Some food for thought: In one of the recent interviews (I believe the last one) NP said more or less textually:
This continued ringing on my ears until today for NP must be aware that the Mild-to-Moderate trial is P2 and still we will need to go trough a P3 trial with more patients before we can obtain approval.
However, we are in a pandemic and new, effective drugs are badly needed.
Let me pose a question: what happens if Leronlimab shows an outstanding efficacy as Dr. Paterson already demonstrated with the data he gathered (IL-6, CD4/CD* ratio, Monocytes count, plasma viral load), supported by data by Dr. Yang (blood CRP plus lymphocytes count) and appendix in NEJM (IL-6 count reduction). Would it be possible that Nader was suggesting an approval after P2 ?. I did some search on this possibility , below some results.
Approvals Based on a Single Trial
https://www.raps.org/news-and-articles/news-a...-2018-reli
"According to a 2014 JAMA study, between 2005 and 2012, FDA approved 188 novel therapeutic agents for 206 indications, and 74 indications (36.8%) were approved on the basis of a single pivotal trial. "
"Nearly all trials were randomized (89.3% [95% CI, 86.4%-92.2%]), double-blinded (79.5% [95% CI, 75.7%-83.2%]), and used either an active or placebo comparator (87.1% [95% CI, 83.9%-90.2%]). The median number of patients enrolled per indication among all pivotal trials was 760 (interquartile range, 270-1550)"
"The approval of a drug by the US Food and Drug Administration (FDA) conveys that the product is safe and effective . An Internet-based survey of a national probability sample of 4316 US adults (2944 respondents [68% response rate]) found that 39% report believing that the FDA approves only “ extremely effective ” drugs and 25% only drugs without serious adverse effects.1 Some physicians make similar assumptions about effectiveness and safety, expecting that patients are likely to benefit from newly approved therapies."
"FDA review of new drug applications is guided by the Federal Food, Drug, and Cosmetic Act, which requires “adequate and well controlled investigations” to determine efficacy. FDA guidance suggests that drug manufacturers submit at least 2 trials, each providing independent evidence of efficacy—such studies are known as “pivotal” efficacy trials— but also implies flexibility, describing circumstances in which a single efficacy trial might be sufficient to support approval. "
"Most recently, IQVIA released a report finding that 25 of 59 (42%) novel drugs approved in 2018 were approved on the basis of only one trial. And one out of eight approvals relied only on Phase 1 or 2 trials, with no Phase 3 trials. But as in previous years, a large portion of the drugs relying on only one trial were new orphan and cancer drugs."
"For instance, AstraZeneca’s orphan drug Lumoxiti (moxetumomab pasudotox-tdfk) was approved in September 2018 based on one trial of less than 100 patients with a rare, slow-growing blood cancer. Stemline Therapeutics also won approval in December 2018 for Elzonris (tagraxofusp-erzx) to treat a rare, rapidly progressing cancer of the bone marrow and blood after conducting one trial of 94 patients in the US."
So, if Leronlimab shows outstanding efficacy (the safety has already been demonstrated) I will think that, due to the extraordinary circumstances we are experimenting and the pressing need to gain time and save lives, that the FDA would be well-advised on considering approving Leronlimab after only one trial . After all, this is not that uncommon.
I don't want to raise false expectations, least with the FDA (I don't trust them to do the right thing) but, what is uncommon are the once-in a lifetime circumstances we are going through. Redemsivir was approved presto and even proposed for SOC. Heck, thousands of people are dying every day, if a drug is SAFE and can help, why would you ask somebody with a helping hand to go through a P3 trial with 100's of patients to delay approval for several months ??
I don't want to be alarmist but one month= 1500X30=45000 (as in American lives lost)
Question: how long can we wait ??? Sure Leronlimab will not save everybody, let's say it saves some ...
Question: how long can we wait ???
Dammit !!!! (pardon my english)
Quote:“The FDA will have the proof of safety and efficacy, this is all they need for approval”.
This continued ringing on my ears until today for NP must be aware that the Mild-to-Moderate trial is P2 and still we will need to go trough a P3 trial with more patients before we can obtain approval.
However, we are in a pandemic and new, effective drugs are badly needed.
Let me pose a question: what happens if Leronlimab shows an outstanding efficacy as Dr. Paterson already demonstrated with the data he gathered (IL-6, CD4/CD* ratio, Monocytes count, plasma viral load), supported by data by Dr. Yang (blood CRP plus lymphocytes count) and appendix in NEJM (IL-6 count reduction). Would it be possible that Nader was suggesting an approval after P2 ?. I did some search on this possibility , below some results.
Approvals Based on a Single Trial
https://www.raps.org/news-and-articles/news-a...-2018-reli
"According to a 2014 JAMA study, between 2005 and 2012, FDA approved 188 novel therapeutic agents for 206 indications, and 74 indications (36.8%) were approved on the basis of a single pivotal trial. "
"Nearly all trials were randomized (89.3% [95% CI, 86.4%-92.2%]), double-blinded (79.5% [95% CI, 75.7%-83.2%]), and used either an active or placebo comparator (87.1% [95% CI, 83.9%-90.2%]). The median number of patients enrolled per indication among all pivotal trials was 760 (interquartile range, 270-1550)"
"The approval of a drug by the US Food and Drug Administration (FDA) conveys that the product is safe and effective . An Internet-based survey of a national probability sample of 4316 US adults (2944 respondents [68% response rate]) found that 39% report believing that the FDA approves only “ extremely effective ” drugs and 25% only drugs without serious adverse effects.1 Some physicians make similar assumptions about effectiveness and safety, expecting that patients are likely to benefit from newly approved therapies."
"FDA review of new drug applications is guided by the Federal Food, Drug, and Cosmetic Act, which requires “adequate and well controlled investigations” to determine efficacy. FDA guidance suggests that drug manufacturers submit at least 2 trials, each providing independent evidence of efficacy—such studies are known as “pivotal” efficacy trials— but also implies flexibility, describing circumstances in which a single efficacy trial might be sufficient to support approval. "
"Most recently, IQVIA released a report finding that 25 of 59 (42%) novel drugs approved in 2018 were approved on the basis of only one trial. And one out of eight approvals relied only on Phase 1 or 2 trials, with no Phase 3 trials. But as in previous years, a large portion of the drugs relying on only one trial were new orphan and cancer drugs."
"For instance, AstraZeneca’s orphan drug Lumoxiti (moxetumomab pasudotox-tdfk) was approved in September 2018 based on one trial of less than 100 patients with a rare, slow-growing blood cancer. Stemline Therapeutics also won approval in December 2018 for Elzonris (tagraxofusp-erzx) to treat a rare, rapidly progressing cancer of the bone marrow and blood after conducting one trial of 94 patients in the US."
So, if Leronlimab shows outstanding efficacy (the safety has already been demonstrated) I will think that, due to the extraordinary circumstances we are experimenting and the pressing need to gain time and save lives, that the FDA would be well-advised on considering approving Leronlimab after only one trial . After all, this is not that uncommon.
I don't want to raise false expectations, least with the FDA (I don't trust them to do the right thing) but, what is uncommon are the once-in a lifetime circumstances we are going through. Redemsivir was approved presto and even proposed for SOC. Heck, thousands of people are dying every day, if a drug is SAFE and can help, why would you ask somebody with a helping hand to go through a P3 trial with 100's of patients to delay approval for several months ??
I don't want to be alarmist but one month= 1500X30=45000 (as in American lives lost)
Question: how long can we wait ??? Sure Leronlimab will not save everybody, let's say it saves some ...
Question: how long can we wait ???
Dammit !!!! (pardon my english)
14
