Fig. 2.

Annual total ERT costs per patient (IOPD; annual currency- and inflation-adjusted [2017 USD])^a. CPI consumer price index, ERT enzyme-replacement therapy, IOPD infantile-onset Pompe disease, OECD Organisation for Economic Cooperation and Development, USD United States dollars. ^aStandardized with respect to currency, costs, and dose (20 mg/kg/2 week). Total ERT costs represent drug acquisition cost only. These costs exclude infusion, supportive care, and other costs associated with ERT for Pompe disease. Annual cost is calculated based on total costs divided by total time alive for all studies except Guo et al. 2012. Annual costs were estimated based on costs and prescriptions dispensed per quarter (prescriptions adjusted to patients based on an assumed regimen of 20 mg/kg/2 weeks) to estimate an annual per-patient cost. Annual cost for 20 mg/kg every 2 weeks per patient calculated as €120,205 [32], £38,324 (Castro-Jaramillo 2012 – England), £41,678 (Castro-Jaramillo 2012—Colombia), $190,488 [36], and £26,025 [37]. Inflation-adjusted from data to year 2017 (inflation-adjusted to 2017 using average annual inflation rate per year reported by the OECD for each country and inflated based on data year) calculated as €135,215 [32], £44,109 (Castro-Jaramillo 2012—England), £50,013 (Castro-Jaramillo 2012—Colombia), $207,604 [36], and £28,852 [37]. OECD (2018), “Inflation consumer price index (CPI) (indicator).” 10.1787/eee82e6e-en (Accessed on 17 Jul 2018). Inflation-adjusted currency converted to USD based on currency reported in the publication. OECD (2018), “Exchange rates (indicator).” 10.1787/037ed317-en (Accessed on 17 Jul 2018)