First it is important to understand what compassionate use is, it is the use of medications that have not been approved by the FDA, but are in earlier stages of development and are allowed to be provided to patients when circumstances call for it. I would say that saving the life of a young child who has already battled cancer four times, should be among those circumstances. In fact, I think that preference should be given to children in compassionate use, but clearly that is not what is happening here. So why is this drug being withheld from Josh?
Is it money? The company behind this drug reports that each compassionate case would cost them $50,000, money that the small company does not have. More than this financial limitation, green-lighting a case like Josh's would take the manpower of all the company's employees, likely contributing to the end of their compassionate use program two years ago. As many of us who are following this story learned as we woke up today, there have been offers to pay the $50,000 for Josh, but those too have been denied.
Is it the principle? When asked by the media about the withholding of this medication, the president of Chimerix, Kenneth Moch (who has vocally proclaimed that he too is a parent) has said that while he is heartbroken, saying yes to Josh's case would essentially open the floodgates to all who apply for compassionate use. He followed that up by saying that if his child had an aggressive adenovirus like Josh, he'd be fighting tooth and nail too. It's no secret that a parent would and should fight to save the life of their child, but I have a sneaking suspicion that should the situation be reversed, Josh's parents would provide access to the drug to save Kenneth Moch's children.
Would compassionate use create a black mark on the drug? More than money and principle, compassionate use has the ability to make a drug look bad in the long run. Of course it goes without saying that those in need of these drugs for compassionate use are in dire situations, and that they may very likely die without the use of the drugs. Chimerix would have to report any poor outcome to the FDA when it applies to market the drug, including those of compassionate use.
Knowing all sides of this case, and even as a biased parent whose daughter lost her life to cancer, when you have the ability to save a child's life, you do it, no matter what. To me, and many people out there, the answer is simple, if you can afford it or not, if you have to go against your principles or not, whether there are consequences to reputation or not, you do whatever is necessary to save the life of a child. Period.
My daughter Alex was on many medications under compassionate use. While they may not have ultimately saved her life, they certainly provided her with more quality time, time to hold her lemonade stands and make a difference in the lives of others. If she would not have had access to these drugs, who knows where we would be now, perhaps not funding millions of dollars of research annually to find better treatments and cures for the very disease that has left Josh fighting for his life.
I understand that this company owes their investors a return on their money, but in the end, we are all human beings, and we owe it to these children to share this potentially lifesaving drug. No matter the obstacles in the way, when a child's life is on the line, you do what you have to. I hope that the president of Chimerix reconsiders, whatever that may mean in the long run - Josh deserves to be given the chance to grow up and it seems to me that through the simple release of Brincidofovir that is completely within the realm of possibility. Let's not let a policy or financial implications come before doing the right thing.
The blog post above was originally posted on the Huffington Post Impact blog on Monday, March, 2014.