Facilitating Expanded Access to Investigational Medicines: Possibilities, Obligations, and Ethical Questions

FDA’s Expanded Access (EA) pathway, and various international analogues, allow patients with serious conditions to access investigational medicines for treatment use outside clinical trials. This approach offers the possibility of hope when patients lack alternatives but it also raises practical and ethical challenges, including issues related to awareness of the pathway and relevant investigational options, fairness in who is granted access, comprehension of the nature and limits of investigational interventions, and blurred lines between clinical care and research. In this panel, we will address these challenges from several angles. The first panelist will consider whether physicians have an ethical obligation to disclose and offer EA to eligible patients, describing when and why this obligation might exist, what it might entail, and whether it could be supported as a legal requirement. The second panelist will present empirical findings from interviews with oncologists practicing in academic medical centers exploring how they make decisions about offering EA, how they navigate the pathway across interactions with companies, FDA, and IRBs, and how they perceive the utility of data collected from EA patients, leading to ethical consideration of what resources should be devoted to supporting EA. Finally, the third panelist will address the growing trend of companies, regulators, and payers relying on – and sometimes soliciting – data from EA programs, discussing ethical issues related to data quality, payment, transparency, and IRB oversight, as well as whether these approaches have the potential to improve patient access.