Project 460621

Systemic Sclerosis (scleroderma; SSc) is a rare musculoskeletal and skin disease characterized by thickening of the skin and fibrosis of internal organs. The burden and impact of SSc on quality of life are high, yet there are few high-quality clinical trials of psychosocial, educational, rehabilitation, or disease management programs to support quality of life in SSc. The Scleroderma Patient-centered Intervention Network (SPIN), an international collaboration of patient organizations, clinicians, researchers, and people with SSc has developed an infrastructure that can be used to study problems prioritized by people with SSc and develop, test, and disseminate accessible programs. Importantly, active engagement of individuals with SSc and patient organizations in the planning and conduct of SPIN's research has been central to its success. Although effective patient engagement is recognized as a key factor in maximizing the relevance, feasibility, acceptability, quality, and dissemination of health research, how best to do this requires evidence on strategies to foster patient engagement, approaches for collaboration with patients or organizations, consideration of patient partner characteristics and equity, and potential barriers to patient engagement, along with strategies to overcome identified barriers. SPIN's approach thus far has been extensive but "ad hoc" and would be strengthened by a careful review of evidence on patient engagement and by engaging patients themselves to evaluate our efforts on patient engagement, use our experiences and evidence to inform a more structured and robust framework for effective patient engagement, and to disseminate knowledge gained. We are seeking funds for planning to address gaps in patient engagement by bringing together people with SSc, patient organization leaders, researchers, and clinicians to jointly develop an evidence-based framework for a patient engagement program in musculoskeletal and rare disease research.