Evidence · subcategory

Trials

Individual randomised trials: design, population, endpoints, results, and limitations.

33 topics1,574 posts10 solved Moderators: j.mwangi, bench_notes About this category

randomised trial estimand intention to treat discontinuation & dropout open label surrogate endpoints

33 topics

Topic	Participants	Replies	Views	Activity
Reading a trial's population section before its results — does this still hold? randomised trial open label intention to treat discontinuation & dropout Closed Reading a trial's population section before its results — does this still hold? I have a specific reason for asking rather than idle curiosity, and the context is below. I have seen STEP 1 ( N Engl J Med ,…	+140	154	5.4k	14mo
Adjudicated events and why the definition matters — the long version intention to treat estimand randomised trial discontinuation & dropout 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. Comparing SURPASS-4 ( Lancet , 2021) with STEP 4 ( JAMA…	+125	146	13k	15mo
Run-in periods and the population they select surrogate endpoints discontinuation & dropout estimand randomised trial On the subject in the title: Run-in periods and the population they select Working notes rather than a conclusion. Session topic: PIONEER 6 ( N Engl J Med , 2019). Please read it before posting; the…	+130	142	7.3k	1mo
Reading a trial's population section before its results estimand surrogate endpoints discontinuation & dropout randomised trial Solved On the subject in the title: Reading a trial's population section before its results Working notes rather than a conclusion. I have seen STEP 2 ( Lancet , 2021) cited in support of a claim I do not think it…	+91	109	22k	2mo
Reading a supplementary appendix and finding the interesting part open label discontinuation & dropout intention to treat estimand Reading a supplementary appendix and finding the interesting part — setting out what I have, and where I think it stops being reliable. I have seen FLOW ( N Engl J Med , 2024) cited in support of a claim I do…	+87	92	1.1k	17mo
Coming back to: Sample size calculations, read backwards from the published number estimand discontinuation & dropout surrogate endpoints randomised trial On the subject in the title: Sample size calculations, read backwards from the published number Working notes rather than a conclusion. Session topic: PIONEER 6 ( N Engl J Med , 2019). Please read it before…	+82	88	1.8k	11mo
How to read a forest plot, properly, from scratch randomised trial intention to treat surrogate endpoints estimand Solved 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. Session topic: STEP 4 ( JAMA , 2021). Please read it before…	+73	81	17k	7mo
Comparators chosen for regulatory reasons rather than clinical ones — one year on randomised trial intention to treat open label estimand Solved 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. Session topic: SURMOUNT-2 ( Lancet , 2023). Please read…	+82	87	1.1k	12mo
[2026 update] Composite endpoints and the component doing the work surrogate endpoints discontinuation & dropout estimand On the subject in the title: Composite endpoints and the component doing the work Working notes rather than a conclusion. Comparing SURMOUNT-1 ( N Engl J Med , 2022) with SURPASS-4 ( Lancet , 2021) and…	+76	87	994	3mo
Primary endpoint hierarchies and why order matters — one year on surrogate endpoints discontinuation & dropout estimand randomised trial Solved On the subject in the title: Primary endpoint hierarchies and why order matters — one year on Working notes rather than a conclusion. Comparing SELECT ( N Engl J Med , 2023) with SURPASS-2 ( N Engl J Med ,…	+56	61	37k	11h
Subgroup analyses: pre-specified versus discovered — a second dataset open label discontinuation & dropout estimand randomised trial On the subject in the title: Subgroup analyses: pre-specified versus discovered — a second dataset Working notes rather than a conclusion. Comparing SURMOUNT-1 ( N Engl J Med , 2022) with SURPASS-4 ( Lancet ,…	+71	79	906	3mo
Revisiting: Primary endpoint hierarchies and why order matters estimand surrogate endpoints discontinuation & dropout intention to treat Posting this under the heading it deserves: Revisiting: Primary endpoint hierarchies and why order matters Everything below is what sits behind that. Comparing SURMOUNT-4 ( JAMA , 2024) with SURPASS-2 ( N…	+21	25	46k	3mo
Second pass at: Trial registration and comparing the protocol with the paper estimand discontinuation & dropout randomised trial open label 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. I have seen SCALE ( N Engl J Med , 2015)…	+30	34	33k	13mo
Interim analyses and stopping rules — does this still hold? estimand discontinuation & dropout open label surrogate endpoints Solved 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. Comparing SCALE ( N Engl J Med , 2015) with SURMOUNT-4 (…	+30	34	29k	13mo
Non-inferiority margins: how they are chosen and how they are abused open label surrogate endpoints estimand intention to treat 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. Comparing SURPASS-2 ( N Engl J Med , 2021) with…	+42	46	10k	5mo
