Navigating Methodological Challenges and Informed Consent in Pediatric Research: A Scoping Literature Review

OBJECTIVES: In the European health policy space, unmet medical needs are increasingly recognised as essential factors to steer pharmaceutical research and development towards a patient-centred healthcare system. However, the needs of challenging populations, such as paediatric patients, are often overlooked, resulting in substantial unmet needs. This study aims to provide an overview of challenges reported in literature on unmet health related needs in paediatric patients, as well as informed consent forms (ICFs) used in paediatric research.

METHODS: This scoping literature review is a case study on paediatric blood cancer patients, following the PRISMA extension for scoping reviews. Two databases were included, PubMed and Embase. Descriptive data as well as qualitative data on methodological challenges and quantitative data on ICF was obtained from the included articles.

RESULTS: In total, 52 articles were included for analysis. Methodological challenges in research on paediatric patients can be categorised by research stage. During recruitment, obtaining a representative sample can be challenging. Moreover, regarding data retrieval, discrepancies may arise between parent and child perspectives and researchers must be aware of parental dominance when interviewing children. Lastly, distinguishing development and disease-related changes can be challenging.

ICFs were analysed in relation to the patients’ ages. In studies addressing minors, there was a tendency to involve adolescents rather than younger children. In most cases, parental informed consent (IC) was obtained, complemented with patients’ assent or IC. When studies focused on the needs of younger children, parents typically served as the respondents, either alone or with their child. In the latter case, an equal number of studies obtained IC from parents alone and complemented with the patients’ IC or assent.

CONCLUSIONS: This study provides an overview of methodological challenges and ICFs used in studies regarding research on identifying unmet health related needs in paediatric patients.