Our summary
Real-world evidence can offer new research paradigms for the small cohorts common in precision medicine where dramatic responses to targeted therapy can be observed in very specific, biologically-informed groups of patients. In these settings, traditional clinical trials are challenging due to slow enrollment timelines and ethical considerations. Thus, even small real-world cohorts of these rare patients are valuable sources of insight that can inform regulatory and development decisions, as well as personalized care.
However, the size of small RWE datasets require special attention to the analysis and interpretation of the data. Using an example from oncology, the authors describe quantitative and qualitative methodological and analytical tenets by which small-cohort RWE can contribute mightily to precision medicine research and treatment.
Why this matters
Generating meaningful clinical data in rare diseases has been a long-standing challenge, given the difficulties in conducting prospective research within these populations. This article discusses the deployment of RWE to tackle this challenge, specifically in the realm of oncology precision medicine, where new evidence generation paradigms may be needed to deliver therapeutic advances to patients with rare genetically defined diseases.