Pompe disease places a substantial economic burden on patients, families, healthcare systems, and society. |
The majority of costs associated with the management of Pompe disease are driven by enzyme-replacement therapy (ERT), the only currently approved treatment. |
The incremental cost-effectiveness ratio (incremental cost per quality-adjusted life-year gained) of ERT ranges widely, but is consistently high when compared with established cost-effectiveness thresholds. |
1 Background
2 Methods
2.1 Search Strategy
2.2 Selection Criteria
2.3 Quality Assessment
2.4 Data Extraction and Analysis
3 Results
3.1 Overview
Publication (quality rating) | Study objective | Methods | Economic data | Outcome(s) evaluated |
---|---|---|---|---|
Studies reporting on economic burden of IOPD | ||||
Castro-Jaramillo (2012) [29] (3/9) | Estimate the cost-effectiveness of ERT vs no ERT (supportive therapy) in two different settings: England and Colombia | Deterministic Markov (annual cycles) model using published literature from a health system’s perspective over a 20-year time horizon (n = NR). Discount rate of 5% on costs and effects | ERT treatment, administration, complications Supportive care EQ-5D utilities | Cost-effectiveness based on cost per QALY gained |
Kanters et al. (2014) [30] (5/9) | Estimate the cost-effectiveness of ERT vs no ERT (supportive therapy) in a Dutch population | Patient-level simulation model (6-month cycles) using patient-level data from a societal perspective over a lifetime time horizon (n = 12). Discount rate of 4% on costs and 1.5% on effects | ERT treatment, administration Other HCRU, informal care EQ-5D utilities | Cost-effectiveness based on cost per QALY gained |
Studies reporting on economic burden of LOPD | ||||
Kanters et al. (2011) [31] (4/9) | Estimate burden of illness of patient not on ERT including societal costs, use of home care and informal care, productivity losses, and losses in HRQoL in a Dutch population | Longitudinal study (January 2005 to October 2009) of 92 patients seen at Erasmus Medical Center. Patients included those not on ERT. Data collected via questionnaire every 6 months and monetized using Dutch unit costs (n = 80) | Hospitalization, ambulatory visits Non-ERT meds Labs, devices Informal care, productivity loss EQ-5D utilities | Cost of supportive care Health utilities |
Kanters et al. (2015) [32] (3/9) | Assess properties of two measures to estimate health state preferences, the EQ-5D and the SF-6D in a Dutch population | Longitudinal study (January 2005 to August 2011) of 110 patients seen at Erasmus Medical Center. All Dutch patients included data collection of EQ-5D and SF-36 (n = 110) | EQ-5D utilities Mapped SF-6D utilities | Health utilities |
Winquist et al. (2014) [33] (2/9) | Assess the validity to apply a standardized policy framework to fairly evaluate rare disease drugs in Ontario, Canada | Retrospective observational cohort study by the DRDWG to apply to policy framework to 7 rare diseases (n = NR) | ERT treatment | Cost per patient Budget impact |
Kanters et al. (2017) [36] (5/9) | Estimate the cost-effectiveness of ERT vs no ERT (supportive therapy) in a Dutch population | Patient-level simulation model using patient-level data from a societal perspective over a lifetime time horizon (n = 283). Discount rate of 4% on costs and 1.5% on effects | ERT treatment, administration Hospitalization, ambulatory visits Home care, diagnostics, Medical aids Informal care, productivity loss EQ-5D utilities | Cost-effectiveness based on cost per QALY gained |
Studies reporting on economic burden of IOPD plus LOPD | ||||
Guo et al. (2012) [34] (2/9) | Describe the associated drug utilization and spending trends in the US Medicaid Program | Retrospective analysis using the National Medicaid pharmacy claims database from 2nd quarter of 2006 through 2nd quarter of 2011 (n = NR) | ERT treatment per prescription | Cost per prescription Budget impact |
Wyatt et al. (2012) [35] (2/9) | Estimate burden of illness of patient including societal costs, use of home care and informal care, productivity losses, and losses in HRQoL in England | Cohort study including prospective and retrospective clinical- and patient-reported data (LOPD, n = 65; IOPD, n = 12) | ERT treatment, administration Other HCRU, informal care EQ-5D utilities | Total cost of care Health utilities |
3.2 Health Economic Evidence of IOPD
3.2.1 Management of IOPD is Associated with a Substantial Economic Burden
3.2.2 The Costs of Treating IOPD with ERT is Substantial
3.2.3 Cost-Effectiveness of ERT in IOPD
Author, year | Currency | Country | Treatment (Myozyme) | ICER | Inflation-adjusted from data year to 2017a | Currency and inflation-adjusted (2017 USD)a |
---|---|---|---|---|---|---|
Kanters et al. (2014) [30] | 2009 euros | Netherlands | 40 mg/kg/week | €1,043,868 per QALY gained | €1,174,210 | $1,323,207 |
Kanters et al. (2014) [30] | 2009 euros | Netherlands | 20 mg/kg/2 week | €286,114 per QALY gained | € 321,840 | $362,678 |
Castro-Jaramillo (2012) [29] | 2010 GBP | England | 20 mg/kg/2 week | £234,308 per QALY gained | £269,674 | $347,070 |
Castro-Jaramillo (2012) [29] | 2010 GBP | Columbia | 20 mg/kg/2 week | £109,991 per QALY gained | £145,183 | $186,851 |
3.3 Health Economic Evidence of LOPD
3.3.1 LOPD is Associated with a Substantial Economic Burden
Author, year | Currency | Country | Costs included | Reported mean annual costs | Inflation-adjusted mean annual costs from data year to 2017a | Currency and inflation-adjusted mean annual costs (2017 USD)a |
---|---|---|---|---|---|---|
Kanters et al. (2011) [31]b | 2009 euros | Netherlands | Medical: Hospital days, ICU, nursing home, ambulatory care, home care, medication, tests, procedures, devices Direct non-medical: Transportation, other non-medical costs Indirect: informal care, productivity losses | Overall: €22,475 Direct medical: €13,679 Direct non-medical: €421 Indirect: €8374 | Overall: €25,281 Direct medical: €15,387 Overall indirect: €9420 Indirect (no productivity): €6955 | Overall: $28,489 Direct medical: $17,340 Overall indirect: $10,615 Indirect (no productivity): $7838 |
Wyatt et al. (2012) (adult) [35] | 2011 GBP | UK | Medical: Hospital days, outpatient visits, day cases, accident and ED visits, GP visits, nurse visits, therapists Direct non-medical: Social workers, home help, care attendant, community support worker, housing worker | Overall: £6300 Direct medical: £4501 Direct non-medical: £1799 | Overall: £6984 Direct medical: £4990 Overall indirect: £1994 | Overall: $8989 Direct medical: $6422 Overall indirect: $2567 |
Wyatt et al. (2012) (child) [35] | 2011 GBP | UK | Medical: Hospital days, outpatient visits, day cases, accident and ED visits, GP visits, nurse visits, therapists Direct non-medical: Social workers, home help, care attendant, community support worker, housing worker | Overall: £10,080 Direct medical: £10,023 Direct non-medical: £57 | Overall: £11,175 Direct medical: £11,112 Overall indirect: £63 | Overall: $14,382 Direct medical: $14,301 Overall indirect: $81 |