Research project on stakeholders in rare disease health care

During the last decades all over the world policy programs have been established aiming to support the health care provision for patients with rare diseases (RD). Major efforts have been made to create better conditions for research, to build networks of researchers, physicians, and patient organizations, and to introduce orphan drug regulations that lower the threshold to bring new drugs to market. From an overarching perspective, it seems like all involved stakeholders are pulling together to improve the field of RD. However, it should not be overseen that these stakeholders are likely to have various self-interests. Indeed, we know little about how they interact with each other, how they exchange their viewpoints and how their self-interests are aligned. Under conditions of scarce health care resources, expensive research, and extremely high costs for specific drugs conflicts of interest seem inevitable.

Aim of the study is to understand the similarities and the differences of the stakeholders’ positions and self-interests regarding the healthcare provision for rare diseases. We intend to close this research gap and furthermore identify overseen problems as well as deepen our understanding of still prevailing challenges in the field of RD. We will conduct heterogenous focus groups, in which we bring together relevant stakeholders to discuss the most urgent problems in the healthcare provision for patients with RD with a specific focus on the approval, research and funding as well as reimbursement of orphan drugs.