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

Autor: Kogetsu A; Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan. kogetsu@eth.med.osaka-u.ac.jp., Isono M; Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan., Aikyo T; Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan.; School of Medicine, Hiroshima University, Hiroshima, Japan., Furuta J; Department of Medical Informatics and Management, Institute of Medicine, University of Tsukuba, Tsukuba, Ibaraki, Japan., Goto D; Department of Stem Cell Therapy Science, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Hamakawa N; Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan., Hide M; Department of Dermatology, Graduate School of Biomedical and Health Sciences, Hiroshima University, Hiroshima, Japan.; Japanese Society of Tuberous Sclerosis Complex Family Net Committee, Yokohama, Kanagawa, Japan., Hori R; Department of Dermatology, Hiroshima Citizens Hospital, Hiroshima, Japan., Ikeda N; Commons Project, Osaka University, Suita, Osaka, Japan., Inoi K; NPO Japan Marfan Association, Kuwana, Mie, Japan., Kawagoe N; MECP2 Duplication Syndrome Family Association, Suita, Osaka, Japan., Kubota T; Department of Clinical Laboratory and Biomedical Sciences, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Manabe S; Department of Transformative System for Medical Information, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Matsumura Y; Osaka National Hospital, Osaka, Japan.; Department of Medical Informatics, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Matsuyama K; Commons Project, Osaka University, Suita, Osaka, Japan., Nakai T; Japanese Huntington's Disease Network (JHDN), Tokyo, Japan., Nakao I; Shirasagi-aiai-kai, Himeji, Hyogo, Japan., Saito Y; Commons Project, Osaka University, Suita, Osaka, Japan., Senoo M; NPO Myotonic Dystrophy Patients' Group of Japan (DM-Family), Tokyo, Japan., Takahashi MP; Department of Clinical Laboratory and Biomedical Sciences, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Takeda T; Department of Medical Informatics, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Takei M; Japanese Society of Tuberous Sclerosis Complex Family Net Committee, Yokohama, Kanagawa, Japan., Tamai K; Department of Stem Cell Therapy Science, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan., Tanaka A; Department of Dermatology, Graduate School of Biomedical and Health Sciences, Hiroshima University, Hiroshima, Japan., Torashima Y; Department of Surgery, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan., Tsuchida Y; NPO Myotonic Dystrophy Patients' Group of Japan (DM-Family), Tokyo, Japan., Yamasaki C; Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan.; Center for Intractable Diseases and ImmunoGenomics (CiDIC), Health and Nutrition (NIBIOHN), National Institutes of Biomedical Innovation, Ibaraki, Osaka, Japan., Yamamoto BA; HAEJ, Non-Profit Patient Organization for Hereditary Angioedema in Japan, Kakogawa, Hyogo, Japan.; HAEi, Non-Profit International Patient Organization for Hereditary Angioedema Registered in the US, Fairfax City, VA, USA.; Graduate School of Human Sciences, Osaka University, Suita, Osaka, Japan., Kato K; Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan. kato@eth.med.osaka-u.ac.jp.
Jazyk: angličtina
Zdroj: Research involvement and engagement [Res Involv Engagem] 2023 Nov 29; Vol. 9 (1), pp. 107. Date of Electronic Publication: 2023 Nov 29.
DOI: 10.1186/s40900-023-00515-5
Abstrakt: 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.
(© 2023. The Author(s).)
Databáze: MEDLINE
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