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Navigating Compassionate Use: From Patients to Policies and Beyond

Two publications from the New York Academy of Sciences examine pre-approval access to investigational drugs from a range of stakeholders and perspectives.

Published June 09, 2016

Navigating Compassionate Use: From Patients to Policies and Beyond

The Food and Drug Administration (FDA) has granted the use of investigational drugs outside of clinical trials for decades, but in the past several years this practice has attracted significant attention in the news and on social media. Under expanded access (also called compassionate use), patients who suffer from serious or immediately life-threatening diseases for whom no comparable or satisfactory alternative therapy is available can access drugs and medical devices that are not approved by the FDA and are currently being tested in clinical trials. A total of 29 states have also passed "right-to-try" laws allowing terminally ill patients to access experimental therapies, but there are many questions about the safety and efficacy of such treatments that are not FDA-approved.

Recently the FDA announced significant changes to shorten and simplify the application process used by physicians to request expanded access to investigational drugs for individual patients. Some are predicting that the FDA may not approve a drug to treat Duchenne muscular dystrophy that is currently under review, but will allow compassionate use while additional studies are conducted. Regardless of the FDA decision, the debate over compassionate use will continue and evolve as additional treatments are brought to the forefront of experimental medicine and research.


Bioethics Meets R&D: The Ethics of Pre-approval Access

June 2, 2016
Patients with life-threatening illnesses face challenges in accessing potential therapies at the cutting-edge of R&D which have not yet been proven in a clinical trial. This podcast will explore the provocative and emotional stories of patients, family members, advocates, researchers, physicians, and the regulators charged with keeping medicines in the marketplace safe and effective.


Pre-Approval Access: Can Compassion, Business, and Medicine Coexist?

January 25, 2016
This eBriefing from the conference Pre-Approval Access: Can Compassion, Business, and Medicine Coexist? explores the issue of pre-approval access to investigational medicines, in which the prospective benefits may outweigh the potential risks for critically ill patients. Representatives of governments, pharmaceutical companies, patient groups, NGOs, and foundations discussed ethical and safety challenges for pre-approval access and compassionate use programs in a series of debates. While the path going forward with compassionate use is still unclear, there is a consensus from all stakeholders on a need for change.