Is Pediatric Indication an Implicit Driver of the French HTA Appraisal Outcomes?

OBJECTIVES: A previously published analysis of meeting transcripts of the Haute Autorité de Santé (HAS) suggested that products indicated for children tended to obtain more favorable ratings of clinical added value (ASMR). This study compared the ASMR ratings between products indicated for pediatrics and adults using structured data extraction from Transparency Committee reports.

METHODS: This study was performed using data from HAS appraisals made between 2018 and 2023, recorded in the NaviHTA database. First, the frequencies of different ASMR ratings for products with pediatric and other indications were analyzed. Second, the evidence submitted to HAS and appraisal outcomes were compared between pediatrics and adults for products appraised in both indications.

RESULTS: There were 163 appraisals for pediatric indications and 559 for adults. The percentage of ASMR V (no improvement) ratings was 58.28% for children vs. 68.71% for adults. This difference was more pronounced in appraisals based on single-arm trials (SAT), where 18.75% of pediatric indications achieved ASMR II-III vs. 3.23% achieving ASMR III; and 59.38% achieved ASMR V for children vs. 90.32% for adults. Eleven drugs were assessed for both populations for the same indication, with randomized controlled trials available for 10/11 adult cases and 5/11 pediatric cases. Pediatric ASMR ratings were either equal to (7/11) or more favorable (4/11) than adult ratings. Of the 4 cases where pediatric ASMR was more favorable, three relied solely on evidence from SAT without external control.

CONCLUSIONS: Medicines indicated for children are more likely to receive favorable ASMR ratings from the HAS, suggesting a greater perceived medical need or benefit in the pediatric population. These findings may encourage manufacturers to submit for indication extensions to pediatrics even when available evidence is limited.