estimand

Non-inferiority margins: how they are chosen and how they are abused — a second dataset — setting out what I have, and where I think it stops being reliable. Posting a small dataset on Non-inferiority…

On the subject in the title: Reading a supplementary appendix and finding the interesting part Working notes rather than a conclusion. Collecting what is known about Reading a supplementary appendix in one…

Posting this under the heading it deserves: Non-inferiority margins: how they are chosen and how they are abused Everything below is what sits behind that. A question about non-inferiority margins that I…

Journal club: semaglutide in MASH, and surrogate endpoints — does this still hold? I have a specific reason for asking rather than idle curiosity, and the context is below. A narrow question about semaglutide…

The question in the title: How to read a forest plot, properly, from scratch I will give what I have already checked below so nobody repeats it. Reading STEP 4 ( JAMA , 2021) for the population rather than…

Journal club: the retatrutide phase 2 obesity paper — setting out what I have, and where I think it stops being reliable. Collecting what is known about retatrutide phase 2 obesity paper in one place, because…

Journal club: how we handle discontinuation in obesity trials — what changed since — setting out what I have, and where I think it stops being reliable. Session topic: STEP 4 ( JAMA , 2021). Please read it…

Second pass at: Journal club: STEP 4 and what a withdrawal design can prove Writing it up because I had to work it out twice and would rather nobody else did. The question about STEP 4 that I actually want…

Posting this under the heading it deserves: Journal club: indirect comparison between two programmes, defended and attacked — one year on Everything below is what sits behind that. Asking about indirect…

On the subject in the title: Journal club: STEP-HFpEF and symptom endpoints — a second dataset Working notes rather than a conclusion. Posting a small dataset on step-hfpef and symptom endpoints. It is mine,…

Posting this under the heading it deserves: Journal club: orforglipron phase 2 and the non-peptide question Everything below is what sits behind that. A follow-up question about orforglipron phase 2 that I…

On the subject in the title: Sample size calculations, read backwards from the published number Working notes rather than a conclusion. Sample size calculations — I have the observation and I do not trust my…

Journal club: STEP-HFpEF and symptom endpoints — setting out what I have, and where I think it stops being reliable. The question about step-hfpef and symptom endpoints that I actually want answered is the…

Comparators chosen for regulatory reasons rather than clinical ones — one year on — setting out what I have, and where I think it stops being reliable. Posting a small dataset on Comparators chosen for…

Journal club: STEP 8 and the fairness of the comparator dose — setting out what I have, and where I think it stops being reliable. Session topic: STEP 8 ( JAMA , 2022). Please read it before posting; the…

Individual randomised trials: design, population, endpoints, results, and limitations. This post is a community wiki: any member at trust level 3 or above can edit it, and every edit is recorded with its…

Journal club: SURMOUNT-1, read without the press release Writing it up because I had to work it out twice and would rather nobody else did. Posting a small dataset on SURMOUNT-1. It is mine, it is…

Journal club: the CagriSema phase 2 combination paper — a second dataset — setting out what I have, and where I think it stops being reliable. Posting a small dataset on CagriSema phase 2 combination paper.…

Posting this under the heading it deserves: Journal club: SURMOUNT-4 and continuation versus withdrawal — a second dataset Everything below is what sits behind that. Posting a small dataset on SURMOUNT-4 and…

Interim analyses and stopping rules — does this still hold? — that is the question, and I have not found it answered plainly anywhere I have looked. Interim analyses and stopping rules, from the point of view…

Posting this under the heading it deserves: Second pass at: Trial registration and comparing the protocol with the paper Everything below is what sits behind that. Two things I would like separated before…

On the subject in the title: Comparators chosen for regulatory reasons rather than clinical ones Working notes rather than a conclusion. An honest uncertainty about comparators chosen for regulatory reasons…

Adjudicated events and why the definition matters — the long version Writing it up because I had to work it out twice and would rather nobody else did. A narrow question about Adjudicated events, deliberately…

Journal club: STEP 4 and what a withdrawal design can prove — one year on Writing it up because I had to work it out twice and would rather nobody else did. Session topic: STEP 4 ( JAMA , 2021). Please read…

On the subject in the title: Journal club: the retatrutide phase 2 obesity paper Working notes rather than a conclusion. A follow-up question about retatrutide phase 2 obesity paper that I did not know to ask…

Open-label extensions: what survives and what does not — setting out what I have, and where I think it stops being reliable. I have spent a fortnight trying to pin Open-label extensions down and I want to set…

The question in the title: Journal club: SUSTAIN 6 and its retinopathy signal — does this still hold? I will give what I have already checked below so nobody repeats it. Reading SUSTAIN 6 ( N Engl J Med ,…

Our recurring session. One named paper per topic, methods first, conclusions last. Everyone reads before posting. This post is a community wiki: any member at trust level 3 or above can edit it, and every…

Posting this under the heading it deserves: Journal club: SURMOUNT-OSA and a hard endpoint in a soft field Everything below is what sits behind that. Collecting what is known about SURMOUNT-OSA in one place,…

Journal club: SURPASS-2 and the semaglutide 1 mg comparator Writing it up because I had to work it out twice and would rather nobody else did. Session topic: SURPASS-2 ( N Engl J Med , 2021). Please read it…