What is “Expanded Access” Program or Compassionate Use Clinical Trial

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For several terminally ill patients drugs available through expanded access program are the only hope left. Source: capewayne.org

Sometime back we got an interesting query from a lady in Blacksburg, Virginia. Her mother, who is suffering from Hepatic cancer (cancer of the liver), would like to try some of the new forms of the treatments that are being investigated; however, since she is also a diabetic and asthmatic, she is not eligible to participate in most of the clinical trials. This lady wanted to know if there is any way through which her mother could gain access to these new treatments. Well, of course there is, it is known as “expanded access” of the investigational drug or treatment procedure. This blog post (a part of clinical primer series) aims at providing more information to all our readers on what expanded access means and how it is used in clinical trials.

As evident in the case mentioned earlier there are several times when people cannot participate in a controlled clinical trial, either due to co-existing health problems, age factors, or at times due to long commute distances. However, there are several among such patients who have no hope left and none of the available modes of treatment can help them in any way. Under such circumstances, if the patient wishes to, (s)he can be treated using a drug or treatment procedure that is still under trial and not available to the general public. The aim of administration here however, would not be to test the efficacy or safety of the treatment procedure but purely to treat the patient. The logic applied here is that since the patient has nothing to lose, administration of the study drug or treatment can not harm the patient any further. The process through which an under-trial drug or treatment is provided to a patient for the purpose of treatment of the person is known as “expanded access,” and the trial is known as “compassionate use clinical trial.”

However, like all other aspects of a clinical trial, the drug companies and agencies conducting the clinical trial have to abide by strict FDA rules before giving expanded access to any of the study drugs or treatments. Certain important aspects of the FDA rules for expanded access are:

The patient must be suffering from a fatal condition that has no known cure or management procedure or the available modes of treatment have failed to show any results on the patient. In such a situation if the potential benefits of expanded access surpass the potential risks of the trial drug, then the study drug may be given an expanded access status and allowed to be provided to the patient.
The drug companies can charge the patients for providing expanded access drugs to the patients. However, FDA has a list of rules as to what drugs and procedures the pharma companies can charge and which ones it cannot.
Since, the clinical trial agencies are allowed to collect efficacy and safety data from the patients receiving treatments via expanded access, there are rules restricting the agencies from using this as a more economical means of conducting trials and collecting data. The data collected from the patients receiving expanded access treatment is always considered as adjunct information and cannot substitute the actual clinical trial data.

Thus, the aptly named, compassionate use trials provide a an opportunity to terminally ill patients to gain access to new means of treatment.

Rob Rapoza

I just wanted to comment on this article relative to the availability of expanded access as I don’t think that the article correctly portrays the difficulty involved in obtaining access to unapproved therapies.

While Compassionate Use Clinical Trials do occur, from a drug development perspective they are problematic. Clinical trials are premised on a series of controls and well-defined study populations. Clinical trials are also extremely expensive to run. Compassionate use, while seemingly ethically positive, is extremely expensive/resource-ladden, potentially contains the risk of negative, uncontrolled data impacting an otherwise scientifically sound drug development process, and ultimately could result in the delay of an efficacious drug reaching the general population (thus putting the ethics of the practice into question).

Therefore, I would hasten to suggest that your statements of, “Well, of course there is, it is known as “expanded access” of the investigational drug or treatment procedure.”, and “Under such circumstances, if the patient wishes to, (s)he can be treated using a drug or treatment procedure that is still under trial and not available to the general public.” are not necessarily accurate. Yes, this type of clinical trial does exist, but it would be unusual for a drug development company to freely offer any unapproved drug for use in anything other than a controlled clinical trial. Further, the decision to access unapproved medication isn’t that of the patient who wishes to access it. Significantly more work than readers likely anticipate would have to take place to convince the appropriate person at the drug development company that compassionate use is warranted.

Therefore, access to copassionate use of unapproved therapies does not come without a lot of hard work and a whole lot of good luck on the part of the patient who hopes to obtain access.

Good luck!

Cure Talk Editor

Rob, Thanks for your detailed comment and insight. Yes you are right there are still hurdles in getting access to experimental treatments under the “compassionate use” program

Please keep providing such useful insights for our blog readers