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Kaspar Rufibach Profile picture
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10 Mar, 4 tweets, 2 min read
1/n

Clinical trials in patients with hematological malignancies often present unique challenges for trial design due to complexity of tmt options and existence of potential curative but highly risky procedures, for example, SCT or tmt sequence across different phases.
2/n

I am very happy to see our paper

Estimands in hematologic oncology trials

now online at Pharm Stat: doi.org/10.1002/pst.21…

The paper illustrates how to apply the estimand framework in hematological clinical trials and how the estimand framework can address...
3/n
... potential difficulties in trial result interpretation. Three phase 3 RCTs are used to illustrate different scientific questions and the consequences of the estimand choice for

- trial design,
- data collection,
- analysis,
- and interpretation.
4/n
Template language for describing estimands in study protocols and statistical analysis plans is suggested in the paper’s appendix.

This paper is another result of the X-industry working group "estimands in oncology" (oncoestimand.org)

#estimand #hematology #oncology

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More from @numbersman77

Kaspar Rufibach Profile picture
10 Mar
1/n

Stop the abuse: plea for more principled approach to analysis of AEs.

A (long) thread. Please RT.

@tim_friede @ADAlthousePhD @VR_Cornelius @RachPips @statsepi

How are AEs typically reported in an RCT? Along the lines of the table below.

From: nejm.org/doi/full/10.10… Image
2/n

So what is the purpose of such tables? I see two:

1) "Signal detection" --> find AEs that differ between arms.

2) Estimate true probability of an AE of interest, P(AE).

I will focus on 2) below.
3/n Assume we are interested in P(AE) - this is our estimand.

What do proportions in above table estimate? The incidence proportion:

IP = P(AE happening in [0, t] and that this AE is observed).
Read 12 tweets
Kaspar Rufibach Profile picture
31 Jan
In response to this Q:



I give a list of activities that were kicked off by the ICH E9 estimands addendum (I believe) and that I am aware of (and/or involved in). I am sure there is much more, so no ambition at all to be comprehensive.

Please RT!
1) NPH: arxiv.org/abs/1908.07112 (and several more papers, e.g. on max-combo tests).

2) Special interests groups:

oncology: oncoestimand.org (PSI / EFSPI and ASA biopharmaceutical section), browse events and publications

neuroscience: psiweb.org/sigs-special-i….
EFPIA / PSI on estimands implementation (I am not sure they have a webpage already)

3) Many events, see e.g.

bbs.ceb-institute.org

psiweb.org/events/event-i…

oncoestimand.github.io/oncowg_webpage…

efpia.eu/news-events/ev…

cytel.com/webinar-replay…
Read 7 tweets
Kaspar Rufibach Profile picture
5 Jan
1/3
sciencedirect.com/science/articl…

Very interesting paper on carbon footprint of pharma industry. Some takeaways:

1) Pharma significantly *more* emission intensive than automotive industry.

2) Highest and lowest intensity emitter in 2015 differ by factor of 5.5!
2/3
3) From 2012-2015 "...the leader of the pack,
namely @Roche, achieved both highest increase in revenues as well as highest reduction in emissions, suggesting, albeit on anecdotal basis, that financial and environmental performances are far from being exclusive."
3/3
4) Roche, J&J, and Amgen are already today below the 2025 target emission according to the Paris climate agreement.

There are many reasons to work for Roche, but this is definitively one more, and not the least important for me!
Read 4 tweets
Kaspar Rufibach Profile picture
11 Nov 20
1) Why do we run group-sequential trials in drug development?

2) How does the effect we power at relate to the effect(s) needed to stop?

3) Is stopping such a trial early (like the one for the Pfizer vaccine) "cheating"?
4) If stopping early the effect estimate may be biased. Is this an issue?

5) What happens operationally if a trial stops early?
A thread from a pharma statistician who has developed, run, analyzed, and taught courses about trials with such designs.

@statsepi @lakens @MaartenvSmeden @stevesphd @ADAlthousePhD @DominicMagirr @thomas_jaki
Read 20 tweets
Kaspar Rufibach Profile picture
9 Nov 20
1/n I was asked to give an industry statistician's view on A) below. Disclaimer: I do not know about the exact regulations (which might also be region-specific). What I offer is a 1st hand experience of what happens around a trial stopping early. A thread.
2/n @ADAlthousePhD @MaartenvSmeden @KertViele @raj_mehta @doublerobust
3/n I was the trial statistician for this trial: nejm.org/doi/full/10.10…

Planned efficacy interim after ~245 PFS events.
1) Data with sponsor, except for tmt assignment (=rando codes).
2) Rando codes with IxRS vendor.
3) Indep. Stat. Reporting Group (ISRG) coordinates.
Read 11 tweets
Kaspar Rufibach Profile picture
5 Oct 20
Registration still open for EFSPI regulatory statistics webinars. Currently we have 627 and 534 registrations for the two webinars. Webex can handle 1000 people dialing in, so go ahead and register! 😉

Program and registration: efspi.org/Documents/Even…

On Day 2 we now added...
...two talks discussing the impact of COVID-19 on clinical trials and their estimands:

Yongming Gu (Eli Lilly): Using a mix of strategies in handling intercurrent events and missing values for studies impacted by the COVID-19 pandemic

...and...
Guenther Mueller-Velten, Yi Wang, Melanie Wright (Novartis): Impact of COVID-19 and risk mitigation in a global cardiovascular outcomes trial

@EFSPItweet #estimands #regulatorystatistics #statstwitter #biostatistics
Read 4 tweets

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