Our new @vkprasadlab article is now out in Cancer Reports

We tackle the frequency (spoiler alert: BRISK) of assessment of progression in RCTs of cancer drugs

Want to learn more?
onlinelibrary.wiley.com/doi/epdf/10.10…
[thread]
First, remember that progression free survival is a time to event, composite endpoint

It is typically the time until 1 of 4 things happen
Death, New lesions, Growth (without shrinkage) or Growth (from Nadir).

Watch my free lecture series to learn more:
As a result PFS is binned

It has a stair-step pattern b/c imaging is assessed not continuously, but at pre-specified time points
One can imagine that as a result: how often you assess the endpoint is important.

Very frequent assessment is rare in the messy reality of life, but might be common in trials

This can turn even trivial changes in tumor growth in stat. significant p values of <0.05!

aka $
We assessed how often PFS was checked in trials in our paper

Indeed it is checked frequently. Often less than 8 weeks is mandated for protocol specified scans.

Relentless scanning may be a tactic to find significant results for small gains
In many cases, the frequency of scanning in trials is more than guideline recommendations
Our study could not find a difference in the hazard ratio comparing different trials with different scan intervals, but this caveat is important to consider 👇👇
A future study should compare real world scan intervals to trial scan intervals. If anyone has access to data & is interested; email our lab, we would love to collaborate
@vkprasadlab

Read the full paper here:
onlinelibrary.wiley.com/doi/epdf/10.10…

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More from @VPrasadMDMPH

23 Dec
Our new paper is now out in European Journal of Cancer

We analyze 55 cancer drugs that target genomic abnormalities & assess the evidence

Only 24% reported an improvement in survival 👇

A seductive mechanism of action apparently means low levels of evidence

[thread]
Modern oncology has several classes of drugs
Cytotoxic drugs
Checkpoint inhibitors
CAR-T therapies
General targeted drugs &
Drugs that target specific cancer genomic abnormalities
(Genome drugs!)
Genome drugs get outsized attention; Previously we found that (best case scenario) 13% of US cancer patients were eligible for these drugs; leaving 87% not eligible

pubmed.ncbi.nlm.nih.gov/33862157/
Read 10 tweets
21 Dec
In our new paper in @JAMANetworkOpen we take a deep look into cost-effectiveness (CEA) studies of cancer drugs

Bottom line: If a CEA study is funded by pharma, it is 40x (OMG!) more likely to find the drug is cost effective

A 🧵 explaining what we found
jamanetwork.com/journals/jaman…
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)
Read 8 tweets
19 Dec
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

Here is the explanation 🧵
onlinelibrary.wiley.com/doi/abs/10.111…
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)

Let's start on the harms side
Read 14 tweets
18 Dec
Swimmers, Spider, & Waterfall Plots are everywhere in Oncology

Led by @mlythoe & Olivier
We offer an improvement in our new paper
The Iceberg Plot

Let me explain why it is preferable & teach you about all 4....
[Tweetorial]
ejcancer.com/article/S0959-… Image
All other plots we use in oncology
Tell you what happens AFTER you start the protocol

A swimmers plot shows when treatment was given, and when response and progression occurred for each individual Image
A spider plot shows the tumor size for each patient, every time they were assessed, over time. Image
Read 11 tweets
16 Dec
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…
2
Read 4 tweets
14 Dec
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

jamanetwork.com/journals/jaman…
First we find, more drug approvals over time!

More approvals means more innovation, right?
Next we show how many drug approvals are truly innovative

The dark bar shows the first approval of a new MOA across tumor types, or within a tumor type (bottom pane)

(bottom pane) the brown bar is moving to an earlier line
light blue = next in class
Read 7 tweets

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