Eline M Bunnik1, Nikkie Aarts2, Suzanne van de Vathorst3. 1. Erasmus MC, University Medical Centre Rotterdam, Department of Medical Ethics and Philosophy of Medicine, Wytemaweg 80, 3015 CN, Rotterdam, The Netherlands. Electronic address: e.bunnik@erasmusmc.nl. 2. Erasmus MC, University Medical Centre Rotterdam, Department of Medical Ethics and Philosophy of Medicine, Wytemaweg 80, 3015 CN, Rotterdam, The Netherlands. Electronic address: n.aarts@erasmusmc.nl. 3. Erasmus MC, University Medical Centre Rotterdam, Department of Medical Ethics and Philosophy of Medicine, Wytemaweg 80, 3015 CN, Rotterdam, The Netherlands. Electronic address: s.vandevathorst@erasmusmc.nl.
Abstract
BACKGROUND: Today, public and private bodies around the world are trying to facilitate and increase expanded access to unapproved, investigational drugs for patients with unmet medical needs. METHODS: This paper discusses three major shifts in the field of expanded access and presents an argumentative account of ethical issues connected with those shifts, based on a literature study and unstructured interviews with 35 stakeholders in the Netherlands. RESULTS AND DISCUSSION: Traditionally, expanded access has been based on three key principles: 1) it is exceptional, 2) it is done 'out of compassion', and 3) it has a therapeutic aim. Current efforts to facilitate expanded access affect these key principles, rendering expanded access a default option, allowing companies to charge for investigational drugs and gather data on its outcomes. These shifts may generate new ethical issues, including false hope, safety concerns and funding issues, which must be anticipated by physicians, pharmaceutical companies, payers and policymakers. CONCLUSION: Healthcare systems allow for the use of promising unapproved drugs in exceptional circumstances, but do not always assist patients with unmet medical needs in getting access. It is time to replace the current patchwork of practices with systems for expanded access in which criteria are clearly described, responsibilities are assigned and arrangements are made, so that patients will know what (not) to expect from expanded access.
BACKGROUND: Today, public and private bodies around the world are trying to facilitate and increase expanded access to unapproved, investigational drugs for patients with unmet medical needs. METHODS: This paper discusses three major shifts in the field of expanded access and presents an argumentative account of ethical issues connected with those shifts, based on a literature study and unstructured interviews with 35 stakeholders in the Netherlands. RESULTS AND DISCUSSION: Traditionally, expanded access has been based on three key principles: 1) it is exceptional, 2) it is done 'out of compassion', and 3) it has a therapeutic aim. Current efforts to facilitate expanded access affect these key principles, rendering expanded access a default option, allowing companies to charge for investigational drugs and gather data on its outcomes. These shifts may generate new ethical issues, including false hope, safety concerns and funding issues, which must be anticipated by physicians, pharmaceutical companies, payers and policymakers. CONCLUSION: Healthcare systems allow for the use of promising unapproved drugs in exceptional circumstances, but do not always assist patients with unmet medical needs in getting access. It is time to replace the current patchwork of practices with systems for expanded access in which criteria are clearly described, responsibilities are assigned and arrangements are made, so that patients will know what (not) to expect from expanded access.
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