The Ethics of Patient and Public Engagement in Drug Development, Approval, and Access

Abstract: Patients have long sought to have their humanity recognized in drug development, approval, and access decisions, demanding “nothing about us, without us.” Patient engagement has become an ethical expectation but also raises important challenges. How much influence should patient voices carry, particularly when their preferences conflict with public health goals? For example, patients sometimes advocate for weaker standards for FDA approval, expressing willingness to accept uncertainty in favor of speedier access to therapies. In other cases, patients advocate for payer coverage of high-priced drugs despite limited evidence of benefit. Further, current approaches to including “the patient voice” risk eschewing heterogenous patient perspectives. In this session, experts in political science, anthropology, law, and bioethics will bring their diverse perspectives to bear on these questions, alongside lived experience in patient advocacy. The moderator will provide opening remarks describing foundational issues, followed by discussion of unique tensions arising in rare disease patient advocacy around research and drug development. Next, we will discuss new empirical data examining patient engagement in FDA drug approval decisions, including involvement in advisory committees as voting members and as contributors during public comment periods. Finally, we will discuss patient engagement with payers, sharing qualitative analysis of 10,000+ comments submitted by patients, families, and the public to the Centers for Medicare and Medicaid Services in response to its proposal to limit coverage for new Alzheimer’s drugs. Across each of these domains, we will consider pros and cons of patient engagement and strategies for balancing relevant ethical considerations.