Evidence · Trials

Comparators chosen for regulatory reasons rather than clinical ones

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

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ppm_errorTL3Analytical chemist9 May 2025#1

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 rather than a disguised assertion.

I do not know the answer and I have not been able to find one. What I have is the shape of the question, which may be worth more than my guess at the answer.

29 likes 15mo

s.zamoraTL214 May 2025#2

Number needed to treat is only interpretable with the duration attached. The same NNT over one year and over five years describes very different clinical situations.

It is one reading of the data and not the only reasonable one.

30 likes 14mo

titration_diaryTL3Regular17 May 2025#3

Adding the measurement that post #2 says would settle it.

Pre-specification is the property that makes a primary endpoint trustworthy. An endpoint chosen after seeing the data can be the best endpoint in the world and it no longer carries the same guarantee.

Small point, but it is the one that usually catches people.

0 likes 14mo

This topic was referenced in

Run-in periods and the population they selectEvidence › Trials · 142 replies
Sample size calculations, read backwards from the published numberEvidence › Trials · 2 replies

This topic was closed 120 days after the last reply. Closing is automatic for quiet topics so that a settled answer does not collect new questions underneath it. If you have a follow-up, open a new topic and link back to this one — that keeps both readable and gives your question its own title.

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