Randomized controlled trials remain the gold standards for generating new novel medical evidence. To be meaningful the evidence generated must be generalizable to the real-world population. The generalisability of a trial is partially determined by its population representativeness, which measures the coverage of the trial participants within the real-world patient population. This form of representativeness – referred to as aposteriori representativeness, has been evaluated for multiple trials (both individually and collectively) involving other patients. However, such analyses for paediatric trials are very limited.
Assessing the population representativeness for paediatric trials was selected as a use case for the Global Paediatric Data Forum (referred to as GLOPAD). The GLOPAD was formed following a conect4children (c4c) workshop on ‘Increasing the Harmonization and Standardization of Disease-Specific Paediatric Trial Data’, held in Rome in May 2022. The GLOPAD aims to (1) bring together key stakeholders in the domain of disease-related terminology and data interoperability, and (2) raise awareness of the need to avoid siloing data and to optimally use and reuse data from vulnerable populations. The GLOPAD meets quarterly to discuss recent advances and possibility of collaboration between members to avoid duplication of effort. Members include research organizations, large initiatives, individuals, projects, and industrial partners working with paediatric data (particularly real-world data, rare disease, implementation of FAIR principles and data standardization).
Smaller working groups within GLOPAD focus on different areas of expertise – one of which is population representativeness in paediatric clinical trials.
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