Opinion The Latest Trends in Patient and Public Involvement (PPI) - A Growth Phase of Patient and Public Involvement

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Akiko Yoshida, Senior Researcher, Pharmaceutical Industry Policy Institute

1. Introduction

Almost two and a half years ago, in Policy Research Institute News No. 58 (November 2019), we introduced recent developments in Japan regarding PPI, stating that the terms Patient and Public Involvement (PPI) and Patient and Public Involvement (PPI) are beginning to catch on in Japan. In light of the current situation where awareness of PPI is generally low, it is necessary to steadily promote PPI, starting with awareness-raising activities, The concept of "Responsible Research & Innovation (RRI)" is also expected to be necessary for effective social ^{implementation1)}.

Since then, the movement related to PPI has become even more active and expansive. This paper will introduce recent developments related to PPI in Japan and in the UK, a European country with a long history of PPI, and discuss expectations and issues for Japan in the future based on these developments.

2. What is PPI?

PPI is a concept first adopted in the UK, and was originally defined as "research conducted with or by patients/citizens" rather than "research conducted for or about patients/citizens," a definition originally leaning toward the research field (medical research, clinical trials, etc. ²⁾. In recent years, however, the concept has evolved to include the participation of patients and citizens in the decision-making process, not only in the field of research, but also in healthcare policy in general. In light of this direction, this paper will consider PPI in a broader sense. Before beginning this paper, the main terms and definitions used in connection with PPI and related topics are summarized in Table 1.

3. Recent PPI initiatives in Japan

Future ^issues1) summarized in the Policy Research Institute News approximately two and a half years ago are presented in Table 2. The following sections 3.1-3.3 describe examples of major efforts that have been made in the face of these challenges.

3.1 AMED (Japan Agency for Medical Research and Development)

AMED has been promoting PPI initiatives for research through ^{a survey3)} on trends in patient and public involvement in clinical research, etc.

In recent years, an introductory ^video4) has been created to raise awareness of PPI initiatives in AMED projects, and an online contact ^point5) has been established to provide consultation on PPI from researchers and others.

In addition, the "Basic Research for Visualization of Subjective Symptoms of Hay Fever and Elucidation of Factors Contributing to Severe Symptoms Using Smartphone Applications with Patient and Public Participation" (Juntendo University), a research project for practical application to allergic diseases, realized application research incorporating patient and public ^feedback6) and developed a hay fever prevention application called The application has been put into practical use as "AlleruSearch®" ⁷⁾, a pollen allergy prevention application.

Based on AMED's definition, medical institutions and research institutes that engage in patient and citizen participation in research are expanding in various regions. Juntendo University Hospital is one such institution that is promoting PPI as a core clinical research ^hospital8).

Furthermore, as of October 1, 2021, AMED established the "Research Fairness and Social Co-creation Section" to "address ethical, legal, and social issues (ELSI) arising from medical research and development," "promote diversity to realize diverse well-being," including PPI, and "promote the development of new research and development in the field of medical research. AMED's recent representative efforts for "Social Co-creation" include the Infectious Disease Research and Development ELSI Program and the Medical Research and Development ELSI Program. The ELSI Program for Research and Development of Infectious Diseases is one of AMED's recent representative "social co-creation" efforts. In response to the new type of coronavirus infection, this program conducted a survey aimed at creating knowledge and technologies that contribute to medical research and development of infectious diseases, and in adopting the survey, the evaluation committee members representing the patients' perspective were engaged in the evaluation ^work9). In addition, in the "AMED Research Project for Practical Application of Intractable Diseases," members of patient groups participated in the preliminary evaluation of the second round of applications in ^FY202010).

AMED's efforts are not limited to the developer side, which was considered to have few opportunities to obtain information on the significance of PPI and how to put it into practice, but also include activities from the perspectives of informing, spreading, and nurturing patients and citizens, which has had a ripple effect on society as a whole.

3.2 Initiatives of PMDA (Pharmaceuticals and Medical Devices Agency): Patient Involvement Guidance issued

The PMDA aims to improve the quality of its work by reflecting the voices and opinions of patients, as well as to improve patients' understanding of and satisfaction with pharmaceuticals and medical devices, and has compiled a set of activity guidelines for the PMDA and its officers and employees to refer to, the "Pharmaceuticals and Medical Devices Agency The "Guidance for Patient Engagement" ⁽ hereinafter referred to as the "Patient Engagement Guidance") was published on September 7, 2021, which outlines guidelines for activities to be referred to by the PMDA and its officers and employees. The table of contents and overview of the Patient Engagement Guidance are shown in Figure 1. The basic policy of the Patient Engagement Guidance is to promote patient engagement by facilitating the exchange of information, as indicated by the following statement: "The PMDA will actively collect information from patients and other stakeholders to improve the quality of its work and enhance the provision of information to patients and other stakeholders to improve their understanding of the PMDA's work and pharmaceutical administration. The text of the Guidance also states that the Guidance is intended to promote patient engagement by facilitating the exchange of information. The text also focuses on information collection, reflection, and provision. The following is a summary of the text.

