A systematic review to identify how children and young people were included in the development of pediatric core outcome sets.

OBJECTIVES: Core outcome sets (COS) are an agreed upon set of outcomes that should be evaluated in a clinical trial, allowing data from similar areas of health care to be compared and pooled in meta-analyses, informing whether treatments work and/or harm. Pediatric COS have been developed by identifying outcomes that are important to relevant stakeholders, and recently the importance of including children and young people (CYP) as stakeholders has been recognized. While research on the overall development of pediatric COS exists, no comprehensive review has examined methods implemented by COS developers to include CYP in the development process. This systematic review aims to establish current practice and identify issues in including CYP in developing a pediatric COS, particularly in the context of rare conditions.

STUDY DESIGN AND SETTING: We conducted a systematic review of studies in the Core Outcome Measures in Effectiveness Trials (COMET) initiative database that developed pediatric COS for children and young people (CYP; 0-18 years). Data were extracted on methods that used to include CYP, barriers to their inclusion and stakeholder participation. Authors were contacted when methodological details were unclear or insufficiently reported.

RESULTS: A total of 70 articles corresponding to 53 pediatric COS were analyzed, 18 (34%) included CYP and 16 (30%) were developed for a rare condition/condition with a rare form. CYP were included in advisory groups (six of 18 COS), pilot surveys/questionnaires (three of 18 COS), focus groups (two of 18 COS), workshop (one of 18 COS), qualitative interviews (eight of 18 COS), Delphi (and other) surveys (15 of 18 COS), and consensus meetings (five of 18 COS). COS developers adapted methods for patient and public involvement and engagement work to improve participation in COS, include CYP in generating outcomes, and reach consensus on the most important outcomes to include in the COS. Barriers to including CYP were age and the condition/area of COS development.

CONCLUSION: Our findings show that with appropriate adaptations, CYP can be included in COS development, regardless of age and communication needs, or whether conditions are rare or common. This review proposes adaptations to refine current pediatric COS methodology to increase CYP inclusion.

PLAIN LANGUAGE SUMMARY: Core outcome sets (COS) are lists of the most important things to measure in a clinical trial-such as symptoms, side effects, or any changes in a person's health-to help show whether a treatment works. When a COS is used in clinical trials for a particular condition, the results are more helpful because they can be combined and compared. This makes it easier to see whether a treatment is helping or harming the people who have that condition. A COS is developed in different stages; first, a list of important things to measure, called outcomes, is put together. One way to make this list is by asking the people who know the condition best-such as patients who live with it and the health-care professionals or researchers who treat or study it-, and what they think should be included. Next, everyone involved votes on which outcomes they think are the most important. The outcomes that receive the most support across all groups are then brought together to form the final COS. To make a COS useful for a condition that affects children and young people (CYP), it is very important to involve CYP with the condition themselves. Some studies described the general steps of creating a COS for CYP, but there is very little information about exactly how COS developers adjusted the methods at each stage so that CYP could take part. This study examines this aspect more closely. We looked at COS that have been created for CYP aged 0 to 18 years and gathered information on how developers adjusted the methods to include CYP and what challenges they faced. Where details were missing or unclear, we contacted the COS developer. Our results showed that one-third of the COS analyzed included CYP at different stages of COS development. Developers used approaches that helped CYP express what outcome mattered most to them, agree on priorities with other groups, and help develop better ways for CYP to take part in research. CYP were not included in COS development when they were young and when their condition made it difficult to participate. Our study shows that by making appropriate adjustments, CYP can be included in COS development, irrespective of age, communication differences, or the type of condition. We propose adaptations to refine current methods to make CYP participation easier and more effective.