Evidence map›Paper›PMID 39115134›Full record

ArticlePharmaceutical statistics

Helle Lynggaard, Oliver N Keene, Tobias Mütze, Sunita Rehal

Abstract readEnglish Abstract
In one paragraph

Article in Pharmaceutical statistics. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Helle LynggaardNovo Nordisk A/S, Bagsvaerd, Denmark.ORCID 0000-0002-2006-3928
Oliver N KeeneKeeneONStatistics, Maidenhead, UK.ORCID 0000-0003-0016-9773
Tobias MützeNovartis Pharma AG, Basel, Switzerland.ORCID 0000-0002-4111-1941
Sunita RehalGlaxoSmithKline plc, Brentford, UK.ORCID 0000-0002-2578-5778

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Most published applications of the estimand framework have focused on superiority trials. However, non-inferiority trials present specific challenges compared to superiority trials. The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use notes in their addendum on estimands and sensitivity analysis in clinical trials that there may be special considerations to the implementation of estimands in clinical trials with a non-inferiority objective yet provides little guidance. This paper discusses considerations that trial teams should make when defining estimands for a clinical trial with a non-inferiority objective. We discuss how the pre-addendum way of establishing non-inferiority can be embraced by the estimand framework including a discussion of the role of the Per Protocol analysis set. We examine what clinical questions of interest can be formulated in the context of non-inferiority trials and outline why we do not think it is sensible to describe an estimand as 'conservative'. The impact of the estimand framework on key considerations in non-inferiority trials such as whether trials should have more than one primary estimand, the choice of non-inferiority margin, assay sensitivity, switching from non-inferiority to superiority and estimation are discussed. We conclude by providing a list of recommendations, and important considerations for defining estimands for trials with a non-inferiority objective.

Indexed as

Equivalence Trials as TopicResearch DesignClinical Trials as TopicData Interpretation, StatisticalHumansModels, Statisticalclinical question of interestestimand frameworkICH E9(R1)non‐inferiorityregulatory guidancesuperiority

Identifiers

PMID39115134
PMCPMC11602919

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.