cost-effectiveness assessment system in Japan began to be discussed by the Central Social Insurance Medical Council (hereinafter referred to as "Chuikyo") around 2012 as an issue for the NHI drug price system, and after a trial introduction in April 2016, the system was officially institutionalized in April of this year. ¹⁾ While referring to systems in other countries, the system should be consistent with Japan's unique NHI drug price system. 1) The design of the system was studied with reference to the systems of other countries, while taking into consideration the compatibility with Japan's unique drug price system and the fact that it would not impede patient access to medicines and other products. Compared to other countries, Japan's system is characterized by the use of evaluation results for price adjustment of the NHI Drug Price Standards, which are official prices (Tables 1 and ²⁾²⁾. For details of this system, please refer to other papers. This section summarizes the current status of cost-effectiveness evaluation in Japan, focusing on key points based on discussions at domestic academic conferences held this summer and other events. The views of the pharmaceutical industry (Pharmaceutical Manufacturers Association of Japan) on this system are expressed in "Institutionalization of Cost-Effectiveness Evaluation " ³⁾.

2-1. selection criteria for target items

The criteria for selecting items are shown in Figure ¹⁴⁾. Simply put, drugs that are highly innovative and have a large fiscal impact are the main targets of this system, and are classified into five categories, H1 to H5, according to the NHI drug price calculation method and selection criteria. At present, the maximum number of items that can be evaluated annually is said to be around 10, but as the human resources of public organizations expand and experience is gained, the number of eligible items is expected to grow. Four products have been selected as of the end of August 2019 (Table 2) ^.5) For Kymriah, with a drug price of approximately 33 million yen per patient, the expected market size is approximately 7 billion yen per year at its peak, but it was selected as H3 because its unit price was judged to be significantly higher by the Chuikyo.

2-2. evaluation method

In cost-effectiveness evaluation, QALY (Quality-adjusted Life Year) is used as the effectiveness index, and the incremental cost-effectiveness ratio (ICER), which is the incremental cost per QALY gained, is used to evaluate the cost-effectiveness (Figure 2). (Figure 2). The use of this indicator is similar to that of other countries that have preceded it, but the handling of the standard value is different. Under this system, a standard value of 5 million yen/QALY (upper limit, 10 million yen/QALY) ^{6) is} set, and if the calculated ICER value exceeds this standard value, the price of the additional portion is adjusted down in steps according to the ^value7) (Figure 3). 7) (Figure 3) Although various calculation methods have been considered for the appropriateness of this standard value, such as referring to willingness to pay surveys and GDP per capita, there is no sufficient basis for explaining it, and even among experts, there are differences in opinion. Furthermore, considering the possibility that the number of innovative therapeutic drugs such as gene and cell therapy drugs, which are expected to have high total product costs, will continue to increase in the future, it is possible that discussions regarding the appropriateness of this standard value and its handling will continue.

2-3. evaluation system and process

Once a product is selected as a target product, analysis and evaluation will begin promptly, and a comprehensive judgment will be made during the overall evaluation, taking into account perspectives other than ^ICER9). It then takes about one to one and a half years for the final evaluation and pricing decision to be made (Figure 4). In addition, as a public professional organization, an organization called the Center for Health Economics and Evaluation Research (commonly known as C2H) was established in April 2018 at the National Institute of Health Sciences; as of September 2019, this center is composed of six members. Comparing the way institutional evaluations are reflected in other countries, it is difficult to say that the comprehensive evaluation in this system has a system that adequately reflects factors other than ICER. ⁽ In the U.K., a system that involves patients and reflects their voices in the evaluation process has been introduced. ¹⁰⁾ Furthermore, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), an international academic society, and other organizations have also introduced a system to incorporate the patient perspective into the process of medical technology evaluation. ¹⁰⁾ In addition, the International Society for Pharmacoeconomics and Outcomes Research ⁽ ISPOR), an international academic society, has been discussing how the patient perspective should be incorporated into the evaluation process of medical ^{technology11)}. In addition, it may be necessary to create a forum for the pharmaceutical industry, academia, and academic societies to cooperate in discussing issues and modifications to the system, and to propose measures for improvement.

3. future utilization of medical technology assessment (HTA)

The above is an overview of the cost-effectiveness evaluation system for pharmaceuticals in Japan, which focuses on "price adjustment" of pharmaceuticals. However, cost-effectiveness analysis is not limited to pharmaceuticals alone. Cost-effectiveness is one of the most important perspectives when considering HTA, and it is also one of the decision-making methods for more efficient allocation of limited medical resources. ¹²⁾,13 Therefore, from here on, from the viewpoint of efficient allocation of medical resources, we will discuss the future utilization of HTA, including cost-effectiveness, in the field of pharmaceuticals, and by extension, in the field of medical health. The following section discusses the future utilization of HTA, including its cost-effectiveness, from the viewpoint of efficient allocation of medical resources in the field of medicine and health. From this perspective, the important elements required for HTA are "appropriate evaluation of value" and "judgment on the efficiency of medical care," and we have examined the issue from this perspective.

