Breast cancer, the patient is the real one (and not the ideal one of the studies)

Anyone who has participated in a clinical trial knows it: there are criteria for joining a trial. The reason is simple: to establish the efficacy and safety of a new treatment, it is necessary to compare the data of groups of patients as uniform as possible, who do not present other pathologies or too many “confounding factors”, because they could alter the result. In a word, they are groups of “selected” patients placed in ideal “protected” situations.

These are still real patients, of course, but this selection means that the patient populations on which a new drug is tested – as well as a surgical or radiation treatment – do not exactly correspond to those found in any oncology department. One example among all: elderly or frail patients, who are most affected by oncological diseases, including breast cancer, are often excluded.

The limitations of clinical trials

“Many drugs are approved based on randomized trials where patient selection is very rigorous and not always representative of the full spectrum of clinical presentations the oncologist encounters in real life,” he explains. Joseph CuriglianoFull Professor of Medical Oncology at the University of Milan and Director of the New Drug Development Division for Innovative Therapies, European Institute of Oncology in Milan – So, often, they are not comparable with the patients we see in our clinics and in our daily clinical activity”.

What are reality data

That’s why data from “ideal” patients from clinical trials, which are essential for drug approval, need to be integrated with data from “real” patients. And precisely for this reason there is more and more talk of Real World Data (RWD): i.e. patient data usually collected in electronic medical records, in the databases of healthcare facilities and in pathology registers. Real World Evidence (RWE) data are in turn based on these, i.e. clinical evidence on the use, benefits and risks of a specific drug prescribed no longer within a clinical trial, but in everyday life.

What are they for?

From this point of view, Real World Evidence is part of personalized medicine and can make pharmaceutical spending more efficient, as well as becoming an innovative health governance tool, if used in a standard, rigorous and consistent way. “Real world data is not enough to approve a drug, because a study with a control arm with standard therapy is always needed. But they can be used to complement the scientific evidence,” he explains Alessandra Gennari, Full Professor of Oncology at the Eastern Piedmont University and Director of the University Structure of Medical Oncology at the Maggiore Hospital of Novara. This applies to efficacy, which may be slightly different, but above all to the safety profile of a given drug. Only by observing what happens in clinical practice can we realize what the real tolerability of a drug is, and also observe the appearance of adverse events, which are rarer in randomized clinical trials.

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The real world study in breast cancer

A recent example of the use of real-world data in oncology, and specifically in the metastatic breast cancer setting, is the P-REALITY-X study, which investigated the clinical use of first-line combination therapy with palbociclib ( an inhibitor of CDK cyclin-dependent kinases 4 and 6) and with an aromatase inhibitor. “The study evaluated the real efficacy of palbociclib and demonstrated an advantage, with an extension of median survival from 43 months to 57 months,” concludes Curigliano. Real world data, that is, confirmed what had emerged in clinical studies, and give doctors additional indications on how to manage therapies. Especially when you have several available, as in the case of breast cancer.

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