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Commentary

Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI

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Pages 1285-1293 | Accepted 12 Mar 2009, Published online: 07 Apr 2009

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Maurizio Scarpa, Cinzia Bellettato & Christina Lampe. 2016. Drug Discovery and Evaluation: Pharmacological Assays. Drug Discovery and Evaluation: Pharmacological Assays 4053 4069 .
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Aris Angelis, David Tordrup & Panos Kanavos. (2015) Socio-economic burden of rare diseases: A systematic review of cost of illness evidence. Health Policy 119:7, pages 964-979.
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Rita FerlaPamela ClaudianiGabriella CotugnoPaola SacconeElvira De LeonibusAlberto Auricchio. (2014) Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease. Human Gene Therapy 25:7, pages 609-618.
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Eline Picavet, Thomas Morel, David Cassiman & Steven Simoens. (2014) Shining a light in the black box of orphan drug pricing. Orphanet Journal of Rare Diseases 9:1, pages 62.
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Sandra Jester, Julia Larsson, Erik A Eklund, Domniki Papadopoulou, Jan-Eric Månsson, Albert N Békássy, Dominik Turkiewicz, Jacek Toporski & Ingrid Øra. (2013) Haploidentical stem cell transplantation in two children with mucopolysaccharidosis VI: clinical and biochemical outcome. Orphanet Journal of Rare Diseases 8:1.
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Anuradha Ganesh, Zandre Bruwer & Khalid Al-Thihli. (2013) An update on ocular involvement in mucopolysaccharidoses. Current Opinion in Ophthalmology 24:5, pages 379-388.
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Eun Cho, Eun-Cheol Park & Myoung Sheen Kang. (2013) Pitfalls in Reimbursement Decisions for Oncology Drugs in South Korea: Need for Addressing the Ethical Dimensions in Technology Assessment. Asian Pacific Journal of Cancer Prevention 14:6, pages 3785-3792.
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Jennifer A. GorzelanyMark P. de Souza. (2013) Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?. Science Translational Medicine 5:178.
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Wilcken Bridget. (2012) Screening for disease in the newborn: the evidence base for blood-spot screening. Pathology 44:2, pages 73-79.
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Joseph Muenzer, Olaf Bodamer, Barbara Burton, Lorne Clarke, Gudrun Schulze Frenking, Roberto Giugliani, Simon Jones, Maria Verónica Muñoz Rojas, Maurizio Scarpa, Michael Beck & Paul Harmatz. (2011) The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus. European Journal of Pediatrics 171:1, pages 181-188.
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EMILY A. LARGENT & STEVEN D. PEARSON. (2014) Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases. Hastings Center Report 42:1, pages 27-34.
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Pamela Holtzclaw Williams. (2011) Policy Framework for Rare Disease Health Disparities. Policy, Politics, & Nursing Practice 12:2, pages 114-118.
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Alain Denis, Lut Mergaert, Christel Fostier, Irina Cleemput & Steven Simoens. (2010) Issues Surrounding Orphan Disease and Orphan Drug Policies in Europe. Applied Health Economics and Health Policy 8:5, pages 343-350.
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Ciaran O’Neill. (2010) How can the risk that orphan drugs present to budgets be managed better?. Clinical Therapeutics 32:9, pages 1640-1641.
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Gregory M Pastores. (2010) Galsulfase: enzyme therapy for mucopolysaccharidosis type VI (Maroteaux–Lamy syndrome). Pediatric Health 4:1, pages 17-24.
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Bryan A. LiangTim Mackey. (2010) Reforming Off-Label Promotion to Enhance Orphan Disease Treatment. Science 327:5963, pages 273-274.
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