Organizacje pacjentów i choroby rzadkie: od problemu medycznego do polityki lekowej państwa. Aspekty społeczne, ekonomiczne i polityczne

Rare Diseases Patient Organizations: From Medical Challenges to the National Drug Policy

The book examines the activities of patient organizations in the field of rare diseases (RDPOs), their evolution, and their role within the healthcare system. Drawing on many years of research, the author explores the origins of RDPOs, their organizational identity, and transformation from communities united by a lived experience of illness into knowledge-based activism. The book further addresses the relationship-building and development of networks with stakeholders at both the national and European Union levels and assesses the extent to which RDPOs influence national drug and health policy contributing to legal framework changes. The study presents RDPOs as key drivers of movements within their field and co-creators of the doctrine of social participation, with a tangible impact on the democratization of decision-making processes and ensuring equitable access to the advances of medicine. The monograph fills a significant gap in existing research. Its value includes empirical evidence drawn from interviews, observation, and analyses of documentary sources, complemented by practical guidelines for patient organizations and decision makers.

Keywords: patient organizations, rare diseases, social participation, democratization of decision-making processes, health policy

Agnieszka Stasiewicz-Swinney holds a PhD in Management and Quality Sciences from the University of Warsaw. Her research interests encompass the interdisciplinary field of management and health sciences, with a particular focus on patient organizations as a unique type of non-profit sector entity, as well as on their institutional and social impact shaping the development of social participation and the democratization of decision-making processes in healthcare in Poland and Europe. She has more than twenty years of experience working in advisory teams within national and international business environments, with expertise spanning management, health economics, public relations, and public affairs. Her academic and professional work is grounded in many years of experience as a physician, practicing in both Poland and Finland. In 2025, Agnieszka Stasiewicz-Swinney was awarded the Prime Minister’s Award for an Outstanding Doctoral Dissertation for her research on the functioning of Polish Rare Disease Patient Organizations in particular, for designing and conducting a complex research project that enabled an interdisciplinary and comprehensive examination of organizations, which constitute a fundamental support for people living with rare diseases.