Patient requests for access to drugs and biologics prior to their approval have long created a dilemma for biotechnology companies. Typically, these requests come from seriously ill patients or their families or caregivers who believe an experimental product could save or prolong their lives. Biotech companies may be challenged by such requests because, in the first instance, they want to respond positively, but at the same time they must consider the impact of such a response on their ability to complete their development of the product, move it successfully through the regulatory process to receive marketing approval, and thus make it available to the many patients whose unmet medical need the product promises to address.
This issue has become increasingly visible and difficult in recent times, as views about the inherent right of patients in certain situations to unapproved products have been expressed by patients and their advocates, state legislators, and some members of Congress. In addition, the increasing use of social media has brought individual cases more prominently into the public eye and rallied hundreds or thousands of people to the cause of each patient. Clearly, the very public pressure on companies — and on the FDA — to fill the requests can become almost unendurable.
The BIO Bioethics Committee has explored the issues surrounding early access to biotech products. This document represents the Committee's deliberations and provides "Points to Consider" for biotechnology companies confronting these issues. It is the Committee's hope that these Points will help companies analyze many of the challenges, including ethical challenges, raised by expanded access programs.
Read the document (175 KB PDF)