ObjectiveWe sought to determine the characteristics of “expanded access” and “compassionate use” programs registered in ClinicalTrials.gov and to determine the percentage of drugs provided through these programs that ultimately received FDA marketing approval.ResultsWe identified 398 expanded access and compassionate use programs (hereafter referred to as expanded access programs) registered on ClinicalTrials.gov. Industry funded 61% (n = 241) of programs individually or collaboratively, while NIH and the US Federal Government rarely funded programs (3% [n = 11] and 2% [n = 6], respectively). Most programs provided access to drugs (71% [n = 282]), 11% to biologics (n = 43), and 10% to medical devices (n = 40). These programs covered 460 unique conditions, the most common being HIV (n = 26), leukemia (22), and multiple myeloma (n = 14). Only 2% of programs reported results in ClinicalTrials.gov. Most programs (82%) were open to enrolling adults and seniors (n = 326). These programs provided access to 210 unique experimental drugs, of which 76% have received FDA approval.

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Increasingly, the objectives and clinical trial practices of companies pursing the development of new medicines are intersecting and conflicting with the interests and activities of patient advocates and patients who are the potential beneficiaries of these experimental medicines-perhaps nowhere more clearly than in the issue of expanded access, often referred to as compassionate use (4, 5). This intersection sets the lengthy and complex process of developing new medicines for future patients in conflict with the immediate needs of current patients. For me and for others in leadership positions in biotechnology companies, the questions raised by this conflict can be trajectory altering for our experimental medicines: Whose life should we focus on saving? How should these decisions be made? Who should make these decisions? In the era of social media, where people can express their opinions and interact with others in real-time, the ethical issues created by these situations are complicated by a hyper-immediacy that increases the intensity and scrutiny under which these issues must be addressed. Some will argue that the moment that you can save a life, no matter where your experimental medicine is in its development pathway, you must save that specific life. Others will argue that you must do whatever you can to secure regulatory approval for the new medicine in order to save the largest number of people. This article is based on my experiences as the Chief Executive Officer (CEO) of a small biotechnology company, Chimerix,

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