04 November 2016
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Federa award: Small clinical studies for huge breakthroughs in cancer research and personalized medicine

Federa Award to David J. Stewart and Razelle Kurzrock


Stewart and Kurzrock’s idea radically broke with traditional reasoning in medicine that led to slow tedious and too limited progress of anticancer treatment.

Professors Stewart (University of Ottawa, Canada) and Kurzrock (UCSD Moores Cancer Center, San Diego, California, USA) proposed a radical change of strategy for anticancer research in 2009.
Clinical trials should aim to find large differences in small groups of patients, and not small differences in large groups. The success of medicines is not measured in statistical significance but in the clinical relevant benefits for the treated patients.

Statistical significant or clinical relevant

Stewart and Kurzrock showed that many so-called successful therapies prolonged survival only for mere weeks, but were hailed for being statistically significant. They also showed that breakthrough medicines did have clear benefits in hard data, such as progression- or disease-free survival for at least 6 months. Clinical studies aiming for hard and large therapeutic differences in the results do not need to be randomized controlled, since placebo effects are never large in hard outcomes.

Simpler testing of more medicines

Stewart and Kurzrock argued that clinical research should focus at treatments that offer relevant differences in survival for the patients. If therapy has a large survival benefit for patients with a poor prognosis, pharmaceutical testing does not need inappropriately high safety testing – such testing would only slow progress. Reducing size and requirements of clinical studies allows the testing of many different agents in small numbers of patients, for example grouped by biomarkers (personal medicine). The bold change of strategy was supported by research into the fate of FDA-approved oncology drugs for which no large randomized controlled trials have been done.

Stewart and Kurzrock’s idea radically broke with traditional reasoning in medicine that led to slow tedious and too limited progress of anticancer treatment. Their concept has opened the way to personalized medicine.

"Four Federa award laureates were awarded with the Nobel Prize, 1 to 25 years later."

This year the prestigious Federa Award will be given for the 40th time to scientists. Four Federa award laureates were awarded with the Nobel Prize, 1 to 25 years later. In lieu of the Federa Award Prof Stewart will give a lecture at the FederaDag, November 11th at the Radboud UMC, Nijmegen, The Netherlands.

Key publication:DJ Stewart, R Kurzrock. 2009 Cancer The Road to Amiens. J. Clin. Oncol. 27:329-333