5 ms·

Most (all?) current right to try legislation involves drugs that have already passed safety protocols, but are not approved for treatment of a specific conditio

Most (all?) current right to try legislation involves drugs that have already passed safety protocols, but are not approved for treatment of a specific condition, or drugs currently approved in other countries for treatments. To deny a terminally ill patient a drug that may save her life and is unlikely to cause further harm is the height of regulatory hubris.

olliej 8y ago
This is literally exactly the scenario you're talking about: The drug looked like it would work, so much so that someone decides to spend $6 billion to buy it before it completed stage 3 trials. Now it turns out that not only does it not work, it also actively made things worse. So your "terminally ill patient" and the "drug that may save her life" turns out not to be "unlikely to cause further harm". The existing regulatory regimes allow for the patient to end up in a trial for a drug like this, but the "right to try" law now means the subject can be 1) charged for it and 2) not provide useful information for other patients because they're no longer in a blinded trial environment.
- djrogers 8y ago
  Ending up in a trial != getting the treatment, and fewer than 3 percent of terminally ill patients gain access to investigational treatments through clinical trials. Current 'compassionate use' exceptions are even worse - only 1,200 of those are granted each year to the lucky few, out of hundreds of thousands of people who could use them. Also, don't you think it's rather hypocritical to imply that being in a trial (if you win that lottery) is safe, while getting the drugs unfer RTT is not? Yes, there will no doubt be failures, but to deny a terminally ill patient the right to choose what to do with their own body seems rather crazy to me.
  - aaavl2821 8y ago
    Perhaps a better solution would be better / more open clinical trials? Terminally ill patients and their families are very vulnerable and clinical data is difficult to parse even for doctors, and their are many people who would prey upon them if given the chance
  - olliej 8y ago
    No i don't think it's hypocritical. Right to try laws allow companies to monetise desperate terminally ill patients. That's been shown repeatedly throughout history (and these days in the 'supplements' industry) to result in abuse of dying patients. And you get things like https://www.statnews.com/2017/11/21/herpes-vaccine-hotel-rooms/ https://www.statnews.com/2017/11/21/herpes-vaccine-hotel-roo... which was clearly a great plan - and yeah that's not "terminal", but the exact same kind of thing can happen for terminal diseases as well. The trial methodology removes the financial incentive, so that now you can't just take all of a dying persons money. By keeping the treatment in the trial model you also mean that we get data that can actually help -- maybe find out that the drug does not in fact work, or maybe finding out that it does! A patient who is not being treated under trial conditions is much less useful as a data point (if at all) because the placebo effect, and a bunch of other related ones, have repeatedly been found to lead to incorrect feedback - the person says that the drug makes them more comfortable, even though they didn't live any longer, is that because it actually made them feel better? Or was it placebo? Or was it a desire to be "helpful"? If you use them as a data point you may end up with a drug that doesn't actually help, and that drug gets sold. for money. to people who it doesn't help. Cool.
- smsm42 8y ago
  Yes, this is definitely a possibility - a drug can make a patient worse. Moreover, this is the case even with approved drugs, and with any medical care in general - medical error is third major cause of death in the US, just behind cancer and heart disease. Thus, it is completely unreasonable to say that because occasionally there could be drugs that are harmful, patients should be denied right to try them, especially if their only alternative is literally dying. It's like saying because medical care could "make things worse", we should ban doctors and medicine. Nobody is insane enough to propose this, but apparently there is a lot of people who are ready to say "because there's one drug that proved harmful we should let people die but not allow them to try other drugs that might be somewhat risky". Equally insane, IMO.
  - Vivian11 8y ago
    My name is Vivian Anderson I lives in the United States help me thank Dr. ODIDI who helped cured me from HERPES SIMPLEX VIRUS in just one (1) week. I got to know him from a friend who he helped, at first I doubt if his herbal product would work until my friend put her car at stake that if it doesn't work she would give me her car. After which I decided to try to God be the glory am now a living testimony, all thanks to DR. ODIDI if not him I would not have been healed so easily. You can reach him via odidispelltemple@gmail.com or via whatsapp at +2348022741942, you can also contact him on cure on 1. HSV 1&2 2. EX BACK 3. ALS CURE 4. STROKE 5. DIABETES MELLITUS 6. SIMPLEX HERPES 7. RINGING EAR .
    - smsm42 8y ago
      If you are naive enough to be tricked by this, you have no money anyway as it's already invested in Nigerian Princes. FDA doesn't and can't help these people because they'd buy stuff from "reputible Cannadian pharmecy" anyway.
aaavl2821 8y ago
In the last few years FDA has already moved closer to this with accelerated approval for mostly cancer drugs. Accelerated approval lets you get approved based on surrogate endpoints from smaller studies for patients facing fatal illness. Companies have to conduct full effectiveness studies, but can do this after the product is on the market. So FDA has already been doing a lot to help terminally ill patients get drugs that may save his / her life Also "safety" is relative to the clinical benefit. The CAR-T drugs recently approved for lymphomas have horrible, sometimes fatal side effects. But they also save lives that would otherwise be lost. The risk / benefit would be very different in things like diabetes. Also, safety is a moving target. Late stage studies are large and expensive bc a lot of safety events are rare. So early studies dont give you a full picture of safety People forget that FDA exists in the first place to 1) protect people from dying from shoddily studied drugs (ie incompetence) and 2) snake oil salesmen (malintent). While in theory less regulation could enable more drugs to get to patients faster, more likely it would just incentivize more snake oil sales and poor science

olliej 8y ago

This is literally exactly the scenario you're talking about: The drug looked like it would work, so much so that someone decides to spend $6 billion to buy it before it completed stage 3 trials. Now it turns out that not only does it not work, it also actively made things worse. So your "terminally ill patient" and the "drug that may save her life" turns out not to be "unlikely to cause further harm". The existing regulatory regimes allow for the patient to end up in a trial for a drug like this, but the "right to try" law now means the subject can be 1) charged for it and 2) not provide useful information for other patients because they're no longer in a blinded trial environment.

djrogers 8y ago
Ending up in a trial != getting the treatment, and fewer than 3 percent of terminally ill patients gain access to investigational treatments through clinical trials. Current 'compassionate use' exceptions are even worse - only 1,200 of those are granted each year to the lucky few, out of hundreds of thousands of people who could use them. Also, don't you think it's rather hypocritical to imply that being in a trial (if you win that lottery) is safe, while getting the drugs unfer RTT is not? Yes, there will no doubt be failures, but to deny a terminally ill patient the right to choose what to do with their own body seems rather crazy to me.
- aaavl2821 8y ago
  Perhaps a better solution would be better / more open clinical trials? Terminally ill patients and their families are very vulnerable and clinical data is difficult to parse even for doctors, and their are many people who would prey upon them if given the chance
- olliej 8y ago
  No i don't think it's hypocritical. Right to try laws allow companies to monetise desperate terminally ill patients. That's been shown repeatedly throughout history (and these days in the 'supplements' industry) to result in abuse of dying patients. And you get things like https://www.statnews.com/2017/11/21/herpes-vaccine-hotel-rooms/ https://www.statnews.com/2017/11/21/herpes-vaccine-hotel-roo... which was clearly a great plan - and yeah that's not "terminal", but the exact same kind of thing can happen for terminal diseases as well. The trial methodology removes the financial incentive, so that now you can't just take all of a dying persons money. By keeping the treatment in the trial model you also mean that we get data that can actually help -- maybe find out that the drug does not in fact work, or maybe finding out that it does! A patient who is not being treated under trial conditions is much less useful as a data point (if at all) because the placebo effect, and a bunch of other related ones, have repeatedly been found to lead to incorrect feedback - the person says that the drug makes them more comfortable, even though they didn't live any longer, is that because it actually made them feel better? Or was it placebo? Or was it a desire to be "helpful"? If you use them as a data point you may end up with a drug that doesn't actually help, and that drug gets sold. for money. to people who it doesn't help. Cool.
smsm42 8y ago
Yes, this is definitely a possibility - a drug can make a patient worse. Moreover, this is the case even with approved drugs, and with any medical care in general - medical error is third major cause of death in the US, just behind cancer and heart disease. Thus, it is completely unreasonable to say that because occasionally there could be drugs that are harmful, patients should be denied right to try them, especially if their only alternative is literally dying. It's like saying because medical care could "make things worse", we should ban doctors and medicine. Nobody is insane enough to propose this, but apparently there is a lot of people who are ready to say "because there's one drug that proved harmful we should let people die but not allow them to try other drugs that might be somewhat risky". Equally insane, IMO.
- Vivian11 8y ago
  My name is Vivian Anderson I lives in the United States help me thank Dr. ODIDI who helped cured me from HERPES SIMPLEX VIRUS in just one (1) week. I got to know him from a friend who he helped, at first I doubt if his herbal product would work until my friend put her car at stake that if it doesn't work she would give me her car. After which I decided to try to God be the glory am now a living testimony, all thanks to DR. ODIDI if not him I would not have been healed so easily. You can reach him via odidispelltemple@gmail.com or via whatsapp at +2348022741942, you can also contact him on cure on 1. HSV 1&2 2. EX BACK 3. ALS CURE 4. STROKE 5. DIABETES MELLITUS 6. SIMPLEX HERPES 7. RINGING EAR .
  - smsm42 8y ago
    If you are naive enough to be tricked by this, you have no money anyway as it's already invested in Nigerian Princes. FDA doesn't and can't help these people because they'd buy stuff from "reputible Cannadian pharmecy" anyway.

aaavl2821 8y ago

In the last few years FDA has already moved closer to this with accelerated approval for mostly cancer drugs. Accelerated approval lets you get approved based on surrogate endpoints from smaller studies for patients facing fatal illness. Companies have to conduct full effectiveness studies, but can do this after the product is on the market. So FDA has already been doing a lot to help terminally ill patients get drugs that may save his / her life Also "safety" is relative to the clinical benefit. The CAR-T drugs recently approved for lymphomas have horrible, sometimes fatal side effects. But they also save lives that would otherwise be lost. The risk / benefit would be very different in things like diabetes. Also, safety is a moving target. Late stage studies are large and expensive bc a lot of safety events are rare. So early studies dont give you a full picture of safety People forget that FDA exists in the first place to 1) protect people from dying from shoddily studied drugs (ie incompetence) and 2) snake oil salesmen (malintent). While in theory less regulation could enable more drugs to get to patients faster, more likely it would just incentivize more snake oil sales and poor science