Observational Studies and Real World Evidence


Great advances were made in medicine by the simple fact of observing and quantifying phenomena. The year 1662 saw John Graunt, the first mortality tables were proposed by the London merchant. He experiments an epidemiological monitoring system to alert the authorities about the start of plague epidemics. In 1834, Pierre Charles Alexandre Louis demonstrates the uselessness of bloodletting in pneumonia with his numerical method. In 1854, John Snow control of the London cholera epidemic by observing the distribution of the city's water and the number of deaths household by household. Observation plays a central role in the progression of medical thought.

Claude Bernard made it one of the bases of its experimental medicine, the observation having to generate hypotheses. Today, the so-called "observational" studies come in addition to clinical studies in the evaluation of the drug. These so-called "real-life" studies provide essential post-marketing information on the description and typology of the populations reached by a product or in the evaluation of the impact of a treatment on morbidity and mortality and its safety of use.


Real-life data (Real World data "RWD") can be defined as any data collected outside of a clinical trial. Although the randomized clinical trial remains the gold standard for evaluating the efficacy of a therapy, its lack of external validity can be overcome by real-life studies. In a randomized clinical trial, the evaluator places himself in the ideal situation of comparing the drug studied to a comparator (a placebo or an active comparator) by minimizing selection, information and confounding biases. The robustness of the results is linked to a high internal validity, at the expense of external validity.

These data can be collected from hospital databases (Electronic Health Records "EHR"), health insurance databases (Système National des Données de Santé " SNDS ") and databases of the Health Data Hub. Other sources of real-life data include data from cohort studies, case-control studies, epidemiological registries or digital data from connected objects and health applications. Their analyses make it possible to identify risk factors, describe care pathways, describe the populations reached by a treatment, evaluate the impact of a treatment on morbidity or mortality, and assess patients' quality of life.

The use of this data for decision-making purposes is the Real World Evidence (RWE), an emerging scientific discipline that is increasingly used to facilitate drug approval and market access decisions.

Post-registration studies, often requested by health authorities, can be treated as observational studies, with real-life data collection.


Kappa Santé masters the various study designs as well as the modeling and statistical techniques specific to this type of study (propensity score, multivariate modelling).

Since its creation, Kappa Santé has conducted more than a hundred observational studies. In this context, we offer the following services:

  • Choice of study design
  • Writing the protocol and CRFs
  • Regulatory procedures
  • Selection of investigators and feasibility study
  • Investigator training
  • Study management and clinical operations
  • Monitoring
  • Data management and biometrics
  • Pharmacovigilance management
  • Report writing and scientific exploitation
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