Topics Forum organized by the Policy Research Institute "The Value of Pharmaceuticals and DTx for the Japanese Public

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Akiko Yoshida, Senior Research Fellow, Pharmaceutical and Industrial Policy Research Institute
Junya Tsujii, Senior Research Fellow, Pharmaceutical and Industrial Policy Research Institute
Yuki Miura, Senior Research Fellow, Pharmaceutical and Industrial Policy Research Institute
Hiroshi Azuma, Senior Research Fellow, Pharmaceutical and Industrial Policy Research Institute

1. Introduction

The value brought by innovations in pharmaceuticals and digital therapeutics (hereinafter referred to as "DTx") has been increasingly discussed, and from the standpoint of the pharmaceutical industry, it is important that this value is properly evaluated and returned to the medical field and contributed to the creation of a prosperous society through the creation of the next innovation. The current situation is that the value of pharmaceutical products is not properly evaluated by the medical community. Currently, the evaluation of the value of pharmaceuticals is based on a medical perspective, and DTx is at the stage where the reimbursement system is beginning to be considered, and has not yet fully reached the point of discussing diverse values. Therefore, the Institute for Pharmaceutical Industry Policy Research (hereinafter referred to as "IIPIPR") has organized this forum to bring together the topics of pharmaceuticals and DTx and to discuss the various elements of value of pharmaceuticals and DTx, including their differences and similarities, as well as to provide a forum for discussion on measures to make the public aware of the values that have been identified. The Forum was held on August 21, 2023, in a hybrid format of on-site and webcast.

This report provides a summary of the presentations made at each session of the forum and the discussions that took place during the plenary sessions.

Outline of the Workshop

After a brief overview by Senior Research Fellow Hiroshi Azuma, the first half of the forum consisted of lectures by outside speakers and researchers, followed by a panel discussion in the second half in which speakers took the stage.

First, on the theme of value from the public/patient perspective, Senior Researcher Akiko Yoshida of the Policy Research Institute gave a report on "Consideration of the Value of Pharmaceuticals that the Public Values. Next, Ms. Naomi Sakurai, President and Representative Director of Cancer Solutions K.K., gave a presentation on "Pharmaceuticals and DTx that are close to the Patient Journey.

Focusing on the specific value evaluation of pharmaceuticals, Dr. Yuki Miura, Senior Researcher of the Policy Research Institute, gave a report on "Various Values of Pharmaceuticals from the Viewpoint of Value Evaluation in the UK". Dr. Naka Igarashi, Associate Professor of Public Health, Yokohama City University School of Medicine, and Visiting Associate Professor of Pharmaceutical Policy, Graduate School of Pharmaceutical Sciences, The University of Tokyo, gave a lecture entitled "What are the various values of pharmaceuticals and DTx from the perspective of academia? The lecture was followed by a presentation on the value of DTx.

Then, Mr. Junya Tsujii, Senior Researcher of the Policy Research Institute, gave a report on "Various Values of DTx" on the subject of values in DTx. Mr. Seigo Hara, CEO of MICIN Corporation, gave a presentation on "DTx opens the way to the future of healthcare.

Finally, following the first half of the session, Kenji Yamada, President of the Firm, moderated a general discussion among the six speakers on the various values of pharmaceuticals and DTx and their evaluation.

Session 1: Diverse Values of Pharmaceuticals and DTx

Lecture Parts

Here is a summary of each speaker's presentation.

Lecture 1: Consideration of the value of medicines that the public values - from the results of a web-based survey -
Akiko Yoshida, Senior Research Fellow

Based on the results of a web-based survey of 2,118 adult men and women (survey on attitudes toward the price, system, and value of drugs) conducted by the Institute for Policy Studies in June 2022, we discussed the value of drugs (Figure 1) that the public attaches importance to.

