Table 3.

Full description of the eleven Case Studies

Case study	Stage of research (research priorities; research design, and planning; research conduct and operations; dissemination, communication, post-approval)	Type of involvement	Who was involved	Outcomes	Things that need improving (gaps in practice)	Link to full Case Study
Funding decision-making for a digital solution for abdominal pain in children and young people	Research priorities	Virtual funding panel consisting of adult professionals including pediatricians, innovations leads, psychologists and 2 young people	2 young people (17 and 21, both female) from an existing Young Person’s Advisory Group	Active representation of youth members on a panel to select projects that received funding for a health application that was intended for use by children	Timing of meetings predetermined by project timetable and panel availability. Professional working week conflicts with YPAG availability requiring them to be absent from school/university	https://bit.ly/3tRyJDH
Identification of unmet medical needs from pediatric patients with a rare condition	Research priorities	Focus group with teenager patients living with an ultrarare disease	7 female patients from 12 to 16 years old	● Description of main characteristics of the patient journey ● Formulation preferences from the patients ● Acceptability of placebo ● Home nurse service ● Medical assessments were potentially included in the study. ● Acceptability of the screening process. ● Assessment of the tool to collect feedback about the palatability of the drug.	● The activity was performed with patients from only one hospital. Diversity was not included in the concept design of this activity. The sponsor didn’t see any concern about it ● Still awaiting feedback from the sponsor about if the outcomes were implemented so they can be shared with the patients.	https://bit.ly/3k5UlZJ
Feasibility study	Research design and planning; research conduct and operations; dissemination and communication.	Virtual meetings were held with parents who had experience of neonatal care or paediatric intensive care, Study Management Group Membership, and consensus meeting attendance.	Nine parents (five PICU parents, two NNU parents and two parents having had experience of both NNUs and PICUs)	● Provided valuable insights into the important issues for parents and carers for a future study ● Revision of the lay summary. ● Co-produced Patient Information Sheets, and interview questions. ● Co-produced a summary of the research findings in a visual abstract format and disseminated widely among the PICU and NNU parent communities.	Meeting face to face was extremely challenging for this group of parents. Study teams need to adapt their approaches to suit the demanding lifestyles of parents if they are to be involved meaningfully.	https://bit.ly/3k7DDZC
Core outcome setting for children with chronic kidney disease	Research priorities	Delphi Survey with chronic kidney disease patients. Two to three rounds of surveys were completed sequentially	72 patients around the world, 66% female age range : 8–14 (24, 12%), 15–18 (25, 12%), 19–21 (16, 9%) including French participants.	● Provide reliable consensus on core outcome sets across a range of chronic kidney disease, with an appropriate and dedicated methodology. ● Experts including children and young people, caregivers, and professionals. ● Establishment of a consensus core outcome set for paediatric nephrology trials, to improve quality of reporting and relevance of trails conducted in children and adolescents with chronic kidney disease.	● Challenging to obtain enough children and young people with CKD disease notably because of late participation of France in the process. ● More information developed by and for children and young people to better target them to participate was required. ● Evaluation of the patient’s experience is needed.	https://bit.ly/3kdKr8s
Core outcome set development	Research priorities	2 workshops were held during the International Children’s Advisory Network (iCAN) Research and Advocacy Summit held in 2018	70 young people (aged 10-18 years with a mix of healthy young people and those with experience of acute and chronic conditions).	● Children and young people provided valuable insights and recommendations to optimise core outcome set development with children and young people. ● Feedback from the workshops identified ways to better engage children and young people as participants in core outcome set studies, which will potentially improve the quality of such studies in the future.	Despite many children and young people taking part in the workshops, most of these came from high-income socio economic backgrounds within countries across Europe and North America. Future workshops or consultations would benefit from including children and young people from rarely included groups, such as black and minority ethnic groups and those from socio-economically disadvantaged backgrounds	https://bit.ly/3tJvwWz
Age-appropriate Patient-Reported Outcomes and Quality of Life Scales	Research priorities	Focus group with teenager patients living with an euro muscular rare condition and their parents	4 teenagers (11-18 years old, all female),and 5parents (4female, 1 male).	● Description of the patient journey. ● Preferences about treatment doses and administration. ● Preferences on QoL and PROMs scales. ● Information and educational materials for patients/families.	● All the patients and parents were from the same hospital. Diversity was not a key element in the design of this activity due to the limitations to recruit patients with his condition. ● Need for further information and materials to support the involvement of patients and parents in the activity.	https://bit.ly/2Xm7EMX
Randomised Controlled Trial investigating the use of a diagnostic technique to predict asthma exacerbations in children	Research design and planning; research conduct and operations; dissemination and communication.	YPAG (Scotcrn) 50%of the group had been recruited from a birth cohort that had studied the prevalence of asthma and wheeze since birth.	Group age range was 14–18 years.	Involved from planning stages and provided advice on logo, description of methodology, recruitment, patient information and lay summary.	No formal evaluation or record of the PPI experience from both CTU and YPAG perspective.	https://bit.ly/3Ce4yth
