No Statistics – No Precision Medicine

A screening program tailored to my risk profile. Only receiving a treatment from which I will benefit. That is what we all want. Can big data help to achieve this goal? Yes it can. The reason is that big data allow identification of homogeneous patient groups. Within a group, patients will benefit from a specific treatment. Across groups, patients probably need different treatments. Within the research domain, studies are often underpowered. The problem is that underpowered studies yield inconclusive results. What is the reason for this? Studies aim to identify biological mechanisms or to assess the effect of treatment. When effect sizes are a priori overestimated the number of subjects will be too small. Can big data help here? Yes: Information from data resources improves study design because effect sizes can be better guesstimated. Is the Health Sector ready for big data? Recently a report was published in The Netherlands where several challenges were mentioned. These need to be addressed to enable big data applications in health care: 1) new technology for collecting, sharing, analyzing and storing data 2) standardization of data 3) access to the data and 4) privacy. No doubt these innovations are important and necessary. But how do we come from data to risk profiles? We use quantitative methods. Specifically machine learning and statistical tools. Apparently, it is assumed that innovations which are required to scale...

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