Expanded Access Very interesting Thor - thanks. So it`s not ‘Right to Try’ but ‘Expanded Access' that we should be after. The barriers don`t look particularly high nor the timelines very long:
‘One reason for the limited impact of Right to Try thus far is that its proponents vastly exaggerated the barriers that the FDA placed between terminally ill patients and experimental drugs. The agency already had an “expanded access” pathway for experimental treatments. To get treatment through that route, a patient must have a serious illness, lack a way to get the treatment via a clinical trial and lack a legally available alternative treatment. (Patients might be shut out of a clinical trial because of previous participation in a different trial or because their disease has progressed too far, among other reasons.) The main distinction between expanded access and Right to Try is that, in the former, the FDA is more in the loop: Companies must submit safety reports to the agency that include copious data about efficacy, side effects and “adverse events” (the most grave side effects). This information is used in determining whether a drug should be approved for general use.
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Many patients get drugs through expanded access. In 2018, for example, the FDA processed more than 1,500 applications (which can include multiple individuals) for expanded access to unproven treatments — and approved more than 95 percent of them, typically within hours to days.’