3 ms·

How is this different than the existing Compassionate Use [1], program? Under this program my dad had access to try treatments for his terminal cancer. What doe

by dwyerm 8y ago

How is this different than the existing Compassionate Use [1], program? Under this program my dad had access to try treatments for his terminal cancer. What does Right To Try offer over that? [1] https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAcc...

lozenge 8y ago
The ability to bypass the FDA's regulations process entirely.
mercurialshark 8y ago
Thanks for sharing. I'm not making a comparison to existing programs, just countering the articles underlying argument that because treatment may cause more harm than good or they may provide false hope, that patients with advanced forms of illnesses should have limited options. With sufficient guidance and information, when available, I'm simply saying that informed patients may opt for highly speculative treatment options, on a case-by-case basis. I can't speak to your question directly, will check it out...
nordsieck 8y ago
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...

by dwyerm 8y ago

lozenge 8y ago

The ability to bypass the FDA's regulations process entirely.

mercurialshark 8y ago

Thanks for sharing. I'm not making a comparison to existing programs, just countering the articles underlying argument that because treatment may cause more harm than good or they may provide false hope, that patients with advanced forms of illnesses should have limited options. With sufficient guidance and information, when available, I'm simply saying that informed patients may opt for highly speculative treatment options, on a case-by-case basis. I can't speak to your question directly, will check it out...

nordsieck 8y ago

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...