Table 1

The key issues encountered and recommendations for developing and conducting the study

ThemeIssueRecommendation
Developing the study teamDeciding on the team membersEven if organically developed, once the core team is formed, it would be helpful to quickly identify what further input is needed and which professional or patient groups to involve. Agree in advance the roles and responsibilities of each team member and decide on whether or how new members will be approached or included. Team members from different disciplines may make the study stronger, but the team needs to be small enough to resolve conflicts and act quickly.
Having an advisory groupConsider having an external study advisory board with experts in the field, but be clear about their roles and remit, and how they will be credited in the publications. Using the group as a sounding board may help in terms of interpreting the data as they come in.
Patient and Public Involvement (PPI)Involve PPI early. Having pre-existing PPI members involved in disease-specific research is helpful because they would have had the required training and experience to be able to offer their input within high-pressure, fast-pace, research encounters. PPI members can help develop a Plain English Summary and comment on concerns about questionnaire fatigue. Develop one early, so that it can be used for publicity to help improve recruitment, but also for funding bodies, ethics applications and dissemination. PPI members also help researchers keep the needs of the patient at the forefront.
Two people per roleHaving two people per role (eg, two neurologists, two psychologists, two Register staff) created some differences in opinion, but also ensured continuity (in case people became ill). While this doubling up of roles is helpful, clear primary and deputy roles and responsibilities need to be agreed in advance of the study commencing.
Conducting the studySurvey platformUse pre-existing disease-specific national registers to host new studies where possible; however, be aware that their pre-existing workload may delay new studies. Where registers exist, consider whether they can be adapted to include ‘control’ participants’ data also (where this is not available as part of the register). Where registers do not exist, consider developing local registries. Explore which platforms (eg, REDCap and Qaultrics) have been recently used successfully by members of your study team.
Ethical approvalConsider and enquire with relevant ethics committees whether amendments to previous ethical approvals will be sufficient for the new study, rather than having to apply for fresh ethical approval (which could be time-consuming). Explore whether universities and institutions have any emergency, fast-track ethical approval processes.
Ever-growing questionnaire – adding new questionsBe prepared for the questionnaire needing to change—so prepare for the change—and reflect on what needs to be put in place to enable or facilitate the change. There are bound to be gaps in questionnaires. Weigh up the benefits of asking certain questions and the risks of increasing the length of the survey and resultant respondent fatigue. Beware of and manage ‘mission creep’.