Opinion Patient and Public Involvement in the Healthcare Sector
Koji Tamura, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute
1. Introduction
The Institute of Pharmaceutical Industry and Policy Studies (IPPI) is a non-profit organization that provides a forum for the public to exchange ideas and opinions about the patient-centered medicine, shared decision-making1), value-based evaluation, and drug safety monitoring and improvement using real-world data. In Japan, the term "Patient and Public Involvement (PPI)" has been gaining attention in the context of the growing awareness of the need for a consumer-centered approach, including the evaluation of patient satisfaction, in medical practice and drug discovery and development. With these changes in the social environment and technological advances and implementations in precision medicine/healthcare and digital therapeutics (DTx), the pharmaceutical industry and companies are shifting the concept of innovation and value from the development and provision of pharmaceuticals as products to the development and provision of therapeutic tools/modalities as solutions. As a result, the pharmaceutical industry and companies recognize the need to change the concept of innovation and value from the development and provision of drugs as objects to the development and provision of therapeutic tools/modalities as solutions, and to create and nurture drugs from the patient perspective while correctly understanding the needs and satisfaction level of patients, the ultimate recipients of value2), 3.
In this paper, we would like to introduce recent developments in Japan regarding PPIs and consider their expectations and challenges.
What is Patient and Public Involvement (PPI)?
PPI is a concept first adopted in the U.K. and defined as "research conducted with or by patients/citizens, rather than for or about patients/citizens " 4). In addition, recently, the concept has expanded to include the involvement of patients and citizens in the decision-making process, not only in the field of research (medical research, clinical trials, etc.), but also in healthcare policy in general. This paper will consider PPI in a broader sense, based on the direction of involving patients and citizens in decision-making in all aspects of healthcare policy, while keeping the scope of PPI centered on the research field5).
3. Targets of "Patients" and "Citizens" and "Stages" of Involvement
The Japan Agency for Medical Research and Development (AMED) defines patient/citizen involvement in medical research and clinical research as "the process in which researchers refer to the findings of patients and citizens as part of the medical research and clinical trial process," where patients and citizens are assumed to be "patients, family members, former patients (survivors) and future patients (6). The National Institute for Health Research (NIHR) in the U.K. states that anyone can be eligible for participation in research, and examples include "patients, caregivers, (non-health professional) citizens, community groups, former patients and survivors, charity providers, patient support groups, family members, schools," etc. All of them include a wide range of supporters other than patients, although their "partyhood" differs, and it would not be a great mistake to consider the parties involved in PPI (in a broad sense) as beneficiaries of healthcare (in a broad sense), together with the meaning that everyone will one day become a patient.
For PPI, there are "stages" (steps) to reach the goal7) (Figure 1). In the research field, the first step is "patients/citizens becoming research participants," such as when they become subjects in a clinical trial, which is called "participation. The second stage is "the sharing of information and knowledge of research results by researchers with society after the research is completed," which includes the case where information such as clinical trial results is provided to subjects and patient groups after the research is completed. The third step is "Involvement," which means that patients and citizens are involved in the planning, design, management, evaluation, and dissemination of results as "research partners. Involvement also includes active involvement as stakeholders in the planning, monitoring, and evaluation of healthcare-related policies, etc., as well as in the study of solutions to various issues in the practice of medicine outside the research field.
Recent trends in Japan
In Japan, the importance of PPI has been recognized by some parties concerned, and momentum for PPI initiatives is gradually building, but the reality is that only a small number of people are involved in voluntary trials.
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(1)Government Policy, etc.
In the Third Basic Plan for the Promotion of Cancer Control (approved by the Cabinet on October 24, 2017), it is stated as a measure to be taken in the future that "AMED will establish a system to enable patients and cancer survivors to participate in the design and evaluation of research in Japan as well as in overseas research systems, Efforts to promote cancer research through the participation of people with cancer experience will be initiated. In addition, the government will begin to develop a program to educate patients who can participate in the planning and evaluation of research" and the "Report Contributing to the Formulation of a 10-Year Strategy for Immunological Allergy Research" by the Research Group on "Establishment of Research Infrastructure for Allergy Disease Control" of the Health and Labor Sciences Special Research Project (March 30, 2018). (March 30, 2018), the "Research on the Promotion of Patient Public Involvement (PPI)" was presented as one of the action plans to achieve "the establishment of an international research and development infrastructure for immunological allergic diseases that involves all citizens, including patients, and places importance on the contribution of each and every one of them". However, the relevant laws, regulations, guidelines, and However, related laws, guidelines, and public training programs have not yet been developed.
