OBJECTIVES: Determining the expected national PICOs is crucial for the EU HTA process, but it can also be highly complex. During the EUnetHTA21 PICO exercise for cipaglucosidase alfa, 9 PICOs were identified using input from 10 (unknown) EU Member States. This analysis aims to reproduce the findings of EUnetHTA21 exercise using input from 16 Member States (Germany, Denmark, France, Italy, Poland, Spain, Ireland, Cyprus, Malta, Greece, Sweden, Norway, Netherlands, Belgium, Luxembourg, Austria), and investigates, how the results are impacted when using a subsample of different payer archetypes.

METHODS: For cipaglucosidase alfa, used in the treatment of adult patients with late-onset Pompe disease, national PICOs were identified from 16 different Member States. These PICOs were consolidated and compared to the results of the EUnetHTA21 PICO exercise. In a second step, this procedure was repeated using a sample of 8 Member States representing different payer archetypes.

RESULTS: Consolidating PICOs from 16 EU Member States resulted in a total of 13 PICOs. 8 out of 9 PICOs identified in the EUnetHTA21 PICO exercise could be reproduced. Our consolidation also identified 5 additional PICOs related to further subpopulations and comparators not included in the original PICO exercise.

CONCLUSIONS: Although Pompe disease is a rare genetic disorder with only a few medicinal products authorized in the EU, input from 16 EU Member States resulted in a higher number of PICOs for cipaglucosidase alfa compared to the EUnetHTA21 PICO exercise. Relying on PICOs from a limited number of representative payer archetypes is not an adequate shortcut. To anticipate all PICOs, involvement of all Member States is necessary.