Top News Press Conference of the President of the Pharmaceutical Manufacturers Association of Japan held.
On January 27, 2020, the "Pharmaceutical Manufacturers Association of Japan Press Conference" was held at Nomura Conference Plaza Nihonbashi (Chuo-ku, Tokyo). The press conference was attended by 38 members of the media, who engaged in a lively exchange of questions.
Scene at the venue
Presentation Agenda
Mr. Joji Nakayama, Chairman of the Pharmaceutical Manufacturers Association of Japan (PMAJ), gave a presentation titled "PMAJ's Initiatives: Progress and Future - Pursuit of Innovation and Resolution of Social Issues" on "Creating an Environment for Innovation Promotion" and "Appropriate Evaluation of Innovation" in the "PMAJ Policy Recommendations" released in January 2019. The presentation focused on the efforts related to the "Pharmaceutical Manufacturers Association of Japan Policy Proposal 2019," which was announced in January 2019, and explained the progress to date and the policy for future efforts ( Fig. 1 ). The following is a summary of the contents of the meeting.
Chairman, Pharmaceutical Manufacturers Association of Japan
Fig. 1: The progress and future of the Pharmaceutical Manufacturers Association of Japan (PMAJ)
I. Creation of an environment for innovation promotion
Progress to 2020 and policy for future initiatives
Since we announced the progress of our policy proposal in January 2019, we have continued conversations with the government through public-private dialogues and advisory meetings. As a result, we believe that our requests have been taken into account and that measures that can be expected to lead to further industry-academia-government collaboration have been included in the Kotta Policy 2019 and the next draft of the Health and Medical Strategy. We have also begun to consider joint research with relevant stakeholders on individual initiatives. We have already formulated a highly concrete research plan and hope to begin the implementation phase this year.
In addition, the Pharmaceutical Cooperative Association has also been participating in the study of the "Action Plan to Promote Genomic Medicine," which was initiated in 2019. As a result, the first version of the "Action Plan for Whole Genome Analysis, etc." has been prepared. It is necessary to discuss more detailed parts of the plan in the future, and the Pharmaceutical Manufacturers Association will continue to actively participate in the discussions.
Regarding the R&D taxation system, several requests from the industry side were reflected when the system was revised in April 2019. We will continue to actively utilize the expanded tax system and communicate with the government to ensure that the time-limited measure is extended to 2021.
1. progress on the Pharmaceutical Manufacturers Association Policy Proposal 2019
The policy recommendations were developed with the goal of creating a virtuous cycle in which innovation enables people to extend their healthy life expectancy and economic growth, which in turn leads to the next round of innovation. We have consulted with the government and relevant stakeholders on three issues
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(1)Early practical application of preventive and preemptive medical solutions
In order to realize prevention and early intervention, a series of long-term data from healthy state to disease onset and recovery, as well as data obtained by comparing healthy people with patients, are important. To this end, the Pharmaceutical Cooperative Association is planning a cohort study in collaboration with Tohoku University Tohoku Medical Megabank Organization (ToMMo) and the National Center for Advanced Medical Specialties (National Center, NC) by inviting interested participating companies.
We signed a confidentiality agreement with ToMMo in 2019 and have been discussing the details of the study. We are first looking to conduct a small, short-term pilot study and hope to sign the agreement around March. We are also considering separately with NC and plan to conclude a joint research agreement during FY2020. We will conduct the research in the areas of psychiatry/neurology and immuno-inflammation, where there is a high unmet medical need, and will consider expanding the target areas in the future.
Since ToMMo is unique in that it includes data from many healthy individuals, and the consent of the individuals has been obtained to allow industry to use the data, we intend to actively utilize the data. This project was originally planned to continue until FY2020, and the Pharmaceutical Manufacturers Association of Japan (PMAJ) requested its continuation in light of the importance of the project. Although the specific project plan will be discussed in the future, the Pharmaceutical Consortium sincerely hopes that the biobank will be enhanced through large-scale whole genome analysis and the collection of data that can confirm long-term changes over time. We also hope that the biobank will promote collaboration with other genome projects and establish a foundation for integrated analysis.
