FDA Ethics Working Group Consensus Statement on the Pediatric Advisory Subcommittee’s September 11, 2000 Meeting
(placebo controlled trials)
1. Placebo controlled trials may be acceptable if there are no approved or adequately studied therapies for children with the condition under study. For serious or life threatening conditions, a Data Monitoring Committee (DMC) should be used with planned interim monitoring and stopping rules to permit early termination of a study that has shown clear evidence of effectiveness or unacceptable risks prior to completion of planned studies. In studies of symptomatic therapy for less serious conditions, it may be helpful to build in individual patient discontinuation criteria, (early escape) so that exposure to ineffective treatment is limited.