10 ms·
> Most of the time it doesnt and patients are better off getting standard of care The context for "right to try" is a person with a terminal condition that doe
by nordsieck 8y ago
> Most of the time it doesnt and patients are better off getting standard of care
The context for "right to try" is a person with a terminal condition that does not respond to any conventional therapies. In essence, the choice is between experimental treatments and hospice.
> If an effective approved drug exists, usually that has to be the control arm, not a placebo.
There will always be a placebo in a stage 3 trial.
- aaavl2821 8y agoThere is not always a placebo in Phase 3 studies. You can have an active control. Here's an example [0]: active arm is upadacitinib, control is methotrexate (the standard of care) FDA guidelines are you need "at least two adequate and well-controlled studies, each convincing on its own, to establish effectiveness" [1]. Well controlled can be placebo controlled or active control [0]: https://www.prnewswire.com/news-releases/upadacitinib-monotherapy-meets-all-primary-and-ranked-secondary-endpoints-versus-methotrexate-in-a-phase-3-study-in-rheumatoid-arthritis-300659605.html https://www.prnewswire.com/news-releases/upadacitinib-monoth... [1]: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072008.pdf https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegula...