Measuring the effect of therapy in rheumatoid arthritis clinical trials from the patient's perspective.
OBJECTIVE
Health measurements used to evaluate the effectiveness of rheumatoid arthritis (RA) therapies often fail to reflect patients' priorities, despite recommendations towards more patient-centered assessments. The goals of the current review are: (1) to present guidelines, tools, and required steps for successful implementation of patient-reported outcome (PRO) measurement in RA clinical trials; and (2) to identify gaps between recommendations and current practices.


METHODS
The first objective was addressed by reviewing existing frameworks for assessment of health-related quality of life among patients with RA and guidelines on the evaluation of PRO instruments, with a focus on evidence required to demonstrate the adequacy of PRO-based labeling claims. The second goal was addressed by conducting an empirical investigation of the overlap between patients' perspectives and current practices regarding PROs in RA studies, elaborated from systematic literature searches. The first search identified qualitative studies that reported direct input from patients with RA, while the second identified the main health outcomes measured in RA trials, with a focus on biologic therapy.


RESULTS
Our review revealed a set of outcomes that have thus far not been widely used to assess treatment benefit in RA, despite evidence of their importance to patients. The psychometric properties of PRO instruments used to evaluate commonly assessed domains are presented, as are recommendations for PRO tools that assess domains less often measured in RA studies.


CONCLUSIONS
Although the validity of some PRO tools among patients with RA is well established, further work needs to be done in several health domains which have traditionally received insufficient attention.
QualityMetric Inc. , Lincoln, RI , USA.
Regina
R
Rendas-Baum
Martha
M
Bayliss
Mark
M
Kosinski
Aditya
A
Raju
Samuel H
SH
Zwillich
Gene V
GV
Wallenstein
Tamas
T
Koncz
D018501:Antirheumatic Agents / Q000627:therapeutic use*; D001172:Arthritis, Rheumatoid / Q000188:drug therapy*; D002986:Clinical Trials as Topic; D006801:Humans; D063868:Patient Outcome Assessment; D017060:Patient Satisfaction; D017410:Practice Guidelines as Topic; D011594:Psychometrics; D011788:Quality of Life
D018501:Antirheumatic Agents
Disease-modifying antirheumatic drugs; Health-related quality of life; Patient-reported outcomes; Rheumatoid arthritis; Treatment response