Table 1 Public spending on orphan medicines reported in included literature
From: Public spending on orphan medicines: a review of the literature
Authors and year of publication | Aim of the study | Type of survey and expenditure data sources | Included country/countries | Year of data | Spending data | Sector | OM spending | Limitations reported in the studies | |
|---|---|---|---|---|---|---|---|---|---|
In absolute terms | As percentage | ||||||||
Kanters TA, Steenhoek A, Hakkaart L (2014) [23] | To assess uptake and budget impact of OM | Secondary data analysis of GIP database (OP) and information by ph. c. or Monitor Expensive Drugs and FarmInform (IP) | NL | 2006–2012 | Yes (OP) and no (IP), BI (predicttions) | OP and IP | 2006 OP: 52.7 mill. EUR IP: 61.2 mill. EUR 2007 OP: 68.7 mill. EUR IP: 97.9 mill. EUR 2008 OP: 97.8 mill. EUR IP: 158.6 mill. EUR 2009 OP: 118.1 mill. EUR IP: 192.7 mill. EUR 2010 OP: 141.6 mill. EUR IP: 225.9 mill. EUR 2011 OP: 156.2 mill. EUR IP: 241.4 mill. EUR 2012 OP: 175.2 mill. EUR IP: 260.4 mill. EUR | 2006: 1.1% of TPE 2007: 1.6% of TPE 2008: 2.6% of TPE 2009: 3.0% of TPE 2010: 3.6% of TPE 2011: 3.8% of TPE 2012: 4.2% of TPE | - Limited observation period (7 years) |
Orofino J, Soto J, Casado MA, Oyagüez I (2010) [27] | - To describe the status of orphan medicines in 2007 in the five countries in the EU with the greatest pharmaceutical expenditure - To estimate the mean annual cost per patient and indication in relation to orphan medicines - To determine the percentage contribution of orphan medicines to overall spending on medicines in each of these five countries in 2007 | Secondary data analysis of IMS Health, MIDAS database | DE, ES, FR, IT, UK | 2007 | No, sales data | n.a. | - | 2007 FR: 1.7% of overall pharmaceutical expenditure DE: 2.1% of overall pharmaceutical expenditure IT: 1.5% of overall pharmaceutical expenditure ES: 2.0% of overall pharmaceutical expenditure UK: 1.0% of overall pharmaceutical expenditure | - Pharmaceutical costs only had been considered (no direct or indirect treatment costs) - Calculations based on regimen in SPC - Short assessment period of pharmaceutical expenditure (1 year) - Prevalence data not completely trustworthy - Standardised information provided by IMS Health, MIDAS database (data collection method used in each country could be biased) |
Hutchings A, Schey C, Dutton R, Achana F, Antonov K (2014) [28] | - To examine historical trends in OM designation, market authorization, sales and budget impact from 2000 to 2012 - To predict the evolution in OM use for existing diseases and new indications between 2013 and 2020 | Secondary data analysis of GERS (France) and IMS Health, MIDAS database (France) | FR, SE | 2004 2006 2012a | No, sales data | n.a. | - | FR: 2012: 3.1% of TPS SE: 2006: 0.7% of TPS 2012: 2.5% of total pharm. market value | - Forecasting assumptions |
Schey C, Milanova T, Hutchings A (2011) [25] | To estimate the European budget impact of orphan medicines as a percentage of total pharmaceutical expenditure, between 2010 and 2020, based upon 10 years of orphan medicine experience in Europe. | Secondary data analysis, data source not indicated | AT, BE, CY, DE, EE, ES, FI, FR, EL, IE, IT, LU, MT, NL, PT, SK, SI, UK | 2010 | n.a. | n.a. | - | 2010: Cumulative for all countries 3.3% of total pharmaceutical spending | - Orphan disease rather than the individual orphan medicine used for modelling - Prevalence data might be weak due to data source - Used ex-factory prices may not reflect effective price paid - Predictability of prices after patent expiry - Pharmaceutical market growth rate, success rate and uptake rate may be uncertain |
Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S (2010) [22] | - To calculate the impact of OM for 2008 - To forecast its impact over the following 5 years | Secondary data analysis of data in ministerial decrees, via NIHDI, Ministry of Economic Affairs, IMS Health | BE | 2008 | Expenses estimated based on treatment costs | n.a. | 2008: 66.2 mill. EUR | 2008: 1.9% of TPE | - One product excluded due to missing information - Pharmaceutical expenditure only (no total treatment costs considered) - Products financed by a special fund not considered - Possible lower prices in future not considered |
