Measuring the effect of therapy in rheumatoid arthritis clinical trials from the patient’s perspective

Abstract

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.

Keywords::

• Disease-modifying antirheumatic drugs
• Health-related quality of life
• Patient-reported outcomes
• Rheumatoid arthritis
• Treatment response

Transparency

Declaration of funding

Support for this study and manuscript development was provided by Pfizer Inc.

Declaration of financial/other relationships

T.K., S.H.Z., and G.V.W. have disclosed that they are employees of Pfizer Inc., the sponsor of this study. R.R.-B., M.B., A.R., and M.K. have disclosed that they are employees of QualityMetric and acted as paid consultants to Pfizer Inc. in connection with the development of this manuscript.

CMRO peer reviewers may have received honoraria for their review work. The peer reviewers on this manuscript have disclosed that they have no relevant financial relationships.

Acknowledgments

This study was sponsored by Pfizer Inc. Editorial assistance was provided by Kate Silverthorne PhD of Complete Medical Communications and funded by Pfizer Inc.