Great to be at an in person médical conference, on Alzheimers disease, in Boston. This particular conference was founded in 2008.
Quite some excitement that we are now in the therapeutic era for AD.
But so striking the conference kicked off by a panel of old men.
/1
It is notable how the leaders of the field haven't really changed in 13 years. Some have died of course. But leadership seems very unrepresentative of the research base.
It is about time conference organizes avoided keynote panels limited to old white men.
/2
And we now get @jasonkarlawish who has criticised the FDA accelerated approve of aducanumab.
"we have set a new standard [for approval] that people don't quite believe in".
He is speaking remotely. Hopefully not because he would have felt uncomfortable here.
Killer audience question
"two years after the [aducanumab] results were presented at this conference we still haven't seen a peer reviewed publication".
No response from panel. Absolute silence from the audience.
Quite a moment.
/4
Another killer question from a former senior executive at Pfizer.
"we have learned the hard way to do things a certain way that were not done in this case [aducanumab]. For example the need for replication" (biogen did not have 2 pivotal trial that met primary endpoints).
/5
"I am sorry there a no further time for questions"
Dry laughter from the audience.
Really tense atmosphere after those questions from the audience.
À seriously y controversial decision by FDA.
/6
And it was impressive that the FDA's Billy Dunn who has received a lot of criticism for his division's decision on aducanumab, was in the front row during the session.
(circled)
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A whistle blower reports poor compliance with Good Clinical Practice at a CRO managing 3 US sites in the Pfizer study.
There is no evidence that these errors would have undermined the scientific integrity of the trial results.
Example 1: needle disposal
/2
The Texas CRO being criticised was managed by large global CRO icon. The article tells us icon raised concerns about turn around time. This is the system working.
In any large clinical trial there are numerous little arrow made at sites. This trial was done at huge speed.
Once again medicines regulations are becoming politically charged in the UK. Late last year it was rapid / hasty (depending in your view) MHRA authorization of COVID vaccines. Now it is about cancer treatments.
Brief thread on regulatory approval and market access.
/1
For your doctor to prescribe you a medicine, two things must be true (outside a clinical trial)
1. The medicine can legally be sold in the place you live (regulatory marketing authorization) 2. Someone (hopefully by you) is willing to pay for that medicine (reimbursement)
/2
The regulators make sure that medicines are only sold if there is strong evidence that the balance of benefit to risk is favourable. Their goal is to stop companies marketing medicines that are unsafe or don't work.
Tough choices for governments in managing COVID-19.
With apologies to @olivierveran here are some sketches which I think illustrate options. We are plotting number of cases on y against time on x.
Firstly the do nothing option is the blue curve in this graph.
The reason that is so bad is that at peak the number if cases would be well above the capacity - red line - of the healthcare system to cope. Insufficent ventilators, beds and staff would lead to higher proportion of cases dying than would otherwise be the case.
/2
Govs want to "flatten the curve" so cases develop like the green curve. Even if the total number of cases were the same as in the blue curve, the fatality rate would be lower as hospitals could cope better: the max number of cases never exceeds healthcare capacity red line).
/3