Image of the value of pharmaceuticals and the value of pharmaceuticals that is important to you

The survey question asked "what items you expect from medicines" and elicited responses (multiple answers) from 12 options. While many respondents focused on "medical value," which is familiar to them, a certain percentage of respondents selected "social ripple effect value," indicating that there are people who place importance on "social ripple effect value. The survey was conducted among the general public, and 45% of the respondents, or slightly less than half, had no medical conditions to receive medical care. Therefore, we presented explanatory statements and asked respondents to recall having had three diseases (hypertension, rheumatoid arthritis, and cancer) that had different effects on their quality of life and mental health, and saw how they changed. For example, "innovation" was more common when respondents recalled "cancer," and "convenience," "return to society," "less burden on caregivers," and "less burden on medical personnel" were more common when respondents recalled "cancer" and "rheumatoid arthritis," respectively. The results indicate that there were differences in responses depending on whether or not the respondents recalled the disease and the disease they recalled. (Figure 2)

Analysis of the characteristics of the group that values "social ripple effect value

We analyzed the characteristics of the groups that placed importance on "social ripple value. The results of the multiple regression analysis showed that the group 2, i.e., the group that selected "productivity," "return to work/return to society," and "reduction in burden of caregiving" as important among the "social ripple value" (the group that selected all the answers of productivity, return to work/return to society, and reduction in burden of caregiving), was more common among those in their 30s to 50s, especially among those in their 40s. The results of the multiple regression analysis revealed that the respondents were more likely to be female, employed, have a higher level of final education, have a greater sense of the burden of medical expenses, and have a lower level of perceived health.

Limited number of cases showing the decrease in "productivity" for patients and their families and the usefulness of medicines for them

Taking productivity as an example of social spillover value, we investigated how "productivity" was reduced for patients and their families as a result of the disease, and the cases that showed the usefulness of the drugs for this purpose. The results showed that the number of cases was limited. (Figure 3) In particular, there may not be enough examples of clinical trials or post-marketing studies showing that the drug has "social spillover value".

Consideration of the value of medicines that the public values

Lecture 2: Pharmaceuticals and DTx that accompany the Patient Journey
Naomi Sakurai, President, Cancer Solutions, Inc.

From the standpoint of a patient, I would like to describe my impressions and future expectations for digital

1. Digital environment in Japan

On March 28 of this year, the Cabinet approved the 4th Basic Plan for the Promotion of Cancer Control. Based on this plan, various cancer control measures will be promoted for the next six years, and "promotion of digitalization" is specified in No. 4 (infrastructure to support these measures). In this section, the measures to be undertaken include the promotion of PHRs, in other words, to enable the development of more various services with the patient at the center. I expect that the use of digitalization will increase convenience from the patient's perspective. As someone who developed cancer in the AYA (Adolescent & Young Adult) generation, I am interested in how the treatment I received in the past can be used in my subsequent health management and how I can control my health. They receive only paper copies of test results and are not in a position to manage their own health, and they feel that this situation should be improved. In addition to medical care, it is also desirable to have a flow of information and a system that allows people to obtain information without having to apply for it (without telling anyone) in various situations such as welfare, health services, nursing care, and cancer treatment for the elderly.

What about Europe and the United States? First of all, in Europe, as the European Union (EU), there is a movement among the 20 or so member countries to work together to promote things, which is a good point. There is a movement to create a European health data platform that will allow patients to receive treatment in other countries with a single passport, and I think Japan is being left behind in many areas.

For example, the French case (common electronic health record infrastructure) is shown. As a patient, I have high expectations that PHR (Personal Health Record) and EHR (Electronic Health Recor) will be merged and used. I sincerely hope that such a platform can be established in Japan. The Cabinet Office's Council for Regulatory Reform has included the phrase "Patient Journey," which refers to the process of a healthy person becoming aware of an illness, seeking medical treatment, and recovering from the illness. We would like to see the stockpiling of the collected data be realized. I also think it is important to promote awareness so that the appropriate use and benefits can be conveyed to patients, regardless of language, educational background, etc.

At the 2023 ASCO Annual Meeting, there were many sessions related to digital technology, and there were reports of improvements and reductions in side effects through its use. It would be good to see such reports from Japan as well. For example, it is reported that applications and approvals for comparative reference values in clinical trials and generic drugs are actually being conducted using real-world data such as PROs.

2.Patient Journey and DTx needs

Patient Journey is considered to vary from disease to disease. Patients would like to be able to use various applications depending on the disease and situation at the time, but ultimately, they would like to be able to retrieve data in a unified manner.