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(2)Development and utilization of healthcare big data and AI
We expect that by utilizing genome information, we can conduct research and development that will contribute to prevention and personalized medicine. We have been participating in several government meetings on genome-related issues, and I would like to explain our requests for the "Cancer Genome Information Center (C-CAT) to play a central role" and the "whole genome analysis project being considered by the Ministry of Health, Labor and Welfare," which are two of the most important measures.
First of all, the necessary systems are being discussed so that the data accumulated by the C-CAT project will be available to industry from FY2021. Accumulation of cases and high-quality, substantial clinical information are important for drug discovery research. The Cancer Genome Medicine Consortium Steering Committee is discussing how to improve the efficiency of clinical information collection in consideration of reducing the burden on medical institutions.
Next, regarding the whole genome analysis project, at the end of 2019, the government formulated the first version of the "Whole Genome Analysis Action Plan" for cancer and intractable diseases. The first version of the plan provides rough numerical targets for advanced analysis, but some important issues, such as the establishment of an operational structure and industry-academia collaboration system, will be discussed in the future. The Pharmaceutical Manufacturers Association of Japan (PMAJ) will continue to cooperate in the discussions so that a concrete plan can be formulated. The establishment of databases and mechanisms that meet the needs of industry will lead to the promotion of data utilization and the securing of user fees. It is extremely important to create a virtuous cycle that leads to the continuous collection and further utilization of data. To this end, we believe it is important to proactively incorporate the needs of industry from an early stage.
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(3)Establishment of an ecosystem for healthcare innovation creation
The next health and medical care strategy was discussed and a draft was released at the 2019 Councilors' Meeting. It is expected to promote more collaboration between industry, academia, and government, including the sharing of equipment and devices, the promotion of cohort studies, the establishment of the Japan Agency for Medical Research and Development (AMED) Fund, and the creation of a platform for public-private partnerships in the field of dementia. The pharmaceutical industry also hopes to be actively involved in industry-academia-government collaboration projects.
One of the concrete steps we are considering is the establishment of a corporation around April 2020 that will manage industry-led projects in a flexible manner. One of the characteristics of the policy advocacy effort is that multiple companies will participate in a single project. As a result, it will be necessary to "consolidate and manage funds, data, and intellectual property" and "unify contracts," etc. We believe that the new corporation will be able to smoothly promote policy advocacy by working in cooperation with the Pharmaceutical Manufacturers Association.
2. research and development taxation
The R&D tax system is the most important tax system for the pharmaceutical industry as it promotes innovation, and the April 2019 revision reflects industry requests, including a two-year extension of the aggregate tax holiday and expansion of the open innovation tax holiday. The time limit expires in April 2021, and we would like to request an extension of the time limit in 2020.
We would like to make more active use of the expanded open innovation type, but there are some requirements that hinder its use and prevent it from being used more effectively. We will have conversations with the government side about easing the regulatory requirements.
II. Appropriate evaluation of innovation
Progress to 2020 and policy for future initiatives
As part of our efforts to promote the proper use of pharmaceuticals, we have been co-hosting a seminar on drug resistance (AMR) with the Japan Medical Association, a public interest incorporated association, and working with the Council on the Proper Use of Drugs to create public awareness materials on polypharmacy. In the future, we will further strengthen and promote initiatives for proper use of drugs, such as holding media forums related to polypharmacy.
With regard to NHI drug price reform, we have requested that the NHI drug price system be revised to appropriately reflect the medical value of pharmaceuticals, such as by improving the additional payment for new drug creation, etc., in preparation for the FY2020 NHI drug price reform. We will continue to study the "reorganization of the evaluation system for new drugs" and "establishment of an evaluation system that is easy for the public to understand" for the FY2022 NHI drug price reform. We will also move forward with "organizing the thinking behind the consideration of an all-generation social security system" ( Figure 2 ).
