OBJECTIVES: Drug development for children and young people (CYP) is complex due to methodological, operational, and ethical challenges which are perpetuated in health technology assessments (HTA). Most drugs for CYP are used off label or off license reflecting the current pediatric treatment landscape, although several recent regulatory initiatives aimed to change this trend. We aimed to assess National Institute of Health and Care Excellence (NICE) technology appraisals (TA) for CYP and to identify methodological challenges unique to this population.

METHODS: A search was conducted using the NICE search interface for TAs targeting CYP from July 2013 to February 2023. Terminated appraisals and medical device appraisals were excluded. Data extraction was standardized and included general information, clinical and economic assessments, and committee critiques.

RESULTS: The search yielded 17 published TAs, of which 13 were included (three terminated and one on medical devices). Seven TAs received positive recommendations, one was rejected, and five were recommended with conditions (i.e., until further data collection or with a restricted patient population). The TAs spanned 10 diseases, including epilepsy (n=3) and psoriasis (n=2). The main criticism centered on utility values in economic modeling: absence of a mapping algorithm to convert EQ-5D-Y, lack of EQ-5D population norms, and extrapolation from adult utility value sets. Additional concerns were related to the trial design of CYP treatments and confidence in data generalizability such as uncertainty in long-term benefits due to small sample sizes, short follow-ups, and notably patient heterogeneity reflecting wider inclusion criteria.

CONCLUSIONS: Few technologies for CYP were assessed by NICE although most were approved. This may reflect slower drug development for this population or longer delays in the HTA process. Unique input challenges for economic modeling and data extrapolation from adult studies create assessment uncertainties. Equity issues for CYP remain an under-researched HTA topic.