Good Practice Action 2: Pre-funding design assessments

Questions for funders to ask research teams

Guidance

What is the research question?

Describe the research question in a Participant-Intervention-Comparator-Outcome (PICO) format. The PICO components provide specific who, what, when, where and how, of an evidence-based health-care research question. 

Where will the trial be done?

Which settings (e.g. community, secondary care) and locations (e.g. countries, sites) will the trial be conducted in. Explain why these are appropriate for the study.

How will patients or service users be involved in trial design?

Describe the plans for involving patients, service users, and/or other public contributors in the design of the trial.

Who will be the participants, and why?

Good quality data collection starts with having the right people in the trial. Describe who the trial participants will be. This should include reference to the disease or condition prevalence and severity, and who can benefit from improved care. A table of eligibility criteria is useful, including a core set of sociodemographic characteristics (a helpful resource for this is PRO EDI. Also mention whether other relevant parties such as healthcare staff and patients have been involved in deciding who needs to be in the trial. For a collection of helpful resources to support the design of inclusive trials, see Inclusive Trials Resources.

How many participants will be needed, and why?

Describe how the sample size was determined, including all assumptions supporting the sample size calculation. If there are targets within the overall sample (e.g. that at least 45% of participants will be female) describe them and explain why these targets were chosen.

Who is the person(s) acting as guarantor for the sample size (and statistical analysis more generally)?

Give the name of the person with competence in trial statistics who acts as guarantor for thestatistical analysis of the trial.

Describe the intervention and comparator

Describe the intervention and comparator in sufficient detail to allow replication, including how, when, and by whom they will be administered. Explain the choice of comparator. Describe whether any concomitant care and interventions are allowed or not during the trial. A useful framework to describe the intervention is the TIDieR reporting standard.

What is/are your primary outcome(s)?

Give the primary outcome(s), including the specific variable being measured (e.g. systolic blood pressure); the analysis metric (e.g. change from baseline, final value, time to event); how this will be summarised (e.g., median, proportion); and time point for each outcome. Describe any measures to ensure the quality of the data.

What other outcomes will you collect?

Give the secondary outcome(s) and provide the same information as above for the primary outcome(s).

Are there any potential harms?

Describe how expected and unexpected harms are defined and how they will be assessed (e.g. systematically, non-systematically). Note whether you will apply any standardised language (e.g. MedDRA). Specify whether you will report all harms recorded in trial publications or only selected harms that meet certain criteria.

How do you know that these are the right outcomes to measure, especially the primary?

Describe how the outcome choice was made. Describe who has given input to this decision process (e.g. patients, health professionals, managers), including reference to Core Outcome Sets (COS) which allow harmonised reporting and minimise research waste: https://www.comet-initiative.org/

Trial teams often spend substantially more time on secondary outcomes than they do on the primary(s) (see Gardner et al., 2022) despite few if any secondary outcomes being considered in the sample size justification. Consider whether each of the secondary outcomes are needed to answer the research question.

When will outcomes be measured?

Describe the time schedule for participants. This should include when the intervention (including any run-ins and washouts) is delivered, and the timing of visits and outcome assessments. A diagram like that recommended by SPIRIT is highly recommended, and can be found at: https://www.consort-spirit.org

How will participants be allocated to intervention and comparator?

Describe the trial design (e.g. parallel group, crossover); type of randomisation, allocation ratio, framework (e.g. superiority, equivalence, non-inferiority, exploratory); who will generate the allocation sequence, how allocation will be concealed; and who has access to the random allocation sequence.

It is worth assessing the proposed allocation and blinding approach using relevant parts of a risk of bias assessment tool, such as ROBUST-RCT. See also the Cochrane Risk of Bias resources.

Who and how will blinding be achieved?

Describe who will be blinded, and whether those who assess outcomes are blinded. If blinding is not possible, explain what measures are being taken to protect against bias and ensure the validity of the trial results. Describe when unblinding is permissible.

What recruitment strategies will you use to ensure the trial recruits enough of the relevant people?

Describe the trial recruitment strategies. What is the evidence of effectiveness? How are these strategies evaluated?

A useful resource about barriers and strategies for effective recruitment and retention of participants in clinical research is the paper by Klompstra et al., 2025. See also a list of potential costs to be considered for recruitment and retention.

Who will take informed consent?

Describe who will obtain informed consent or assent from potential trial participants or authorised proxies, and how.

What retention strategies will you use to ensure you are able to collect outcome data from the people in the trial?

Describe the trial retention strategies. What is the evidence of effectiveness? How are these strategies evaluated?

A useful resource about barriers and strategies for effective recruitment and retention of participants in clinical research is the paper by Klompstra et al., 2025. See also a list of potential costs to be considered for recruitment and retention.

How has the feasibility of your proposed recruitment and retention strategies been discussed or tested with people such as health professionals and potential trial participants?

Describe how the approach to recruitment and retention has been discussed and tested together with those needed to make the trial feasible. These individuals could be potential trial participants, PPI groups, health professionals, managers etc.

What are the statistical methods you will use for the primary and secondary outcomes?

Describe the statistical methods that will be used to compare groups for primary and secondary outcomes. Describe who will be included in each analysis (e.g. all randomised participants).

How will missing data be handled?

Describe how missing data will be handled.  Justify the chosen techniques.

Are there any additional analyses?

Describe any additional analyses, such as subgroup analyses.

Will there be an interim analysis?

Describe any interim analyses and stopping guidelines, including who will have access to interim results and make the final decision to terminate the trial.