Outraged Strangers Beg Drug Company To Save 7-Year-Old’s Life

safe_imageMost people would be appalled to hear that a pharmaceutical company is withholding a potentially life-saving drug from a dying 7-year-old child. The drug isn’t on the market yet, but could be administered under a “compassionate use” exception from the Food and Drug Administration. The situation is a lot more complex than “big mean company keeps drug away from little kid.” Update: Chimerix and the FDA found a way to let Josh Hardy access the drug.

The 7-year-old is currently being treated at St. Jude’s Children’s Research Hospital. He’s been seriously ill since he was an infant, eventually going into remission from cancer that began in his kidneys and spread to other parts of his body. He has received a bone marrow transplant, and is critically ill from a virus that people who aren’t on anti-rejection drugs would fight off easily. The drug that he needs to defeat the virus without further destroying his kidneys is the oral form of an antiviral called brincidofovir. The problem is that oral brincidofovir, made by a company called Chimerix, isn’t supposed to go on the market until 2016.

Drugs that are still considered “experimental” can go to patients who will die without them: during earlier phases of testing the drug, Chimerix granted hundreds of exceptions. They ended that program, and say that they will not make any more exceptions. One company executive told CNN that even employees’ own family members who could benefit from the drug can’t get it.

The company claims that evaluating compassionate use cases and manufacturing the drug for those patients wouldn’t be an effective use of company resources. Chimerix only has fifty employees. They estimate that it costs $50,000 to provide it to one patient, since insurance generally doesn’t cover experimental treatments. Spending staff hours and money on compassionate use cases means that time and money can’t be spent getting the product on the market.

The argument from Chimerix effectively is, “If we give the drug to Josh, we’ll have to consider every other patient who could benefit, too.” If the company makes one exception due to a social media campaign, would hundreds of other such campaigns follow?

Bioethicist Art Caplan tells CNN that a compromise is possible: the company could consider taking on a limited number of compassionate use cases each year, or limiting them to the most dire cases.

Update, 9:30 PM: Chimerix and the FDA have found a way to let the drug be used in this case: they announced a new clinical trial that will begin tomorrow and enroll Josh Hardy as the first subject. The trial will let the company use the drug on future patients with compromised immune systems who are infected with adenoviruses.

Company denies drug to dying child [CNN]
Save Josh [Facebook]

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  1. furiousd says:

    Perhaps people would be more willing to see both sides of the situation if supporting a social media campaign meant agreeing to contribute to cover the costs of what they claim to support. Far too much credibility is given to ‘likes’ when it’s so simple for someone to appear to care.

  2. JoeBlow says:

    I thought CNN presented this story almost fairly, with only a little slant towards the fact that there is a dying child involved. But when you say:
    The argument from Chimerix effectively is, “If we give the drug to little Josh, we’ll have to give it to everyone else, too.” If the company makes one exception due to a social media campaign, would hundreds of other such campaigns follow?
    That is an irresponsible oversimplification of the explanation from Chimerix. The argument is that as a business with 50 employees, as well as investors they are responsible to, it isn’t in the company’s best interest to either dedicate some of those employees, or hire more employees to field and evaluate requests for compassionate use cases, each of which if approved would cost the company an estimated $50,000, and would involve more manpower to compile medical records and send follow up information to the FDA. The results of which could result in the drug being delayed (therefore kept out of the hands of other patients,) as negative events suffered by the patient while on the drug are reported, regardless of whether or not the event is caused by the experimental drug, or the original underlying condition. The “ethical” suggestion to limit compassionate cases to the most dire would exacerbate this facet of the issue. The company participated in compassionate use cases when there was still more to be learned from them. Now that they have a drug that they believe can be approved by the FDA, further cases won’t help that process, and can stand to hurt the approval of the drug. Basically, to attempt to save Josh, you have to risk the drug company, as well as the lives of any future patients that might have been helped by the drug if it is delayed or rejected based on potential negative outcomes.