Compassionate Drug Use and the FDA

I've done a bit of history research about the FDA and compassionate drug use approval. I've seen the question asked here and seen some interesting responses. I am putting together a few questions for some contacts I have that might receive interesting responses but I wanted to run it by you folks first because you folks always think of things I miss.

I made a list of the questions that I think are key to understanding compassionate drug use, The conversation arose from a friend who is very advanced about why some drugs do get released for compassionate use and some do not. Abiraterone Acetate (Zytiga) is a good example of a drug while on trial received a go ahead to cancel the control arm (placebo) and release the drug for both arms. This was because the evidece was convincing that survival benefits were realized in the incomplete trial. Eventually the trial did reach its end points and we have it regularly in use today. But what about other drugs that have shown survival benefits but were never released by the FDA? That fact is that it's not usually the FDA that stops the release and I found that to be a surprise.

Here is my list of questions about compassionate care use of experimental drugs, for the purpose of these questions assume that the requesting patients are terminally ill and no other drugs are working:

1.Why not release any drug on trial?
2.What drugs do receive compassionate approval?
3.What criteria is used when a drug is reviewed for compassionate use?
4.What's different in the US than what's in Europe, Thailand, China, or elsewhere?
5.What's the role of the drug maker in compassionate use?
6.Why do some drugs that show the drug effectively working in trials not get approval for compassionate use?
7.Does the phase a drug trial is currently in matter?

I will ask these questions to my liaison at SWOG and get his reply. If you have any additional questions what questions would you like to add? I'll include them...

As I said above, I did some basic research and found that the FDA does not actually or fully control compassionate drug use. When the FDA allows a group ~ such as we saw for Abiraterone Acetate when they allowed the control arm to stop the placebo and take the actual investigational drug ~ they actually allowed it not because of compassionate use reasons, but rather to allow the study to continue without the placebo.

Interestingly this topic was a big one in 2001 when the FDA was called to testify before congress because of out cry that the FDA was being too restrictive. The FDA responded in the following testimony was follows (The entire transcript is the link I highlighted the official FDA position as represented in 2001 by quoting the text below):

FDA Testimony to Congress in 2001:
http://www.fda.gov/NewsEvents/Testimony/ucm115209.htm

"We understand that patients and their family members are often unfamiliar with FDA's legal and regulatory responsibilities. Often they are unaware that FDA cannot compel a company to supply an individual patient with an investigational drug outside of its planned clinical trials. The manufacturer or sponsor makes the final decision to provide an experimental drug or therapy to a patient. The sponsor may consider many factors, including the amount of information available about the drug, the amount of drug available, and how best to use its resources to optimize development of the drug for marketing. This maximizes the availability of the drug to patients who can benefit from it. In some cases, the sponsor is unwilling to provide the product outside of clinical trials, especially relatively early in drug development. Patients are sometimes confused or angered by this situation and misinterpret the company's unwillingness to provide the product as an FDA action.

FDA may not allow treatment uses because of safety concerns. Generally, however, if a physician makes a request for treatment use of an experimental drug, in a patient for whom no effective therapy exists, and there is an ongoing study of the drug and a sponsor agrees to provide the product, FDA does not object to the treatment use."


Tony Crispino
SWOG Patient Advocate - Prostate Cancer
Chapter President - UsTOO Las Vegas
Prostate Cancer Education and Support
(702) 917-7779
www.swog.org

I'm no expert on this.
One idea: if the drug company allows compassionate drug use , that might be a disincentive for patients to join a double blind clinical trial that has a placebo component. Just conjecture on my part.

A question: Can the drug company charge for compassionate use?