This slideshow presentation from Roche in a meeting earlier this month seem to point toward some hopeful results. The conclusions apparently require some informed inferences to effectively compare the test groups, but it seemed newsworthy.
Slide 15 puts it like this:
"The comparison of PASADENA and PPMI data suggests potential benefit in slowing motor
progression in favour of prasinezumab on multiple endpoints.
The 2 approaches (propensity score and disease progression modelling) demonstrated
consistent results:
--Slowing of progression on MDS-UPDRS Part III (clinician-rated motor examination) OFF and ON symptomatic medication state, consistent with previous data analyses
--Slowing of progression on MDS-UPDRS Part II (patient-reported motor experiences of daily living) emerges after the effect on Part III
These findings are exploratory and need to be confirmed in an independent trial such as the Phase IIb PADOVA study and its OLE
I retired before starting the trial because I saw the article of the trial failure. I wrote to the doctor who wtote the results, and he confirmed me the failure.
I describe it on my book 'waiting for Michael J Fox'.
The argument Roche seems to be exploring is that the initial results were based on measurements that were just too coarse--that early in PD, the MDS-UPDRS scoring would tend to not reveal much differential because the course of progression only renders smaller changes anyway.
They saw, apparently, that certain secondary endpoints were displaying a more noteworthy difference, and began to wonder if, over a longer time, the "spread" between the control group and the treated group would begin to widen. Apparently, that seems to be happening.
No magic bullet, but if it turns out that we could cut progression by half or a third, that could translate to a very different quality of life over many years. Maybe...
If we end up with a situation where there is detailed discussions about statistics, then it’s not for me. I’m sure most of us are waiting for something that makes enough of a difference that you don’t need to do any maths, to know you’re doing better.
For not the first time in response to a comment of yours expressing the same sentiment: it's not about doing better, it's about slowing the rate at which you progressively get worse. We will need a data set and an analysis for that since you absolutely cannot measure such things at the n=1 level.
I’m flattered to hear that you’re are keeping such a close eye on all my posts. The point I’m making is that there are a lot of people in our community who don’t feel too good or live too well and the promise of novel drugs that keep them in the same place, aren't especially welcome. I think there is a frustration that progress is so slow in developing therapies that make a positive difference to peoples lives, so you’ll have to excuse me for not getting excited about a new drug that probably has weak data and weak patient feedback. In the UK, health care is rationed by necessity, and I would also worry that unless there is clear benefit from new drugs, then approval from NICE may not be forthcoming anyway.
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