FDA agrees to let patients get controversial drug I have mixed feelings on this one. On the one hand, I am all for patients being allowed to try experimental treatments, especially when all approved options have run out and they are not eligible for clinical trials. On the other hand, some protection of the patient is needed.
I would propose a few things before I allowed this to be made available:
1. Release all of the data on the clinical trials that have been run. They have not been published. Unofficial sources have said that there were over 100 trials of this treatment, and 99 of them showed negative results. 1 showed good results. Statistically, at the level of proof most commonly used, .05%, if you ran 100 trials, you would expect 5 of them to come out positive by chance alone. Having only 1 positive and 99 negative trials leads me to believe this is not the miracle drug it is touted to be.
2. I understand a traditional clinical trial can not be run on this group of patients, however, that does not mean that their experiances should go to waste. All patients who use a compassionate use treatment should be tracked. Our "Brain tumor virtual trial" is a perfect vehicle for this. An individual success is meaningless. About 10% of kids and a higher % of adults with brainstem gliomas do well for a long time on the standard treatments. If you take 3 patients, there is a good chance that 1 of them, and a small chance that 2 or 3 of them, would do well no matter what you did to them. However, if you had 50 patients tracked from before they start the treatment, the chances that 25 of them do well is pretty low if the treatment is not working. Not as convincing proof as a randomized blinded trial, but good enough for me and the people who have no other options.
