It seems to me that the patient treated under compassionate use had already been given a death sentence by the disease, the fact that the PLX treatment couldn't overcome it should have been expected. It would have been a spectacular outcome had the treatment worked, but it didn't.
Compassionate use shouldn't work against a company, nor should the right to try, which seems to be the current term for what was previously called compassionate use. One reason many companies don't permit their products to be used in this manner is that in the past bad outcomes have occasionally delayed trials. No one should be surprised when someone who's dying actually dies, yet some people make a big deal of it in spite of the fact that asking for the right to try only occurs when the Doctors know of nothing better to try.
I frankly wish the FDA would establish a way of pricing drugs under right to try that was a percentage of what the list price was expected to be, ideally 50% or less. I suspect that if the price were established, and if the insurance company had to pay, a lot more uses of right to try would be permitted. Essentially this would apply to drugs in, or beyond, Phase 3 Trials to be used while an approval decision was approaching consideration. In every case, the outcome should be reported, but only in a database where the patient isn't identified, but the disease being treated and the outcome is.
Gary
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