Time to improve the clarity of clinical trial reports by including estimands

Cro, Suzie

doi:10.1136/bmj.o2108

Cited by 3 publications

(2 citation statements)

References 2 publications

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“…In other contexts, for example to define the adherence to medications, there is less flexibility to discuss the item [ 29 ]. There were new suggested items such as estimands that showcase possibilities for patient and public involvement in statistical and numerical aspects and work is ongoing to generate best practice [ 30 ]. Discussions about these items and the scope for PPI should be ongoing within trial teams and include public partners.…”

Section: Discussionmentioning

confidence: 99%

Involving patients and the public In sTatistIcal Analysis pLans (INITIAL): A delphi survey

Goulão,

Morris,

Blazeby

et al. 2023

PLoS ONE

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Background Patient and public involvement (PPI) in trials aims to enhance research by improving its relevance and transparency. Planning for statistical analysis begins at the design stage of a trial within the protocol and is refined and detailed in a Statistical Analysis Plan (SAP). While PPI is common in design and protocol development it is less common within SAPs. This study aimed to reach consensus on the most important and relevant statistical analysis items within an SAP to involve patients and the public. Methods We developed a UK-based, two-round Delphi survey through an iterative consultation with public partners, statisticians, and trialists. The consultation process started with 55 items from international guidance for statistical analysis plans. We aimed to recruit at least 20 participants per key stakeholder group for inclusion in the final analysis of the Delphi survey. Participants were asked to vote on each item using a Likert scale from 1 to 9, where a rating of 1 to 3 was labelled as having ‘limited importance’; 4 to 6 as ‘important but not critical’ and 7 to 9 as ‘critical’ to involve patients and the public. Results from the second round determined consensus on critical items for PPI. Results The consultation exercise led to the inclusion of 15 statistical items in the Delphi survey. We recruited 179 participants, of whom 72% (129: 36 statisticians, 29 patients or public partners, 25 clinical researchers or methodologists, 27 trial managers, and 12 PPI coordinators) completed both rounds. Participants were on average 48 years old, 60% were female, 84% were White, 64% were based in England and 84% had at least five years’ experience in trials. Four items reached consensus regarding critical importance for patient and public involvement: presentation of results to trial participants; summary and presentation of harms; interpretation and presentation of findings in an academic setting; factors impacting how well a treatment works. No consensus was reached for the remaining 11 items. In general, the results were consistent across stakeholder groups. Discussion We identified four critical items to involve patients and the public in statistical analysis plans. The remaining 11 items did not reach consensus and need to be considered in a case-by-case basis with most responders considering patient and public involvement important (but not critical). Our research provides a platform to enable focused future efforts to improve patient and public involvement in trials and enhance the relevance of statistical analyses to patients and the public.

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Section: Discussionmentioning

confidence: 99%

Involving patients and the public In sTatistIcal Analysis pLans (INITIAL): A delphi survey

Goulão,

Morris,

Blazeby

et al. 2023

PLoS ONE

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show abstract

“…However, in trials, as in routine health practice, treatments are not always taken by patients exactly as prescribed [ 1 , 2 ]. When this happens, by looking at trial data in various ways, different (and potentially more relevant) questions can be addressed such as: ‘does the treatment improve health outcomes for all patients even if it is not taken as instructed?’ or ‘does the treatment improve health outcomes only for patients who take treatment as prescribed?’ Because the answers to different questions can sometimes lead to alternative conclusions on treatment benefits, when planning a trial, it is important to have clarity on exactly what questions the trial needs to address [ 3 , 4 ]. The trial can be subsequently designed, conducted, and analysed to ensure the key clinical questions of interest are addressed.…”

Section: Introductionmentioning

confidence: 99%

Starting a conversation about estimands with public partners involved in clinical trials: a co-developed tool

Cro

Kahan

Patel

et al. 2023

Trials

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Background Clinical trials aim to draw conclusions about the effects of treatments, but a trial can address many different potential questions. For example, does the treatment work well for patients who take it as prescribed? Or does it work regardless of whether patients take it exactly as prescribed? Since different questions can lead to different conclusions on treatment benefit, it is important to clearly understand what treatment effect a trial aims to investigate—this is called the ‘estimand’. Using estimands helps to ensure trials are designed and analysed to answer the questions of interest to different stakeholders, including patients and public. However, there is uncertainty about whether patients and public would like to be involved in defining estimands and how to do so. Public partners are patients and/or members of the public who are part of, or advise, the research team. We aimed to (i) co-develop a tool with public partners that helps explain what an estimand is and (ii) explore public partner’s perspectives on the importance of discussing estimands during trial design. Methods An online consultation meeting was held with 5 public partners of mixed age, gender and ethnicities, from various regions of the UK. Public partner opinions were collected and a practical tool describing estimands, drafted before the meeting by the research team, was developed. Afterwards, the tool was refined, and additional feedback sought via email. Results Public partners want to be involved in estimand discussions. They found an introductory tool, to be presented and described to them by a researcher, helpful for starting a discussion about estimands in a trial design context. They recommended storytelling, analogies and visual aids within the tool. Four topics related to public partners’ involvement in defining estimands were identified: (i) the importance of addressing questions that are relevant to patients and public in trials, (ii) involving public partners early on, (iii) a need for education and communication for all stakeholders and (iv) public partners and researchers working together. Conclusions We co-developed a tool for researchers and public partners to use to facilitate the involvement of public partners in estimand discussions.

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The importance of clinical importance when determining the target difference in sample size calculations

Parker

Cook

2023

Trials

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Recently, it was argued that clinically important differences should play no role in sample size calculations. Instead, it was proposed that sample size calculations should focus on setting realistic estimates of treatment benefit. We disagree, and argue in this article that considering the importance of a target difference is necessary in the context of randomised controlled trials of effectiveness, particularly definitive phase III trials. Ignoring clinical importance could have serious ethical and practical consequences.

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Time to improve the clarity of clinical trial reports by including estimands

Cited by 3 publications

References 2 publications

Involving patients and the public In sTatistIcal Analysis pLans (INITIAL): A delphi survey

Involving patients and the public In sTatistIcal Analysis pLans (INITIAL): A delphi survey

Starting a conversation about estimands with public partners involved in clinical trials: a co-developed tool

The importance of clinical importance when determining the target difference in sample size calculations

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