PhD defence T.B. (Tobias) Polak

Below is a brief summary of the dissertation:

Expanded access is a pathway to access unregistered medicines if there are no registered treatments available and patients cannot enroll in clinical trials. For patients who currently are in dire need of treatment options and cannot await drug development processes, or for patient who may benefit from treatments that are not (or not anymore) registered in their jurisdiction, expanded access may serve as a last resort. Unregistered medicine can be acquired via named-patient pathways (‘Leveren op Artsenverklaring’) or via group programs (‘Compassione Use Programma’s). We describe the origins of expanded access and its daily practice in the Netherlands. We observe an increasing trend in expanded access requests. Expanded access enables physicians to facilitate access for patients with unmet medical needs. The potential risks these treatments provide, the option to cease further treatment and the preferences of individual patients should all inform the decision whether or not to pursue expanded access.