Compassionate Use – What’s in a Name?

Compassionate Use – What’s in a Name?

In Romeo and Juliet, by Wiliam Shakespeare, Juliet said:

“What’s in a name? That which we call a rose by any other name would smell as sweet.”

Patients suffering from serious and life threatening diseases may not always qualify to take part in ongoing clinical trials. The inclusion and exclusion criteria for clinical trials can tend to exclude all but the patient in the middle ground of any disease. Unfortunately, this can tend to exclude real life patients.

I remember sitting in a meeting for the development of a treatment for Lupus. Lupus is a disease that is relatively common in black women.  During analysis of the clinical study results, the company executives discussed why the patient population consisted of so few black women despite them having a large prevalence of this devastating disease.  The issue was the seriousness of their disease. In an effort to ensure a successful clinical trial, the company had effectively excluded the very people that they should have been including in the clinical trial.  The company did not want to put the success of the clinical program at risk and made a concerted decision not to actively recruit this population of patients. However, I digress….

Patients with serious and life threatening diseases that find themselves excluded from an ongoing clinical trial for a drug that has not yet been approved, and that appears to be producing good results in clinical trials, can ask their physician to approach the company concerned. The company concerned is under no obligation to provide the drug on a compassionate basis. The officers of the company have to consider many factors before making a final decision on whether or not to make the drug available to any patient on a compassionate basis.

A decision not to provide the drug under a compassionate basis may not appear compassionate if you are the patient, or if it is your child that might benefit from the drug.  However, denying the drug may be the most compassionate action for the firm to take.

The company has to consider if the patient would derive benefit from the investigational drug. The company also has to consider if the patient might suffer harm if the drug is administered to them on a compassionate basis. If the drug is in a late stage of development, the firm may best serve that patient and all other patients by not placing that success at risk by giving it to any patients outside of clinical trials before it is approved.

If a patient were to suffer a serious reaction to the drug, the drug’s future could be put into jeopardy.  Outside of a formal clinical trial, it is not always straightforward to determine if the serious reaction was due to the drug, the underlying disease, or occurred just by chance. In the absence of any clear causality, the drug could be considered possibly causally related to the adverse outcome.

Another consideration is that if too many patients are placed into a compassionate use program, the number of patients available to go into the ongoing studies could be detrimentally affected, especially in the case of rare diseases. This would slow down the availability of the drug for all patients.

When denied access to a drug on a compassionate use basis, patients have been known to start a war with the company, on social media. The goal in this situation is to get the drug by any means necessary, and to embarrass the company into making it available, usually to one specific “special” patient.

Social media is a very powerful tool. People who spend a lot of time on social media, which appears to be much of society these days, tend to be swept along in wave after wave of hysteria and social activism on social media. In fact, it can sometimes feel as though we are all being swept from one social activist project to another, on Facebook, Twitter, Instagram, …..

When this happens to a pharmaceutical company the effect can be detrimental to careers, patients, and even the drug under development. If competent people lose their jobs because it becomes untenable for them to remain with the company as a result of a social media campaign, that could be the very person needed to bring the drug to market quickly.

A very real and important consideration in whether or not to make a drug available on a compassionate use basis, is the need to be seen to be fair. Pharmaceutical companies have to give investigational drugs on a compassionate use basis to the patients who will benefit the most. These may not be the patients who are the most articulate and socially connected, or who can can garner support on social media and from high profile celebrities.

It is truly unfortunate that these programs are called compassionate use programs. If denied access to a drug under a compassionate use program, the decision can appear to be most uncompassionate, but this denial may be the true act of compassion.

Lorna Speid, Ph.D.


Putting Rare Diseases Patients First!

Dr. Speid is the author of Clinical Trials: What Patients and Healthy Volunteers Need to Know. It is published by Oxford University Press.

1 Comment

  1. review 4 years ago

    This is really useful, thanks.

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