Abstract
Seriously ill patients who have exhausted all approved treatment regimens and who cannot be enrolled in clinical trials may resort to expanded access programmes in order to gain access to unapproved, investigational drugs. It seems that in some countries, expanded access to investigational drugs is offered in clinical practice on a more routine basis than in other countries. This study is the first to investigate the experiences of physicians with expanded access to investigational drugs in different healthcare systems, with a focus on factors that facilitate or hinder expanded access. Semi-structured interviews (n = 36) were carried out with medical specialists in the Netherlands (n = 14), Turkey (n = 9) and the United States of America (n = 13), and analysed thematically. This study identifies five sets of factors pointed out by physicians that determine the degree to which expanded access to investigational drugs is deemed feasible in clinical practice: the suitability of investigational treatments, the application process, hospital policies, support by pharmaceutical companies, and funding and reimbursement arrangements. Based on the interviews conducted, we conclude that, while legally allowed and technically possible, expanded access is not always feasible for—and not always considered an option by—treating physicians. This is mainly due to lack of familiarity with expanded access, the extensive time and effort required for the application process, willingness or ability of pharmaceutical companies to supply the drugs, and funding issues.
Highlights
After exhausting all approved medical treatments for patients who are suffering from serious or life-threatening diseases and ineligible for clinical trials, physicians may— under strict conditions—resort to expanded access to unapproved, investigational drugs: the process of prescribing investigational drugs that are not registered by regulatory authorities, outside of the context of clinical trials (Falit and Gross, 2008; Darrow et al, 2015; Caplan et al, 2019), as a final therapeutic option
Little is known about the factors affecting uptake from an international perspective or from the perspective of treating physicians engaging in expanded access to investigational drugs
In order to capture a wide range of experiences with expanded access to investigational drugs in this interview study, we recruited physicians working in different countries, different types of hospitals, and with different medical specialties, who treat patients with unmet medical needs
Summary
After exhausting all approved medical treatments for patients who are suffering from serious or life-threatening diseases and ineligible for clinical trials, physicians may— under strict conditions—resort to expanded access to unapproved, investigational drugs: the process of prescribing investigational drugs that are not (yet) registered by regulatory authorities, outside of the context of clinical trials (Falit and Gross, 2008; Darrow et al, 2015; Caplan et al, 2019), as a final therapeutic option. In most countries, expanded access can only be granted when patients satisfy three criteria: (a) they must be suffering from serious or life-threatening diseases, (b) they must have exhausted all applicable approved alternatives and (c) they must not be eligible for clinical trials (either because there are none in which they can reasonably or logistically participate, or because they do not meet the in- or exclusion criteria). What are the factors that hinder or facilitate this process? How do doctors decide whether or not to pursue expanded access to investigational drugs for their patients?
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