International crossed borders reopened and people can travel around freely now, recovery is inevitable for retail malls, airlines, hotels, transportation and Genting.
How to use external controls: FDA spells out thinking in new draft guidance
The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.
The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.
A recent study co-authored by BioMarin employees identified 45 approvals between 2000 and 2019 where the FDA accepted external control data as part of the investigational drug’s benefit/risk assessment. The authors noted, “they did so for many reasons including the rare nature of the disease, ethical concerns regarding use of a placebo or no-treatment arm, the seriousness of the condition, and the high unmet medical need.” advertisement advertisement
In its new 17-page draft guidance, the FDA makes clear that bias is a major concern with any externally controlled trial, noting:
Although unmeasured confounding, lack of blinding, and other sources of bias cannot be eliminated in externally controlled trials, an assessment of the extent of confounding and bias, along with analytic methods to reduce the impact of such bias, are critically important in the conduct of such trials.
So when might externally controlled trials provide convincing results? The FDA says “when the effect size on a well-characterized outcome of interest is anticipated to be large.” How “large” is defined remains to be seen.
The agency also makes clear in a footnote that unknown unknowns are as much an issue as known unknowns.
Scenarios that would not be suitable for externally controlled trials include when the natural history of the disease of interest is not understood sufficiently or when the disease course is considered well-understood but is variable.
The agency also points to changes in the understanding of the natural history of a disease introducing bias.
“For example, diagnosis of patients with a genetic disorder may have been based historically on the development of signs and symptoms, whereas the development and increased use of genetic testing in clinical trials can diagnose patients at earlier stages of disease,” FDA clarified. “In such situations, a historical control arm would have shorter diagnosis-to-death intervals than a treatment arm, even if the drug of interest has no impact on survival.” ----------------------------------------------
This will affect Taurx's new trial design. Non-placebo trial VS ADNI historical data of placebo patients is considered an externally controlled trial. Generally to pass a trial for AD, if can slowdown by abt 25% can pass liow but the drug effect is super marginal, no meaningful effect at all cannot see any difference in real life application.
********The FDA says “when the effect size on a well-characterized outcome of interest is anticipated to be large.” How “large” is defined remains to be seen.**********
I personally think FDA could be looking at a slowdown of at least 50% for cognitive decline under this new guideline for externally controlled trials. But not to worry HMTM can easily pass this new guideline as this drug can almost totally halt disease progression (almost 90% cognitive slowdown on the average for all the trial patient groups namely MCI, MILD, MODERATE). HMTM can also reverse and improve cognition for Mild Cognitive Impairment (MCI) patient group.
FDA’s Billy Dunn, key figure in approval of Aduhelm, to leave agency
This is actually very good news for Taurx. Billy Dunn is a hardcore amyloid hypothesis supporter and it is so obvious he is in the pocket of amyloid based companies. He even directed that Lucidity trial needs to meet the requirement of all trial patients reaching a certain level of being Beta Amyloid positive. Only these patients can then be accepted into the Lucidity trial. We are a tau based company focusing on clearing toxic tau aggregates inside brain neurons, beta amyloid has nothing to do with our drug action and tau hypothesis but that was his requirement. What is this!!!???
Now with Janet Woodcock and Billy Dunn gone, I think there is an improved chance of HMTM getting a FDA CA but its still a very tough battle, not a very high probability thing. Anyway, I will stay disciplined, sticking to my earlier plan and try to clear another 20% of my Taurx shares soon hopefully at a better price.
Waiting for useless fool LAMPAR EMPTYVESSELi to publicly beg KYY again to buy and post his HOLLAND charts on his wannabe trader blog Hahaha....
Posted by emsvsi > 2 weeks ago | Report Abuse YES RM5 ULTIMATE SUPPORT BUY WITH ULTIMATE CONFIDENCE YES WAIT FOR THE NEXT SURGE ! YES HOLDING STEADY ! PREPARING FOR LIFTOFF SKYROCKET STRONG CLOSING UPTREND IS INTACT AND UNSTOPPABLE OWNINGGG STRONG CLOSING ! NEXT WEEK SKYROCKET YES BUY THE DIP ! YES CONSOLIDATION IS ALMOST OVER ! SKYROCKET SOON
*********************************************************************************** Lowest price achieved on 24/02/2023 was 4.57. OMG Skyrocket to HOLLAND!!! ***********************************************************************************
ATK, When Taurx could start selling ? -------------------------------------
I think better ask EMPTYVESSELi to answer any Taurx queries next time, I think he can give you the best answer like SKYROCKET TO THE MOON APPROVAL VERY SOON...........
All key metrics are now pointed to surge on travel demand according to airline and airport Q result. GenB with 3 mega resort RWS, RWG and RWLV accounted 80% of the overall revenue must have very good earning for the next couple years. Enjoy the show.
In the near term, only looking forward to China tourists Based on survey, only close to 20% China citizens are going to travel in Q1 2023, with the bulk of them planning for Q3. Q1 QR should be interesting for Malaysia and Singapore, but for the US region, I'll keep a close watch.
Catskills increased loss partially was partly due to increased stake from the recent purchase of stake from RPT This has totally negated the increase in profit from RWLV, for your reference
Don’t completely discount HMTM! I have a contact at one of the big Pharma companies in US involved in drug trials and deals regularly with FDA…according to him whilst FDA are picky HMTM is promising despite the placebo issue and may get approval providing test result are replicated over a period. So it is very possible!
"YES WELCOME ALL NAYSAYERS TO GENTING 3182 THE NUMBAWAN TOP PICK SHURE WIN STOCK IN CASINO DE BURSA" @emsvsi, I bought Genting at slightly over RM5.00 a share just before the pandemic struck. Didn't make any money on this counter. Bought Maybank at about the same time at around RM8.50 a share. Including dividends, return from Maybank is so much better.
Thailand recorded 2.14 million foreign tourist arrivals in January, a surge from 133,828 in the same period a year earlier, tourism ministry data showed on Monday, as the vital tourism sector gathered pace with the return of Chinese travellers.
Buying Genting Berhad, you indirectly own Genting Singapore and get GenM/ Genting Plantation for FREE. You could see how undervalued is Genting Berhad!
Price at first price target of inverted head n shoulders. Very normal for professional to close some positions. Only thing can do is to wait it out! Zz..Zz...
This book is the result of the author's many years of experience and observation throughout his 26 years in the stockbroking industry. It was written for general public to learn to invest based on facts and not on fantasies or hearsay....
neohts
2,470 posts
Posted by neohts > 2023-02-28 19:14 | Report Abuse
Closing time 4.66 very big buy volume. Syndicate accumulating