Unlocking Value: Assessing the Cost-Effectiveness of Pitolisant Wakix®in Portugal
Author(s)
Daniel Figueiredo1, Joana Rita Abrantes, MsC2.
1AIBILI - Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal, 2AIBILI, Coimbra, Portugal.
1AIBILI - Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal, 2AIBILI, Coimbra, Portugal.
OBJECTIVES: Pitolisant (Wakix®) is an orphan medicine indicated for the treatment of narcolepsy, with (type I) or without cataplexy. As a novel medicine, no other treatments are approved in Portugal for this indication, although some exists in other countries. In the Portuguese context, a pharmacotherapeutic evaluation revealed a major added therapeutic value compared to the best supportive care for treating type I narcolepsy. In parallel, an economic evaluation was performed. To support reimbursement decisions by the Portuguese regulator, a cost-effectiveness analysis adjusted to the Portuguese population was carried out. The study population considered were adults with narcolepsy type I.
METHODS: The cost-effectiveness analysis model used is a decision tree model, developed according the Portuguese legislation, clinical protocols and medical practice. Given that this disease remains underdiagnosed and underreported in Portugal, we consulted an expert panel of physicians treating narcoleptic patients in Portugal . Their input was considered to build, validate and populate the model. The effectiveness outcome was the reduction of cataplexy events, weighted against incremental cost to the Portuguese Health System to calculate the ICER.
RESULTS: The mean age at diagnosis is 36.4 years for Portuguese patients with type I narcolepsy, with equal gender distribution. The model outcomes estimated a cost difference of 18,668 € between Pitolisant (Wakix®) and its comparator (best supportive care) over a patient's lifetime for Portuguese Health System and that. Pitolisant (Wakix®) prevents, on average, 1853 cataplexy events over lifetime, indicating an ICER of €10.08 per cataplexy event prevented.
CONCLUSIONS: The results suggest a favourable cost-effectiveness profile especially considering th high burden of disease burden and that no therapeutic alternatives are available. While uncertainty remains due to limited local epidemiological data, expert input and sensitivity analyses help mitigate it. This study presents a new model and highlights the need to generate and integrate local data into health technology assessments.
METHODS: The cost-effectiveness analysis model used is a decision tree model, developed according the Portuguese legislation, clinical protocols and medical practice. Given that this disease remains underdiagnosed and underreported in Portugal, we consulted an expert panel of physicians treating narcoleptic patients in Portugal . Their input was considered to build, validate and populate the model. The effectiveness outcome was the reduction of cataplexy events, weighted against incremental cost to the Portuguese Health System to calculate the ICER.
RESULTS: The mean age at diagnosis is 36.4 years for Portuguese patients with type I narcolepsy, with equal gender distribution. The model outcomes estimated a cost difference of 18,668 € between Pitolisant (Wakix®) and its comparator (best supportive care) over a patient's lifetime for Portuguese Health System and that. Pitolisant (Wakix®) prevents, on average, 1853 cataplexy events over lifetime, indicating an ICER of €10.08 per cataplexy event prevented.
CONCLUSIONS: The results suggest a favourable cost-effectiveness profile especially considering th high burden of disease burden and that no therapeutic alternatives are available. While uncertainty remains due to limited local epidemiological data, expert input and sensitivity analyses help mitigate it. This study presents a new model and highlights the need to generate and integrate local data into health technology assessments.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE744
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Rare & Orphan Diseases