18 ms·

Thoughts on AbbVie’s Rova-T Implosion

22 points by aaavl2821 8y ago

mercurialshark 8y ago
As a counterpoint: New Immunotherapy Treatment Removes All Tumors In Woman With Advanced Metastatic Breast Cancer https://www.cancer.gov/news-events/press-releases/2018/immunotherapy-targets-breast-cancer-case-report https://www.cancer.gov/news-events/press-releases/2018/immun... https://clinicaltrials.gov/ct2/show/NCT01174121 https://clinicaltrials.gov/ct2/show/NCT01174121 https://www.forbes.com/sites/victoriaforster/2018/06/04/new-immunotherapy-treatment-removes-all-tumors-in-woman-with-advanced-metastatic-breast-cancer/#963363100550 https://www.forbes.com/sites/victoriaforster/2018/06/04/new-... https://www.cbsnews.com/news/immunotherapy-treatment-saves-life-of-woman-with-late-stage-breast-cancer/ https://www.cbsnews.com/news/immunotherapy-treatment-saves-l...
- 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...
oh_sigh 8y ago
Derek's argument seems to be that some drugs may actually harm patients, and so Right to Try is bad. Isn't that known, and just something terminal patients need to balance with their doctors? If I had 6 months to live, I would probably roll the dice for a cure even if 50% of the time the drug would actually only give me 3 months to live. I doubt anyone believes that trying experimental drugs can only help, and never hurt the patient. If that was the case we'd have been allowing experimental drug usage for a long time.
- AnimalMuppet 8y ago
  There's two ways to look at this. If I've got six months to live, would I take a 50/50 gamble on living 20 years, where the downside is that I might only live 3 months? Probably - the expectation value of that gamble is highly positive. On the other hand, if you only have 6 months to live, 3 months is really a lot to lose... But I agree. This isn't a valid argument against Right to Try.
  - nordsieck 8y ago
    The proper way to look at this is a dynamic analysis, not a static analysis. For the first person to try the drug, it's a big gamble, but as each additional "right to try" participant takes the drug, everyone else gains knowledge as to its effectiveness. Is this as good as stage 3 trials at assuring safety? No. But you have to count all the people who could have been helped but weren't because of delays against a no right to try policy.
    - jcranmer 8y ago
      > For the first person to try the drug, it's a big gamble, but as each additional "right to try" participant takes the drug, everyone else gains knowledge as to its effectiveness. The plural of anecdote is not data. We have come to understand what it takes to run a good study for effectiveness of a drug: double-blinded, randomized, high-power control studies, with further guards against p-hacking (did you fail a phase III but find a subgroup that was responsive? great, do another phase III on that subgroup; turns out it's mostly spurious). The data that comes from right-to-try is mostly not useful because it's not going to tell you how much of the benefit comes from things other than the drug (such as the placebo effect).
masterleep 8y ago
The idea that bureaucrats and scientists should be able to decide what experiments a terminally ill patient should be allowed to participate in is a deeply evil one.
- beat 8y ago
  The idea that lay people should decide instead is a deeply incompetent one.
  - syshum 8y ago
    So you reject the idea of Informed Consent? Hell why stop at right to try let simply take away all medical choices for everyone, If the doctor says something you will be compelled to do it, Second Opinion, no way, clearly the doctor is the professional and you as a individual are too stupid to make your own choices for your life
    - smt88 8y ago
      How is informed consent possible when the information required is a deep understanding of both medicine and statistics?
      - syshum 8y ago
        Wow... I really thought I would die before humanity would embrace a dystopian technocracy where all Self Agency is removed completely from the individual...
        smt88 8y ago
        That's a very extreme reading of my implication that laypeople aren't able to just take a crash course in a particular disease or drug and make a well-informed decision. Do you disagree with all individual restrictions? Should we eliminate the concept of prescription drugs, so that anyone can go buy Adderall or Accutane without a doctor's approval?
Shivetya 8y ago
Can we please change the title to match the article instead of being sensationalized/ politicized ?
- asdsa5325 8y ago
  ^ This.
djrogers 8y ago
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
2RTZZSro 8y ago
In some sense we are all terminally ill so I'm interested to see how far they will push the definition of a terminal illness.
aaavl2821 8y ago
Another pretty harmful potential downside of "right to try" is that while FDA may no longer hold as much sway, payers will still be gatekeepers. Payers will decide what to pay based on evidence and will not pay for drugs with little evidence to support their effectiveness. So patients will be stuck with the costs, and end up paying more than they do now There is precedent for this in the world of molecular diagnostics. Certain tests can be marketed without FDA approval. However few companies are successful because insurers wont pay and patients wont pay enough
jennybush 8y ago
I was cured of HIV with the used of natural cure. I loves Traditional Medicine so much. Most times, injection and drugs are just a waste of time and money. I was cured 2yrs ago, i suffered from HIV for 13yrs but with the help of Dr. TEBOR herbal medicine, i was cured within few weeks of drinking the herbs he sent to me through courier delivery service. This same man also cured my Aunt from herpes, as soon as i heard she had herpes, i referred her to the doctor and she was cured after drinking his herbs.I have referred more than 15 persons and they were all cured from their various illness. Have you taken herbs before?. You have spent so much money on drugs,injections,surgeries etc and yet you have no good result to show for it. This man is a herbalist doctor, i assured you of cure if you drink his natural herbs. He have herbs that cures Hiv, Herpes, diabetics, asthma, hepatitis, etc. Contact Dr. TEBOR on email: TEBORHERBALCENTER@GMAIL.COM Phone/Whatsap no: +2348153052125
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