The concept of survivorship (the idea of living better after an illness) includes services from pre-symptomatic (before the onset of illness), during treatment (acute phase), follow-up (extended phase), chronic phase, and in old age, and the part shown in blue is the service in medical care. (Figure 4) From the perspective of promoting qualified medical care, including reimbursement, digital technology is probably at its best in areas such as pathological diagnosis and surgical procedures using images. What impact acute care will have on the chronic phase may be unknown to us, but we also believe that it will lead to future patients, and the accumulation of such data is an area where the body will especially benefit from digital possibilities and direct benefits for the future patient.

I would like to see not only the problems of the most vocal patients and those with access to data be solved, but also those that are not visualized, those that are under the iceberg, to be made visible digitally. It is important and our hope is that by utilizing digital technology, we can switch our way of thinking that there are many disparities in cancer (disparities in life expectancy based on income, disparities in education due to reduced educational opportunities, etc.), and that the use of digital technology will be beneficial to narrow these disparities as well. (Figure 5)

3. giving back advanced science and technology to society - so that no one is left behind

We also hope that the use of digital technology will lead to improved services. For example, consultation support is something that is outside the realm of expertise and difficult for medical professionals to handle. We are trying our best to provide communication skills training, etc., but we have also found that using the chat GPT function was surprisingly good. I believe that this is a point that needs to be considered, including the reform of work styles. When we analyzed how patients acted in response to appearance changes, which is one of the aspects of consultation support, by segment using behavioral economics, we found that 20 to 30% of the patients said that the issues were multi-layered and difficult to solve only digitally, and that human resources were needed, while 70% said that the issues could not be solved by digital technology, such as apps. The hypothesis that digital technology can solve these problems is also derived. Research to implement these hypotheses is also needed. (Figure 6).

As a patient, I welcome the introduction of digital technology into clinical practice, but I am concerned about the cost. If the services are to be provided within the reimbursement system, we will have to pay for them ourselves (within the 30% co-payment). I think it is important to show more and more to the society how much the service provision will meet the patients' expectations and the fact that this kind of thing can be realized (fact). In addition, in Japan, there seems to be a lack of awareness of contribution to the community to which one belongs. It is necessary to raise awareness that providing information on one's disease can improve the community to which one belongs, and even Japan as a whole.

Lecture 3: The Diverse Value of Pharmaceuticals and DTx from a UK Valuation Perspective
Yuki Miura, Senior Research Fellow

Pharmaceuticals have diverse values, but the range of value elements evaluated differs according to the framework of the evaluation system. Of these, the cost-effectiveness evaluation system in place in Japan mainly focuses on those that can be measured in terms of Cost/QALYs. This is because the cost-effectiveness system itself is intended to supplement the NHI drug price system by targeting some items that have a large fiscal impact and adjusting the price of the additional portion. On the other hand, the Health Technology Assessment (HTA) of the National Institute for Health and Care Excellence (NICE) in the U.K. is used to make decisions on whether or not to reimburse pharmaceutical products. It comprehensively evaluates various factors other than cost-effectiveness. In the case of HST, the factors that cannot be captured by Cost/QALY are The factors considered in determining reimbursement are presented in Table 1.

The survey subjects were all "recommended" and introduced that "magnitude of innovation," "burden of care," and other factors were evaluated as values. He then introduced the factors that were individually considered with respect to "magnitude of innovation" and "burden of caregiving" (Table 2).

Drugs for diseases with high unmet needs and unforeseen costs (e.g., psychological impact) are aspects that are difficult to be reflected in ICERs, and were taken into account in decision-making.

In discussing the various values of pharmaceuticals, as in the NICE case, utility values that cannot be captured by Cost/QALY and values that cannot be fully evaluated by the system may be important elements in discussing the value of pharmaceuticals. However, it is difficult to evaluate all values without omission from the viewpoint of measurability (whether the index is suitable for measurement, whether all products can be measured uniformly, etc.), and it is important to be able to explain "elements recognized as value" to people in various positions and to gain their understanding in order to promote future value evaluation discussions. We believe it is important to be able to "explain" the "elements of perceived value" to people in various positions and to obtain their "sense of conviction" in order to promote future discussions on value evaluation.

