The cost-effectiveness of enzyme replacement therapy (ERT) for the infantile form of Pompe disease: comparing a high-income country’s approach (England) to that of a middleincome one (Colombia)

Castro Jaramillo, Héctor Eduardo

The cost-effectiveness of enzyme replacement therapy (ERT) for the infantile form of Pompe disease: comparing a high-income country’s approach (England) to that of a middleincome one (Colombia)

dc.contributor.author	Castro Jaramillo, Héctor Eduardo
dc.contributor.corporatename	Pontificia Universidad Javeriana. Facultad de Medicina. Departamento de Epidemiología Clínica y Bioestadística
dc.coverage.spatial	Colombia	spa
dc.date.accessioned	2020-04-24T20:39:38Z
dc.date.accessioned	2020-05-08T15:26:01Z
dc.date.available	2020-04-24T20:39:38Z
dc.date.available	2020-05-08T15:26:01Z
dc.date.created	2012
dc.description.abstractenglish	Objectives Determining the cost-effectiveness of enzyme replacement therapy (ERT) for the classical infantile form of Pompe disease (complete acid α-glucosidase defi ciency-related) in two different settings: England and Colombia. Pompe disease is very rare (1:40,000 births incidence). Methods A literature review was made and historic databases searched for National Health Service (NHS) reimbursed costs in England and by health insurers in Colombia; expert opinion was elicited. Two Markov models were constructed for comparing both countries; alive with symptoms and dead were the transition states used. Patients aged ≤ 6 months receiving ERT were assumed to have 75 % survival rate and better health-related quality of life (HR-QoL) compared to those without treatment (0.700 HR- QoL using the EQ-5D scale). Results The incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained was £234,307.7 for England and £109,991 for Colombia. Uncertainty about fi nal HR-QoL with ERT, disease progression and cost from palliative care had the biggest impact on the ICER in both models. If ERT costs were reduced to 10,000 times per dose and HR-QoL was 0.750-0.820 ICER, then £165,000 could be attainable for England and £65,000 for Colombia. Transaction costs per case in Colombia were high. Conclusions ERT was more effective than no ERT in treating infantile Pompe disease, but high levels of uncertainty still remain about survival and progression rates and QoL in the long-run. ICERs were high compared to CE thresholds. Manufacturers’ ERT costs and monopoly had a major impact on fi nal CEA results.	spa
dc.description.paginas	143-155	spa
dc.description.quartilescopus	Q3	spa
dc.format	PDF	spa
dc.format.mimetype	application/pdf	spa
dc.format.soporte	Papel / Electrónico	spa
dc.identifier	https://revistas.unal.edu.co/index.php/revsaludpublica/article/view/17802	spa
dc.identifier.issn	0124-0064 / 2539-3596 (Electrónico)	spa
dc.identifier.uri	http://hdl.handle.net/10554/48427
dc.language	spa	spa
dc.rights.licence	Atribución-NoComercial 4.0 Internacional	*
dc.rights.uri	http://creativecommons.org/licenses/by-nc/4.0/	*
dc.source	Revista de Salud Pública; Vol. 14 Núm. 1 (2012)	spa
dc.subject	Enfermedad de Almacenamiento de Glucógeno Tipo II	spa
dc.subject	Glucogenosis generalizada	spa
dc.subject	Glucogenosis 2	spa
dc.subject	Enfermedad por deﬁciencia de maltasa ácida	spa
dc.subject	Enfermedad de Pompe	spa
dc.subject	Enfermedad por deﬁciencia de lisosoma alfa-1	spa
dc.subject	4-Glucosidasa	spa
dc.subject	Análisis costo-beneﬁcio	spa
dc.subject	Costo efectividad	spa
dc.subject	Calidad de vida	spa
dc.subject.keyword	Glycogen storage disease type II	spa
dc.subject.keyword	Pompe disease	spa
dc.subject.keyword	Cost and cost analysis	spa
dc.subject.keyword	Cost-benefit analysis	spa
dc.subject.keyword	Quality of life	spa
dc.title	The cost-effectiveness of enzyme replacement therapy (ERT) for the infantile form of Pompe disease: comparing a high-income country’s approach (England) to that of a middleincome one (Colombia)	spa
dc.type	info:eu-repo/semantics/article
dc.type.hasversion	http://purl.org/coar/version/c_ab4af688f83e57aa
dc.type.local	Artículo de revista	spa