Compassionate Use — The Fairness Principle

Compassionate Use — The Fairness Principle

When a new drug is under development, it has to be studied in patients in well designed clinical trials.  There are many patients that fall outside of the criteria set for inclusion. When a drug that is being studied has been demonstrated to be effective, patients with diseases that the new drug could treat can approach the pharmaceutical company and ask to be given the drug on a compassionate basis. The pharmaceutical company must then decide whether or not to provide the drug compassionately. As stated in my previous Blog post there are important considerations to be taken into account before the drug can be provided to any patient compassionately.

One important principle is the need to be fair.  The pharmaceutical company should be free from bias – i.e. it should not provide the drug compassionately to someone with lots of money, and refuse to give it compassionately to someone that is not affluent.  The company must not give the drug to someone who is well spoken, and refuse to give it to someone who is not. Too often we hear of situations where pharmaceutical companies have come under undue pressure to make an investigational drug available to one specific child because the parent was able to coordinate an influential social media campaign. The pharmaceutical industry becomes the bad guy that does not want to save the child’s life. When this happens the reputation of the pharmaceutical industry is damaged, the company concerned is denigrated in the media, and there are no winners.

One pharmaceutical company, Janssen, is being proactive about these situations. It recognizes that these decisions are ethical. They have formed a collaboration with Professor Art Caplan, an ethicist at New York University (NYU).  They have worked together to create a model which I am hopeful will be a template for the pharmaceutical industry for compassionate use decisions.

The model involves the creation of a committee, called a Compac, that is independent of the pharmaceutical company. The Compac consists of experts and patient advocates. The Compac sits outside the company and will receive and consider the requests for compassionate use that are received by the Janssen. With this approach, the patient may feel assured that the response was considered in relation to well thought out and independent procedures. By being a step removed from the process, the company will be less susceptible to accusations of unfairness.

The Compac’s first drug,  daratumumab, is being developed for the treatment of multiple myeloma. This is a rare disease. Patients must have failed other treatment options to be considered for compassionate use, and not be eligible for clinical trial enrollment. The requests must be sent by the patient’s physician. The Compac has received requests from all over the world for this investigational drug.

This appears to be a model that could, and perhaps even should, be adopted by other pharmaceutical and biotech companies, small and large.

Lorna Speid, Ph.D.

Lorna is the author of Clinical Trials: What Patients and Healthy Volunteers Need to Know, which is published by Oxford University Press, and available in major bookstores and online at Amazon.com, and barnesandnoble.com.


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