Regarding the collection and reflection of information from patients, PMDA's work in regulatory review and safety measures is based on scientific discussion, and the policy states that it is necessary to be conscious of scientific validity when collecting and reflecting patients' opinions. The policy also states that, when "exchanging opinions and holding study sessions with patient groups," which is supposed to be actively implemented as a framework for collecting the opinions of patients, etc., a certain degree of fairness and transparency should be considered, and conflicts of interest should be appropriately controlled, since a wide participation of patient groups is required based on their size and disease areas, etc. The document also states that a certain degree of fairness and transparency must be considered and conflicts of interest must be appropriately controlled. With regard to responses to information from patients, it is necessary to understand the needs and concerns of patients who are the target population of pharmaceuticals in the development of pharmaceuticals, etc., and efforts to utilize the opinions of patients, etc., from the planning stage in the implementation of clinical trials, etc., are becoming active, It will be noted that in the future, it is expected that the number of applications for approval including the results of clinical trials that include Patient Reported Outcome (PRO) as a part of the evaluation items will increase. It also clarifies the stance that the purpose of PROs is to obtain information that contributes to the scientific evaluation of the subject product, assuming the participation of patients and others on the consulting side in face-to-face advice, etc.

The PRO is "a useful tool for evaluating the benefits to patients in the review process, and its use is considered to contribute to the efficient development of drugs and other products whose clinical significance is supported by patients. On the other hand, "In using a PRO scale, it is important to confirm that the PRO scale has been developed and language validated based on the scientific method indicated in guidelines, etc., and the clinical significance of the evaluation by the PRO scale should be considered when interpreting the results of the PRO scale. The clinical significance of the PRO scale should also be taken into account when interpreting the results. Therefore, when such data are submitted as part of the application materials for approval, the handling of such data should be considered, paying attention to the implementation status of language validation and the results of domestic and international use, including description in clinical evaluation guidelines. The Guidelines indicate that the following is required

The PMDA will consider how to provide information that can be understood by the general public, in addition to its usual activities of providing information to patients. The report also states that PMDA needs to consider how to provide information that can be understood by the general public, in addition to its usual information provision activities, given its limited experience in direct contact with patient groups.

In addition to ensuring transparency and appropriate disclosure of information on the review process of pharmaceutical products, such as information provided on the PMDA website, and providing easy-to-understand information on side effects and defects, etc., as "basic information dissemination related to pharmaceutical administration," information that is beneficial to patients, etc., should also be provided through various media. In addition, it will be stated that information that is beneficial to patients, etc., should be widely provided using various media. It also states that the PMDA website will be listed as one of the most important media and will be enhanced to improve usability from the patient perspective, and that the use of social media, e-mail and video sites, and distribution through publications will also be considered as methods that are easy for patients to access.

The PMDA's publication of the Patient Involvement Guidance is expected to promote patient involvement in drug development, approval review, and post-marketing activities such as safety measures, and will greatly expand the scope of PPI in the drug discovery and drug development process. It is also expected to provide an opportunity for not only PMDA but also researchers, developers, and patients to obtain information on the significance of PPI and how it is practiced, which will lead to better understanding and development cases in the future.

3.3 Activation of various other activities

PPI Japan (Patient and Public Involvement Consortium) ¹²⁾ is engaged in various activities with the aim of becoming a mother body for realizing true "industry-government-academia" collaboration. As a first step to educate patients and citizens about medical care and drug development, PPI Japan concluded a basic agreement on partnership with ^EUPATI13) in November 2020 and published the Japanese version of EUPATIToolBox on its website. ¹⁴⁾ The Japanese version of the EUPATI ToolBox is designed to provide information about the drug development process in the EU. The Japanese version of the EUPATI ToolBox allows users to search for questions and obtain necessary information on research and development and patient/citizen engagement, although the approval procedures, etc. are based on the European system. The site also provides many examples of patient and citizen involvement as well as drug development.

PPI Japan has also held several patient and citizen engagement (PPI) study groups, dialogue meetings for members, and the "Let's learn about PPI activities in Japan" seminar series, among others, to promote dialogue through planning, create opportunities for communication between industry interested in working with patients and citizens, and patients and citizens interested in medical and drug development, and to promote their participation. It is expected to develop further as a platform for co-creation and collaboration for the development and diffusion of PPI.

The Japan Institute for Healthcare Research and Development (Ji4pe) ¹⁵⁾, established in June 2020, aims to share understanding of drug development and proper use with patients, patient groups, patient support groups, the general public, and industry, government, and academia, and to promote medical evaluation based on evidence and values. The website shows that DIA is promoting education and training programs to promote understanding and awareness, and that its lineup and the range of participants are expanding.

