Monday, April 7, 2014

Dying patients denied experimental drugs - what can be done?

CNN     At first, Sandy Barker decided to behave nicely and sit silently in the audience as an official from the Food and Drug Administration extolled the virtues of a program to get experimental drugs to desperately ill patients.

Then she couldn't take it anymore. Barker's hand shot up.

"I've been sitting here for the past hour trying to be quiet, but I want to tell you what happened to my son," she said.

Barker looked down at a picture of Christian on her lap. She started to cry, but regained enough composure to describe how her son was diagnosed eight years ago with a rare form of leukemia when he was 13. A bone marrow transplant was supposed to help, but instead the donor's cells attacked Christian's body.

Christian's graft-versus-host disease was quickly getting worse. His life was on the line. Nothing was working. 

The Barkers searched for studies he could join but found none. Christian's doctors desperately wanted to try an experimental drug, but first the FDA had to give its blessing. 

The Barkers and their doctors begged the agency to allow Christian to use the medicine. By the time permission was given, more than three weeks had passed, and the graft-versus-host disease had moved to stage 4, the most severe stage. 

Christian died two months later.

During a panel discussion at a conference on rare diseases, Barker says the FDA official noted it can be helpful to lobby one's congressman to get access to experimental drugs.[...]

Last month the parents of a 7-year-old boy did just that and made headlines around the world. Josh Hardy's parents took to Twitter and Facebook when the drug company Chimerix denied their request for an experimental antiviral drug to save Josh's life. After receiving death threats from "Josh's army" -- executives had to hire security guards -- Chimerix reversed its position and granted Josh and other patients like him access to the drug.

Now that he's had the medicine, the virus that nearly killed Josh is gone and he's been moved out of the intensive care unit.[...]


  1. Well, on the one hand, lives can be saved through new medication. On the other hand, there are risks when experiencing with new medication, and lives can also be lost due to unexpected side effects.

    If the FDA autorises the use of drug that is not safe, the scandal is even worse than if a patient cannot be treated because the drug is not yet availabe.

    They cannot compromise on security, and therefore, media should not present such one-sides stories without presenting the other side of the medal...

  2. I feel so sorry for this mother Sandy Baker and her son, but the FDA has rules for testing safety and efficacy of drugs for good reasons. For example, FDA rules prevented American babies from joining the 10,000 babies who were born deformed as a result of thalidomide.

    Furthermore, there are already rules for "expanded access" or "compassionate use" that were successfully used in the case of Josh Hardy. Thus, there is no real reason to change the rules as they currently are.

    A mother would pay any price to someone who claims to heal their child, even if they are selling snake oil. Witness the tragic case of Refael Elisha Cohen, whose trusting parents waged a war for a deadly and ineffective treatment. The quack doctor they're supporting has taken advantage of lax FDA rules for decades by classifying his treatment as experimental, as a USA Today investigation revealed.

    See also the likely preventable death of Joey Hofbauer, whose father convinced a judge to let him try a quack treatment on his little boy in the 1970s.

    A breast cancer surgeon who knows something about these proposed "right to try" laws explains further why newly proposed rules are impractical. The critical point is that "only 5% of all cancer drugs that enter clinical testing are ultimately approved for patient use."

    Thus, a balance must be struck between patient safety and patient rights. Otherwise, all sorts of crazy drugs will be allowed to hit the market, such as the bleach enemas discussed here.


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