For the benefit of those who may not make it through this thread, as the RCH4 Web site states, it's a charity, not a clinical trial. There has been no IRB approval of a protocol, and there is no independent Data Monitoring Committee to monitor whether the drug is working as hypothesized, whether patients are at risk, etc.
In short, there is no independent scientific oversight of these experiments, and they will never qualify as evidence to take the compound to market. That's why the Web site says "friends" instead of "patients."
Anyone entering this charitable program should be aware of these facts, which are both acknowledged and muddled on the Web site. The fact that patient-reported outcomes (answers to questions) are part of the program, as Tim mentioned, as in all drug development, does not mitigate the differences between this and a clinical trial, or even authorized Compassionate Use as we have discussed in other threads.
RCH4 patients have no tangible protection from adverse outcomes and are by implication trusting in the good intentions and scientific competence of anonymous principals.
Best,
Laurie