Introduction: Background and outline of this thesis 13 Figure 1: Relative number of expanded access requests over a product’s life cycle. The strict common definition of expanded access would be ‘access to a medicine that is unregistered for any indication within its jurisdiction’. What is typically not considered expanded access? It is important to differentiate between expanded access and other types of unconventional product usage. Off-label usage is not the same as expanded access, as the former occurs when a label is approved in a different indication, and the latter is only possible when the product lacks any regulatory approval. Furthermore, expanded access programs are strictly speaking not synonymous with programs that aim to bridge the time between regulatory approval and reimbursement. Expanded access usually precedes regulatory approval and is not directly linked to reimbursement status. Expanded access is not limited to pre-approval access, as it can also be used post-withdrawal. As these ‘peri-approval, peri-reimbursement’ situations are simultaneously different and similar, some stakeholders use these terms interchangeably. Understandably, industry frequently employs one ‘managed access’ desk to handle all request from patients seeking access to drugs that are not available, regardless of whether this is due to a lack of registration or funding.
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