Addressing All PICOs in the JCA Dossier – Is It Feasible?

OBJECTIVES: Consolidating national PICOs from 16 EU Member States for cipaglucosidase alfa for the treatment of adult patients with late onset Pompe disease resulted in 13 PICOs. The aim of this research is to investigate whether it is feasible to address all identified PICOs in the Joint Clinical Assessment (JCA) dossier with currently available evidence.

METHODS: A systematic literature review was performed to identify randomized controlled trials that allow a direct or indirect treatment comparison for cipaglucosidase alfa in Pompe disease for all consolidated PICOs.

RESULTS: In the systematic literature review, only a small number of randomized controlled trials in Pompe disease was identified.

Although a direct treatment comparison for cipaglucosidase alfa is possible in some of the specified (sub-)populations, many PICOs cannot be addressed via a direct treatment comparison. Due to the different subpopulations and comparators, many PICOs have to be addressed via an indirect treatment comparison. Nevertheless, for some PICOs no adequate evidence for neither direct nor indirect treatment comparison is available, especially for subpopulations requiring ITC, in which the relevant patient subpopulation was not reported separately.

CONCLUSIONS: Due to a high number of expected PICOs, indirect treatment comparisons will be more frequently required for the JCA dossier. However, in order to address all PICOs and adequately prepare for the statistical analyses, a timely review of existing evidence as well as an identification of possible evidence gaps is essential for preparing the JCA dossier. Still, a relevant number of PICOs will remain without relevant evidence.