Policy Research Institute page Forum on "The Value of Pharmaceuticals and DTx to the Public" organized by the Institute for Policy Studies

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The value brought by innovations in pharmaceuticals and digital therapeutics (hereinafter referred to as "DTx") is becoming increasingly discussed, and from the standpoint of the pharmaceutical industry, it is important that this value is properly evaluated and that it is returned to the medical community and contributes to the creation of a prosperous society through the creation of the next innovation. We believe that the pharmaceutical industry should contribute to the creation of an affluent society through the creation of the next innovation. Currently, the evaluation of the value of pharmaceuticals is based on a medical perspective, and DTx is at the stage where its inclusion in the reimbursement system is just beginning to be considered, so we have not yet fully reached the point of discussing its various values. Therefore, the Pharmaceutical and Drug Industry Policy Institute (PIPI) 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 to discuss 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 venue (Nihonbashi Life Science Building, Chuo-ku, Tokyo) and webcast.

Overview of the Forum

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

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

Focusing on the specific value evaluation of pharmaceuticals, Yuki Miura, Senior Researcher of the Policy Research Institute, gave a report on "The 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, 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 of Japan, gave a report on "The Various Values of DTx" on the subject of values in DTx. Next, Mr. Seigo Hara, CEO of MICIN, gave a presentation on "The Future of Healthcare Pioneered by DTx.

Finally, following the first half of the session, Kenji Yamada, Director of the Policy Research Institute, moderated a plenary discussion among the six speakers on the various values of pharmaceuticals and DTx and their evaluation. 　The following is a summary of the points discussed in the plenary discussion*1.

Plenary Discussion "The various values of pharmaceuticals and DTx and their evaluation

Topic 1] Organizing the various types of value

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

Mr. Igarashi first mentioned the proximity of the concepts of wellbeing and QOL, and the integration and substitution risks in measuring them. In the UK evaluation mentioned in Senior Researcher Miura's lecture, there are two categories of benefits that do not fit into the concept of QOL, and factors that fall inside the concept of QOL but cannot be fully measured by the EQ-5D or other measures. In his presentation, Mr. Igarashi pointed out that while there is a danger of arguing that what cannot be measured is not value, there is also a danger that if a scale to measure something is standardized, what cannot be measured by that scale may be deemed not to be included.

He also pointed out that, with regard to well-being, which overlaps with the concept of QOL, some attempts to measure well-being by QOL values are being considered, but the idea that all elements can be measured by creating a scale is an illusion, and it is precisely because the discussion is gaining momentum that we must be careful about it. In addition, he said that we must be careful about what elements cannot be fully measured by well-being because it is a new measure.

As an example of the challenges in accessing treatment, Dr. Sakurai cited the case of patients with psychiatric disorders who are sometimes unable to receive satisfactory treatment due to a shortage of specialists or incompatibility of doctors. He stated that the expansion of the use of DTx is expected to bring new joys such as the ability to receive personalized and appropriate treatment and to participate in treatment without coming out to society. He pointed out that it is important for these concerned patients and citizens to participate in DTx development and visualize the advantages of DTx.

Mr. Hara expressed his views from two perspectives. First, "how to visualize the non-therapeutic value": according to a physician familiar with DTx, patients tend to be more receptive to DTx before starting medication or adding a drug. We can imagine that patients at this point in their lives may perceive the value of DTx in some way, and we need to visualize this kind of invisible value. Another point of view is to communicate the value of the digital ecosystem to patients on a real-world basis. For example, the spread of the use of DTx products is expected to lead to the evolution of DTx products, understanding of patient groups with high therapeutic efficacy, and so on. Mr. Hara pointed out that it is important to accumulate such examples and help 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 pointed out both immediate and underlying issues regarding the current situation. The immediate issue is to promote verification based on actual cases. The results of a numerical estimation of only those drugs that had been launched in the past and for which evidence had emerged at that point in time showed a negative result of approximately 90% for anticancer drugs. On the other hand, the case of the hepatitis C drug was a successful case due to a combination of various factors. It cannot be said that other drugs can be verified in the same way. Mr. Igarashi stated that if the evaluation is to include cases that are difficult to verify, it will be necessary to include qualitative aspects to some extent.

The challenge that is diametrically opposed to this is whether pharmaceutical companies are ready to align themselves and advocate value-based pricing. As objective value validation is discussed, not all cases will be evaluated positively; some will be positive and some will be negative. In some cases, the values that have been constructed may come to nothing. Mr. Igarashi pointed out that the key to seriously advancing the value discussion is whether or not we can tolerate such a situation.

Next, Mr. Sakurai shared his views on the value of prevention. The current discussion, including pharmaceuticals, starts after the onset of disease, and prevention is not on the table for discussion. From a digital perspective, there could be value in improving the health of those who are not yet ill and preventing serious illness, but this has not yet been discussed or visualized. Overseas, accessibility to medical care is worse than in Japan, and apps are being used to manage one's own health and symptoms. Mr. Sakurai commented that he expects digital to intervene even before the onset of disease, and that a wide range of support will be needed, including not only from the pharmaceutical industry but also from the insurance industry and others.

Mr. Hara expressed his belief that there is room for improvement in resource allocation based on outcomes. He pointed out that in discussions on how to score reimbursement, discussions on resource allocation are partly adjusted based on stakeholder interests, and that outcome-based discussions are not always sufficiently conducted. He stated that although there are currently difficulties with the existing system in place, it would be good to eventually have a system in which 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?

Regarding this question, Mr. Sakurai explained the situation overseas, where the value to patients is sometimes reflected in the implementation of 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 measuring the 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 Ministry of Health, Labor and Welfare in June 2023?

