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Can non-fundable trials be conducted anyway? The case for open, randomised, actively controlled trials in rheumatology

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Clinicians are encouraged to practice “evidence-based medicine”, a term implying that a practitioner can access, summarise, and apply information from the literature to day to day clinical problems and questions.1 But how convincing should the evidence be for a clinician to accept the data and adopt the approach into his or her practice? Undoubtedly the double blind, placebo controlled, randomised trial remains as the ultimate tool for gathering the strongest evidence of therapeutic efficacy and safety. However, this study design is at times unfeasible, unethical or may not even be appropriate for obtaining the information needed (for example, long term outcome), and less stringent alternatives have to be considered.2 3 In rheumatology, where the optimal therapeutic approach to many conditions is still uncertain, approved drugs are frequently used “off label” (implying that the agent is being used to treat a disease for which it has not gained formal approval, or at a dosage level other than that recommended on the label). Diverse combinations of drugs are also commonly tried as clinicians attempt to retain or reclaim a higher functional outcome and quality of life for their patients. Understandably, one frequently encounters conflicting data when searching for evidence about the clinical utility of these new approaches.4 Furthermore, the likelihood of a pivotal trial ever being done to establish unequivocal evidence of effectiveness and safety may be remote.

Less rigorous designs may not produce evidence as compelling to the practitioner as a pivotal study, but they can provide at least some insight into therapeutic effectiveness and safety where otherwise none would exist. One such approach is the open, randomised, actively controlled trial (ORACT), a mainstay of haematology-oncology research studies of the US based Pediatric Oncology Group (POG) and Children’s Cancer Study Group.5 Virtually all newly diagnosed children with …

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