Figure 2: Appropriate Evaluation of Innovation
1. efforts to promote appropriate use
As reported at previous press conferences, the Pharmaceutical Manufacturers Association of Japan (PMAJ) has established a "Subcommittee for the Promotion of Appropriate Use" and has been promoting studies. This subcommittee has been studying measures to promote proper use of medicines beyond the conventional framework, with regard to the global health issue of AMR, polypharmacy, and drug residuals. With regard to the promotion of proper use, the Pharmaceutical Manufacturers Association of Japan (PMAJ) has been promoting studies and activities on four promotion issues that we would like to strengthen our efforts. Of these, this section describes the details of our studies and activities to address the AMR and polypharmacy issues.
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(1)Tackling the issue of drug resistance (AMR)
As part of our efforts to address the AMR issue, we co-hosted an AMR seminar with the Japan Medical Association. The seminar featured lectures by physicians, government officials, pharmaceutical organizations, and others on issues and approaches to infectious disease countermeasures from their respective perspectives. The participants also engaged in lively discussions, which we believe deepened their understanding of AMR countermeasures. In addition, we have been promoting educational activities such as projecting the "STOP AMR" anti-AMR video at Quorn Pharmacy. We intend to further promote AMR awareness-raising activities and verify their effectiveness in the future.
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(2)Promotion of polypharmacy to the public and patients
With regard to activities to promote polypharmacy to the public and patients, we have been working with the Council for the Proper Use of Drugs to prepare a pamphlet for the public on the "Guidelines for the Proper Use of Drugs for the Elderly" formulated by the Ministry of Health, Labour and Welfare. The pamphlet is scheduled to be completed by the end of February 2020, and we will promote educational activities using this pamphlet from March onward. We also plan to hold media forums. In addition, we will also strengthen measures to raise awareness of polypharmacy among member companies by preparing training materials for them. The preparation of training materials is already underway and is scheduled to be completed by April 2020. The materials will be distributed to member companies, and will also be made available on the website of the Pharmaceutical Manufacturers Association of Japan (PMAJ) for wider use. We are also planning to hold a briefing session for those in charge of training, and are making preparations to promote initiatives that will contribute to raising employee awareness at participating companies.
2. review of FY2020 drug price reform and considerations for FY2022 drug price reform
In the reform of the NHI drug price system for FY2020, we have requested three points: "Improvement of the additional payment for new drug creation," "Reorganization of the additional payment system for new drugs," and "Review of criteria for selection of similar drugs. The Central Social Insurance Medical Council (Chuikyo) has recently compiled a framework for reform of the NHI drug price system for FY2020, and we will provide our assessment of the contents related to each of these requests.
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(1)Improvement of Additional Allowance for New Drug Creation
We have requested that the requirements for items be expanded based on the evaluation of high medical necessity, innovation, and usefulness, and for the company requirements, we have requested a system in which the NHI price of eligible items is maintained regardless of the size of the company, as long as the company is involved in the development of innovative new drugs. While the reform of the NHI drug pricing system in FY2020 has resulted in improvements in both areas, we believe that these improvements have been limited. We believe that the basic concept of this system is to properly identify truly effective drugs, evaluate innovation, and promote R&D investment. We will continue to carefully monitor whether the review of the system will contribute to the promotion of R&D investment by each company.
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(2)Restructuring of the additional payment system for new drugs (medical value)
As for evaluation at the time of NHI drug price listing, medical value that contributes to improvement of the quality of treatment will be evaluated as a case equivalent to the usefulness addition "c. improvement of treatment methods. We believe that our request was approved to a certain extent. We hope that this will promote the research and development of drugs with medical value that contribute to improving the quality of treatment.
