Oversight of Right-to-Try and Expanded Access Requests for Off-Trial Access to Investigational Drugs.

For decades, the U.S. Food and Drug Administration (FDA) has provided an "expanded access" pathway that allows patients who meet qualifying conditions to gain access outside a clinical trial to an investigational medical product being tested to see if it is safe and effective for a specific use.

Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials.

Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or 'preapproval', access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time.

Institutional Review Boards as Arbiters of Expanded Access to Unapproved Drugs: Time for a Change?

Institutional review boards (IRBs) are one of the bodies charged with prospectively reviewing compassionate use, the hopefully therapeutic use of an unapproved drug in a seriously ill or dying patient who has no other treatment options. However, there are ethical issues in assigning this role to a body whose primary purpose is to review research proposals. The role of IRBs with regard to compassionate use must be examined and potentially revised.

Safety issues in cell-based intervention trials.

We report on the deliberations of an interdisciplinary group of experts in science, law, and philosophy who convened to discuss novel ethical and policy challenges in stem cell research. In this report we discuss the ethical and policy implications of safety concerns in the transition from basic laboratory research to clinical applications of cell-based therapies derived from stem cells. Although many features of this transition from lab to clinic are common to other therapies, three aspects of stem cell biology pose unique challenges.