Enhancing evidence-informed policymaking in medicine and healthcare: stakeholder involvement in the Commons Project for rare diseases in Japan.

BACKGROUND: Although stakeholder involvement in policymaking is attracting attention in the fields of medicine and healthcare, a practical methodology has not yet been established. Rare-disease policy, specifically research priority setting for the allocation of limited research resources, is an area where evidence generation through stakeholder involvement is expected to be effective. We generated evidence for rare-disease policymaking through stakeholder involvement and explored effective collaboration among stakeholders. METHODS: We constructed a space called 'Evidence-generating Commons', where patients, family members, researchers, and former policymakers can share their knowledge and experiences and engage in continual deliberations on evidence generation. Ten rare diseases were consequently represented. In the 'Commons', 25 consecutive workshops were held predominantly online, from 2019 to 2021. These workshops focused on (1) clarification of difficulties faced by rare-disease patients, (2) development and selection of criteria for priority setting, and (3) priority setting through the application of the criteria. For the first step, an on-site workshop using sticky notes was held. The data were analysed based on KJ method. For the second and third steps, workshops on specific themes were held to build consensus. The workshop agendas and methods were modified based on participants' feedback. RESULTS: The 'Commons' was established with 43 participants, resulting in positive effects such as capacity building, opportunities for interactions, mutual understanding, and empathy among the participants. The difficulties faced by patients with rare diseases were classified into 10 categories. Seven research topics were identified as priority issues to be addressed including 'impediments to daily life', 'financial burden', 'anxiety', and 'burden of hospital visits'. This was performed by synthesising the results of the application of the two criteria that were particularly important to strengthen future research on rare diseases. We also clarified high-priority research topics by using criteria valued more by patients and family members than by researchers and former policymakers, and criteria with specific perspectives. CONCLUSION: We generated evidence for policymaking in the field of rare diseases. This study's insights into stakeholder involvement can enhance evidence-informed policymaking. We engaged in comprehensive discussions with policymakers regarding policy implementation and planned analysis of the participants' experiences in this project.

Stakeholder involvement is significant for effective policymaking in the field of rare diseases. However, practical methods for this involvement have not yet been established. Therefore, we developed the 'Commons project' to generate valuable policymaking information and explore effective ways for stakeholders' collaboration. This article explains the process and results of 25 continuous workshops, held from 2019 to 2021 with 43 participants, including patients, family members, researchers, and former policymakers. The main achievements of the discussion that took place in the 'Commons' included a presentation of the overview of the difficulties faced by patients with rare diseases and formulation of high priority research topics.First, the difficulties faced by patients with rare diseases were grouped into 10 categories. Second, seven research topics were identified as priority issues including 'impediments to daily life', 'financial burden', 'anxiety', and 'burden of hospital visits'. During the project process, positive effects such as capacity building, opportunities for interactions, mutual understanding, and empathy among the participants, were identified. Beyond the context of the field of rare diseases and science of policy, these findings are useful for the future of society, including co-creation among stakeholders and patient and public involvement. Based on this study's results, we have initiated communications with policy stakeholders in the field of rare diseases, with the aim of policy implementation.

Perspectives on artificial intelligence in healthcare from a Patient and Public Involvement Panel in Japan: an exploratory study.

Patients and members of the public are the end users of healthcare, but little is known about their views on the use of artificial intelligence (AI) in healthcare, particularly in the Japanese context. This paper reports on an exploratory two-part workshop conducted with members of a Patient and Public Involvement Panel in Japan, which was designed to identify their expectations and concerns about the use of AI in healthcare broadly. 55 expectations and 52 concerns were elicited from workshop participants, who were then asked to cluster and title these expectations and concerns. Thematic content analysis was used to identify 12 major themes from this data. Participants had notable expectations around improved hospital administration, improved quality of care and patient experience, and positive changes in roles and relationships, and reductions in costs and disparities. These were counterbalanced by concerns about problematic changes to healthcare and a potential loss of autonomy, as well as risks around accountability and data management, and the possible emergence of new disparities. The findings reflect participants' expectations for AI as a possible solution for long-standing issues in healthcare, though their overall balanced view of AI mirrors findings reported in other contexts. Thus, this paper offers initial, novel insights into perspectives on AI in healthcare from the Japanese context. Moreover, the findings are used to argue for the importance of involving patient and public stakeholders in deliberation on AI in healthcare.

Evaluating standards for 'serious' disease for preimplantation genetic testing: a multi-case study on regulatory frameworks in Japan, the UK, and Western Australia.