On the other hand, the reimbursement mechanism has been further strengthened without any expansion of the evaluation after the drug price is listed on the NHI drug price list. We are concerned that the strengthening of the reimbursement mechanism will lead to a decrease in motivation to develop additional indications. From the perspective of promoting the addition of innovative and useful indications, we believe that the enhancement of the evaluation of innovation after the NHI drug price listing needs to be improved and is a matter for consideration in the next reform of the NHI drug price system.
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(3)Review of criteria for selection of similar drugs
Although the system was not reviewed, it was decided to first accumulate cases with similar medical realities, such as clinical positioning, and then consider the issue. From the perspective of increasing the transparency and acceptability of NHI drug price calculations, the industry will actively work to accumulate case studies in preparation for the next reform of the NHI drug price system.
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(4)Items to be achieved in the NHI price reform in FY2022
Based on the details of the FY2020 NHI drug price reform, we have reorganized the items that we hope to achieve in the FY2022 NHI drug price reform ( Figure 3 ). Of these, "reorganization of the new drug evaluation system" will be studied with three specific issues: "evaluation of social value," "evaluation of post-listing innovation," and "review of criteria for selection of similar drugs.
Figure 3: Appropriate Evaluation of Innovation in the NHI Drug Price System
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(5)Status of consideration of "evaluation of social value
The social ripple effects brought about by pharmaceuticals can be considered to be varied if one broadens one's perspective. We believe that some medicines can contribute to the sustainability of social security by increasing the "supporters" of the economy and society, or by leading to more efficient consumption of medical resources. We believe that pharmaceuticals with such value should be evaluated to a certain degree because they will ultimately be returned to the sustainability of social security.
On the other hand, we recognize that there are issues to be considered with respect to these "social values," such as the definition of value and methods of evaluation. Therefore, we have begun research and surveys on outcome indicators and measurement methods, with the visualization of the various values of pharmaceuticals as our main task.
The Task Force of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), an international academic organization that promotes awareness and dissemination of pharmacoeconomics and outcomes research, has reported on the various values of pharmaceuticals, and we are currently studying how the values reported by ISPOR are defined, We are currently working on the definition of the values reported by the ISPOR and how they compare to the Japanese pharmaceuticals system.
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(6)Evaluation of innovation after NHI drug price listing
In the reform of the NHI drug price system in FY2020, the reimbursement system will be further strengthened. This will significantly reduce the predictability of the overall business of companies, and there is concern that it will lead to a decrease in motivation for development of additional indications. Adding new indications after the NHI drug price listing contributes to the improvement of the quality of medical care from the perspective of increasing the number of drug treatment options according to the patient's condition. From the viewpoint of promoting the addition of indications with high innovation and usefulness, we will promote consideration of the enhancement of the evaluation of innovation after NHI drug price listing and request improvements in the next NHI drug price system reform.
With regard to the revision of criteria for selection of similar drugs, it will be possible to select a wide range of similar drugs, which will lead to a reduction in calculations based on the cost accounting method. Although the NHI drug price reform in FY2020 will continue to consider this issue, the Pharmaceutical Manufacturers Association of Japan (PMAJ) would like to actively cooperate in the accumulation of case studies in order to create a better system for the next NHI drug price system. As we have explained at previous press conferences, we are also looking forward to the continued consideration of NHI reform in FY2020.
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(7)Improvement of the evaluation process for new drugs
As I have explained at previous press conferences, we would like to establish an evaluation system through the use of evaluation reports. This is based on the premise that through an evaluation report prepared by a third party, the selection of comparator drugs and the value of drugs as explained by companies will be objectively evaluated and described, and the contents will be made public. If the drug price is calculated based on this evaluation report, it will be easier to understand the basis on which the drug price of a new drug is based, including the company's thinking.
The "reorganization of the new drug evaluation system" and "improvement of the new drug evaluation process" are expected to reduce the number of cases of calculation based on the cost accounting method and eliminate the black box nature of NHI price calculation. By establishing an "evaluation system that is easy for the public to understand," we hope to increase the public's confidence in NHI drug prices and the convincing nature of NHI drug price calculations.
