Phase 1 clinical trials in oncology are designed to test safety, identify dose recommended for further testing and probe the pharmacologic and pharmacodynamic performance of new treatments. The probability that new cancer drugs tested in Phase 1 will reach regulatory approval is reported to be around 3–7%. No estimates exist for oncology agents targeting pediatric patients during Phase 1 trials. Children are a unique group of participants and are considered vulnerable. Pediatric trials are evaluated with enhanced caution and must adhere to stricter standards than their counterparts with adult participants. Most regulations allow only minimal risk or require the prospect of direct benefit for participants.

At the same time all drug development trials must fulfill social value requirement. For example, Council for International Organizations of Medical Sciences (CIOMS) guidelines requires social and scientific value for all studies. It states that studies have to be “scientifically sound, build on an adequate prior knowledge base and (…) likely to generate valuable information”. Since (beside identifying toxicities) the goal of Phase 1 studies is to develop clinically useful treatments, one key proxy for measuring social value is the extent to which treatments tested in Phase 1 trials advance to later phases and/or regulatory approval. A recent meta-analysis of Phase 1 trials in pediatric oncology showed that they provide limited direct benefit for participants. On average every participant experienced at least one serious adverse event that is associated with treatment, and 1 in 50 died from such an event. Therefore the justification of risk rests heavily on demonstrating social value.

The social value of pediatric Phase 1 trials has not, to our knowledge, been subject to systematic analysis. In what follows we assess clinical development success rates and other proxies of social value for a sample of pediatric Phase 1 trials in oncology to examine how frequently such trials influence clinical development.