Ethical Crossroads: Expanded Access, Patient Advocacy, and the #SaveJosh Social Media Campaign

Abstract

Expanded access programs raise complex ethical dilemmas regarding the use of experimental medicines to treat life-threatening medical conditions – issues for which there are no simple, monolithic solutions. Beyond the risks to an individual, how does society or a company balance the immediate needs of a critically ill individual versus the potential needs of many future patients? This article offers insights into and learning experiences from the case of a 7-year-old boy whose family sought access to an experimental antiviral medicine being developed by Chimerix, where the author was Chief Executive Officer. The high-profile #SaveJosh social media campaign helped to catalyze and crystalize the international debate on issues of ethics and equity in expanded access, raising questions regarding the role of patient advocacy and the impact of social media on healthcare and the biopharmaceutical industry. Additionally, the #SaveJosh campaign demonstrated how easily thoughtful dialogue can be overwhelmed by a hyper-immediacy that increases the intensity and scrutiny under which these issues must be addressed. Given that the decision to grant an expanded access request lies solely with the leadership of the company developing the experimental medicine, management must evaluate and balance a request against what is known about the safety and efficacy of the compound, where it is in its testing pathway, and any other complexities or risks identified during the development process. Furthermore, companies must craft and be prepared to explain their rationale, including the right not to make an experimental medicine available, to regulators, legislators, patient advocates, and patients in need.