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Ever planned RCT w/ PFS and OS as formal endpoints?
Ever simulated PFS and OS for same set of patients?
In both cases: How did you model the fact that PFS and OS are highly dependent? E.g. we know that PFS <= OS and P(PFS = OS) > 0.
A (long) thread - but it' s worth it!
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Ever simulated PFS and OS for same set of patients?
In both cases: How did you model the fact that PFS and OS are highly dependent? E.g. we know that PFS <= OS and P(PFS = OS) > 0.
A (long) thread - but it' s worth it!
1/n
We are very used to model dependency of an endpoint over time (= group-sequential designs) or over nested populations (= enrichment designs), b/c then it's not difficult to write down correlation between test statistics.
However, typically in trial planning PFS and OS...
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However, typically in trial planning PFS and OS...
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are treated as if they were independent and proportional hazards (PH) is assumed for both.
We propose to model dependency between PFS and OS using an illness-death multistate model (IDM), building on the work of Meller et al (2019) doi.org/10.1002/sim.82…
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We propose to model dependency between PFS and OS using an illness-death multistate model (IDM), building on the work of Meller et al (2019) doi.org/10.1002/sim.82…
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