You do understand that those kids were taking LESS or underdosing of steroids? So the issue is whether they need any steroids not is it was daily or weekly underdosing shots? MG/KG stayed the same over 4 years., NOT increased with weight. Just because Farkas was flippant with data does not make it a real concern, just his poor craftsmanship.
They are not going to get it from current EXON51, How many parents would want to risk placebo and a wheelchair for their KIDs? I would be out of the placebo in a flash and into some other trial.
It's can't be done now. Natural History has to be the only way. The FDA wants lots of biopsies they can't read. Big trials with doomed placebo kids and data they don't know how to read.
The FDA want bigs drama, kids in placebo falling down and into wheelchairs to make a BIG data point. Then they can point and say "well we have a great placebo trial".
How many kids do they want to sacrifice? 50? 100? 1500? 1-2 Billion $ more for limited new data?
$SRPT $FDA Start a signature drive to have DMD, rare DX expert on drug evaluations. Not Farkas again for any DMD,FDA with record release. Please join me.
A placebo control trial cannot be done with this drug. At 24 weeks they would know because of improved ADL's and no longer blinded.
If you want kids to prove a drug by unethically keeping them off drug until a wheelchair is needed. NOT for Children. Adults maybe, they have a legal choice, not 7-9 year old children. It's unethical now. They know the cause and effects. No way to keep it blinded. 4 year natural history required. Then Farkas playing games with his ego to prove his point that only big placebo trials should cross his desk.
He should change or resign, no DMD drug maker is going to trust him again.
What need to change with orphan drugs under review? FDA needs QUALIFIED Reviewers for rare diseases. With these rare diseases, we must have at least one Expert or panel in "that area" working with the FDA for drug review. With current drug review, we have second rate reviewers that have NO experience with these diseases reviewing and making life and death decisions that they are not qualified in their background or training.
With this FDA reviewer Farkas, it seems personality clashes over ethically run trial were the problem. The FDA/Farkas requiring placebo kids to stay on placebo even if that mean those children lost ambulation forever. The vilified CEO CG was "Retired" for sticking up for those same kids. Now Farkas with his "GOT YA" revenge in the review for the notion that messy ethics should be part of his Data. Farkas main takeaway? FDA needs for a large placebo control data no matter the cost to children. He slammed all data to make his point. A Gold star for him.
Farkas, BABY killer. Oh wait lets collect data on that...how many kids can we put on Farkas head. %1 and all delayed exons...at least 50 a year.
So now all rare DX trials will require dead or disabled kids to be a success? Big extended placebo trials will require many someones to sacrifice their lifes. No natural history unless agreed on BEFORE the trial.
That’s health care cost. Hospital used to take losses on Labor and Delivery. It was a seller for future family returns to those Hospitals.
To blame Socialized medicine is a quack answer. Completive Health Care is driving up the Cost. Better machines, buildings, staff, expensive medications, maximum treatment for low returns. That what runs up cost. Without insurance smaller Hospitals were failing nationwide. You would be stuck with a few Larger Hospitals with only a few people paying all the cost. Everyone would get free care and Hospital would eat the cost. That was yesterday's System funded by a well-paid middle class. That’s gone. Just Welfare Corporations now.
I think the letter campaign may have had results. Major DMD centers supporting EXONDYS 51. It would put Fafkus's reputation down the Toilet amongst DMD leaders for a lifetime. Demand he be removed.
Now we know Farkas was the one at the FDA the Chris Garabedian resigned over. He's still holding a grudge. Stains the FDA corruption with his "Got Ya" review. Farkas, He should resign.
This was about Farkas and his "You don't know the whole story". Got you SRPT. Farkas presented data that he knew would screw over SRPT. He's getting his revenge. This was not about KIDS, just FDA petty officials making a point. Did you see any Positives from Farkus or Dunn? Nope..l.they were attacking not with analyzed data but with dirty got yours. Kindergartner actions from Farkas.
The trial would have been unethical, Farkas had no problem with kids proving his trial with subject falling into wheelchairs. That would have been a good trial. CEO CG has the ethics to say NO. Farkas should be removed..
Now we know Farkas was the one at the FDA the Chris Garabedian resigned over. He's still holding a grudge. Stains the FDA corruption
Farkas had it out to get SRPT, he may have been the problem all along with CG. Farkas did not lay out data, he laid out an unreasonable attack.
So now what? New Placebo with biopsies trial. Extended trial until they to over way over 18? or whatever Farkas changes the new goals too? All over Again, biopsies from "same" muscle group. With three independent reviewers? Or whatever Farkas wants next? Reports on PT and steroid diaries? Same type or all over the place age groups like BMRN's failed trial.
Farkas option to change goal as he likes on Becker and other data? Farkas should just resign.