DIA (DIA Japan ¹⁶⁾ (hereafter, DIA) also holds DIA Patient Engagement Webinars as a forum for discussion to promote the four parties: industry, government, academia, and patients.

The fourth Patient Engagement Webinar (December 2021) was held to provide an opportunity for both companies and patients to learn about the value and metrics of PPI that regulators and companies have. While an increasing number of companies are implementing PPI in drug development, some companies say that they are reluctant to implement PPI for clinical trial plans and consent documents because of the burden of implementing PPI in the start-up phase of trials, and some patients say that the value of PPI is difficult to see because of insufficient feedback after implementation. The participants were also able to learn about current issues, such as patients' opinions that the feedback after PPI implementation is not sufficient and that it is difficult to see the value of PPI. In addition, a patient engagement representative from the European Medicines Agency (EMA) shared directly with the audience "the value of patient engagement from the EMA's perspective. It was shared that opportunities for patient engagement are visibly increasing and diversifying in Europe, and that the results of these efforts include patient feedback that has led to deeper discussions and some changes. The value of patient engagement is that it enables discussion of regulations that take into account the everyday aspects of people living with a disease, and that the outputs (research, development, and regulatory outcomes) are beneficial to patients. It is important to continue to improve awareness, understanding, and credibility. In the future, "everyone should play a role" in responding to new data collection including real-world data, expanding the scope of patient data collection, and improving training and support systems.

The increase in various activities by the private sector and others confirms that activities to inform, disseminate, and nurture, especially on the part of patients and citizens, are being held continuously and extensively, and will gradually have a greater impact on society as a whole.

3.4 Recent PPI initiatives in Japan (summary)

As mentioned above, we have seen the current status of a great many initiatives being undertaken over the past few years. The main examples of recent initiatives and their results are summarized in Table 3. Although it is not possible to measure the changes in our country in recent years using any indicators, many of the major initiatives listed are novel, as if the initiatives themselves had never existed before, and it can be seen that the foundations of PPI are being laid and that we are in a growth phase.

On the researcher side, where opportunities to obtain information were considered limited, activities centered on AMED to inform, disseminate, and cultivate are bearing fruit. On the developer side, where there are few opportunities to obtain information, the activities of companies are becoming more ^active17), which are not mentioned in this paper, and the PMDA has issued guidance on patient involvement, which raises expectations for the creation of a PPI system and culture for development and post-launch activities. On the patient/citizen side, where the number of people willing and prepared to participate in PPI was considered to be low due to the lack of educational opportunities on PPI, activities to inform, promote, and foster PPI by the industry, affected public, and private sectors are becoming more active, and are beginning to have an impact on society as a whole, where interest and commitment to PPI activities were generally considered insufficient.

4. Recent PPI initiatives in the UK

Then, as a recent PPI initiative in the UK, the Medicinesand Healthcare Products Regulatory Agency ("MHRA"), the UK's Medicines and Healthcare Products Regulatory Agency, issued on May 24, 2021, the ProposedPatient and Public Involvement Strategy 2020-25 ("PPI Strategy") ¹⁸⁾ issued on May 24, 2021 by the UK's Medicinesand Healthcare Products Regulatory Agency ("MHRA").

The history of PPI in the UK and other European countries is over 20 years since the first patient involvement. So while systematic patient involvement in regulatory decisions was already the norm, the PPI Strategy is the first equivalent of a formal regulatory framework. The PPI Strategy states that the MHRA views the involvement of patients and the public in its decision-making as very important and wants to listen to their concerns and opinions at every opportunity. The MHRA then sets five goals and clearly outlines what it will do and by when. An overview is provided in Figure 2, and the main points are presented in the following sections.

Goal 1: PPI

In creating the process, the document includes how to clarify the patient engagement process, including measuring expected outcomes, being mindful of health disparities, ensuring equity by creating opportunities for groups that are not actively participating in patient groups (e.g., minorities, the elderly, people with learning disabilities, and non-native English speakers) to interact with the MHRA, and actively working with other regulatory agencies and The document also describes how the patient engagement process will be clarified, including a willingness to work with other regulators and to provide training and resources to patient groups.

It also describes a flexible attitude going forward, such as taking into account the views of children and young people who may have different perceptions of medical problems and their treatment, and considering referring to and incorporating international standards and good practices in PPI.

Specific examples include: making the method more informative for patients and citizens to make informed decisions; conducting research on citizens' understanding of risks associated with medicines and vaccines after the recent COVID-19 epidemic and how risks are best communicated; and involving patients and citizens in regulatory decision-making processes, committees, and governance in a more systematic way. Developing processes to more systematically involve patients and the public in regulatory decision-making processes, committees, and governance. It also states that the use of PROs, which has become increasingly used in recent years, should be developed and incorporated into all approval/permission decisions.