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(2)Japan Agency for Medical Research and Development (AMED)
The AMED has positioned PPI as an essential concept in the promotion of R&D in healthcare (Figure 2).
One of the backdrops for AMED's promotion of PPI is the "Plan for the Promotion of Research and Development in the Medical Field" (February 17, 2017, partially revised), which was decided on July 22, 2014 by the Cabinet's Headquarters for Health and Medical Care Strategic Promotion. In this plan, the importance of "efforts to collaborate with patients and to educate the public" is pointed out, and the need to "promote the participation of subjects and patients (←changed from the original "collaboration") in clinical research and clinical trials from the planning stage" is clearly stated.
AMED has established a website8) on PPI, and has stated the need to "promote patient and public involvement (PPI) in medical research and clinical trials with the aim of bringing the results of research in the medical field to practical use and to patients and their families as quickly as possible, while staying close to each patient and supporting his/her LIFE (life, life, and life). (PPI) initiatives in medical research and clinical trials." In 2017, a committee on "Trends in Patient and Public Involvement in Clinical Research, etc." consisting of researchers and representatives of patient groups was established (Table 1), and in March 2019, the committee compiled a report on "Trends in Patient and Public Involvement in Clinical Research, etc." and published a booklet for researchers, " Patient and Public Involvement (PPI) Guidebook" and a leaflet "Patient and Public Involvement (PPI) in Medical Research and Clinical Trials" were prepared. In addition, starting with the 2019 public call for proposals, the R&D proposals will be required to include a description of PPI initiatives (although this is optional at this time).
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(3)Pharmaceuticals and Medical Devices Agency (PMDA)
Yasuhiro Fujiwara, who was the chairman of the AMED "Survey on Trends in Patient and Public Involvement in Clinical Research, etc." committee mentioned above, became the new president of PMDA in April this year, and in order to take the initiative in addressing the four "Fs" (Firsts) 9), the "Patient Involvement Study The "Patient Involvement Study WG" was organized in May of this year as part of the first "Patient First" activity. The WG will "examine PMDA's efforts to promote patient engagement and collaboration, including examining public relations for patients and developing guidance on patient activities and PMDA's involvement in them. Although specific activities have not been announced at this time, many related departments are listed, including the Corporate Planning Department, International Affairs Department, Review-related Departments, Safety Affairs Departments, Research Support and Promotion Department, etc., and we expect that the study will be actively promoted from a variety of perspectives, not limited to the above.
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(4)Patient and Public Involvement Consortium (PPI Japan)
On July 1, 2019, the "Patient and Public Involvement Consortium (Patient and Public Involvement Consortium)" will be established as a platform for patients and citizens interested in medical and drug development, academia, and the pharmaceutical industry to discuss together and disseminate information on the concept and process of medical and drug development, as well as ethical and highly transparent collaboration among the parties involved. The Patient and Public Involvement Consortium in Japan was established on July 1, 201910). The Consortium "aims to promote mutual understanding and collaboration among patient groups, patient support groups, the general public, and industry, government, and academia, which are essential stakeholders in medical and drug development, and to become the mother body for realizing true "industry-public-involvement" collaboration by formulating activity policies and plans based on careful communication, while uncovering the needs of the relevant parties. The JCMT aims to be the mother body for realizing true "industry-government-academia" collaboration.
