A few years ago, my brother lost a child to a terrible genetic disease. In this case, there was nothing to do; when the diagnostic was established, there was nothing we could do. Not a single experimental treatment. Well, when this Precious Little Sunshine was conceived, she was handed a bad set of genes and that's it.
A week ago, my sister sent me an article that was published in the newspaper about a family's battle for their little one. The child has the same disease our Little Sunshine had. In the last few years, research had progressed. There is currently an experimental treatment available in Canada for the condition. Unfortunately, this child was born 17 days too early to fit the criteria, so was ineligible. I am taking shortcuts here, but let's just say, the parents didn't take no for an answer, got the magic formula, asked some lab, that is making drugs for animal (and not human), to synthesize the medication and travelled to Mexico to have it injected in their child. And now, they have to go to Mexico every 45 days or so to give it to the child, since no physician here or in United States would agree to do it.
I felt this article was more an editorial about big bad pharma and the health care system not trying to save this baby's life. Maybe it was not the journalist intention, but it is how I felt it. And I felt criticized. I worked for almost a decade in clinical trials. I believe I have some ideas on how things work and why they work that way.
I do not have to agree or disagree on how the parents handled the situation. I do not judge if they did right or wrong. They did the best in this situation for their family. Would I be in the same situation, I have no idea what I would do. All I have to say is that I am impressed with their creativity and resilience.
Even though sometimes, from the outside of this closed little world that is clinical trials, we can have the impression that the decisions are made only to save money and bring the drug to the market as soon as possible, it is not exactly how the decisions are made. First, even though we are treating patients one by one, the decisions are taken at another level. The investigator (physician who treats patients) follows a protocol designed to demonstrate if the drug works. Each criteria is there for a reason and we have to trust the judgement of the statistician and specialist physician involved in the design of the protocol.
There is a pretty solid safety net around research as well. Regulatory bodies (Health Canada or FDA) and the institution ethic committee review the protocol and decide if it is ethical and if the benefit that can be expect outweight the risks taken. These people approve a protocol, and therefore it is not to be taken lightly or not followed.
I feel sad that people always complain about pharma, one way or another. When you are in a situation, you see one tree but not the whole forest. A trial is done with the forest in mind. Decisions cannot be made on the basis of only one individual. If the investigator do not comply with the protocol, the results won't be admissible.
Unfortunately for this child, he was ineligible for this trial. But, when I think of all the other babies that can be treated if this medication works and get to the market sooner because the investigators were compliant with the protocol and there was no delay, I understand the reason.
I am not giving my blessing to everything the pharma world does, far from it. I try to keep a critical mind. I do not have all the specifics about this case, so it is very difficult to comment or make a judgement... but there is a possibility for compassionate use of a drug for this kind of situation. From the outside, it looks like it was a perfect case for this kind of program. Why the child couldn't receive the drug this way, I have no clues. And I hope from the bottom of my heart that a viable long term solution will be find very soon for the child to receive the drug that seems to help him.
When someone asked my brother what he thought of the situation, his answer was: "I do not necessarily agree with the way they did it, but when you are there, you could do anything. However, it shows that the research is progressing and this is a good news." I am so proud of you! I love you, Brother!