Social Innovations

In ITINERARE, we acknowledge the far-reaching societal impact of science and innovation. Therefore, social innovation (ELSI and clinical trial design) are an integral aspect of our research program. Rare disease research poses two principal challenges: i) the low prevalence of individual rare disease cases, and ii) the high costs associated with complex therapies. Moreover, fast-paced developments in medical research have created a lag with appropriate ethical-legal and health-economic frameworks. ITINERARE takes the initiative to close this gap through an unprecedented combination of expertise. WP3 analyzes the risks and benefits of treating genetic diseases in a vulnerable population. WP3 is closely involved with WP1-2 in joint development and clinical translation of novel therapies, including psychosocial and reimbursement issues, starting in phase I of ITINERARE, but continuing through the whole clinical trial development process in phase II and III. Moreover, we will provide recommendations to improve healthcare access to treatments, one of the key concerns of the National Rare Disease Policy.

The challenges and implications associated to an equitable treatment access for rare disease patients will be investigated from the perspective of constitutional law. In addition, we will address legal questions concerning human research, data access, and privacy protection.

The ethical and psychosocial issues faced by rare disease patients and their families, such as access to diagnosis and treatment, incurred costs, or the effects on social life, will be investigated. Ethical perspectives will be discussed in the context of healthcare and criteria for research prioritization in will be developed.