In terms of information provision and collection for patient groups, the MHRA's Executive Committee will regularly attend the activities of the Patient Groups Council, which will be the central channel through which the voices of patients and citizens will be conveyed to the MHRA, and the MHRA's regulatory system, decision-making processes, committees, and governance will be transparent, with reports on how patient and citizen involvement has had an impact on the MHRA; seeking patient and public input on communication concepts and materials at appropriate times in the decision-making process and aligning them with their needs and preferences as much as possible; and introducing regular (e.g., twice a year) public meetings. Furthermore, the MHRA will actively seek the views of patients and the public on issues that the MHRA is considering or that patients and the public would like to raise with the MHRA regarding drugs and medical devices, and will seek their cooperation in determining the MHRA's priorities.

Goal 2: Responsiveness, responsiveness

It is stated that services will be designed and provided in a way that meets the needs of patients and citizens, including making the process more agile and regular in reviewing high-risk issues and implementing a system to flag when patient groups need to be more involved. Specific examples will include the establishment of a customer service center.

Goal 3: Internal culture

Our core focus will beon patients and citizens. （Our core focus will beon patients and the public, placing them at the heartof our thinking. The company is committed to transforming its internal culture by developing and introducing new systems, processes, and training, with patients and the public at the center.

Specific examples include teaching new agency employees about patient and citizen engagement at induction ceremonies, having patients and citizens talk about the importance of PPI, and reflecting this in the MHRA staff goal plans.

Goal 4: Measuring Results

This section clearly states that the Agency will ensure that progress and outcomes are measured in order to realize the vision. In measuring patient engagement outcomes, it is important to consider patient engagement in its broadest sense, beyond the scope included in the PPI strategy, and it includes three outcome frameworks for trust in the MHRA and for trust in medicines and other products.

First, whether patient involvement in signal ^{management19)} is increasing; second, whether the number of clinical trial protocols in which the MHRA has recommended incorporating PROs is increasing; third, whether the MHRA is considering and acting upon the input received from patients and the public; and third, whether the MHRA is taking into account and acting on the input of patients and the public, and whether these actions are tangible and increasing. Means of measuring these achievements include, for example, conducting objective interviews and surveys on a regular basis and publishing the results, and conducting pulse checks with patient groups through patient organizations. And it also states that it will refer to and incorporate developments in other organizations and regulatory authorities outside the UK.

Goal 5: Collaboration

In order to maximize the return of results to patients and the public, it is stated that a collaboration plan will be developed and implemented that will bring stakeholders closer together than ever before, and it is declared that efforts will focus on two points where this can create significant value.

The first point is the "exchange of insights. The increased opportunities for participation by patients and citizens, such as workshops and forums, have brought together a wealth of information and other resources, but it is necessary to bring them together. In other words, at present, it appears that systematic organization and utilization are not sufficient. The MHRA plans to consolidate information on the MHRA as a whole into a new project for efficient and effective utilization. The MHRA will also share the aggregated information with partners in the healthcare field in a bidirectional manner to maximize the use of patient and citizen feedback.

The second point is the "patient participation mechanism. The plan calls for reviewing the existing patient group councils to increase their representativeness and diversity, and in this regard, it will consider whether it is possible to collaborate with information and other assets held by partners in the health and medical care sector.

5. Summary: Challenges and Expectations for PPI in Japan

Japan's PPI system is being established with the publication of the Patient Involvement Guidance, while expanding its scope. In addition, materials, experiences, and knowledge that can be utilized for PPI are gradually being accumulated. And it is truly a period of growth, with enhanced recognition and learning opportunities (often online) for patients, the public, regulators, developers, and others. Coincidentally, the last two years have been a COVID-19 boom period, and it is possible that the accelerated shift to online events has lowered the barriers to participation, which has helped to fuel PPI growth. This growth is expected to continue in the future.

In order for PPI in Japan to achieve further growth in the future, we believe that the "collaboration" set forth by the U.K. will be helpful. We expect that the efficient and effective use of case studies and experiences through collaboration among patients and citizens, the pharmaceutical industry conducting research and development, regulatory authorities, and other stakeholders (industry, patients, government, and the private sector), as well as increasing patient representation and diversity, will promote further growth of PPIs.

6. Conclusion

PPI in Japan is expanding and deepening, particularly in the drug discovery and drug development process. On the other hand, patient and public participation in the evaluation of the value of pharmaceutical products is not taking place, and there is a possibility that values that only patients can understand, their weight, etc., are not fully evaluated and overlooked.

In order to improve this situation, it would be desirable for the evaluation of the value of pharmaceutical products to be based on both scientific objective evaluation and subjective evaluation (including patients' voices).

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