Just scanning Twitter I came across this story of 7 year old Josh who is a 4-time cancer patient now dying of a virus for which there is a cure. Here is a beautiful video about Josh and an interview with his mom on CNN:
[I’m having trouble embedding the CNN video, so included the link.]
The drug brincidofovir is being developed by @chimerix and is in de last phases prior to marketing authorization, which is expected by 2016. This company is denying this drug to Josh because if it helps one patient, it must help others as well. I suspect that the compassionate use of this drug in this phase could result in the risk that the drug is not accepted by the FDA and other authorities. This is because all ‘adverse events’ – from minor side effects to deaths of patients – have to be reported. When patients are close to dying and get this drug, if they die, it may prevent the drug from being approved even if the deaths are not caused by the drug. But there is a way out of this catch 22.
There is a movement that could make a difference for patients with incurable cancer and for the drug companies that would help them if they could: it is called myTomorrows. This social enterprise aims to make drugs available to patients who need them when these drugs may not (yet) be approved for use. What if myTomorrows were able to provide brincidofovir on behalf of Chimerix? I’m just speculating now, and of course money is needed to be augment the limited supply that Chimerix has currently. I believe this could be a win-win situation.