As the first step, they will establish an operating body in Japan in partnership with the European Patients' Academy on Therapeutic Innovation (EUPATI)11), 12) and promote practical operation of activities in Japan. The first step is to establish an operating entity in Japan in partnership with the European Patients' Academy on Therapeutic Innovation (EUPATI) 11) and 12 ) and to promote practical management of the activities. As the next step, they will plan forums and workshops to promote dialogue, create opportunities for communication between industry interested in collaboration with patients and citizens, and patients and citizens interested in medical and pharmaceutical development, develop human resources to coordinate these activities, and establish related rules. The consortium is expected to carry out activities related to the following areas. The consortium has founders from industry, academia, government, and the private sector (Table 2), and is expected to develop as a platform for co-creation and collaboration for the development and diffusion of PPI.
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(5)Initiatives in academia and the private sector (examples)
Although PPI-related activities have been initiated by some lethal disease-related academic societies, starting with the activities of the Japanese Association for Cancer Therapy, there are not many initiatives in other disease fields, except for some voluntary efforts.
Regarding patient/citizen participation in the creation of medical practice guidelines, the Patient/Citizen Specialized Subcommittee of the EBM Dissemination and Promotion Project (Minds) of the Japan Institute for Health Care Excellence published "Basic Concept of 'Patient/Citizen Participation in the Creation of Medical Practice Guidelines'" 13) on November 15, 2016. The "basic idea" here is that medical practice guidelines are "documents that provide recommendations for decision-making based on the values and wishes of patients and the medical judgment of healthcare providers. About 10 years before the Minds version, the MHLW Research Group developed "Guidelines for Patient and Supporter Involvement in the Process of Medical Guideline Development (PIGL)," which outlines the procedures and support measures for patient and supporter participation in the development of medical guidelines, and the Japan Patients Association Information Center participated in this research group (14). 14), 15, which participated in the research group.
In addition to the above, the Center for Medical and Human Rights (COML), a certified NPO, has established a basic course "Training Course for Citizens Supporting Medical Care" and an advanced course "Training Course for General Members of Medical-Related Councils" for those who have completed all of the courses, in order to foster human resources who can play a sufficient role in councils, etc.16) The advanced course "Training Course for General Members of Medical Councils " is designed for those who have completed all of the advanced courses.) Those who have completed the advanced course and meet certain requirements will be eligible for registration in the general committee candidate bank.
5. Efforts by Pharmaceutical Manufacturers' Associations (Examples)
The Pharmaceutical Manufacturers Association of Japan (PMAJ) states in its "PMAJ Industry Vision 2025" (released in January 2016) that it aims to realize patient-participatory medicine. With reference to the opinions and recommendations of the Advisory Board of the Committee for the Promotion of Collaboration among Patient Organizations, the Committee and the Clinical Evaluation Subcommittee of the Drug Evaluation Committee will play a central role in identifying issues and collecting information to promote Patient Centricity activities by pharmaceutical companies with patient organizations, with the aim of better utilizing patient feedback in drug development. The Clinical Evaluation Subcommittee of the Drug Evaluation Committee is taking the lead in identifying issues and collecting information in order for pharmaceutical companies to promote Patient Centricity activities with patient groups.
The Clinical Evaluation Subcommittee of the Drug Evaluation Committee has been working on the theme of "Patient Centricity" since FY2016 with the ultimate goal of implementing drug development that utilizes the voice of the patient in all pharmaceutical companies. In June 2018, the "Patient Centricity" report was published17). This report focuses on drug development by pharmaceutical companies, and includes the following topics: trends in regulatory authorities and public-private partnerships for Patient Centricity activities in Japan, the U.S., and Europe; results of a questionnaire survey of companies belonging to the Clinical Evaluation Committee; examples of such activities by each company; proposals and effects for promoting such activities; a survey of such activities by patient groups in Japan, the U.S., and Europe; and a survey of such activities by patient groups in Japan, the U.S., and Europe. In September 2019, to further promote the implementation of these activities in each company, we collected case studies of these activities from each company and published a report entitled "Patient Centricity in the Pharmaceutical Industry", which summarizes the specifics of each case study, implementation procedures, effects, achievements, and points to keep in mind when implementing these activities. A Guidebook for Pharmaceutical Companies to Implement Activities Based on Patient Centricity: Drug Development Utilizing Patients' Voices," and a report on how to communicate with patient groups based on the knowledge and experience gained by the companies concerned. The report "Drug Development Utilizing the Voice of the Patient: A Communication Guidebook for Pharmaceutical Companies to Promote Activities Based on Patient Centricity with Patient Groups" was prepared and published18), 19.
