An Integral Approach to Assess the 3 Human Normal Immunoglobulin Formulations for Primary Immunodeficiency in Portugal: Combining Costs, Patient's Preferences, and HCP Choices

OBJECTIVES: The aim of this study was to evaluate human normal immunoglobulin formulations (facilitated and conventional subcutaneous or intravenous immunoglobulin replacement therapies – fSCIG, cSCIG, IVIG) in the treatment of primary immunodeficiency diseases (PID) in Portugal, considering three dimensions: costs per treatment, patients’ preferences, and Healthcare Professionals’ (HCP) choice.

METHODS: A cost-minimization analysis was performed to compare the costs for IG formulations, assuming equivalent outcomes (efficacy/safety), for both National Healthcare System (NHS) and societal perspectives, based on a literature review and official sources. An expert panel was conducted to validate literature-based assumptions, healthcare resources used and HCP preferences, ensuring its adequacy to the Portuguese reality, in 2024. Patient preferences were taken from the literature. Variables as treatment, travel, productivity, school absenteeism, leisure time costs, treatment criteria, hospital capacity, and patient preference were assessed.

RESULTS: From both NHS and Society’s perspectives, SCIG formulations are less costly than IVIG (adult/pediatric: -498€/-498€ and -714€/-781€ per patient, respectively). An additional cost associated with infusion pumps and consumables can be applied to cSCIG (up to 1000€ per patient), varying by hospitals. In Portugal, cSCIG incurs a burden of around 11 days annually related with administration, whereas IVIG and fSCIG require 9 and 5 days, respectively. Considering HCP’s perspective, fSCIG seems to be the preferred therapy choice, as it enables the combination of most criteria: lower overall cost, lower supply risk, patient preference, outpatient care capacity. From patients’ perspective, treatment convenience, time spent and frequency of administration are particular concerns that must be considered and fCSIG seems to be the best option.

CONCLUSIONS: Considering PID patients in the Portuguese setting, fSCIG formulation presents itself as a cost-saving option for the NHS and societal perspectives. On top of that, fSCIG seems to be the preferred option for patients and is aligned with all HCP treatment preferences.