Project 449979

Rare diseases can negatively affect a patients' quality of life and psychosocial support needs of affected individuals often go unmet. A randomised clinical efficacy trial showed a newly developed, peer-delivered psychosocial support program led to improved quality of life (Depping et al. JAMA Psychiatry, 2021). As the program is independent of patients' location and disease, it has the potential to reach a large group of patients with rare diseases. However, the program is not yet available to patients. This will require confirmation of effectiveness in routine care and implementation preparation. In the proposed project, we intend to initiate the implementation of the program for patients with rare autoimmune liver diseases. We aim to conduct an effectiveness-implementation hybrid trial in routine care in five diverse countries. We will assess effectiveness of the intervention in routine care within a randomized controlled trial by comparing outcomes in patients who receive the intervention (and care-as-usual) with patients who just receive care-as-usual. We will include 200 patients with rare autoimmune liver diseases, who will be randomly assigned to either of the two groups. Our primary outcome will be mental health-related quality of life directly after patients in the intervention group com-pleted the intervention. After the trial, we will assess acceptability and feasibility of the program in a quantitative survey. In addition, we will conduct focus groups with different healthcare stakeholders to identify implementation barriers and derive country-specific implementation strategies. By preparing the implementation in five countries, the study can pave the way for widely available low-threshold psychosocial support for patients with rare diseases and support them in adjusting to the disease and staying mentally healthy.