Clinical trial; Randomisation; Evidence-based medicine; Good Clinical Practice; Personalized medicine
Abstract :
[en] Randomised Controlled Trials (RCTs) represent the cornerstone of Evidence-Based Medicine (EBM). Based upon the rules of Good Clinical Practice (GCP), they offer many strengths but also present some weaknesses. The rigorous methodology used allows avoid bias related to confounding factors (through a control group), selection bias (through randomisation) and interpretation bias (through double blinding). However, patients recruited in clinical trials and study experimental conditions markedly differ from the situation in real life. Furthermore, clinical trials recruit a mix of good and poor responders, so that the average therapeutic response is most often mitigated. Clinical trials must evolve according to the new concepts of personalized medicine to become even more performing. In a near future, they must progress from a statistical analysis on large cohorts of patients to a more individualized analysis guided by patient phenotype and genotype characteristics. [fr] Les essais cliniques contrôlés représentent la pierre
angulaire de la médecine factuelle. Construits selon les règles
des bonnes pratiques cliniques, ils offrent de nombreuses
forces, mais présentent également quelques faiblesses. La
méthodologie rigoureuse utilisée permet, notamment, d’éviter
les biais liés à des facteurs confondants (via un groupe
contrôle), les biais de sélection (via la randomisation) et les
biais d’interprétation (via la technique du double aveugle).
Cependant, les patients recrutés dans les essais cliniques et
les conditions dans lesquelles ceux-ci sont menés diffèrent
fondamentalement de la situation en vie réelle. De plus, les
essais cliniques recrutent un mélange de patients bons et mauvais
répondeurs, ce qui conduit à une réponse thérapeutique
moyenne le plus souvent mitigée. Les essais cliniques devront
s’adapter au développement de la médecine personnalisée
pour devenir encore plus performants. Ils devront, à l’avenir,
passer d’une analyse statistique sur de grands groupes à une
analyse plus individualisée en fonction des caractéristiques
phénotypiques et génotypiques des patients
Disciplines :
Human health sciences: Multidisciplinary, general & others
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