OPINION: Personalised Medicine, Ageing and Rare Diseases

It’s time to validate new statistical approaches for clinical trials if we want to maintain the evidence-based medicine as cornerstone of our drug safety and efficacy evaluation.

The progress of genetic screening has led to the detailed characterization of individuals and it is cancelling the concept of “large population” leading to increasingly smaller groups of individuals based on shared genetic biomarkers.

Similarly, ageing restricts the overall population to small groups afflicted by concomitant pathologies. In fact, the Rare Disease paradigm, of a small patient population, is becoming a very common one.

As matter of fact, the H2020 grand challenges on Personalized Medicine, Rare Disease and Ageing can be said to be all focused on finding a cure for small groups of individuals defined by genetic and/or the concomitant presence of multiple pathologies.

Such a trend makes the need to validate new statistical approaches for clinical trials very urgent if we want to maintain the evidence-based medicine as cornerstone of our drug safety and efficacy evaluation. The Adaptive approach to Clinical Trials is a small step in this direction but more Private-Public Partnership initiatives will be needed to reach a common agreement in a reasonable time.

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