Lecture 4: What is the diverse value of pharmaceuticals and DTx from the viewpoint of academia?
Dr. Naka Igarashi, Associate Professor, Department of Public Health, Yokohama City University School of Medicine, Visiting Associate Professor of Pharmaceutical Policy, Graduate School of Pharmaceutical Sciences, The University of Tokyo

Referring to the content of Chief Researcher Miura's speech, he raised the issue of whether what cannot be measured should not be added in the discussion that the cases in the UK and Japan are different. Using the example of the usefulness addition ratio in the already existing NHI drug price system, he introduced the current system in Japan, which incorporates a pseudo-quantification of what must be evaluated qualitatively. While it is easy to focus on measurement when there are various evaluation criteria and measurement methods, caution must be exercised because it is not analogous to saying that something is not value if it cannot be measured. He introduced specific elements that do not lend themselves to quantification (Figure 7) and stated that whether or not something can be quantified and whether or not it is valuable are two separate issues. It is by no means the case that everything must be quantified.

In addition, he introduced the UK system (Table 3). The UK case is not the same as if these indicators were set according to surveys or GDP, but the system of quantification was created from the idea that factors such as those shown in Figure 7 must also be valued qualitatively. This process of thinking is very important, and if it is misjudged, what could not be measured may go in the direction of something that is not needed.

The questionnaire conducted on the basis of three case studies, referring to the "severity of disease" of the ISPOR (International Society for Pharmacoeconomicsand Outcomes Research) value flower (Figure 8), and which interventions should be prioritized, is presented below. The details are beyond the scope of this report. We will not go into details, but even if Cost/QALY is calculated, the QALY itself may oscillate depending on which intervention is selected. It is easy to assume that the analysis has led to a final conclusion, but the discussion of which diseases are prioritized, for example, is an argument that must be advanced during the value assessment.

In the past, because medical care was so important, the argument could be made that if resources were not available, they would be increased, but now the public is aware and has also realized that increasing medical resources will cause other wrinkles. Now is a very valuable opportunity to discuss value-based pricing.

He continued by presenting examples of actual value evaluations of pharmaceuticals and medical devices. In some cases, companies were fortunate to be able to evaluate products with high expectations, such as those for which they had more than enough time to plan clinical trials or for which they had already built models and had access to detailed clinical research data from similar studies. However, the question was raised as to whether it is possible to quantify all products in the same way.

It is important to consider how to include the qualitative part in the evaluation, from the perspective of whether it can be done with limited resources of time and money, and how to convert the qualitative part into quantity. (Table 4)

Lecture 5: Diverse Values of Digital Therapeutics (DTx)
Junya Tsujii, Senior Research Fellow

The various values that DTx brings were presented in detail with specific examples.

From the viewpoint of its recipients, the value of DTx is broadly classified into two categories: "medical value," which is "value directly felt by patients," and "social value," which is "value that spreads to the patients' surroundings" (Figure 9). Medical value can be further classified into "therapeutic value," which is based on efficacy and safety, and "supportive value," which supports treatment through optimization of the treatment process.

Therapeutic value is value based on the therapeutic effect exerted by the DTx itself, regardless of whether or not it is used in combination with standard treatments (including pharmaceuticals) (Figure 10). For example, Sleepio, used to treat insomnia, provides an Internet-based treatment for patients who have difficulty accessing face-to-face cognitive behavioral therapy, while ^{EndeavorRxⓇ}, used to treat pediatric ADHD, provides a game-based treatment that activates the prefrontal cortex of the brain. Thus, it can be said that DTx is one of the new treatment modalities that will bring therapeutic benefits to patients.

On the other hand, "supportive value" is the value that contributes to the improvement of therapeutic effect or the expansion of therapeutic opportunity and self-management ability by utilizing the characteristics of DTx, such as continuous data acquisition from daily life and therapeutic intervention utilizing digital technology, and can be considered as "DTx-specific value.

