Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.
Kanters TA, van der Ploeg AT, Kruijshaar ME, Rizopoulos D, Redekop WK, Rutten-van MÓ§lken MPMH, Hakkaart-van Roijen L
Orphanet journal of rare diseases, 2017 12 13
Abstract
Pompe disease is a rare, progressive, metabolic disease, and the first treatable inheritable muscle disorder. Enzyme replacement therapy (ERT) with alglucosidase alfa is disease specific and the only medicinal product authorized for the treatment of Pompe disease. Costs of ERT are very high as for most orphan drugs. This study investigates the cost-effectiveness of ERT compared to supportive treatment in adult patients with Pompe disease.