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Randomized controlled trials in systemic lupus erythematosus: what has been done and what do we need to do?

Food and Drug Administration, CDER, Rockville, MD 20850, USA, schiffenbaue{at}cder.fda.gov

L S Simon

Harvard Medical School, Boston, MA 02115, USA

The study of systemic lupus erythematosus (SLE) is a challenging undertaking. It is difficult to assess outcomes in SLE randomized controlled trials (RCTs), and this is illustrated by the lack of new therapies approved for use in lupus. In a disease that is waxing and waning, and requires constantly changing medications, identifying treatment effects of new therapies may be difficult, and the use of potentially toxic therapies requires a rigorous understandingof the benefit to risk ratio. Some issues that need to be consideredby the investigatorin designingthese studies include:1) should the trial focus on patientswith active or inactive disease; 2) which of the measures of disease activity should be used or should preventionof flares be examined; 3) should the study focus on defined organ specific endpoints or utilize one of the available disease activity indices to identify changes in disease activity; 4) should the trial be a superioritytrial or an equivalencetrial. This review summarizes the critical issues involving the design of studies in lupus and provides the reader with suggestionsand recommendations for considerationbefore embarking on trials in this area.

Key Words: disease activity indices (DAIs) • flare • health related quality of life (HRQOL) • lupus • noninferiority trials • randomized controlled trials (RCTs)

Lupus, Vol. 13, No. 5, 398-405 (2004)
DOI: 10.1191/0961203303lu1033oa


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