Evolving Clinical Trial Design and Innovative Approaches

Clinical trial design is challenged in systemic sclerosis by the heterogeneity of the disease, a diversity in natural history of certain manifestations such as skin fibrosis. However, there has been substantial progress in study design and refinement of endpoints, so that therapies can now be successfully evaluated for complications such as lung fibrosis, digital ulceration and pulmonary arterial hypertension. Composite endpoints that reflect more global disease burden together with more disease-specific patient reported outcomes are being developed. These advances will provide a robust foundation for the next generation of clinical trials and will underpin progress in advancing evidence-based treatment.