The former value that contributes to the improvement of treatment efficacy is the value that strengthens or adds to the therapeutic effect of the standard treatment by changing patients' awareness and behavior, managing symptom changes, etc. For example, edupression. ^comⓇ used to treat depression ^{andvorvidaⓇ} used to treat alcoholism can improve patients' health literacy and self-efficacy, resulting in active participation in treatment and reliable response. ^ProHerzⓇ, which supports symptom management and other aspects of heart failure, contributes to the provision of personalized and appropriate treatment and prevention of serious illness through early intervention by providing treatment consistent with guidelines and recognized treatment standards and intervening during gaps in treatment through routine monitoring. Thus, it can be said that DTx has value in improving the therapeutic effect that standard treatment originally has.

The latter value of expanding treatment opportunities and self-management capacity provides patients with appropriate access to treatment, reduced treatment-related burden, and better treatment outcomes. Daylight, which is used to treat Sleepio, generalized anxiety disorder, and other disorders mentioned above, provides a new treatment option for patients who have had limited access to treatment, such as pregnant women and those who refuse to talk to their doctors. In addition, edupression. ^{comⓇ andvorvidaⓇ} can be said to contribute to the maintenance of prognosis health, as one of the purposes of their use is to prevent relapse through improving patients' health literacy and self-efficacy.

In light of the above, it can be said that DTx provides value to help patients start treatment earlier, receive more effective treatment, and stay healthier longer.

However, in order to properly capture the various values that DTx brings, it is essential to consider not only the value directly perceived by the patient, but also the social value, which is the value that spills over to the patient's surroundings, from multiple perspectives. For example, from the family's perspective, the improvement of the patient's productivity (work, schoolwork, daily life, etc.) through the management of symptoms of irritable bowel syndrome and the reduction of the burden of nursing care through continuous blood glucose monitoring for pediatric diabetes can lead to an improvement in the productivity of the entire society (Figure 11). From the perspective of healthcare providers, it is also possible to improve the efficiency of healthcare resources by reducing the response time of therapists for cognitive behavioral therapy and securing time for other tasks through the management of medication dosage in diabetes. From the insurer's perspective, medical costs can be optimized by reducing the number of medical examinations and medications through DTx intervention and by reducing the risk of hospitalization through the sharing of health status with medical professionals. In pharmaceuticals, "social ripple value" was also mentioned, and we believe that DTx similarly has value that is brought to society along with the process of patients' health recovery.

In order to further discuss the various values of DTx, what is first required from the industry side is to organize and present the various values of DTx in concrete terms to patients and other value recipients, and to foster a common understanding of the importance of the various values.

Lecture 6: The Future of Medicine Pioneered by DTx
Mr. Seigo Hara, CEO, MICIN, Inc.

DTx (e.g., therapeutic applications) are products that have the characteristics of software but also have therapeutic effects like pharmaceuticals, and have value not only as stand-alone products but also as "digital ecosystems" (Figure 12). The "digital ecosystem" is a value that consists of four elements: "accumulation of evidence," "utilization and creation of digital biomarkers," "patient/medical practitioner contact," and "optimization of interventions.

Because DTx utilizes digital technology, evidence is accumulated in real time, and the use and creation of digital biomarkers that leverage this evidence will lead to more optimal treatment for each patient. In addition, DTx is expected to be used as a point of contact between patients and healthcare providers and to contribute to the optimization of interventions. While pharmaceuticals and hardware medical devices may go through a similar loop over a long period of time, DTx is unique in that it creates these values in a very fast cycle. Furthermore, DTx has the potential to synergistically increase the value of pharmaceuticals. When considering the value of DTx on a dynamic time axis, the value of the "digital ecosystem" may be a unique value.

To embody the value of the "digital ecosystem," MICIN Corporation is developing DTx products such as the MedBridge series of perioperative care applications and the BSA-01 biometric sound measurement application that uses a smartphone to acquire the patient's breath sounds.

However, there are several issues that need to be addressed in order to promote the widespread use of DTx. Three of them are listed here: "development of the system," "development of technology and environment," and "acceptance by physicians and patients.

