How can we improve patient access to experimental drugs? – WSJ


New approaches are needed for terminally-ill patients

In this article published in the Wall Street Journal by J. D. Rockoff a very important issue is raised: how can we give some hope to terminally-ill patients that could benefit from unregistered experimental drugs?

Johnson & Johnson has found a way with the set-up of an independent panel which will review requests from seriously ill patients. A committee of doctors, bioethicists and patient representatives will be organized and managed by the New York University School of Medicine in order to give a quick and relevant answer to all these requests.

The current law prohibits companies to give out unapproved drugs but the FDA has a “compassionate use” exception where companies can give medicines to patients with serious conditions and who are not participating in clinical trials. Over 1’800 requests reached the FDA in 2014.


From the article: “Amrit Ray, chief medical officer of J&J’s Janssen pharmaceuticals unit, said “the setup should be easier for patients because the company is establishing a single website and toll-free hotline for requests, rather than the current patchwork of entry points across the company and its drug trials”. Dr. Ray expects the committee could issue recommendations within days for urgent requests.”

WSJ Article


More on compassionate use and programs:

FDA Expanded Access Program

Compassionate use of medicinal products in Europe: current status and perspectives

Compassionate use of medicines in the European Union

Navigating the landscape of compassionate use (how to manage expanded access from an industry point of view)

Expanded access programmes: patient interests versus clinical trial integrity – 2015 – The Lancet Oncology (subscription required)

From 100 Hours to 1: FDA Dramatically Simplifies its Compassionate Use Process – 2015 – RAPS


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