Concept paper on the new reflection paper on the clinical investigation of medicinal products for the treatment of systemic sclerosis
HumanScientific guidelines
The European Alliance of Associations for Rheumatology (EULAR) has recently published updated recommendations for the treatment of systemic sclerosis (SSc) (Del Galdo et al, 2025). This publication puts forward that “SSc is a rare connective tissue disorder characterised by the association of autoimmune features with vascular manifestations and culminating in tissue and vascular fibrosis of the skin and internal organs, with highly variable outcomes. Type and severity of organ involvement drive the heterogeneous prognosis, but overall SSc remains the rheumatic disease with the highest morbidity and mortality, despite recent improvement in survival”. The publication also stresses that the high heterogeneity in the presence and severity of skin and visceral involvement is a major challenge in trial design (Del Galdo et al, 2025, Del Galdo et al, 2020).
There is currently no scientific guidance from the European Medicines Agency (EMA) on the clinical investigation of medicinal products for the treatment of SSc. This is needed to guide development and support marketing authorisation applications for this condition.
Keywords: systemic sclerosis, clinical development, guidance