N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine

Authors: Kyr, Svobodnik, Stepanova & Hejnova (2021)

Clinical trials in paediatric oncology are often focused on rare diseases. However, traditional clinical trials (e.g., randomised controlled trials) in the context of paediatric oncology can be challenging. N-of-1 designs have received attention about how they can fit into new paradigms of modern medicine. It is important to understand whether N-of-1 designs can be applied in the field of paediatric oncology and if so, how. In this review paper, Kyr et al. outline the limitations of traditional clinical trials in paediatric personalised oncology; analytical approaches for small populations and their applicability in paediatric oncology; N-of-1 trials in paediatric oncology; and provide a comprehensive table of N-of-1 trial assumptions and how they relate to paediatric oncology. Two applications for N-of-1 trials in paediatric oncology are identified. One is the use of aggregated N-of-1 trials where repeated measures methods are not possible (e.g., where patients have aggressive disease) and the other is employing N-of-1 designs to generate evidence for regulatory approval of new treatments. The authors argue for reshaping the evidence pyramid for rare diseases and personalised medicine, promoting the idea that treatments and evidence sources should be evaluated individually.

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Full citation: Kyr, M., Svobodnik, A., Stepanova, R., & Hejnova, R. (2021). N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review. Cancers, 13, 5428.