OBJECTIVES: The NICE guideline on experience of healthcare for babies, children and young people recognizes the importance of patient involvement in healthcare decisions. Quantifying how conditions, and treatments, affect children's daily lives and overall quality of life (QoL) can help guide those decisions. Conditions affecting paediatric patients also often involve significant caregiver responsibilities resulting in considerable financial, emotional, and even physical burden.

Given potential challenges with collecting data in paediatric populations, it is not clear to what extent quantitative and/or qualitative data are being collected from patients and their caregivers. The use of data to support robust clinical and economic value arguments and their influence on HTA outcomes is also not captured.

METHODS: Assessments of pharmaceutical treatments for paediatric populations (non-oncology indications) conducted by NICE from January 2021 to June 2024 were identified. For each assessment, the instruments capturing paediatric PRO data and caregiver burden/QoL data were identified, along with any associated commentary.

RESULTS: Between January 2021 and June 2024, 37 technology appraisals or HST guidance documents for paediatric populations were published and 24 were in non-oncology populations. Overall, 14 (58%) assessments included at least one paediatric PRO instrument and 7 different generic paediatric PROs were identified, with PedsQL most commonly used (6 assessments). Only 2 (8%) studies included caregiver burden / QoL instruments alongside paediatric PROs. Committee discussions benefit from expert input from clinicians, patients, and families /caregivers to supplement and contextualize clinical data collected. Limitations around data collection, particularly for younger paediatric populations, are recognized.

CONCLUSIONS: Despite the importance of delivering patient-centered that can improve the child's quality of life, quantitative data on patient and caregiver perspectives are often sparse within submissions to HTA agencies such as NICE. Future studies should carefully evaluate ways in which paediatric patient/caregiver data can be collected to support clinical and economic value.