With regard to the "development of systems," following the first time programmed medical devices (SaMD) were discussed as an agenda item at the Council for Promoting Regulatory Reform in October 2020, discussions on systems such as the SaMD working group established under the Central Social Insurance Medical Council's specialized organization for insured medical materials and the publication of "Guidance for appropriate and prompt approval and development of programmed medical devices based on their characteristics," have accelerated. The discussion on the pharmaceutical approval system and other systems has accelerated with the establishment of the SaMD working group under the Central Social Insurance Medical Council and the publication of "Guidance for Appropriate and Prompt Approval and Development of Programmed Medical Devices Based on Their Characteristics. However, there are still some uncertainties regarding the axis of evaluation (value) in terms of insurance, and ongoing discussions are required.

On the other hand, regarding "physician/patient acceptance," a survey of physicians conducted by MICIN, Inc. revealed that only 9.4% of physicians had experience using the system in actual practice, indicating issues in terms of DTx diffusion (Figure 13). However, about half of the physicians were interested in using the therapeutic application, and relatively positive responses were observed, especially among working physicians. The most common reason for lack of interest was not seeing the target patients. Only two DTx products were available at the time of the survey. On the flip side, it can be said that not only the development of the system and acceptance by physicians and patients, but also the "development of technology and environment" by providers remains important.

Session 2: Plenary Discussion on the various values of pharmaceuticals and DTx and their evaluation

Here is a summary of the discussion in the plenary session.

Topic 1: Organizing Diverse Values

Q. What perspectives and perceptions of the value of pharmaceuticals/DTx are important to expand?

Dr. Igarashi first presented the proximity of the concepts of well-being and QOL, and the integration and substitution risks in measuring them. In the UK evaluation, there are two categories of benefits that do not fit into the QOL concept, and factors that fall within the QOL concept but cannot be fully measured by the EQ-5D, etc. He mentioned that this could lead to discussions such as "what cannot be measured is not a value. He pointed out that when a scale to measure something is standardized, there is a danger that what cannot be measured by the scale may be deemed not to be included.

When the discussion of well-being, which overlaps with the concept of quality of life, is raised, some are considering the point that it could be measured by QOL values, or that a questionnaire could be used to ensure these concepts. It was suggested that the idea that all of a certain element can be measured if a scale can be created is an illusion, and that this is a point that must be noted now that the discussion is gaining momentum. In addition, he said that we must be careful about what elements cannot be fully measured by well-being, and whether all of them can be quantitatively evaluated, because it is a new scale.

Dr. Sakurai pointed out, as an example, the issue of access to treatment in the field of psychiatric disorders, where patients are sometimes unable to receive satisfactory treatment due to a lack of specialists or incompatibility of doctors. and new pleasures such as the ability to participate in treatment without coming out to society are expected. He stated that it is important for patients and citizens, who are the parties concerned, to participate in DTx development and visualize the advantages of DTx.

Mr. Hara also expressed his views from two perspectives. One is "how to visualize the value other than treatment. According to a digitally-minded physician, he commented that acceptance of DTx tends to be stronger in patients before they are put on medication or before adding a drug. For patients at this timing, one can imagine that they perceive some value in DTx, and it was stated that it is necessary to visualize this kind of value that is currently invisible. Another point is to "convey the value of the digital ecosystem to patients on a real-world basis. For example, the expansion of the use of DTx products is expected to lead to the evolution of DTx products and the understanding of patient groups with high therapeutic efficacy, etc. He indicated that it is important to accumulate such examples and have patients understand the value that the digital ecosystem can quickly realize.

Q. What is the current status of the perception of the value of pharmaceuticals/DTx in Japan?

Mr. Igarashi introduced the presentation by dividing it into front and back sides. As the front part, he indicated that he would not talk about a new system that reflects more value, but rather verify it based on actual examples. The result of quantifying or estimating only the part of the evidence manifested at that point in time for drugs launched in the past, resulted in a nine-discount for anticancer drugs. On the other hand, the hepatitis C case is a case in which a combination of various factors worked well, and it cannot be said that other drugs can be verified in the same way. He stated that if evaluations are to include cases that are difficult to verify, it is necessary to include qualitative aspects to some extent.

As a back side part, he indicated that he wondered whether pharmaceutical companies are willing to align themselves and advocate value-based pricing. While it is not realistic to expect everything to be valued in a positive way, and the discussion of value is finally becoming possible, the key to seriously advancing the discussion of value is whether we can accept that there are positives and negatives, and that in some cases the knowledge of affordability we have built up may come to naught. This is the key to seriously discussing the value of a product.

