Most 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.