title: Are better endpoints and better design of clinical trials needed?
abstract: For the clinician it is important to know whether a new drug works, and how the new drug performs against other drugs. However, new drugs are typically tested in placebo-controlled trials without active comparison. New drugs are often tested in a population with high levels of disease activity. Clinicians, however, may also seek the optimal treatment for patients with persistent moderate levels of disease activity. An answer may come from clinical trials that compare two effective drugs in patients with moderate disease activity. A main consequence, however, is that trial endpoints are needed that can detect small but relevant differences in efficacy. An ideal endpoint for trials in rheumatic diseases may be a marker that continually follows short-term changes in the disease process. The levels of the marker should be strongly associated with long-term outcome, thus prognosticating the future. When it can be measured truly and feasible, the marker would be useful as an endpoint in trials and for supporting treatment decisions in clinical practice.
authors.affiliation: University Medical Centre Nijmegen, Department of Rheumatology, P.O. Box 9101, 6500 HB Nijmegen, The Netherlands. j.fransen@reuma.umcn.nl
authors.forename: Jaap
authors.initials: J
authors.lastname: Fransen
authors.forename: Piet L C M
authors.initials: PL
authors.lastname: van Riel
mesh_terms: D018501:Antirheumatic Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D018849:Controlled Clinical Trials as Topic; D005260:Female; D006801:Humans; D008297:Male; D018579:Patient Selection; D016032:Randomized Controlled Trials as Topic; D012107:Research Design; D012216:Rheumatic Diseases / Q000175:diagnosis / Q000188:drug therapy*; D012307:Risk Factors; D012680:Sensitivity and Specificity
chemical_list: D018501:Antirheumatic Agents