Patient involvement in clinical trial design can facilitate the recruitment and retention of participants as well as potentially increase the uptake of the tested intervention and the impact of the findings on patient outcomes. Despite these benefits, patients still have very limited involvement in designing and conducting trials in nephrology. Many trials do not address research questions and outcomes that are important to patients, including patient-reported outcomes that reflect how patients feel and function. This limitation can undermine the relevance, reliability and value of trial-based evidence for decision-making in clinical practice and health policy. However, efforts to involve patients with kidney disease are increasing across all stages of the trial process from priority setting, to study design (including selection of outcomes and approaches to improve participant recruitment and retention) and dissemination and implementation of the findings. Harnessing the patient voice in designing trials can ensure that efforts and resources are directed towards patient-centred trials that address the needs, concerns and priorities of patients living with kidney disease with the aim of achieving transformative improvements in care and outcomes.
Patients have limited involvement in designing and conducting trials and are not routinely given positions of agency and power in their organizational and governance structure.
The involvement of patients in the design of clinical trials can help to improve participant recruitment and retention and increase the uptake and impact of the findings.
Patients can be involved in research priority setting, study design (including participant recruitment and retention and selection of outcomes, including patient-reported outcomes), as well as the dissemination and implementation of trial findings.
Resources need to be allocated to provide the administrative, educational, financial and logistical support required to support the meaningful involvement of patients across all stages of the trial.
Patient-centred trials that are designed and conducted with patient involvement can provide the evidence needed for patients to make informed decisions that reflect their desired health outcomes.
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The authors declare no competing interests.
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- Research priority setting
Refers to activities that involve identifying, prioritizing and establishing consensus on research topics or questions that are important to stakeholders.
- Process evaluations
Evaluations that are conducted as part of a trial to explore the process of implementing the intervention. They typically involve ascertaining the trial participants’ perspectives on the intervention and may include investigating contextual factors that affect the intervention and the acceptability of the intervention.
- Choice experiments
A survey approach that is used to assess participant preferences.
- Patient navigator
A person (usually trained non-medical personnel) who assists patients in accessing and navigating the health-care system. They may facilitate access to care and care management in various ways, including through communication, education, care coordination and advocacy.
- Nominal group technique study
A structured method that is used to identify and prioritize ideas generated by a group of participants.
- Data linkage
The process of identifying, matching and combining records that correspond to the same person, usually from multiple data sets. For example, linked administrative data may be used in clinical trials.
- Social marketing
In the context of health, social marketing involves creating, communicating and delivering information using patient-centred and science-based strategies that are designed to influence health behaviour.
- Patient-reported outcomes
Outcomes that are directly reported by the patient without interpretation by a clinician or anyone else, and usually reflect how the patient feels and functions, for example, quality of life, symptoms (pain, fatigue) and functioning.
- Core outcomes
Consensus-based minimum sets of outcomes to be reported in all trials in a specific health or medical area.
- Content validity
The extent to which a patient-reported outcome measure adequately reflects the construct to be measured.
- Construct validity
The degree to which the scores of a patient-reported outcome instrument are consistent with the hypothesis based on the assumption that the instrument measures the intended construct.
- Criterion validity
The extent to which the scores of a patient-reported outcome measure are an adequate reflection of a ‘gold standard.’
- Patient-caregiver partner
A patient or informal caregiver involved in a research study as an equal partner with other members of the research team.
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Tong, A., Scholes-Robertson, N., Hawley, C. et al. Patient-centred clinical trial design. Nat Rev Nephrol 18, 514–523 (2022). https://doi.org/10.1038/s41581-022-00585-w