Vol 6, No 3 (2015)
Review paper
Published online: 2015-12-29

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Drugs for treating patients with chronic lymphocytic leukemia with FDA granted ‘Breakthrough Therapy Status’ — implications for patients

Rafał Zyśk, Dariusz Wołowiec
DOI: 10.5603/Hem.2015.0036
Hematologia 2015;6(3):233-244.


The added value of a new drug can be defined in various ways, but the patient-focused approach obliges the clinical significance of additional health effects to be also considered. For any given new drug, the American Food and Drug Administration (FDA) determines the added value delivered to the current therapy standard by conducting an assessment based on: the extent of the delivered health effect regarding clinically significant endpoints, the duration of that effect and the importance of the clinical effect for treating a serious disease. In principle, preliminary scientific evidence for a new and original drug should clearly indicate an additional benefit compared to currently available therapeutic methods. In order to make such drugs available as quickly as possible to patients, the FDA has implemented programmes since the 1970s aimed at shortening development and approval processes of methods with therapeutic promise, particularly in treating patients suffering from serious diseases. The most recent programme of this type introduced by the FDA, grants an innovative drug the special status of a breakthrough therapy, from which the drug acquires numerous privileges. In the last ten to twenty months the FDA granted several molecules this status, thus significantly shortening the patient waiting time for drug marketing. In the FDA’s opinion, such drugs offer breakthrough progress in treating patients with chronic lymphocytic leukemia.

Hematology in Clinical Practice