Revolutionizing Clinical Trials in Systemic Sclerosis
摘要
Systemic sclerosis (SSc) is a rare, complex autoimmune connective tissue disease with significant morbidity and mortality. Management of SSc has centered on organ-specific manifestations; and recent advances have furthered our understanding of pathophysiology, uncovered new therapeutic targets, and increased drugs available. Despite this, several recent negative clinical trials have dampened enthusiasm for further investigation of potentially beneficial therapies in systemic sclerosis. Outcome measurements are often sensitive to change but benefit with some treatments is very low so trials may be underpowered to determine efficacy. Additionally, the more common subset of SSc is limited cutaneous SSc and overall disease modification in this group has not been subjected to randomized trials except if there is specific organ involvement.
Recent findingsNew articles have discussed improving upon trial design in SSc in order to reduce care gaps (such as having RCTs in prevalent patients with active SSc and the more common lcSSc subset).
SummaryThere have been publications on improving the sensitivity and developing outcome measurements, using pragmatic or basket trial designs, eliminating placebo arms, and increasing inclusion of diverse patients and various subsets of SSc; all of which can reduce SSc unmet needs.
SummaryWe propose that a paradigm shift is needed to revolutionize clinical trials in systemic sclerosis and discuss innovative approaches to clinical trial design that will help transform the research landscape.