22 points by aaavl2821 8y ago

As a counterpoint: New Immunotherapy Treatment Removes All Tumors In Woman With Advanced Metastatic Breast Cancer https://www.cancer.gov/news-events/press-releases/2018/immunotherapy-targets-breast-cancer-case-report https://www.cancer.gov/news-events/press-releases/2018/immun... https://clinicaltrials.gov/ct2/show/NCT01174121 https://clinicaltrials.gov/ct2/show/NCT01174121 https://www.forbes.com/sites/victoriaforster/2018/06/04/new-immunotherapy-treatment-removes-all-tumors-in-woman-with-advanced-metastatic-breast-cancer/#963363100550 https://www.forbes.com/sites/victoriaforster/2018/06/04/new-... https://www.cbsnews.com/news/immunotherapy-treatment-saves-life-of-woman-with-late-stage-breast-cancer/ https://www.cbsnews.com/news/immunotherapy-treatment-saves-l...

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...

oh_sigh 8y ago

Derek's argument seems to be that some drugs may actually harm patients, and so Right to Try is bad. Isn't that known, and just something terminal patients need to balance with their doctors? If I had 6 months to live, I would probably roll the dice for a cure even if 50% of the time the drug would actually only give me 3 months to live. I doubt anyone believes that trying experimental drugs can only help, and never hurt the patient. If that was the case we'd have been allowing experimental drug usage for a long time.

AnimalMuppet 8y ago
There's two ways to look at this. If I've got six months to live, would I take a 50/50 gamble on living 20 years, where the downside is that I might only live 3 months? Probably - the expectation value of that gamble is highly positive. On the other hand, if you only have 6 months to live, 3 months is really a lot to lose... But I agree. This isn't a valid argument against Right to Try.
- nordsieck 8y ago
  The proper way to look at this is a dynamic analysis, not a static analysis. For the first person to try the drug, it's a big gamble, but as each additional "right to try" participant takes the drug, everyone else gains knowledge as to its effectiveness. Is this as good as stage 3 trials at assuring safety? No. But you have to count all the people who could have been helped but weren't because of delays against a no right to try policy.
  - jcranmer 8y ago
    > For the first person to try the drug, it's a big gamble, but as each additional "right to try" participant takes the drug, everyone else gains knowledge as to its effectiveness. The plural of anecdote is not data. We have come to understand what it takes to run a good study for effectiveness of a drug: double-blinded, randomized, high-power control studies, with further guards against p-hacking (did you fail a phase III but find a subgroup that was responsive? great, do another phase III on that subgroup; turns out it's mostly spurious). The data that comes from right-to-try is mostly not useful because it's not going to tell you how much of the benefit comes from things other than the drug (such as the placebo effect).

masterleep 8y ago

The idea that bureaucrats and scientists should be able to decide what experiments a terminally ill patient should be allowed to participate in is a deeply evil one.

beat 8y ago
The idea that lay people should decide instead is a deeply incompetent one.
- syshum 8y ago
  So you reject the idea of Informed Consent? Hell why stop at right to try let simply take away all medical choices for everyone, If the doctor says something you will be compelled to do it, Second Opinion, no way, clearly the doctor is the professional and you as a individual are too stupid to make your own choices for your life
  - smt88 8y ago
    How is informed consent possible when the information required is a deep understanding of both medicine and statistics?
    - syshum 8y ago
      Wow... I really thought I would die before humanity would embrace a dystopian technocracy where all Self Agency is removed completely from the individual...
      - smt88 8y ago
        That's a very extreme reading of my implication that laypeople aren't able to just take a crash course in a particular disease or drug and make a well-informed decision. Do you disagree with all individual restrictions? Should we eliminate the concept of prescription drugs, so that anyone can go buy Adderall or Accutane without a doctor's approval?

Shivetya 8y ago

Can we please change the title to match the article instead of being sensationalized/ politicized ?

asdsa5325 8y ago
^ This.

djrogers 8y ago

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

2RTZZSro 8y ago

In some sense we are all terminally ill so I'm interested to see how far they will push the definition of a terminal illness.

aaavl2821 8y ago

Another pretty harmful potential downside of "right to try" is that while FDA may no longer hold as much sway, payers will still be gatekeepers. Payers will decide what to pay based on evidence and will not pay for drugs with little evidence to support their effectiveness. So patients will be stuck with the costs, and end up paying more than they do now There is precedent for this in the world of molecular diagnostics. Certain tests can be marketed without FDA approval. However few companies are successful because insurers wont pay and patients wont pay enough

jennybush 8y ago

I was cured of HIV with the used of natural cure. I loves Traditional Medicine so much. Most times, injection and drugs are just a waste of time and money. I was cured 2yrs ago, i suffered from HIV for 13yrs but with the help of Dr. TEBOR herbal medicine, i was cured within few weeks of drinking the herbs he sent to me through courier delivery service. This same man also cured my Aunt from herpes, as soon as i heard she had herpes, i referred her to the doctor and she was cured after drinking his herbs.I have referred more than 15 persons and they were all cured from their various illness. Have you taken herbs before?. You have spent so much money on drugs,injections,surgeries etc and yet you have no good result to show for it. This man is a herbalist doctor, i assured you of cure if you drink his natural herbs. He have herbs that cures Hiv, Herpes, diabetics, asthma, hepatitis, etc. Contact Dr. TEBOR on email: TEBORHERBALCENTER@GMAIL.COM Phone/Whatsap no: +2348153052125

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