Interim analyses and stopping rules intention to treat surrogate endpoints open label discontinuation & dropout Interim analyses and stopping rules Writing it up because I had to work it out twice and would rather nobody else did. I have seen SUSTAIN 6 ( N Engl J Med , 2016) cited in support of a claim I do not think…	+25	29	35k	16d
[2026 update] How to read a forest plot, properly, from scratch estimand discontinuation & dropout surrogate endpoints Solved How to read a forest plot, properly, from scratch — that is the question, and I have not found it answered plainly anywhere I have looked. Session topic: SURMOUNT-4 ( JAMA , 2024). Please read it before…	+49	53	643	19mo
[2026 update] Reading a supplementary appendix and finding the interesting part estimand intention to treat open label surrogate endpoints On the subject in the title: Reading a supplementary appendix and finding the interesting part Working notes rather than a conclusion. Comparing SELECT ( N Engl J Med , 2023) with FLOW ( N Engl J Med , 2024)…	+39	44	8.2k	5mo
Trial registration and comparing the protocol with the paper randomised trial open label intention to treat Trial registration and comparing the protocol with the paper — setting out what I have, and where I think it stops being reliable. I have seen LEADER ( N Engl J Med , 2016) cited in support of a claim I do…	+38	44	4.8k	10mo
Composite endpoints and the component doing the work randomised trial surrogate endpoints open label discontinuation & dropout Solved Under review Composite endpoints and the component doing the work Writing it up because I had to work it out twice and would rather nobody else did. I have seen SURPASS-4 ( Lancet , 2021) cited in support of a claim I do…	+3	7	33k	13h
Non-inferiority margins: how they are chosen and how they are abused — a second dataset randomised trial discontinuation & dropout intention to treat 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. Comparing FLOW ( N Engl J Med , 2024) with…	+32	37	574	5mo
About the Trials category randomised trial estimand intention to treat Pinned Wiki 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…	+6	10	27k	12mo
Follow-up: Open-label extensions: what survives and what does not open label intention to treat surrogate endpoints estimand Solved Open-label extensions: what survives and what does not — setting out what I have, and where I think it stops being reliable. Comparing SURMOUNT-2 ( Lancet , 2023) with SURMOUNT-1 ( N Engl J Med , 2022) and…	+22	26	397	16mo
Comparators chosen for regulatory reasons rather than clinical ones surrogate endpoints open label estimand randomised trial Pinned Closed On the subject in the title: Comparators chosen for regulatory reasons rather than clinical ones Working notes rather than a conclusion. Session topic: SURMOUNT-1 ( N Engl J Med , 2022). Please read it before…		2	27k	14mo
Open-label extensions: what survives and what does not discontinuation & dropout randomised trial estimand Closed Posting this under the heading it deserves: Open-label extensions: what survives and what does not Everything below is what sits behind that. Comparing STEP 8 ( JAMA , 2022) with PIONEER 6 ( N Engl J Med ,…		4	22k	3mo
Subgroup analyses: pre-specified versus discovered randomised trial intention to treat surrogate endpoints estimand Subgroup analyses: pre-specified versus discovered — setting out what I have, and where I think it stops being reliable. Comparing SURMOUNT-2 ( Lancet , 2023) with SURPASS-4 ( Lancet , 2021) and finding the…		2	27k	1d
Run-in periods and the population they select — the long version randomised trial estimand intention to treat surrogate endpoints Run-in periods and the population they select — the long version Writing it up because I had to work it out twice and would rather nobody else did. Session topic: SUSTAIN 6 ( N Engl J Med , 2016). Please read…		2	8.9k	4mo
Second pass at: Reading a trial's population section before its results open label intention to treat estimand surrogate endpoints Solved Second pass at: Reading a trial's population section before its results Writing it up because I had to work it out twice and would rather nobody else did. I have seen STEP 1 ( N Engl J Med , 2021) cited in…		4	9.5k	10h
What a statistical analysis plan adds that the paper does not open label discontinuation & dropout randomised trial surrogate endpoints Solved What a statistical analysis plan adds that the paper does not — that is the question, and I have not found it answered plainly anywhere I have looked. I have seen SCALE ( N Engl J Med , 2015) cited in support…		2	6.6k	10h
Primary endpoint hierarchies and why order matters randomised trial intention to treat estimand Primary endpoint hierarchies and why order matters Writing it up because I had to work it out twice and would rather nobody else did. Comparing SURMOUNT-OSA ( N Engl J Med , 2024) with SURPASS-2 ( N Engl J…		4	308	3mo

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