On September 11, 2000, the Pediatric Advisory Subcommittee met and was supplemented by a group of ethicists and international experts. The subcommittee was charged with providing guidance on the ethical considerations of placebo-controlled trials in the pediatric population.

The FDA Pediatric Ethics Working Group has summarized the discussion of this subcommittee meeting. The consensus areas of discussion are as follows:

2) Add on placebo-controlled trials that do not deny any element(s) of the standard of care (SOC) are generally acceptable if individual patient study discontinuation criteria are defined. These studies would require a DMC under the same circumstances as would any other trial.

3) In placebo-controlled studies of minor illnesses and symptomatic conditions, exposure to placebo and patient discomfort can often be minimized by use of a randomized withdrawal design, usually with defined individual patient discontinuation criteria (escape rules) so that the time of exposure to ineffective treatment is minimized. These studies can be used to demonstrate long-term effectiveness when a long-term placebo-controlled trial would be unacceptable. DMC's generally are not needed for studies of symptomatic conditions but would be desirable if there were important safety concerns.