Assessment of Placebo Response in Objective and Subjective Outcome Measures in Rheumatoid Arthritis Clinical Trials

Abstract

Key Points Question Are subjective patient-reported outcomes vs objective biomarkers associated with higher placebo responses in clinical trials? Findings In this cross-sectional study examining the placebo arms of 5 randomized clinical trials of rheumatoid arthritis including 788 patients, objective markers of inflammation and subjective pain ratings improved in a comparable clinically meaningful magnitude. Baseline values were associated with placebo response, suggesting that regression to the mean might dominate response to randomized placebo treatment. Meaning The findings of this study suggest that investigators may need to improve their understanding of natural history and baseline levels of outcomes because these factors can be important contributors to the response in placebo arms.

The effect of atorvastatin on inflammatory markers in sulfur mustard gas induced bronchitis; a randomized double-blinded, placebo-control clinical trial

“…Although the CAT score of our participants met the minimum of clinical important differences (33) of a 2-unit reduction in both groups, its mean differences was not signi cant between groups (mean difference [95% CI]: 0.57 [-2.5, 3.7]). The CAT score reduction in the placebo group can be explained by placebo effect (34). In contrary to our ndings, Maneechotesuwan et al, studied the effect of simvastatin 20 mg daily versus placebo on sputum in ammatory markers, airway in ammation, and CAT score of 30 patients with stable COPD (35), and found clinically signi cant important reduction in CAT score after statin administration.…”

Feasibility of investigating methylphenidate for the treatment of sarcoidosis-associated fatigue (the FaST-MP study): a double-blind, parallel-arm randomised feasibility trial

“…The small sample size makes it difficult to draw conclusions about the performance of either arm, though both subjective and objective measures have been seen to improve in the placebo arms of trials in other conditions. 36 We performed multiple baseline fatigue measurements prior to commencing medications given the subjective nature of fatigue, in line with previous suggestions, 36 though other aspects of the trial design may have influenced the outcomes in the placebo arm. The high level of contact with the study team may have reduced anxiety levels 37 38 ; anxiety is known to moderate fatigue.…”

The effect of atorvastatin on inflammatory markers in sulfur mustard gas induced bronchitis: a randomized double-blinded, placebo-control clinical trial

“…Although the CAT score of our participants met the minimum of clinical important differences [ 33 ] of a 2-unit reduction in both groups, its mean differences was not significant between groups (mean difference [95% CI]: 0.57 [− 2.5, 3.7]). The CAT score reduction in the placebo group can be explained by placebo effect [ 34 ]. In contrary to our findings, Maneechotesuwan et al [ 35 ] studied the effect of simvastatin 20 mg daily versus placebo on sputum inflammatory markers, airway inflammation, and CAT score of 30 patients with stable COPD, and found clinically significant important reduction in CAT score after statin administration.…”