Who Should Get 'Compassionate Use' Drugs?

Arthur Caplan, PhD


May 13, 2015

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I'm Art Caplan, at the Division of Medical Ethics at the New York University (NYU) Langone Medical Center. Compassionate use is something you may have heard of. It refers to situations in which patients are trying to get access to drugs that haven't yet been approved by the US Food and Drug Administration (FDA).

Sometimes those drugs are very early in the development process, tested in animals (and maybe in a few human subjects), and desperately ill people would like to get access to those drugs. They say, "I don't have time to be in a clinical trial. I don't have time to wait for approval. I need it now. I would like to try."

Other situations occur when a drug is in short supply. That can happen when a drug is moving through the testing process but a company isn't ready to make a lot of the drug until it has been approved, so some exists but not enough to meet the demands of everybody who might want to get the drug. There just isn't much of it around.

Recently, Johnson & Johnson reached out to me and said, "We have some drugs that are coming. We think that they are going to be very successful. They are hard to make. What would you advise us about what to do in terms of compassionate-use requests? We know that we are going to get them."

I thought about it and I said, "It is time to try a more systematic approach to compassionate use." Right now, the way people might go out and seek these drugs is to go to their doctors and say, "Can you help me?" Some doctors know that they should turn to the FDA, so they look on their website to see how to apply, and that is useful. Some doctors actually may say to the patient, "This particular drug isn't the right one for you," and that is great, too.

However, presuming that the drug might be beneficial usually falls to the manufacturer, who must decide what to do with the available supply. What we have seen so far is people mounting social media campaigns, or calling their friends who might be connected to a senator or some politician who could intervene with a manufacturer to try to get the drug. I certainly understand why people do that, and I might try to do it myself if I were dying or if my child were very sick and I thought there was something that might help. But that is not a system. It basically rewards the squeakiest wheel, and if you are using social media campaigns or trying to get the local media to cover your situation and help you get access to something, it doesn't stay news for a long time. The media lose interest and people get tired of seeing these appeals on social media.

A better and more fair way to do it is to create a committee. Try to remove the company from the decision and have a group of doctors, bioethicists, and consumer advocates who would weigh in and say, "We are independent; this seems like the fairest approach to us." That is what I recommended to Johnson & Johnson—that we create a pool of 10 people (medical experts, bioethicists, and people who have backgrounds in consumer advocacy), and we say to Johnson & Johnson, "You look at requests that come in from doctors and patients. You tell us whether they are appropriate; whether these drugs might, by any stretch of the imagination, be helpful for these individuals. You make those requests anonymous and send them to the committee. We won't know who they are. Three members of the committee will serve on a panel every week or two to look at the requests and decide what is the best and fairest way to allocate the small supply of drug that is available."

The reason that the system becomes fair is that all requests are anonymous, so it doesn't matter whether you are rich or poor, or American or not American. All that matters is your medical need. No one will know who is going to make the decision, because the pool of three people who will decide in any given week will rotate, so it is hard to pressure or lobby them. They will have the kind of independence and hopefully the breadth of experience that will bring greater wisdom to decisions about what to do when faced with scarcity.

These problems come up a lot. They came up with experimental drugs for Ebola, and people had to decide whether they should be used. This issue will come up for other companies who have scarce supplies of novel drugs. Hopefully, this system, if it works, might be extended throughout Johnson & Johnson and to other companies and even international organizations.

Keep an eye on how this goes. I will be interested in criticism, feedback, and ideas that might come up in terms of how to make this work even better, but I think having a systematic approach to tough decision-making when you are faced with compassionate-use requests is the way to go. We shall see.

I'm Art Caplan, at the NYU Langone Medical Center's Division of Medical Ethics. Thanks for watching.

The opinions expressed herein are those of the author and do not represent the views of Medscape.


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