3-1. appropriate evaluation of value

Evaluation of multifaceted value

As Japan faces a hyper-aged society, medical health is an issue not only for individuals but also for society as a whole. In order to solve social issues, the cooperation of citizens and patients, as well as government and industry, is indispensable. Pharmaceuticals and medical technologies are no exception, and the multifaceted value they bring must be evaluated and incorporated into HTA. As an example, Figure 5 shows the elements of value of pharmaceuticals and medical technology that have been studied so far, and these elements are not limited to the perspective of medical economics. Therefore, there is a limit to evaluating the value of pharmaceuticals and medical technologies only in terms of the aforementioned ICER (QALYs and costs). As for their value, we believe that the value to the patients themselves and the social value beyond the medical value are also important, but it is not easy to evaluate medicines and medical technologies from the standpoint of "value to the patients themselves" and "social value. Because, what outcome indicators should be defined for those elements of value? And how to measure these outcomes? On the other hand, the acquisition and utilization of patient-reported outcomes has been gaining momentum recently, and with the advancement of digital technology, patient data (voice, movement, emotions, etc.), which was previously considered difficult to acquire or utilize, is now available through the use of digital devices and sensors. In addition, digital technology is making it possible to acquire patient data (voice, movement, emotions, etc.) that was previously thought to be difficult to acquire or utilize, by making full use of digital devices and sensors. In addition, the government is taking the lead in developing an environment in which individual medical, health, and nursing care data can be collected and consolidated over the long term. In light of the above, it is expected that new outcome indicators and measurements will become possible in the future, and that the evaluation of the multifaceted value of pharmaceuticals and other products will progress. In anticipation of such progress, measurement technological innovations and data manifestations, such as IoT, sensors, and digital, should also be closely monitored.

Response to innovation

Significant innovations are now beginning to occur in the medical health field. New trends include personalized medicine based on genome data, development of solutions utilizing various medical health-related data, and development and launch of new modalities such as digital therapeutics and gene therapy. How to evaluate such innovative therapies and medical technologies that are difficult to assess based on conventional concepts will be a major challenge in the future. Taking the example of the evaluation of anticancer drugs with cross-organ indications based on genetic mutations, it is possible that the value is not fully evaluated in the method of considering evaluation for each organ-specific indication. In addition, in the process of the spread of cancer genome medicine, it can be assumed that more attention will be paid to the evaluation of value as a solution for cancer genome medicine, including testing and treatment.

^In addition, in the process of the spread of cancer genome medicine, attention is expected to be paid to the evaluation of the value of cancer genome medicine as a solution, including tests and therapies. ⁽ 15 ⁾ ICER, a U.S. nongovernmental HTA organization, has also begun to examine methods for evaluating the value of gene and cell therapies (Single or Short-Term Transformative Therapies: SSTs) in light of the emergence of such therapies. ¹⁶⁾

In the evaluation of the value of these advanced medical treatments and drugs, some current evaluation indices are not necessarily appropriate, and some evaluations require a longer-term perspective than ever before. In such evaluations, it is necessary to develop new evaluation methods.

3-2. decision to improve efficiency of medical care

Judgment of insurance reimbursement, etc.

In Japan, where the urgent issue is to curb the increase in social security costs, it is conceivable that some may argue that cost-effectiveness should be used as a means to determine whether or not a drug or medical device can be reimbursed by insurance and what the reimbursement rate should be. In fact, as shown in Table 1, HTA results are also used to determine insurance reimbursement in the U.K., South Korea, and other countries, and some experts are advocating such use. Since September of this year, the government has been holding the All-Generational Social Security Study Council, and we expect that similar arguments could be made in this Council. From the perspective of improving the efficiency of medical care, it is important to take an approach that determines which medical intervention requires the least cost and effort to achieve the same effect, or which maximizes benefits. However, in Japan, which aims to maintain a universal health insurance system, there are concerns that decisions on reimbursement may impede patient access to drugs and medical devices, and therefore, careful discussion is desirable.

Diffusion to the entire medical and health care field

The use of cost-effectiveness in Japan is biased toward pharmaceuticals, etc., and its use is limited in areas related to medical technology, such as general diagnostic treatment and medical processes. As the importance of interventions for disease prevention and health maintenance ^increases17), it is expected that HTA-based decision making will expand to disease prevention other than vaccination. In general, it is said that it is difficult to demonstrate the effectiveness of disease prevention in reducing healthcare costs, but it is hoped that research on disease prevention measures that are cost-effective will progress through low-cost intervention methods that utilize digital technology, and the need to prove cost-effectiveness will increase. The need to prove the cost-effectiveness of such measures is also expected to increase.

Furthermore, the concept of cost-effectiveness is gradually expanding in the evaluation of medical technologies (diagnosis, treatment, rehabilitation, etc.) in some areas. For example, the "Guidelines for Rehabilitation in Cardiovascular Disease" incorporate the concept of ^{cost-effectiveness18)}, and it is said that some other medical guidelines are also moving toward its introduction. Considering the efficiency and cost burden of medical care and health care in Japan as a whole, it will be necessary in the future to expand the perspective of cost-effectiveness, or HTA, across the medical and health fields, including reimbursement.

4. Conclusion

In the above, we discussed how HTA, including cost-effectiveness, should be utilized in the future in the field of medical care and health, based on the current status of cost-effectiveness evaluation institutionalized in Japan. Currently, the role of cost-effectiveness in Japan for pharmaceuticals is limited to "price adjustment of drug prices. However, as the concept of medical care expands from treatment to prevention and health maintenance toward Precision Medicine/Healthcare, there is a need to examine cost-effectiveness not only within the scope of current treatment, but also across all aspects of health care, and from a social standpoint. In addition, the need to consider cost-effectiveness in the context of overall healthcare and from a social standpoint is emerging.

It is also expected that innovative technologies such as digital technology, IoT, and AI will be actively utilized to expand evaluation indicators and methods. Although there is still a lack of human resources in industry, government, and academia to conduct research that contributes to cost-effectiveness evaluation and ultimately HTA, research for decision-making on how to efficiently invest limited medical resources in the medical health field is extremely important.

It is hoped that HTA efforts, including cost-effectiveness, will spread and develop in the overall medical health field in Japan, so that the discussion does not become one of seeing the forest for the trees.

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