Abstract
In the past 20 years great progress has been made in the development of multidimensional outcome measures (such as the Disease Activity Score and ACR20) to evaluate treatments in rheumatoid arthritis, a process disseminated throughout rheumatic diseases. These outcome measures have standardized the assessment of outcomes in trials, making it possible to evaluate and compare the efficacy of treatments. The methodologic advances have included the selection of pre-existing outcome measures that detected change in a sensitive fashion (in rheumatoid arthritis, this was the Core Set Measures). These measures were then combined into a single multidimensional outcome measure and such outcome measures have been widely adopted in trials and endorsed by the American College of Rheumatology (ACR) and the European League Against Rheumatism (EULAR) and regulatory agencies. The secular improvement in treatment for patients with rheumatoid arthritis has been facilitated in part by these major methodologic advancements. The one element of this effort that has not optimized measurement of outcomes nor made it easier to detect the effect of treatments is the dichotomization of continuous measures of response, creating responders and non-responder definitions (for example, ACR20 responders; EULAR good responders). Dichotomizing response sacrifices statistical power and eliminates variability in response. Future methodologic work will need to focus on improving multidimensional outcome measurement without arbitrarily characterizing some patients as responders while labeling others as non-responders.
Highlights
In the past 20 years great progress has been made in the development of multidimensional outcome measures to evaluate treatments in rheumatoid arthritis, a process disseminated throughout rheumatic diseases
Prior to 1990 in rheumatology and especially in rheumatoid arthritis (RA), trials tested the efficacy of treatments using outcome measures that varied from trial to trial
It was not possible to compare the efficacy of treatments across trials because each trial generally used its own set of outcome measures
Summary
For trial duration ≥1 year and agent being tested as a ‘DMARD’, perform: Radiography or other imaging technique. In the few years after the ACR20 was published, similar efforts were undertaken for juvenile RA, osteoarthritis, low back pain, psoriatic arthritis, and spondyloarthropathies; more recently, efforts for myositis and vasculitis have paralleled earlier efforts with a focus on developing a uniform set of measures for trial outcomes and sometimes defining a threshold for improvement. It is not surprising that the promulgation of a rationally selected core set of outcome measures and its consolidation into one multidimensional measure of response has occurred contemporaneously with the improvement of treatments in rheumatic disease. Meta-analyses have convincingly demonstrated that some new therapies for RA did not work as well as either conventional or new biologic agents [8,9,10] These treatments shown to be less efficacious have lost favor in the marketplace.
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