The FOCUS model: A guide to promote patient partnership in clinical trial designs

Evidence suggests that partnerships between researchers and patient communities result in clinical trials that better reflect the patient experience, but there are few documented and successful models of such partnerships. Within the clinical research landscape, the Duchenne Muscular Dystrophy (DMD) community has emerged as a positive outlier in this regard, having engaged in a research partnership over multiple years and in the approval of several disease-modifying therapies where there were previously none.

The successful partnership factors observed in the DMD community were mapped onto the three levels of the Behavior Change Wheel framework. This mapping created an academic model of patient community readiness for research partnerships. The academic model was further translated into a user-friendly, pragmatic model and blueprint.

The FOCUS Model outlines the development of a research partnership from the ground up, where each theme represents an essential component of the partnership structure. Accompanying this model is a discussion guide that communities can use in meetings or workshops to assess and enhance their readiness for research partnerships.

The FOCUS model and its blueprint offer generalizable approaches that other rare disease patient communities can leverage to foster effective and lasting partnerships with scientific, medical, and regulatory stakeholders. The approach proposed in this study has the potential to help both the clinical research community and rare disease patient communities overcome typical barriers to rare disease clinical development.