Tobias Polak

Introduction: Background and outline of this thesis 11 formalize expanded access pathways in 1987, offering regulated access to experimental drugs to thousands of patients facing a life-threatening illness with no other recourse.12 Similar pathways have since been established around the globe to offer treatment options to patients in need. Over the years, there has been an increasing interest in expanded access, with various factors potentially causing its rise.13 External factors, such as improved understanding of the underlying biological mechanism of diseases through research have resulted in an increase in trials investigating potential treatments, in turn leading increased expanded access requests. Likewise, the rise of internet and social media has heightened awareness among patients and physicians of medicine in development.14 Expanded access itself has also evolved. Over time, familiarity with expanded access has increased, in part due to efforts such as the US ‘Project Facilitate’ which aims to educate patients, physicians, and industry on expanded access, and intends to improve efficiency in requesting expanded access.15,16 Companies must publicly post their expanded access policies online,17,18 and international research databases (e.g., clinicaltrials.gov) offer the ability to register expanded access programs. Before further examining expanded access and research conducted in tandem with it, we first need a clear and universal definition of expanded access. Finding common ground: what is expanded access? There is no globally accepted definition of expanded access. Even more, there is no consensus on the term expanded access itself.19 In English alone, expanded access is known as ‘named-patient use’, ‘compassionate use’, or as ‘managed’, ‘early’ or ‘special’ access, all referring to the provision of unlicensed medicine outside of a trial setting. Non-English speaking countries have implemented local equivalents. For example, the ‘authorisation temporaire d’utilisation’ or ‘accès précoce’ in France,20 the ‘Levering op Artsenverklaring’ in the Netherlands,21 the ‘Heilversuch’ in Austria,22 and ‘el uso compasivo’ in Spain,23 are all analogous to expanded access. Despite these variations, there are common theoretical principles that define expanded access.24 Said access pathways are open to patients who are 1. diagnosed with a seriously debilitating or deadly disease; 2. unable to benefit from registered treatment options; 3. disqualified to participate in clinical trials. The practical process of obtaining access typically involves obtaining informed consent from the patient, a prescription from the treating physician, involvement of a local ethics committee, cooperation from the product manufacturer, and approval from the local regulator.25,26 These

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