Issues in the design of new clinical trials for rheumatoid arthritis therapeutics.
Rheumatoid arthritis (RA) is a chronic, heterogeneous disease, for which there has traditionally been few reliable prognostic indicators or simple clinical outcome measures with which to adequately assess disease status or clinical improvement. These challenges have hindered the assessment of new therapeutic agents. Investigators in rheumatology have, therefore, attempted over the past two decades to develop new approaches and scoring systems to facilitate the study and development of therapies. As a result of these efforts, the efficacy of almost all new agents tested in clinical trials are now assessed with widely accepted scoring systems that measure improvement in three important areas. First, changes in patient signs and symptoms are measured via composite indices of both clinical and laboratory parameters. Second, the progression of structural damage is assessed by radiographic imaging. Finally, long-term improvement in patient function is measured via patient-reported outcomes. These changes to clinical trial design have been adopted by health authorities around the world. Further challenges, however, must be overcome in order for progress to continue.
Schwieterman
William D
WD
Tekgenics, Inc., Mobile, AL 36619, USA. wds@tekgenics.org
D001172:Arthritis, Rheumatoid / Q000188:drug therapy*; D002986:Clinical Trials as Topic / Q000592:standards*; D064420:Drug-Related Side Effects and Adverse Reactions; D006801:Humans; D017063:Outcome Assessment, Health Care; D012107:Research Design; D012219:Rheumatology / Q000639:trends*