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(8)Regarding social security for all generations
Next, I would like to explain the Pharmaceutical Manufacturers Association of Japan (PMAJ)'s position on the consideration of an all-generation social security system. There are three major points.
First, we believe that in order to achieve both the "establishment of a sustainable social security system" and "sustainable economic growth," it is important to consider the issue from a longer-term perspective. Therefore, we hope that the reform will not only take a relatively short-term perspective, such as the period from 2022 to 2025, when the baby boom generation will reach the late elderly, but also look ahead to the year 2040, when the number of elderly people will peak.
Second, we would like the government to present to the public the ideal shape and broad vision of social security in a super-aged society. Based on this overall vision, we hope that it will stimulate national debate on each of the issues.
Thirdly, many experts have spoken about the importance of increasing the number of so-called "supporters" in the social security system. I hope that a wide range of measures to increase the number of "supporters" will be discussed.
An interim report on an all-generation social security system was compiled in December 2019. The "basic concept" and "major reform points" compiled in the report are all based on the basic premise that people should work in "good health. Pharmaceutical companies can contribute to the establishment of an all-generation social security system by extending healthy life expectancy.
The contribution of drug discovery to an all-generation social security is shown here ( Fig. 4 ).
Fig. 4 Contribution of drug discovery to social security for all generations
In the age of 100 years of life, there are various diseases that cause problems at each stage of life. We hope to contribute to the extension of healthy life expectancy and increase the number of people who support it by creating innovative medicines for each stage of life, not only for cancer, which remains an important issue for human life and health, but also for dementia, rare diseases, and others. In addition, we would like to contribute to the social security for all generations by creating drugs for individual disease stages, from prevention and preemptive medicine before disease onset to improvement of quality of life (QOL) after treatment and reintegration into society.
Closing Remarks
In order to bring the benefits of innovative new drugs to Japan and further extend healthy life expectancy, it is necessary to strongly promote both "creation of innovation" and "appropriate evaluation of innovation," not one or the other, but the two wheels of the cart. We have incorporated what is needed to achieve this into our policy recommendations. We will do our utmost to pursue innovation and solve social issues, which is our mission. We ask for your support for innovation and your understanding of the value of innovation.
Main Questions and Answers
Q&A session
Q1 What are the challenges for this year, 2020?
A major theme is the time-limited extension of the aggregate type of R&D taxation. Since it is a very effective stimulant for R&D investment, I believe it is an effective system for promoting R&D investment in the Japanese economy as a whole. In addition, although the open innovation type has been expanded, there are many cases where the maximum deduction is not used due to procedural requirements.
As we move forward with discussions on reforming the NHI drug pricing system, in the long run we would like to broaden the recognition of its social value. For example, if a certain anticancer drug is only 10% effective, a diagnostic agent that identifies this 10% will be extremely valuable. In this way, we would like you to recognize that there are values that affect the entire social security system, and by appropriately evaluating these values, we would like you to create a system that will produce pharmaceuticals that contribute to the sustainability of the entire social security system.
As we face the trend of declining birthrates, an aging society, and increasing social security costs, at the end of the day, the public needs to be convinced about the flow of money. To this end, we would like to increase the transparency of the drug price calculation process and make the system more convincing in terms of price.
Q2 What is the industry's approach from the perspective of not only the NHI drug pricing system but also the approach to all-generation social security?
Although the focus of the debate has been on the immediate future (2022), Japan's infrastructure (social security system) needs to be stable in 2040, when the number of elderly people will peak in Japanese society. To this end, we need to consider the process to realize a society in which the younger generation is healthy and active in 2040. We would like to discuss the issue from this perspective, but to do so, it is necessary to create an environment in which good pharmaceuticals can be produced in Japan. For example, various measures, such as the enhancement of genome databases, must not only be reduced, but good ones must be evaluated and the situation must be such that development in Japan is oriented to include good ones from overseas. This, in turn, will lead to Japanese pharmaceuticals going overseas. In addition, if the efficiency of R&D using genome databases is improved, it will lead to cost reductions, and the profits generated will positively affect the Japanese economy. If such various measures are taken, they will provide a brighter outlook for the future, and will be more likely to produce results than other types of investment.
