In clinical trials on the effectiveness of disease-modifying antirheumatic drugs (DMARDs) in patients with rheumatoid arthritis (RA), it is common to apply a large number of endpoint measures. This practice has several disadvantages. To determine which endpoint measures are most valuable, reports of 32 clinical trials on six DMARDs were reviewed. The frequency with which each endpoint measure was used is described and discussed, as well as the frequency with which the values of each endpoint were significantly different in statistical comparisons within or between groups, thus showing ability to discriminate between drugs not equally effective. The results of this review are discussed and compared with other reports in the literature on the choice of endpoint measures in RA clinical trials. The authors conclude that it is still common practice to evaluate multiple outcome measures. The number of measures could be reduced by using only those that are generally considered important, are sensitive to change, and are able to differentiate between drugs in clinical trials. A joint count, assessment of pain, a questionnaire on functional status, and measurement of erythrocyte sedimentation rate are sufficient.
AimThis article reviews recently published reports of clinical trials on the effectiveness of
DMARD treatment of RA. The impact of each endpoint measure is assessed using the following questions: Which endpoint measures are being used in RA clinical trials?
Which outcomes are most sensitive in detecting effects of treatment by showing statistically significant changes in DMARD-treated groups? Which measures are most decisive in differentiating the effectiveness of various treatments by showing statistically significant differences between groups treated with drugs not equally effective?
van der Heide A, Jacobs JWG, Dinant HJ, Bijlsma JWJ.
Disease Category: Rheumatology
Disease Name: Rheumatoid arthritis
Age Range: Unknown
Sex: Either
Nature of Intervention: Drug
- COS uptake study
- Systematic review
Reports of clinical trials assessing the effectiveness of DMARD therapy published in English from 1986 through 1990 were selected for this review. This search was done using a
CD-ROM computerized bibliography. Trials were included that studied patients with RA
according to the American College of Rheumatology diagnostic criteria of 1958” or 1987’. The frequency with which each endpoint measure was used was determined for all trials and for short-term ( < 24 weeks) and long-term ( > 24 weeks) trials.