Debating Compassionate Access To Experimental Drugs For The Terminally Ill

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An atrium at the headquarters of Johnson & Johnson in New Brunswick, N.J. (Mel Evans/AP)
An atrium at the headquarters of Johnson & Johnson in New Brunswick, N.J. (Mel Evans/AP)

There is a wrenching debate that affects people who are desperately ill and running out of options. At issue: should drug companies give them access to experiment drugs? Treatments that have not been fully tested, but that just might save their lives or grant them a few more years of life?

If you or a loved one is in this position, the answer is easy: yes. But the reality is not so simple. Drug makers often have only a limited supply of these drugs, which leads to difficult decisions over who gets them and who does not. Also, the drugs might not work — they could even cause harm.

In response to this, Johnson & Johnson has become the first drug company working to set up a panel of doctors and bio-ethicists to decide how to respond to requests for so called "compassionate use" of experimental drugs.


George Annas, chair of the Department of Health Law, Bioethics & Human Rights of Boston University's School of Public Health, and a professor in the Boston University School of Medicine and School of Law.

Darshak Sanghavi, chief of pediatric cardiology at UMass Medical School and an associate professor of pediatrics, as well as a former managing director of the Engelberg Center for Health Care Reform at the Brookings Institution.


The Wall Street Journal: J&J Changes ‘Compassionate’ Care

  • "Johnson & Johnson has arranged for an independent panel to review requests from seriously ill patients who want to try an unapproved drug even if they aren’t participating in the drug’s testing."

The New York Times: The Pills Of Last Resort

  • "As a public-health advocate, I know that if we simply let people have access to untested medicines without those trials, we will never learn which ones are effective and how best to use them. But to a physician coming face to face with frightened and desperate patients in the clinic, the case for the greater good seems less compelling. After all, the promising drug may be the patient’s last and only chance. Every now and then, desperation leads to success."

Brookings Institution: Four A's of Expanding Access to Life-Saving Treatments and Regulatory Implications

  • "The most efficient and equitable way to make new effective treatments to the largest number of needy patients is regulatory approval, accelerated or otherwise, following successful demonstration of efficacy and safety for a given indication in a specific population. Until that process is complete, access to an experimental therapy is by definition an additional risk, as the agreed necessary safety and efficacy have not yet been demonstrated."

This segment aired on May 14, 2015.


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