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 middle-income one (Colombia)

Abstract

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.

The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review

“…Agalsidase alfa, agalsidase beta, laronidase, sildenafil, iloprost and bosentan were approved under exceptional circumstances. The majority of the studies included were conducted in the USA [ 14 – 18 ], followed by the UK [ 19 – 23 ], and the Netherlands [ 13 , 24 , 25 ]. The remaining studies have been conducted in Spain [ 26 ], Australia [ 27 ] and Canada 28 ].…”

“…Costs and QALYs were generated for 2 settings: England and Colombia. 16 Castro-Jaramillo [ 19 ] 1: 300 000 Colombia: €153 405 b Eculizumab (Soliris) Paroxysmal Nocturnal Haemoglobinuria 1: 500 000 soc Markov-state-transition model €1 620 256 b Six different consequences from PNH or treatment were modelled. Patients may have more than 1 complication at the same time, resulting in a total of 47 disease states.…”

“…The remaining 11 studies used more sophisticated pharmacoeconomic models to evaluate costs and effects of a certain treatment, of which 7 used a Markov-state-transition model [ 13 , 15 , 18 , 19 , 25 , 26 , 28 ]. Other models used were patient-level simulation models ( N = 3) [ 20 , 24 , 27 ] and decision trees ( N = 1) [ 17 ].…”

“…Thirteen studies applied a sensitivity analysis [ 13 , 15 – 21 , 24 – 28 ]. Eight studies discussed the issue of generalizability for application in a different context [ 13 , 15 – 19 , 21 , 27 ]. CHEC-list scores of individual studies are shown in Additional file 1 .…”

“…Remarkable differences are observed when comparing the ICURs from different economic evaluations in Fabry disease (€351 622 to €3 282 252 per QALY) [ 21 , 25 ], Pompe disease (€153 405 to €1 043 868 per QALY) [ 19 , 24 ], and Gaucher disease (€43 532 to €432 540 per QALY) [ 13 , 14 ]. These differences can be attributed to the assumptions made; studies assuming survival with perfect quality of life [ 14 , 21 ], or a huge increase in survival rate [ 19 ] upon therapy result in lower but less realistic ICURs.…”