Recently, news headlines have surfaced about a 7-year-old boy and his parents' fight to be allowed access to the experimental drug CMX001 to fight off a viral infection which spread due to a bone marrow transplant, weakening his immune system. Tears and tempers ignited worldwide attention on Internet news sites from Chimerix's (the manufacturer) initial denial of “compassionate usage” of the drug. Joyful shouts of praise followed just days later when the company reversed its decision. Was it the right decision?This debate spurred me into reading the underlying animal and Phase 2 human trial studies that this drug has undergone. Both of these studies are relatively short, available free of charge on the Internet, and contain understandable conclusions and analysis.
I am not a medical doctor, but I do understand how to read scholarly material and the underlying statistics behind the conclusions. My own field, Industrial and Organizational Psychology, is always interested in analyzing the far-reaching ethical consequences of business decisions and then applying this knowledge for the betterment of future business practices.
The most significant conclusion from the animal trials was that timing is everything. The drug was very effective when administered within 24 hours of inoculation, barely effective at 48 hours, and completely ineffective when administered 72 hours after inoculation. These results narrowed the focus of the Phase 2 human trials — focusing on the ability of the drug to improve mortality as a first choice of treatment.
Phase 2 human trial results are adequately presented in the company’s press release from last September. The same fundamental conclusions were made as in the animal studies — Phase 3 of human trials will focus exclusively on the drug’s ability to prevent the virus in patients receiving bone transplants. This is where the ethical dilemma comes into play.
The company knows that the drug has marginal, at best, effectiveness as anything besides a preventative measure. Both prior studies have narrowed the focus to the drug’s ability to prevent the spread of the viral infection — not as a treatment for an infection that has already taken hold. At this stage of the illness, the company knows that the efficacy is only slightly better than taking no medicine at all.
The long-term damage of this decision comes from its publicity. Rather than the company emphatically standing behind its own studies and stating that the drug is not effective as a treatment, the company’s actions have created the impression that the drug really is an effective treatment option. Once that kind of impression is engrained into the minds of doctors and patients, it is hard to remove.
Company-created impressions are so dangerous that the FDA has begun to assess enormous fines against companies who give false information about a drug’s effectiveness or promote off-label use of drugs. In this instance, the company has abandoned testing of the drug as a treatment, investigating only its properties as a preventative drug — the company is giving mixed signals as to what the real benefit of the drug will be. While this drug is still in testing, the company is still overstepping its scientific research -- the drug is a “winner” on its own merits, not ones the company or public would like it to have.
The boy might in fact live — and that could actually make the ethical dilemma worse. People occasionally survive the disease without any medication. No one knows what the outcome will be. The company is risking over 4 years of scientific drug studies on the premise that public opinion will not affect future responsible use of the drug. That risk is not worth giving one family a bit of hope.