I have a childhood friend, a life-long friend. She has a husband and three young sons. Her youngest son, age five, was recently diagnosed with Acute Myeloid Leukemia. AML is very aggressive cancer. Today this five year old boy is battling for his young life at Cooks Children’s Medical Center in Fort Worth, and his parents are at battle alongside him. Over ten-thousand people have visited his Caring Bridge web page since it began a little over a month ago.
Just one month ago, this boy and this family of five were carrying out their daily routine of life as we all do. They were hurrying to school and attending church. They were preparing for little league baseball games and attending birthday parties. They were completely unaware of what would be in store for them the rest of this year and most likely the following year. And now their life looks radically different. Mom and Dad are taking turns staying in the hospital with their sick child. They are seeking the balance of being present for their sick child and being at home with their two healthy children. And they are dealing with decisions regarding their son’s treatment, most of which include determining which medicines are best for their son’s diagnosis. All of this is sudden.
According to an article by Elizabeth Cohen, Senior Medical Correspondent at CNN, families seeking alternative medications for severely ill children are sometimes interested in what has become known as “compassionate use” drugs. These drugs must be approved for use by the Food and Drug Administration before administered, and are often administered when traditional treatments are exhausted or unresponsive. The process of obtaining approval from the FDA for these drugs can often take several weeks. Sometimes, experimental compassionate use drugs that might curb side effects or aide in a patient’s recovery process are not approved for use in time to benefit patients.
The Cohen article references a child diagnosed with leukemia at age 13. Before the family could receive permission from the FDA to receive and use the desired drug, the child’s cancer moved to stage four and soon after he died. This process with the FDA to obtain these experimental drugs had only been in progress for three weeks. Three weeks. Had these drugs been available earlier, could they have saved him? The family now has no way of knowing the answer to this question, but I am confident as a mother that it is too painful for them to even consider the possibility.
My empathy to this family is indescribable. I cannot imagine the lengths to which I would go to exhaust every possible option of treatment for my two daughters were they facing a life-threatening disease. I cannot tolerate for a moment, or begin to conceptualize, the level of pain families who have been denied available treatment options for their children, for whatever the reason. No reason is good enough in my book. It seems in this instance with the thirteen year old, that funding or insurance was not the roadblock, and only bureaucratic federal procedures in place prevented this compassionate use drug from being available.
The point I’m making here is too obvious to spell out. It’s imperative that we ask the FDA why we can’t speed up these procedures. And the article points out that the FDA is not the only organization to blame. Often, the drug manufacturers themselves deny more production of a certain drug or the availability of that drug to a supplier.
The article states, “Companies fear that a death — especially if it’s a child — could turn the public and the FDA against them.” However, drug industry executives argue this point. They say they often approve requests for compassionate use drugs to extremely sick patients. And then Cohen says, “But they do agree that servicing compassionate use requests in general can divert them from their goal, which is to get the drug on the market where it can help anyone who needs it.”
Ironic, isn’t it that somehow “compassionate use” requests are not in the same category as “anyone who needs it.” I tend to think that dying would qualify as a need. How about you?
Cohen, E. (2014). Dying Patients Denied Drugs. Retrieved from http://www. cnn.com /2014/04 /05/health/Cohen-compassionate-use/index.html?hpt=hp_c2