The European journal of health economics : HEPAC : health economics in prevention and care
The cost-effectiveness of enzyme replacement therapies versus best supportive care for treating late onset Pompe disease in the UK NHS.
Matthew Walton, Nyanar Jasmine Deng, Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, Helen Fulbright, Chong Yew Tan, Robin Lachmann, Rachel Churchill, Robert Hodgson
Published: 202510.1007/s10198-025-01868-2
Abstract
OBJECTIVE: To assess the cost-effectiveness of enzyme replacement therapy (ERT) compared with best supportive care (BSC) for late-onset Pompe disease (LOPD) in a UK NHS setting. METHODS: A discrete event simulation model was developed using data from…
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