Pragmatic Comparative Effectiveness Trials in Rheumatoid Arthritis-Associated Interstitial Lung Disease

Abstract

Background

Rheumatoid arthritis-associated interstitial lung disease (RA-ILD) portends a devastating prognosis for patients, with survival typically being < 5 to 8 years after diagnosis. Limited clinical trial data exist to guide treatment strategies, and the efficacy of current strategies—immunomodulation and antifibrotics—remains uncertain. Large randomized controlled trials are costly, but pragmatic trial designs could reduce expenses. Establishing equipoise and assessing feasibility from both patient and expert perspectives are essential for developing these trials.

Research Question

What are the perceptions of RA-ILD experts and patients with interstitial lung disease surrounding equipoise, feasibility, and trial design?

Study Design and Methods

A qualitative study involving a panel of 10 RA-ILD experts and 3 patient panels was conducted. Experts were recruited via snowball sampling, and patient panels included 29 individuals with interstitial lung disease or their caregivers. Discussions were transcribed and analyzed using inductive coding, creating a thematic network based on the Attride-Stirling guidelines.

Results

Expert themes included variability in treatment strategies, prioritizing patient-reported outcomes and balancing pragmatism with data collection in trial design. Patient themes highlighted outcomes of importance, participation barriers, and the need for patient-centered research.

Interpretation

Both expert and patient panels endorsed using real-world clinical outcomes and patient-reported outcomes as primary trial end points. Pragmatic trials could reduce costs and expand inclusion criteria, highlighting the potential of patient-centered approaches in RA-ILD research.