6. Conclusion
Efforts related to PPI in Japan may not yet have reached the "engagement" stage, analogous to the "stages" indicated in Figure 1. Considering the fact that awareness of PPI is still generally low, it is necessary to start by steadily raising awareness of the issue. ELSI (Ethical, Legal and Social Issues) However, it is also necessary to promote science and technology in a manner that meets the needs and expectations of society with the participation of diverse stakeholders throughout the entire process from basic research to social implementation, and to promote the RRI (Responsible Research Initiative), which is an initiative to promote science and technology in a manner that meets the needs and expectations of society. The concept of RRI (Responsible Research & Innovation), an initiative to promote science and technology in a manner that meets society's needs and expectations with the participation of diverse stakeholders throughout the entire process from basic research to social implementation, will also be necessary for effective social implementation20).
Finally, we would like to conclude this paper by summarizing the main issues and measures from the "Expectations for Future Efforts" section of the AMED "Survey of Trends in Patient and Public Participation in Clinical Research, etc." Committee's summary of discussions21) ( Table 3).
Reference
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Kaori Muto, "Patient and Public Involvement (PPI) in Medical Research and Clinical Trials
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Kaori Muto, "Patient and Public Involvement in Clinical Trials 'PPI' in Japan" (Weekly Igakukai Shimbun No. 3132, July 06, 2015)
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Ikuko Yamaguchi, "Patient and Public Involvement (PPI) is on the Rise."
- Interview with President Suematsu, to describe patient and citizen engagement initiatives from FY 2019," Nikkei Biotech Online, August 21, 2019.
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Patient and Public Involvement (Patient and Public Involvement) in the Medical Field"
JPMA NEWSLETTER No. 194 (November 2019) -
Medicine and Drugs - Patient-Centered Drug Development - Fall 2019" (Pharmaceutical Affairs Daily, October 07, 2019)
PPI Initiatives Gaining Momentum - Interview with Kaori Muto, Professor, Institute of Medical Science, The University of Tokyo
Drug Development Needs Public Participation - Interview with Kyoko Imamura, Specially Appointed Professor, Graduate School of Pharmaceutical Sciences, The University of Tokyo
Challenges and Prospects for Drug Development - Interview with Naomi Sakurai, President, Cancer Solutions, Inc.
EUPATI Educational Tools in Japan - Interview with Satoshi Miki, Vice President, UCB Japan
Reflecting Patients' Voices in Drug Development
Examples of Experiences of Researchers and Patient Groups - From the "PPI Guidebook" compiled by AMED
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1) Number of reports and countries from which data was obtainedCollaborative Decision Making. Refers to a process in which both patients and physicians participate equally in the medical decision-making process, helping patients make decisions about treatments and other options based on their preferences. (Reference.) https://www.pmda.go.jp/files/000221675.pdf , https://www.england.nhs.uk/shared-decision-making/
https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-guidelines/shared-decision-making -
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5)Synonyms for PPI include Patient Engagement and Research Advocacy. While these terms are used from the patient's point of view, Patient Centricity is used in Japan more from the healthcare provider's point of view.
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11)European Patient Academy for Innovative Therapies The European Patient Academy for Innovative Therapies (EAP) is a non-profit, non-profit organization. Currently in its second phase, more than 60% of the partners are EFPIA-related companies (funds and in-kind contributions). EFPIA Japan also established PASE (Patient Advocacy Support by EFPIA Japan) in 2017 as a project to support patient organizations.
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14)MHLW Science Research Grant, Comprehensive Research Project for Evaluation of Medical Technology, "Research on Infrastructure Development for Appropriate Creation, Use, and Dissemination of Evidence-Based Clinical Practice Guidelines: Toward Promotion of Patient and Medical Staff Participation (Principal Investigator: Takeo Nakayama, Graduate School of Medicine, Kyoto University).
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