Physicians’ vs patients’ global assessments of disease activity in rheumatology and musculoskeletal trials: A meta-research project, 2022, Lynæs et al

[Andy]

Full title: Physicians’ vs patients’ global assessments of disease activity in rheumatology and musculoskeletal trials: A meta-research project with focus on reasons for discrepancies

Abstract

Background
In most rheumatic and musculoskeletal diseases (RMDs), global assessments of disease activity by physicians and patients are ‘anchor outcomes’ in therapeutic trials evaluating whether a treatment is effective.

Objectives
To compare physicians’ vs patients’ global assessments of disease activity in RMD trials and explore reasons for discrepancies between them.

Methods
Eligible trials were sampled from systematic reviews of treatments for RMDs by using the Cochrane database of systematic reviews (i.e., reviews from the Cochrane Musculoskeletal Group, [CMSG]). Randomized controlled trials (RCTs) of interventions for RMDs were eligible if they reported quantitative analyses of both physicians´ and patients´ global assessments at the same time point for the comparison of the same experimental intervention against the same comparator (i.e., placebo, no treatment, or other treatment). We accepted data from trial comparisons for each type of outcome, regardless of the type of intervention and type of RMD within the CMSG. Using mixed-effects meta-regression models, we assigned the dependent variable as the ratio of odds ratios (ROR) of global change with the experimental intervention, versus the control comparator. An ROR>1 would indicate that physicians rated the experimental intervention more favorable than their patients did.

Results
We were able to estimate the ROR (data from both physicians’ and patients’ global assessments) across 70 trials (116 randomized comparisons) in 7 diseases (ankylosing spondylitis, fibromyalgia, psoriatic arthritis, osteoarthritis, rheumatoid arthritis, systemic lupus erythematosus, and gout). The combined ROR across all effectiveness comparisons were rated significantly in favor of the intervention by physicians: ROR=1.15 CI 95% (1.07 to 1.23). This combined ROR was based on a substantial heterogeneity across comparisons (I2=89.1%). Across all the stratified analyses, the type of the RMD was an informative reason for discrepancies, with a statistically significant ROR in rheumatoid arthritis ROR=1.33, CI 95% (1.13 to 1.56), unlike the ROR in all other conditions (ROR=1.04, CI 95% (0.95–1.14).

Conclusion
In comparative effectiveness research on rheumatology, physicians’ global assessments of disease activity, surprisingly, are more in favor of the experimental interventions than are those of the patients.

Open access, https://www.sciencedirect.com/science/article/abs/pii/S0049017222001251

 

[rvallee]

In this meta-research project, we found that on average physicians’ global assessments of disease activity were more in favor of the experimental interventions than those of the patients. However, a subgroup analysis by disease revealed that this result was limited to studies on RA (52 comparisons) alone. There was no apparent source for the discordance between the result of the subgroup analysis for RA studies versus Other RMD conditions. However, some of the discordance could in part be due...

I don't know if we have to pay for the rest but it ends there. A pay-for-speculation model is an odd thing.

I don't even understand what this is about, what "perception of effectiveness of experimental interventions" means. Or how it makes sense to have such a conclusion based on only one sub-group while leaving it unclear in the conclusion. Or what this has to do with "assessment of disease activity".

This is bizarre. Cochrane being Cochrane, I guess.

 

[Hutan]

Ironically, patients´ global assessments might be viewed as a ‘subjective measure’ [19]. However, only patients themselves can provide their perspective on the outcomes of disease and its treatment [17], and for individual experience of disease activity, the patient may be the only source of information. Patients’ global assessments have been shown to be reliable, feasible, and sensitive to change [12] (and inherently have good face validity).

I wonder if this paper aims to bolster the use of patient reported measures as primary outcomes, which would of course make trials of CBT or GET for fatigue that use e.g. the Chalder Fatigue Questionnaire ok.

The context is everything, and we don't see acknowledgement of that in the abstract (and only the Introduction is open access). If physicians are biased towards an intervention and the assessments are subjective and aren't blinded, of course their assessments will favour the intervention. If patients are biased towards an intervention and their assessments aren't blinded, then of course their reports will favour the intervention, especially if the symptoms being assessed are particularly subjective (e.g. fatigue versus counts of explosive diarrhoea) and there is a background of natural improvement or fluctuation.