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Alternative access schemes for pharmaceuticals in Europe: Towards an emerging typology.

Accepted version
Peer-reviewed

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Type

Article

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Authors

Löblová, Olga 
Csanádi, Marcell 
King, Lawrence 

Abstract

European governments employ sophisticated health technology assessment and regulatory procedures to identify which pharmaceuticals to fund publicly. However, there are persisting demands from patients for those drugs excluded from positive reimbursement lists, leading to the emergence of what are here termed "alternative access schemes". This paper presents a purposive review of these schemes based on available scholarly and grey literature, illustrated with real-world examples from recent practice. It puts forward an original typology of alternative access schemes based on their marketing authorization (regulation) and reimbursement (redistribution) status. We describe the complex, multidimensional policy trade-offs between the principles of patient freedom of choice, clinical autonomy, encouragement of innovation, evidence-informed decisions on safety and quality, access to treatment, and financial sustainability, involved in marketing authorization and reimbursement decisions. We discuss the ways in which alternative access schemes differ and conclude that our typology can illuminate salient policy dilemmas raised by alternative access schemes in national drug reimbursement systems.

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Keywords

Early access, Off-label, Oncology, Pharmaceuticals, Pricing and reimbursement, Rare diseases, Compassionate Use Trials, Cost-Benefit Analysis, Europe, Humans, Legislation, Drug, Marketing, Off-Label Use, Pharmaceutical Preparations, Rare Diseases, Reimbursement Mechanisms, Technology Assessment, Biomedical

Journal Title

Health Policy

Conference Name

Journal ISSN

0168-8510
1872-6054

Volume Title

123

Publisher

Elsevier BV