BACKGROUND: A number of countries are leading the way in creating regulatory frameworks for preimplantation genetic testing (PGT). Among these countries, a point of consensus is that PGT may be used to avoid the birth of a child with a serious genetic disease. However, standards for evaluating disease severity in this context are not always clear. Considering the numerous medical and social implications of defining a standard for serious disease, our study sought out to better understand how disease severity for PGT is being defined by analyzing and comparing the regulatory landscapes for PGT in various countries. METHODS: We carried out a multi-case study analysis using policy documents from the UK, Western Australia, and Japan. Documentary analysis was used to analyze and compare these documents in terms of medical indications for PGT, evaluation methods of applications for PGT, and review frameworks used during the evaluation process, which includes the specific medical and social factors that are considered. RESULTS: Within our three case studies, medical indications for PGT are based on an estimated risk of the woman giving birth to a child with a genetic abnormality with known clinical deficits. Evaluation methods for approving applications for PGT include reference to a pre-approved list of genetic conditions (the UK) and case-by-case reviews (all case studies). Review frameworks for case-by-case reviews include reference to a list of considered factors (the UK and Western Australia) and a definition statement of disease severity (Japan), which provide insight into interpretations of disease severity in each context. CONCLUSIONS: The results of this study point to the possible medical and social impacts of PGT regulatory frameworks on multiple stakeholders. Furthermore, it suggests that impacts in this case are not only caused by whether PGT is permitted or not, but also by the circumstances under which it is allowed and how decisions regarding its approval are made. Our results may serve as valuable insights for countries that already have established policy for PGT but are considering revision, countries that are without policy, and for discussions on related genetic and reproductive technologies.

Status of school health programs in Asia: National policy and implementation.

BACKGROUND: The WHO's Health Promoting Schools (HPS) framework is based on an understanding of the reciprocal relationship between health and education, and the need to take a holistic approach to health promotion in schools. We aim to clarify the degree to which the HPS framework is reflected in the national policies of eight target countries and the issues surrounding its successful implementation. METHODS: Date were collected through two expert workshops with participants from eight Asian countries: Cambodia, China, Japan, Korea, Lao PDR, Nepal, the Philippines, and Thailand. In the first workshop, data collected on national policy were mapped against the HPS framework. From this, key issues were identified, and follow-up data collection was conducted in each country for a second workshop. RESULTS: We identified a policy shift toward the HPS framework in six out of the eight countries. Neither Japan nor Korea had changed their national policy frameworks to reflect an HPS approach; however, in the latter, model programs had been introduced at a local level. We identified various barriers to successful implementation, especially in relation to mental health and wellbeing. CONCLUSION: Given the recent shift toward the HPS approach in six out of the eight countries in this study, there is a need to conduct research to assess the impact of this framework on the health and wellbeing of students and school staff. At the same time, we call for more dialog in the context of Japan to explore the possible benefits of introducing the HPS framework into schools.

The practice of active patient involvement in rare disease research using ICT: experiences and lessons from the RUDY JAPAN project.

BACKGROUND: The role of patients in medical research is changing, as more emphasis is being placed on patient involvement, and patient reported outcomes are increasingly contributing to clinical decision-making. Information and communication technology provides new opportunities for patients to actively become involved in research. These trends are particularly noticeable in Europe and the US, but less obvious in Japan. The aim of this study was to investigate the practice of active involvement of patients in medical research in Japan by utilizing a digital platform, and to analyze the outcomes to clarify what specific approaches could be put into practice. METHODS: We developed the RUDY JAPAN system, an ongoing rare disease medical research platform, in collaboration with the Rare and Undiagnosed Diseases Study (RUDY) project in the UK. After 2 years of preparation, RUDY JAPAN was launched in December 2017. Skeletal muscle channelopathies were initially selected as target diseases, and hereditary angioedema was subsequently added. Several approaches for active patient involvement were designed through patient-researcher collaboration, namely the Steering Committee, questionnaire development, dynamic consent, and other communication strategies. We analyzed our practices and experiences focusing on how each approach affected and contributed to the research project. RESULTS: RUDY JAPAN has successfully involved patients in this research project in various ways. While not a part of the initial decision-making phase to launch the project, patients have increasingly been involved since then. A high level of patient involvement was achieved through the Steering Committee, a governance body that has made a major contribution to RUDY JAPAN, and the process of the questionnaire development. The creation of the Patient Network Forum, website and newsletter cultivated dialogue between patients and researchers. The registry itself allowed patient participation through data input and control of data usage through dynamic consent. CONCLUSIONS: We believe the initial outcomes demonstrate the feasibility and utility of active patient involvement in Japan. The collaboration realized through RUDY JAPAN was enabled by digital technologies. It allowed busy patients and researchers to find the space to meet and work together for the Steering Committee, questionnaire development and various communication activities. While the practice of active patient involvement in Japan is still in its early stages, this research confirms its viability if the right conditions are in place. (331 words).