Developing multimedia interventions with children, young people, and families	Research design and planning; research conduct and operations; dissemination and communication.	YPAG (Generation R Liverpool) and formation of a Patient and Parent Advisory Group (PPAG)	12 young people(8–21 years old, 7female, 3 male); 3young adults between 19 and24 years (two female, one male) with long term health conditions, and three parents (all female) of young people with long term health conditions joined the PPAG	● Input and improvement to the patient-facing study documentation. ● Review of multimedia and written content for the multimedia interventions. ● Contributed to decisions regarding study design and governance. ● Promoted the study via social media. ● Contributed to the writing of presentations and publications arising from the TRECA study.	● Meeting face to face is challenging. ● PPAG members would have liked to meet the study team much earlier on in the process to form relationships. ● Paying young people and parents for their time is complicated. Having a payments policy and sharing this with members at the beginning is important. ● Diversity was an issue, involving younger age children was difficult and needs more consideration when planning PPI activities.	https://bit.ly/3tD0dwH
Reviewing documentation for participants to a clinical trial	Research design and planning; Patient information	YPAG Kids France focus group activity, advice on information documents for a future trial in a rare disease condition. Presentation and explanations about he trial and review of the documents	8 young people12 to 19 years old	● Input on the explanations and documentation of assent/consent given to participants to a future trial ● Changes proposed to improve the understanding of the trial design and the conduct of the study. ● Review of the content and readability	● Impact on authorizations was evaluated, but the impact on understanding, and recruitment/retention was not evaluated and missing. ● Experience of the participants and the Principle Investigator about the activity was not evaluated at the end of the involvement.	https://bit.ly/399e9VE
Good Lay Summary Practice- European Guidance	Dissemination, communication, and post approval phase	Face to face meeting with members of existing YPAGs and individual questionnaire	Albania (N = 11) France (N = 8) Italy (N = 7) Scotland (N = 4) Spain (N = 14)	● Recommendations for the pediatric lay summaries ● Visual format is preferable to text format. ● Comics, animations, and infographics were selected as the best format options for children between 12-18 years old. ● A glossary of terms will be useful to clarify and offer extended information about medical terms. ● Seeking the views of young people about the readability will make information more accessible to this age group.	● All the lay summaries were available in English not in the native languages of the 4 to 5 of the YPAGs involved. This can be a limitation to some young people due to their level of competence in the use of English. ● Further involvement of YPAGs from other European countries was desirable. ● Diversity was not a mandatory element in the design of the activity. ● Patient organisations representing pediatric conditions were involved during the public consultation of the guidance after this activity.	https://bit.ly/3nrs2qp
Lay summary of findings	Dissemination, communication, and post approval phase	iCAN completed the first lay summary video supported by one young person from iCAN KIDS Central Ohio	Pediatric Trials Network (PTN), International Children’s Advisory Network, Inc. (iCAN), One young person, a male, age 14.	● Provide valuable insight to researchers regarding the young person's perception, understanding, and clarity of the written lay summary. ● Offer a new method of dissemination of lay summary materials to young people and their families of the content within the lay summary so that it would be more understandable to others.	● Offer additional time to complete lay summary videos. ● Ensure that the young person is included at the beginning of the written lay summary to ensure the appropriateness of reading level, and content. ● Create more opportunities to develop lay summary videos to further advance new communication styles of lay summary to young people and their families.	https://bit.ly/3tJxk1S
Working with Regulators	Research design and planning; research conduct and operations; dissemination and communication.	Face to Face meeting, including a panel of youth members and US Food and Drug Administration (FDA)regulators	6 children with diagnosed rare conditions (including 5 female, 1 male) in the ages of 8-19 years old.	• Youth Input on the patient experience through sharing insight of their own personal rare disease journey. • Youth input focused on patient endpoints. • Heavy focus on accessibility, new medical treatment and medical device needs for all young people. • Feedback from youth members supported improvements within clinical research trial development, regulatory processes, and patient centred care for improved treatments and new medicine development.	• Experience and feedback of patient involvement was not evaluated. • While parents/guardians were invited to chaperone and attend the meetings, it had to be frequently communicated that the panel speakers were to be children (no adults). • Meeting Face to face requires that the patient needs to be supported for physical accommodations. • Young people living with rare diseases may have urgent and unforeseen medical needs that prevent them from traveling, so it is helpful to increase the number of representatives. • During COVID-19, this type of meeting cannot be replicated as face-to-face meetings are not allowable for safety and health reasons.	https://bit.ly/38ZrpvW