Abstract
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.
| Original language | English |
|---|---|
| Article number | 443 |
| Number of pages | 6 |
| Journal | Trials |
| Volume | 24 |
| Issue number | 1 |
| Early online date | 6 Jul 2023 |
| DOIs | |
| Publication status | Published - 6 Jul 2023 |
Bibliographical note
AcknowledgementsWe are thankful to Marie Miller, Imperial Clinical trials Units’ patient and public involvement coordinator who contributed to the set-up the HEALTHY STATS public involvement group. We are also thankful to Imperial College London’s’ Patients Experience and Research Centre (PERC).
Funding
This research was funded by an MRC NIHR TMRP project funding grant and an NIHR advanced research fellowship (Suzie Cro, NIHR300593). The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care. B.C.K. is funded by the UK MRC, grants MC_UU_00004/07 and MC_UU_00004/09.
Keywords
- Humans
- Research Design
- Communication
- Educational Status
- Research Personnel
- Uncertainty
Access to Document
- Cro_etal_T_Starting_A_Conversation_VoR
© The Author(s) 2023. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.