Landscape of Participant-Centric Initiatives for Medical Research in the United States, the United Kingdom, and Japan: Scoping Review.

BACKGROUND: Information and communication technology (ICT) has made remarkable progress in recent years and is being increasingly applied to medical research. This technology has the potential to facilitate the active involvement of research participants. Digital platforms that enable participants to be involved in the research process are called participant-centric initiatives (PCIs). Several PCIs have been reported in the literature, but no scoping reviews have been carried out. Moreover, detailed methods and features to aid in developing a clear definition of PCIs have not been sufficiently elucidated to date. OBJECTIVE: The objective of this scoping review is to describe the recent trends in, and features of, PCIs across the United States, the United Kingdom, and Japan. METHODS: We applied a methodology suggested by Levac et al to conduct this scoping review. We searched electronic databases-MEDLINE (Medical Literature Analysis and Retrieval System Online), Embase (Excerpta Medica Database), CINAHL (Cumulative Index of Nursing and Allied Health Literature), PsycINFO, and Ichushi-Web-and sources of grey literature, as well as internet search engines-Google and Bing. We hand-searched through key journals and reference lists of the relevant articles. Medical research using ICT was eligible for inclusion if there was a description of the active involvement of the participants. RESULTS: Ultimately, 21 PCIs were identified that have implemented practical methods and modes of various communication activities, such as patient forums and use of social media, in the field of medical research. Various methods of decision making that enable participants to become involved in setting the agenda were also evident. CONCLUSIONS: This scoping review is the first study to analyze the detailed features of PCIs and how they are being implemented. By clarifying the modes and methods of various forms of communication and decision making with patients, this review contributes to a better understanding of patient-centric involvement, which can be facilitated by PCIs. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): RR2-10.2196/resprot.7407.

Patient involvement in preparing health research peer-reviewed publications or results summaries: a systematic review and evidence-based recommendations.

BACKGROUND: There are increasing calls for patient involvement in sharing health research results, but no evidence-based recommendations to guide such involvement. Our objectives were to: (1) conduct a systematic review of the evidence on patient involvement in results sharing, (2) propose evidence-based recommendations to help maximize benefits and minimize risks of such involvement and (3) conduct this project with patient authors. METHODS: To avoid research waste, we verified that no systematic reviews were registered or published on this topic. We co-created, with patients, a PRISMA-P-compliant protocol. We included peer-reviewed publications reporting the effects of patient involvement in preparing peer-reviewed publications or results summaries from health research studies. We searched (9/10/2017) MEDLINE, EMBASE and the Cochrane Database of Systematic Reviews, and secondary information sources (until 11/06/2018). We assessed the risk of bias in eligible publications and extracted data using standardized processes. To evaluate patient involvement in this project, we co-created a Patient Authorship Experience Tool. RESULTS: All nine eligible publications reported on patient involvement in preparing publications; none on preparing results summaries. Evidence quality was moderate. A qualitative synthesis of evidence indicated the benefits of patient involvement may outweigh the risks. We have proposed 21 evidence-based recommendations to help maximize the benefits and minimize the risks when involving patients as authors of peer-reviewed publications. The recommendations focus on practical actions patient and non-patient authors can take before (10 recommendations), during (7 recommendations) and after (4 recommendations) manuscript development. Using the Patient Authorship Experience Tool, both patient and non-patient authors rated their experience highly. CONCLUSIONS: Based on a systematic review, we have proposed 21 evidence-based recommendations to help maximize the benefits and minimize the risks of involving patients as authors of peer-reviewed publications.

Associations between childhood experiences of parental corporal punishment and neglectful parenting and undergraduate students' endorsement of corporal punishment as an acceptable parenting strategy.

This study evaluated the effects of childhood experiences of parental corporal punishment (CP) and neglectful parenting (NP) on Japanese university students' endorsement of parental CP (EPP) to discipline children, in relation to subjective happiness (SH). A total of 536 undergraduate students who showed no physical symptoms completed anonymous paper-based questionnaires addressing demographic characteristics, undergraduate classes, and recent health conditions on SF-8 (PCS, MCS). It was found that the proportions of participants who experienced pervasive CP and NP were larger in men than in women (36.5% vs. 19.4% for CP; 22.1% vs. 9.7% for NP). Multiple regression analyses (n = 346) revealed that the CP score was associated with positive EPP (ß = 0.310, p < 0.001). Further, students whose major was nursery education reported significantly lower level of EPP; however, neither SH nor good recent health conditions significantly reduced EPP. The NP score was inversely associated with the SH score (ß = -0.253, p < 0.001) (n = 346). In conclusion, childhood experiences of parental CP may affect adolescents' views related to their own parenting. Further investigation using internationally comparable methodologies, especially in prospective cohort studies, is warranted, not only in Japan but also in other Asian countries.