Twenty-two points to consider for clinical trials in systemic sclerosis, based on EULAR standards

Area of Science:

• Rheumatology
• Clinical Trial Design
• Systemic Sclerosis Research

Background:

• Systemic sclerosis (SSc) is a complex autoimmune disease with significant clinical heterogeneity.
• Existing clinical trial designs and outcome measures for SSc lack uniformity.
• There is a need for evidence-based guidelines to standardize SSc clinical trials.

Purpose of the Study:

• To develop consensus-based Points to Consider (PTCs) for future clinical trials in SSc.
• To establish uniform standards for trial design and outcome measure selection in SSc research.
• To guide the development of higher-quality, more consistent SSc clinical trials.

Main Methods:

• Systematic literature review using PubMed and Cochrane Central Register of Controlled Trials.
• Inclusion of thirteen international SSc clinical trial experts.
• Internet-based Delphi exercise to achieve expert consensus on PTCs.

Main Results:

• A general outline for Points to Consider (PTCs) for SSc clinical trials was developed.
• Consensus was achieved on key aspects of general trial design, inclusion/exclusion criteria, and analysis.
• Specific PTCs for individual organ systems will be published separately.

Conclusions:

• The developed general outline for PTCs provides a framework for uniform SSc clinical trial design.
• Implementation of these PTCs is expected to improve the quality and consistency of SSc trials.
• The guidelines will help identify areas requiring further research in SSc.