Principles and Challenges in Access to Experimental Medicines

May 26, 2015, Vol 313, No. 20

Principles and Challenges in Access to Experimental Medicines
Michael Rosenblatt, MD; Bruce Kuhlik, JD
Efforts by patients to obtain early access to experimental medicines have increased as novel therapies provide new evidence of their potential to treat or cure life-threatening diseases. As drug discovery efforts, particularly for cancer and orphan diseases, are increasingly based on molecular targets, success rates improve, generating further interest in early access to experimental drugs. Devising “expanded access programs” (EAPs), however, presents challenges.1,2 Fairness and ethical issues need to be addressed as do practical matters, such as efficient conduct of clinical trials, adequate drug supply, finances, and geography. The complexity of crafting EAPs is compounded by early, rapid, and broad communication by traditional and social media. This Viewpoint outlines general principles to help balance the competing interests of individuals facing life-threatening illness with practical concerns and broader societal interests in knowing which drugs do or do not work and making them generally available through expeditious regulatory approval…