A patient-informed clinical platform to inform patient-centered decision-making for rare diseases (INFORM-RD)

This project will deliver a platform for collection and use of data on unmet needs of RD patients to assist policy makers, industry and researchers in patient-centred decision-making related to relevant clinical endpoints or innovative therapeutic approaches. Although rare, there are over 6000 RDs that together affect up to 6% of the population. Due to multiple challenges in research on small patient groups, knowledge on RDs lags behind and 95% of RDs cannot be adequately treated. To effectively use resources toward equitable care, defining unmet needs of RD patients is crucial. Experts in ethical, legal and social issues (ELSI) collaborate for this project with the UZ Leuven RD Council within the framework of the new Leuven Institute for Rare Diseases. We will collect and standardize data on unmet needs across the approx. 30,000 RD patients diagnosed and/or cared for at UZ Leuven each year. Specifically, we will (1) develop a platform and questionnaire for unmet needs in RD, (2) collect data from the UZ Leuven cohort, (3) create methodologies to integrate and valorize this data in decision-making and (4) develop a toolbox for ELSI compliance and patient involvement.

Keywords:rare diseases, orphan medicinal product, patient experience data, health policy, unmet medical need

Disciplines:Health and community services, Health promotion and policy, Clinical trials, Health information systems of medical informatics, Public health sciences not elsewhere classified, Bioethics, Social medical sciences not elsewhere classified, Medical informatics, Public health services not elsewhere classified