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Estimands and their estimators for clinical trials impacted by the COVID-19 pandemic: a report from the NISS Ingram Olkin Forum Series on unplanned clinical trial disruptions

Van Lancker K, Tarima S, Bartlett J, Bauer M, Bharani-Dharan B, Bretz F, Flournoy N, Michiels H, Olarte Parra C, Rosenberger JL, Cro S

An introduction to several hypothetical estimand strategies that may be useful to trials impacted by the COVID-19 pandemic and a review of various causal inference and missing data methods, as well as a statistical method that combines unbiased and possibly biased estimators, for estimation.

Statistics in Biopharmaceutical Research

Publication Date:

Jun 26, 2022

Visualising harms in publications of randomised controlled trials: consensus and recommendations

Phillips R, Cro S, Wheeler G, Cornelius V et al.

Recommendations on which visualisations researchers should consider using for harm data in the publication of their main research findings.


Publication Date:

May 15, 2022

Early phase clinical trials extension to guidelines for the content of statistical analysis plans

Victoria Homer, Christina Yap, Simon Bond, Jane Holmes, Deborah Stocken, Katrina Walker, Emily J Robinson, Graham Wheeler, Sarah Brown, Samantha Hinsley, Matthew Schipper, Christopher J Weir, Khadija Rantell, Thomas Prior, Ly-Mee Yu, John Kirkpatrick, Alun Bedding, Carrol Gamble, Piers Gaunt

This paper reports guidelines for the content of statistical analysis plans for early phase clinical trials, ensuring specification of the minimum reporting analysis requirements, by detailing extensions (11 new items) and modifications (25 items) to existing guidance after a review by various stakeholders.


Publication Date:

Feb 7, 2022

Design and analysis features used in small population and rare disease trials: A targeted review

Partington G, Cro S, Mason A, Phillips R, Cornelius V

A targeted review investigating what design and analysis methods are used by trials in Rare disease/small population facing restricted recruitment.

Journal of Clinical Epidemiology

Publication Date:

Jan 18, 2022

Individual participant data meta-analysis versus aggregate data meta-analysis: A case study in eczema and food allergy prevention

Eleanor Van Vogt ,Suzie Cro, Victoria R. Cornelius, Hywel C. Williams, Lisa M. Askie, Rachel Phillips, Maeve M. Kelleher, Robert J. Boyle

This paper compares Aggregate data and IPD meta-analysis. It demonstrates that using IPD enables safety, subgroup and adherence analyses not possible using only aggregate data and certainty of evidence is increased.

Clinical & Experimental Allergy

Publication Date:

Jan 10, 2022

Improving the analysis of adverse event data in randomized controlled trials

Cornelius V, Phillips R

A discussion on how the value of harm data from RCTs could be better realized, including immediate, mid and longer-term strategies for trialists to adopt to support a change in practice.

Journal of Clinical Epidemiology

Publication Date:

Dec 23, 2021

A dose-finding design for dual-agent trials with patient-specific doses for one agent with application to an opiate detoxification trial

Mozgunov P, Cro S, Lingford-Hughes A, Paterson LM, Jaki T

In this paper, a dose-finding design for a dual-agent combination trial motivated by an opiate detoxification trial is proposed. The distinguishing feature of the trial is that the (continuous) dose of one compound is defined externally by the clinicians and is individual for every patient.

Pharmaceutical Statistics

Publication Date:

Dec 10, 2021

The Use of Extreme Value Statistics to Characterize Blood Glucose Curves and Patient Level Risk Assessment of Patients With Type I Diabetes

Szigeti M, Ferenci T, Kovacs L

This article investigates how Extreme Value Statistics can be applied to characterize blood glucose curves and provide patient level risk assessment of hyperglycemia, using data from the REPLACE-BG trial.

Journal of Diabetes Science and Technology

Publication Date:

Nov 24, 2021

Estimands in published protocols of randomised trials: urgent improvement needed

Kahan BC, Morris TP, White IT, Carpenter J, Cro S

This paper described a review of trial protocols published in October 2020 in Trials and BMJ Open. For each protocol is was investigated whether the estimand for the primary outcome was explicitly stated, not stated but inferable (i.e. could be constructed from the information given), or not inferable.

Trials Journal

Publication Date:

Oct 8, 2021

Commentary: Time to improve the reporting of harms in randomized controlled trials

Junqueira DR, Phillips R, Zorzela L, Golder S, Loke Y, Moher D, Ioannidis JPA, Vohra S

This article describes an overview of reviews to analyse changes in the reporting of harms in RCTs, in relation to the milestone of the publication of the CONSORT Harms guidance in 2004. Results showed that, overall, there has been only a slight improvement in the reporting of harms in clinical trials post publication of CONSORT Harms, and the reporting of harms in RCTs re- mains suboptimal as most trials report less than half of the CONSORT Harms items. We call for an update of CONSORT Harms to better align the reporting of harm outcomes with those of efficacy outcomes.

Publication Date:

Jul 31, 2021