OBJECTIVES : When a medicinal product is to be used in the paediatric population for the same indication(s) as those studied and approved in adults, the disease process is similar in adults and paediatric patients, and the outcome of therapy is likely to be comparable. Therefore, extrapolation from adult efficacy data may be appropriate. The aim was to find out on which studies (paediatric/adult) the benefit assessments in Germany, France, and the UK were based, whether an extrapolation was carried out and which benefit decisions were made. The evaluation was based on the active substances blinatumomab, brivaracetam, dolutegravir, teduglutide and vandetanib.

METHODS : The EMA Assessment Reports were used to identify studies relevant to paediatric marketing authorisation procedures. IQVIA HTA Accelerator data were used to analyze the clinical evidence included in the evaluation procedures, whether extrapolation was carried out, and the benefit decisions of G-BA, HAS, and NICE.

RESULTS : The main studies were exclusively paediatric studies across all five paediatric registration procedures. In four out of five procedures, the EMA already addressed an extrapolation approach. For four out of five active substances, the evidence base for the G-BA included an extrapolation. Of these, the active substances dolutegravir, teduglutide and vandetanib were already evaluated (no additional benefit, not-quantifiable, indicator of non-quantifiable added benefit). The HAS evaluated three of the five active substances in the paediatric indications and made solely for dolutegravir a decision based on extrapolation of data. NICE assessed only teduglutide and did not include extrapolation data.

CONCLUSIONS : The evidence base of benefit assessments differs between the decision-making bodies G-BA, HAS, and NICE. G-BA submissions included more often extrapolation data than those of HAS or NICE.