For every cancer drug indication approved between 2015-20, we searched for cost-effectiveness studies
we found between 0 and 9 per drug!!
Some trials were industry sponsored & others neutral
Here are the baseline characteristics of the studies we looked it.
Only 1/2 to 2/3 of drugs have even shown they improve survival
The rest have unknown effects on survival
That is not good enough
It is FDA failure! (these days that's common)
In descriptive analysis, 96% of industry sponsored studies find a drug is cost effective, while this is only true for 30% of non industry sponsored studies
Don't worry! I bet the authors of the 4% of studies sponsored by pharma finding it not cost effective have been fired.
In a multivariate model, being funded by pharma is associated with an odds ratio of 40!!! that a study concludes a drug is cost -effective
This relationship is MASSIVE
Tobacco as a lung carcinogen is only OR 20!!!
Finally, here is a waterfall plot of the incremental cost-effectiveness of drugs BEYOND the threshold
Some cancer drugs are 1 MILLION dollars per QUALY beyond the threshold!!!
This is madness!
We spend so much on drugs that either (a) don't improve survival or quality of life (b) we don't know if they do or (c) do so only marginally
No society can exist funding these drugs
It is untenable
It will bankrupt us all
They say Rome collapsed when half of all days were holidays; perhaps the same fate will happen in the USA when half of GDP is spent on cancer drugs that don't work or work marginally
Our new paper in @EJCI_News argues that Randomized trials are necessary in medicine & PH for interventions w putative benefit & at best MED to LG effect size.
Parachutes & smoking are not good counter examples
Some people argue that b/c we did not need RCTs to know smoking is harmful or Parachutes are life saving, we don't need them to test cloth masking, or the Impella, or some new cancer drug, or HCQ, or <insert ur favorite practice>
But this is based on misunderstanding
There is a huge range of things we can do to someone that might hurt them or save them, imagine the spectrum (below)
Now out in @EJCI_News
Logan Powell & I show where randomized trials necessary
When people say 'we don't need an RCT of smoking (to prove harm) or parachutes (to prove benefit)' does that apply to widespread medical interventions?
🧵 onlinelibrary.wiley.com/doi/10.1111/ec…
Led by Timothee Olivier, our new paper is now out at @JAMANetworkOpen
We analyze 12 years of FDA approvals, and do the hard work of sorting them into
New Mechanism of Action (MOA)
& New MOA for that tumor type
Vs next in class
Few prelim thoughts on this trial (from quick read) #ASH21 1. It is not a 'second line' trial, it is a trial in the worst subset of second line pts & cannot extrapolate beyond
Primary refractory & relapse <12 mo
(TBH, a lot of people doing this already)
As such, it should not generalize to relapse > 12 months
2. That said, for those included, axi-cel seems preferable to chemo then auto; I am not surprised this is true in the most chemo insensitive biology. But a few more thoughts
3. This is Wrong, you are not supposed to do this 👇👇
Standard practice is to take these pts to CAR-T if needed in the control arm; thus, you must compare routine, upfront CAR-T to using CAR-T as salvage when indicated and standard of care.