ステークホルダーが一堂に介して知識を共有する価値　―中間機関の視点から

抄録

In the field of rare and intractable diseases, patients and families face numerous unmet needs due to limited medical resources, insufficient understanding, and social isolation. To address these challenges, intermediary organizations can play a key role in facilitating collaboration among diverse stakeholders.

This study highlights a collaborative research initiative on hereditary transthyretin amyloidosis (ATTRv), conducted by NPO ASrid in cooperation with clinicians, patient groups, and industry partners. Using mixed methods—including standardized QOL assessments and in-depth interviews—this project explored the health-related quality of life (HRQOL), psychological burden, family dynamics, and care experiences of patients and families.

The findings revealed significant variation in patient experiences depending on treatment availability and disease progression, as well as the importance of tailored support and communication strategies. Following the study, ASrid organized feedback sessions and a symposium involving all major stakeholders to disseminate findings and promote co-creation of solutions.

This case underscores the importance of intermediary organizations in integrating patient perspectives into research and policy, and in building inclusive frameworks for evidence-based advocacy.