Q3 You mentioned that the review of the additional subsidy for new drug creation was limited, but it is difficult to see what you want to do with the additional subsidy for new drug creation in terms of recognizing issues for FY2022?
While there have been revisions to both the corporate and item requirements, we are still not fully convinced of some of the details. We would like to present some ideas on this point and discuss them. However, since discussions will not move forward even if we continue as we are now, we would like to explain more appropriately from various angles, including the evaluation of drugs after they are listed on the market. Regarding the item requirements, we will accumulate concrete examples and make efforts to broaden the viewpoint by explaining examples, such as the fact that very important drugs have been created in the past even if they are third or later in the list.
Q4 In the evaluation of innovations after the NHI drug price listing, you mentioned the evaluation of additional indications with high innovation and usefulness, but do you have any specific examples?
Although we do not have specific examples at hand, what we wanted to show here is that the discussion has so far been biased toward lower evaluation, such as reimbursement, etc. If this situation continues, companies will lose motivation to add new indications, so positive evaluation is also necessary. There is a concern that a positive evaluation will lead to a further increase in NHI drug prices, but even if a positive evaluation is included, it is unlikely that NHI drug prices will increase beyond the current level. For items for which drug prices are reduced too much, if there are other indications that are innovative and useful, incentives should be provided for the benefit of patients. That is not a level that would exceed the downward pressure.
Q5 The Ministry of Finance has expressed its opinion on the scope of reimbursement. In an environment where innovative new drugs are being introduced one after another and are becoming more expensive, there is an argument that how long will old drugs continue to be reimbursed under the limited fiscal resources?
The scope of reimbursement is based on the concept of a universal health insurance system, where all insurers have the right to benefit from approved drugs. A national debate is needed to review the scope of such coverage. Even within the scope of the current insurance system, various measures have been taken, such as increasing the weight of generics, and this may be the next discussion after further progress is made. Above all, the rights of insurers should be redefined and discussed. In addition, the scope of such rights should also fully reflect the opinions of physicians, and the extent to which they are possible in treating patients should be discussed.
Q6 In order to generate a positive increase in the main part of reimbursement each time the reimbursement is revised, the NHI price portion is allocated.
Since there is a history of devaluing most or more of the increase in social security costs through drug prices, this should be fundamentally reviewed. On the other hand, the appropriateness of higher reimbursement should be examined in relation to the management of medical institutions, and cannot be directly compared to NHI drug prices. For our part, we would like to discuss only whether or not it is appropriate in relation to the value of the drug.
Q7 How and by when will the industry consider and organize the part of social security for all generations?
Basically, the discussion will be about how much to change the burden on those who support the patient and those who are supported by the patient. 2040, if people who are healthy until the age of 75 can work and those who are 76 or older can be supported, we can draw a picture in which the balance between those who support the patient and those who are supported by the patient is maintained at the same level as it is today. In order to achieve this, it is necessary to consider what is necessary for the health care of people aged 75 and what are the important diseases among them. Although the mortality rate of cancer is improving, some types of cancer are still fatal, so it is possible to change the balance of society as a whole and make it a better place to live by saving these people. This is what I would like to discuss.
Q8 Is it correct to recognize the need to visualize the various values of pharmaceuticals in order to support the infrastructure of the future?
That is right. If all the people are not convinced, the money in the world will not circulate. If we can convince people, we can make good use of it. NHI prices are extremely complicated, and it is difficult even for legislators, who represent the people, to understand everything about them. It is problematic for it to become a central issue in society in such a state. We believe it is extremely important to make transparent the process by which NHI prices are determined and how they are determined.
( Chikara Yoshida, Policy & PR Subcommittee, Public Relations Committee)