Families, including CYP who take glucocorticoid medication, must be included in a meaningful way from the beginning of research studies, in order for their voice to be considered when research is designed. This is particularly so in the case of CYP, in order that their voices remain central to paediatric research. Effective PPI is characterised through the UK National Standards for Public Involvement as: Support & Learning, Impact, Communications, Inclusive Opportunities, Working Together, Governance (6). The authors discuss how many of these features of effective PPI involvement were included within this virtual PPI initiative, whilst highlighting some of the barriers to implementation, and also outline how PPI can be used to inform future co-development of a PROM.
High quality PPI, that meets the participants where they are in terms of knowledge and understanding, has the ability to offer novel and innovative insights into the research process (9, 22). To achieve this, participants should be supported with adequate training and tools to understand the research process (23) and therefore be able to meaningfully contribute to nuanced discussions. In this initiative, the PPI group received training on key aspects of the research process, as well as bias in recruitment and measuring health outcomes. This was found to be one of the most enjoyable aspects of the initiative by the participants. Another strength of the initiative was the range of expertise working alongside the PPI group, including doctors with expertise in participants’ illnesses, and a psychologist with expertise in qualitative and PPI work. Members of both the large group event and the monthly PPI group were offered support from the research team outside of the PPI setting and signposted to further resources, although this was not adopted.
Participants were compensated for their time as per the NIHR INVOLVE guidelines (24) which is the nationally recognised payment for people who are patient experts and who are giving their time to support research. It was important to the research team to compensate the children in the same way as the adults, giving children equal value in the group as co-creators of the research process. Financial cost and time investment are two of the most commonly noted barriers to extensive PPI involvement in clinical studies (2, 25). Some have reported frustration that funding to support PPI prior to funding applications could be difficult to obtain and that local NHS and Higher Education Institution administrative practices may slow down prompt reimbursement and payment (26).
The lack of suitable ways of disseminating research findings to the wider public was identified as a particular barrier by the PPI group. Dissemination may be facilitated by patient groups and charity organisations (27), or through the use of ‘patient advocates’ (28). Large group PPI events, as the one described here, represent another route to disseminate key research findings to relevant patient groups.
The virtual setting of the large group event and monthly meetings presented both opportunities for inclusive research, and also challenges. For example, a benefit was that participants could raise their hand, use the chat box, or shout out when they wanted to contribute. This was then noticed by the facilitator (a medical doctor or psychologist) so CYP were as likely to be able to contribute as adults. However, it may have also meant that younger children missed some of the nuanced discussion because different mediums were used that may not have been accessible to them. It also presented opportunities for greater flexibility, facilitating representation from groups who may be traditionally excluded such as those with greater health needs, poor mobility or young children (29). However, this may have posed to a barrier to participation for those from low socioeconomic backgrounds, those with no access to technology, or those who were technology illiterate. Another strength of the online event was that participants joined from across the country, which would not have been possible in a face-to-face setting due to limited travel funding.
Participants of both the large group PPI event and the monthly PPI group series varied vastly in age, making it challenging to tailor the activities and discussions to all participants. There is a risk of over-burdening CYP with unreasonable expectations of contribution (30). To minimise this, the initiative held multiple short sessions with frequent breaks and encouraged CYP to creatively express themselves in whichever way they felt best, or forego participation if they did not feel ready. It is the role of the research team to find innovative ways in which children with a range of abilities and levels of understanding can meaningfully contribute to the research process (2). Ensuring diversity has been described a salient action for ensuring effective PPI (31) and the participants felt that more diversity within the genders of the CYP would have allowed the group to be more representative.
There was also diversity in previous experience with glucocorticoid medication, which informed participants’ opinions and outlooks. The researchers laid guidelines for respectful communication prior to starting discussion to create a safe and respectful space for people to contribute. The large group event began with online polls and quizzes to facilitate discussion and then split into smaller groups, in Zoom breakout rooms, for further topic exploration. The monthly PPI group began with allowing time for members to get to know each other, independent of their experience with glucocorticoid medication. The diversity of the participants ultimately became one of the strengths of the PPI group and participants reported that they found hearing each other’s experiences and forming positive relationships to be one of the most beneficial aspects of taking part. The research team also reported that they had gained new insight and in this way, PPI initiatives such as this one can help align patient and clinician perspectives.
The use of PROMs in healthcare also fosters a shift towards a patient-centred focus from a clinician/researcher centred-focus. Despite an acknowledgment that patient involvement is necessary to PROM development, few take the approach of this initiative where PPI forms an integral part of every part of the PROM development process. Weiring et.al., reported in a scoping review of patient involvement in PROMs that only 6.7% of studies reviewed featured patient involvement in all aspects of development (8).
Equally, few studies report PPI in identifying the need for PROM development or in determining which outcome to measure (8, 10).The James Lind Alliance uses a Priority Setting Partnership (PSP) approach where healthcare providers and patients work together to determine target areas for future research directions (32). This informed the approach of the PPI group towards determining research priorities in glucocorticoid-associated clinical research. These priorities reflected larger trends in patient-set priorities towards medication use in paediatric rheumatic conditions. A recent PSP in Netherlands exploring JIA also identified seeking alternatives (including diet-based and nutritional alternatives) and minimizing long-term side-effects of medication as research priorities (32).
The monthly PPI group also informed the development of a conceptual model based on their lived experience and designed key aspects of a future study developing a glucocorticoid-associated PROM. The group have advised on the scope of the PROM, identified key barriers to diverse recruitment within this patient group, and designed interviews to maximise value of input from CYP who participate. The research team held honest and open dialogue that the group’s proposals must be balanced with feasibility and scientific rigour. Here the group’s training on how research was conducted and the positive relationships between the research team and the PPI group fostered a collaborative, problem-solving approach. Ongoing input from the PPI group will continue to inform further PROM development, such as analysis of data, design of further studies (e.g. psychometric surveys or cross-cultural validation studies) and dissemination of findings (9).