title: The changing landscape of expanded access to investigational drugs for patients with unmet medical needs: ethical implications.
abstract: When patients are told that standard medical treatment options have been exhausted, their treating physicians may start looking for promising new drugs that are not yet approved, and still under investigation. Some patients can be included in clinical trials, but others cannot. It is not widely known that these patients might still be eligible for trying investigational drugs, in a therapeutic context. Worldwide, public and private parties are seeking to change this by informing patients and physicians about opportunities for expanded access and/or by facilitating its processes. When expanded access becomes available to larger groups of patients, ethical issues gain prominence, including informed consent, funding issues, disparities in access, and potential adverse effects on clinical drug development. Physicians, patients and policy-makers should not shift the responsibility to address these issues to pharmaceutical companies, but work together to resolve them.
authors.affiliation: Department of Medical Ethics and Philosophy of Medicine, Erasmus MC, University Medical Centre Rotterdam, Wytemaweg 80, 3015 CN Rotterdam, The Netherlands.
authors.forename: Eline M
authors.initials: EM
authors.lastname: Bunnik
authors.affiliation: Department of Medical Ethics and Philosophy of Medicine, Erasmus MC, University Medical Centre Rotterdam, Wytemaweg 80, 3015 CN Rotterdam, The Netherlands.
authors.forename: Nikkie
authors.initials: N
authors.lastname: Aarts
authors.affiliation: Department of Medical Ethics and Philosophy of Medicine, Erasmus MC, University Medical Centre Rotterdam, Wytemaweg 80, 3015 CN Rotterdam, The Netherlands.|Amsterdam Medical Centre (AMC), Department of General Practice, Meibergdreef 9, 1105 AZ Amsterdam, The Netherlands.
authors.forename: Suzanne
authors.initials: S
authors.lastname: van de Vathorst
keywords: Compassionate use; Ethical issues; Expanded access; Investigational drugs