Mr. Sakurai mentioned that in the same expert panel, he was able to participate in the discussion as a patient group and deliver his real voice in the resolution of the drug lag and loss issue, and that the report also included "opinions from patient groups".

Q: Many DTx developments are often carried out by financially constrained start-up companies. What challenges and necessary support are there for acquiring evidence to prove various values?

Mr. Hara pointed out that the lack of predictability in how much DTx would be reimbursed by insurance was a difficult point in the development process. He noted that although development is proceeding with a certain degree of risk tolerance because this is a new area, it is important to make the predictability of evaluation a little more visible. From this perspective, he argued that various evaluation cutoffs could be the axis of the foreseeability discussion, and that if the cutoffs for evaluating value were clear, it would be easier for developers to formulate strategies for how to prove their value. However, he added that it is not simply a matter of giving a high score, but that in clarifying the evaluation axis, it is important to make adjustments to address any conflicts with the conventional approach.

Mr. Igarashi pointed out that one of the unique strengths of DTx is the ability to collect real-world post-marketing evidence since the application is in the patient's hands. A particular strength is that DTx can build evidence from collected real-world data, especially when the items (value) to be evaluated and the evaluation axis are not clear. In addition, regarding feedback of results to patients, it was pointed out 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 the full value of any treatment modality at the time of approval, and value-based pricing ultimately means reevaluation, both positive and negative, based on post-approval evidence. He stated that DTx is the most inexpensive way to accomplish this.

In addition, Dr. Sakurai expressed his opinion from the patient's point of view that, for example, digital technology could be used to graphically predict (visualize) changes in appearance caused by anticancer drugs. He believes that such data could be fed back to medical schools for education. On the other hand, in order to promote the secondary use of such data, it is necessary to consolidate the data into a single national policy, rather than individual developers working on their own, and this may facilitate the use of 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 ripple effects. It has been suggested that the groups that place the highest value on social ripple effects (in particular, productivity, return to work, and reduction of the burden of caregiving) are those who are busy with work and household chores and who have some kind of disease. It would be a good idea to show and deliver to the working (household) generation in particular that pharmaceuticals have social ripple effects, such as enabling people to work (or do household chores) while being treated for a disease, returning to work (or do household chores), and reducing the burden of caregiving.

Also, Senior Researcher Miura reiterated the importance of how to visualize the "value that is not yet visible," which came up in the part on organizing various values. He stated that an approach that provides an opportunity to hear from patients undergoing treatment and patient groups about what they want to be able to do that they could not do before, and then feeds this information back to the physicians who interact with the patients, would be an approach to make patients aware of diverse values.

Lastly, Mr. 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. Regarding the latter, there is the problem that patients have difficulty understanding 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 data*2 , which explains the contribution of DTx to medical healthcare and the various values that DTx brings, using plain language and easy-to-understand expressions as much as possible to reach the public directly. He stated that it is important to take a direct approach to the public, using plain language and easy-to-understand expressions as much as possible.

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

Dr. Hara stated that it is important for physicians to feel the therapeutic effects and usefulness of DTx, and to find out what kind of use cases it is effective in, and to develop it.

Q: What are the changes in the public's perception of value compared to 10 years ago, and what actions are being taken by the pharmaceutical industry and academia to increase awareness of value?

Mr. Igarashi pointed out 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. In the past, explanations of the value of pharmaceuticals were directed at members of the Central Social Insurance Medical Council (Chuikyo). However, there is now a change in the need to explain to the public and convince them of the value of the product. He stated that because the number of people to be persuaded has increased, it is now necessary to explain in plainer language and with more precision.

Q: How has the perception of pharmaceuticals, DTx, and healthcare itself changed for patients, and what can the industry and patients do together to raise awareness?

Mr. Sakurai commented that compared to the past, when prescribing and taking medicines were somewhat passive, now patients who would have had a severe medical condition in the past can return to society and feel a wider range of benefits and values from medicines. He concluded by pointing out the importance of continuing discussions on the value and evaluation of medicines and DTx among all stakeholders, including the pharmaceutical industry and patient groups, as was the case in this forum.

Conclusion

The Forum was attended by a large audience, with a total of over 500 people in attendance at the venue and via webcast. In a survey conducted after the meeting, 86% of the respondents answered that their understanding of the various values was "deepened" or "somewhat deepened," while the majority of respondents answered that their understanding of DTx was "deepened" or "somewhat deepened," at 86% and 89%, respectively ( Figure 1 ).

Figure 1: "Has your own understanding of the various values of "pharmaceuticals" and "DTx" deepened?" (N=65)

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

As pointed out in the Forum, the discussion on the value and evaluation of pharmaceuticals is still insufficient, and I felt the need for the pharmaceutical industry to make efforts to further visualize and simplify the value of pharmaceuticals, deepen literacy from the perspective of patients and the public, and demonstrate the value of pharmaceuticals with examples, including doctors, pharmacists, and other medical professionals. We also felt the need to demonstrate the value of the system through case studies, including physicians, pharmacists and other medical professionals.

These issues have been discussed in the past, but through this forum, we could sense that the number of stakeholders interested and participating in the discussions has been increasing in particular. With the launch of various study groups led by the Chuikyo and the Ministry of Health, Labor and Welfare, I feel that there is an increased need to explain the situation with appealing data, making full use of evaluation methods, etc. We would like to utilize the suggestions we gained from this forum in our future research and studies.

Finally, I would like to thank all the speakers and others who contributed to the forum, and all those who attended.

In the same way, for DTx, it is important to accumulate concrete evidence and demonstrate the value of those items that have not yet been visualized even though their value has been realized, and furthermore, the industry side should actively present evaluation axes such as predictability to advance the discussion.

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

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