3 ms·

Having just skimmed the text, it seems like the difference is similar to the difference between "shall issue" and "may issue". Fundamentally the FDA is very ag

Having just skimmed the text, it seems like the difference is similar to the difference between "shall issue" and "may issue". Fundamentally the FDA is very against right to try because they believe, correctly, that it reduces the incentive for patients to participate in medical trials - why risk being in the control group if you don't have to? My (perhaps flawed) understanding is that the FDA doesn't not approve Compassionate Use very often for this reason, although they probably use safety as the official excuse.

aaavl2821 8y ago
The null hypothesis in clinical studies is that the investigational drug doesnt work. Most of the time it doesnt and patients are better off getting standard of care There are also ethics guidelines that deal with control group selection. If an effective approved drug exists, usually that has to be the control arm, not a placebo. So a patient will get a drug that works in the control arm, though maybe not in the active arm
- 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 ago
    There 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...

aaavl2821 8y ago

The null hypothesis in clinical studies is that the investigational drug doesnt work. Most of the time it doesnt and patients are better off getting standard of care There are also ethics guidelines that deal with control group selection. If an effective approved drug exists, usually that has to be the control arm, not a placebo. So a patient will get a drug that works in the control arm, though maybe not in the active arm

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 ago
  There 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...