Next, Dr. Sakurai touched on the value of prevention. He expressed his opinion that current discussions, including those on pharmaceuticals, start from "after the onset of disease" and that prevention is not even on the chopping block of discussion. From a digital perspective, he pointed out that there may be value in promoting the health of those who are not yet ill and preventing serious illness, but that discussion and visualization of this value has not yet been achieved. In other countries, accessibility to medical care is worse than in Japan, and apps are being used to manage one's own health and symptoms. He commented that he expects digital to intervene before the onset of disease, and that a wide range of support will be needed from the pharmaceutical industry as well as the insurance industry and others, including the uninsured.

Dr. Hara suggested that there is room for improvement in resource allocation based on outcomes. First of all, he felt that in discussions on how to assign points (for reimbursement), there is an aspect where the authorities and companies work in unison to build a logic toward the target price, but this aspect is difficult to see. He raised the issue that discussions on resource allocation are often structured in such a way that stakeholders' interests are coordinated, and that outcome-based discussions may not be sufficiently conducted. He expressed his hope that the world will become a place where resources are allocated based on value.

Topic 2: Evaluation of Value

Q. What are the differences between Japan and other countries in reflecting patient/public opinions in value assessment?

Dr. Sakurai shared an overseas example of reflecting the value to patients when implementing generics and biosimilars. As expectations for the future, including overseas, he mentioned the realization of clinical trials without placebo by utilizing data, visualization of continuous data, realization of QOL evaluation including quantity (survival period), and the possibility of seeing value that had been apparent through evaluation between treatments.

Q. What is your response to the "Report of the Expert Panel on Comprehensive Measures to Achieve a Rapid and Stable Supply of Pharmaceuticals" issued by the MHLW in June 2023?

Mr. Sakurai mentioned that he was able to participate in the discussion as a patient group and deliver his voice to the Expert Panel to solve the drug lag and loss problem, and expressed his surprise and gratitude that his opinion was included in the report as a statement.

Q. Many DTx developments are often carried out by financially constrained start-ups. What challenges and necessary support do you see for acquiring evidence to prove diverse values?

Mr. Hara pointed out that the lack of predictability in how much DTx will be evaluated (in terms of insurance reimbursement) is a difficult point in the development process. Since this is a new area, development is proceeding with a certain degree of risk tolerance, but we feel it is important to make the possibility of evaluation a little more visible. From this perspective, he believes that various value cutoffs can be the axis of discussions on foreseeability, and he argued that clarifying the value to be evaluated will also make it easier for developers to formulate strategies on how to prove that value. He added, however, that the purpose is not to simply give a high score, but to clarify the evaluation axis, and that it is important to organize the conflicts with the conventional way of doing things.

Dr. Igarashi also pointed out that one of the unique strengths of DTx, which is in the hands of patients, is the ability to collect real-world post-marketing evidence. In particular, in cases where the items (values) to be evaluated or the axis of evaluation is not clear, a major strength of DTx is its ability to build evidence from already collected data. In addition, regarding feedback of results to patients, it was also commented that DTx is a tool that can return results to patients in an individualized and specific manner, which cannot be overlooked. It is impossible to prove everything at the time of approval, and value-based pricing ultimately means reevaluating the results, positive or negative, based on post-approval evidence. He stated that DTx is the most inexpensive way to accomplish this.

In addition, Dr. Sakurai commented that from the patient perspective, for example, it may be possible to predict (visualize) changes in appearance caused by anticancer drugs graphically using digital data. He also mentioned the possibility of feeding this data back to those who are educated in medical schools. On the other hand, he raised the issue that it is necessary not only for each developer to work on the data individually, but also for a national policy to consolidate patient data into a single database, which would make it easier to use the data at various points in the patient journey.

Topic 3: How to make the public aware of diverse values?

Q. What is the approach from the industry side to raise more awareness of value among the public?

Senior Researcher Yoshida stated that pharmaceutical companies need to take the initiative, materialize, and firmly demonstrate to the public that pharmaceuticals have various values, especially social spillover value. The results of the study suggest that the groups that place the highest value on social ripple effects (in particular, productivity, return to work/return to society, and reduced burden of caregiving) are those who are busy with work and household chores and those who have some kind of disease. It is one idea to show and deliver to the working (household) generation (citizens) in particular that pharmaceuticals have social ripple effects, such as enabling people to work (household chores) while being treated for a disease, returning to work (household chores), and reducing the burden of caregiving.

In addition, Senior Researcher Miura stated that it is important to visualize the values that have yet to be seen, which came out in the section on organizing various values. He suggested that an approach that provides opportunities to hear from patients undergoing treatment and patient groups about their experiences of being able to do things they could not do before, and then feeds this information back to physicians who work with patients, would be an approach to make patients aware of the various values.

Lastly, Dr. Tsujii mentioned two approaches in DTx: "indirect awareness-raising through physicians, etc." and "direct awareness-raising. In the former, it is important to present the value of DTx to physicians and others with evidence. In the latter, on the other hand, he stated that from the patients' perspective, there is the problem that it is difficult for them to understand what is being said in technical terms. For example, this spring, the Pharmaceutical Manufacturers Association of Japan (PMAJ) prepared and released an educational booklet for the public on the use of health and medical ^data1). The presentation was based on the following points

Q. What measures can be taken to make physicians themselves aware of the value or to make patients aware of the value?

Dr. Hara commented that it is important for physicians to feel the therapeutic effects and usefulness of DTx, to find out in what kind of use cases DTx is effective, and to develop DTx, and that the community among physicians and various other forms of expansion will be a trigger for this.

Q. What are the changes in public awareness compared to 10 years ago, and what actions are being taken by the pharmaceutical industry and academia to increase awareness of the value of pharmaceuticals and Drugs?

Mr. Igarashi mentioned that it is not so much that the world's view of value has changed, but rather that the stakeholders to whom explanations are given have changed between then and now. He stated that at that time, explanations were given to the members of the Chuikyo, but now there is a change in that explanations must be given to the public and must be acceptable to them. He stated that the time when pharmaceuticals were prescribed without question, as in those days, has changed, and now they have to devise various means to get people to share (resources) and prioritize them, and they have to explain in plainer language because there are more people to persuade than before.

Q. How have patients' perceptions of pharmaceuticals, DTx, and medical care changed?

Dr. Sakurai commented that compared to the past, when prescribing and taking medicines were somewhat passive, now, for example, patients who used to be very sick can return to society and feel a wider range of benefits and values from medicines. He concluded by emphasizing the importance of continued discussions among all stakeholders, including patient groups and the pharmaceutical industry, on the value and evaluation of drugs and DTx in general.

Conclusion

The forum was well attended by the audience, with over 500 participants in total, both at the venue and via webcast. A questionnaire conducted after the meeting was tabulated, and the results showed that the majority (86% and 89%) of the participants said that their understanding of the diverse values of both pharmaceuticals and DTx had been deepened or somewhat deepened. (Figure 14)

In addition, the following comments were received in the free-text section of the survey on value. (excerpts)

The following are suggestions that the authors obtained through the discussions in this forum. Discussions on the value and evaluation of pharmaceuticals are still insufficient, as was the case in this forum, and we felt the need for the pharmaceutical industry to make efforts to make them more visible and plain, to deepen literacy from the perspective of patients and the public, or to demonstrate their value with examples, including medical professionals such as physicians and pharmacists.

Similarly, for DTx, it is important to accumulate concrete evidence and demonstrate its value to those who are aware of it but have yet to visualize it, and furthermore, the industry side should actively present evaluation axes such as predictability in order to advance the discussion.

These issues have been discussed in the past, but through this forum, we could sense that the number of stakeholders interested in and participating in the discussions has been increasing. With the Central Social Insurance Medical Council (Chuikyo) and various study groups led by the Ministry of Health, Labor and Welfare (MHLW) now in place, we feel that it is increasingly necessary to explain the situation with appealing data, making full use of evaluation methods, etc. We intend to make use of the suggestions we gained from this forum in our future research and studies.

Finally, we would like to express our gratitude to all the speakers and other participants for their efforts and cooperation in organizing this forum.

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