Iskrov G, Jessop E, Miteva-Katrandzhieva T, Stefanov R. (2015) [29] | To estimate the impact of OM on NHIF total pharmaceutical budget between 2011 and 2014 | Secondary data analysis of NHIF | BG | 2011 2014b | Yes | n.a. | 2011: 31.6 mill. BGN 2014: 74.5 mill. BGN | 2011: 6.0% of TPE 2014: 7.8% of TPE | None reported |
Iskrov GG, Jakovljevic MM, Stefanov SS (2018) [30] | To estimate the budgetary impact of rare disease medicines’ therapies from NHIF perspective for 2014 and 2016 - To compare the main cost drivers for this period | Secondary data analysis of NHIF | BG | 2014 2016 | Yes | OP and IPc | - | 2014: 9.39% of TPE 2016: 9.25% of TPE | - Included both orphan and non-orphan medicines (rare disease indications used for analysis) - Analysis with official list prices, therefore BI might be overestimated |
Logviss K, Krievins D, Purvina S (2016) [31] | To assess the budget impact of OM in Latvia and compare it with other European countries | Secondary data analysis of NHS | LV | 2010–2014 | Yes | n.a. | 2010: 2.1 mill. EUR 2011: 2.6 mill. EUR 2012: 3.1 mill. EUR 2013: 2.1 mill. EUR 2014: 2.6 mill. EUR | 2010: 1.95% of TPM 2011: 2.16% of TPM 2012: 2.62% of TPM 2013: 1.83% of TPM 2014: 2.16% of TPM | - Payers’ expenditure perspective only - Product costs exceeding a yearly limit of NHS are not considered (costs might be higher) - Different approach for estimating the number of patients |
Divino V, DeKoven M, Kleinrock M, Wade RL, Kaura S (2016) [32] | To estimate the economic impact of OM in the period 2007–2013 - To extrapolate orphan medicine spending up to 2018 | Secondary data analysis of IMS Health, MIDAS database | US | 2007–2013 | No, sales data | n.a. | 2007: 15.0 bill. USD 2008: 17.1 bill. USD 2009: 19.4 bill. USD 2010: 23.1 bill. USD 2011: 26.1 bill. USD 2012: 28.0 bill. USD 2013: 30.0 bill. USD | 2007: 4.8% of TPS 2008: 5.5% of TPS 2009: 6.0% of TPS 2010: 6.8% of TPS 2011: 7.5% of TPS 2012: 8.5% of TPS 2013: 8.9% of TPS | - No stratification between therapies for chronic and acute illnesses (potential long-term impact on payers’ expenditure) - IMS Health, MIDAS database do not cover 100% of the market - No generic orphan medicines considered - Potential off-label use of orphan medicines not considered |
Divino V, DeKoven M, Kleinrock M, Wade RL, Kim T, Kaura S (2016) [33] | - To estimate the financial impact of OM on the TPE from 2007 to 2013 in Canada - To extrapolate orphan medicine spend up to 2018 | Secondary data analysis of IMS Health, MIDAS database | CA | 2007–2013 | No, sales data | OP and IP | 2007: 610.2 mill. CAD f2008: 669.2 mill. CAD 2009: 743.7 mill. CAD 2010: 818.1 mill. CAD 2011: 880.5 mill. CAD 2012: 989.6 mill. CAD 2013: 1,100.0 mill. CAD | 2007: 3.3% of TPS 2008: 3.4% of TPS 2009: 3.6% of TPS 2010: 4.0% of TPS 2011: 4.4% of TPS 2012: 5.0% of TPS 2013: 5.6% of TPS | - IMS Health, MIDAS database does not cover 100 % of the market - Custom methodologies - Possible changes through policy adoption not considered - Potential differences in indication approvals (no approval in the USA, not accounted for in the study) - No generic orphan medicines considered - Potential off-label use not considered |
Kockaya G, Wertheimer AI, Kilic P, Tanyeri P, Vural IM, Akbulat A, Artiran G, Kerman S (2014) [34] | To shed light on the use of OM in Turkey to aid further classifications of rare diseases and assessments of orphan medicines in the country | Secondary data analysis of IMS Turkey and TITCK | TR | 2008–2010 | No, sales data | n.a. | 2008: 135.7 mill. EUR 2009: 182.4 mill. EUR 2010: 208.5 mill. EUR | 2008: 2% of TPE 2010: 3% of TPE | None reported |
Hsu JC, Wu H-C, Feng W-C, Chou C-H, Lai EC-C, Lu CY (2018) [2] | To examine 2003–2014 longitudinal trends in the prevalence and expenditure of rare disease s in Taiwan | Secondary data analysis of NHIRD | TW | 2003–2014 | Yes | n.a. | 2003: 13.2 mill. USD 2004: 17.7 mill. USD 2005: 21.5 mill. USD 2006: 30.8 mill. USD 2007: 41.3 mill. USD 2008: 49.2 mill. USD 2009: 54.6 mill. USD 2010: 61.8 mill. USD 2011: 72.7 mill. USD 2012: 91.5 mill. USD 2013: 104.9 mill. USD 2014: 122.0 mill. USD | 2003: 0.35% of TPE 2004: 0.41% of TPE 2005: 0.50% of TPE 2006: 0.73% of TPE 2007: 0.99% of TPE 2008: 1.14% of TPE 2009: 1.21% of TPE 2010: 1.37% of TPE 2011: 1.51% of TPE 2012: 1.92% of TPE 2013: 2.06% of TPE 2014: 2.31% of TPE | - Nationwide approach instead of individual patients (no out-of-pocket payments or clinical outcomes considered) - Focus on rare diseases in general (no analysis with regard to certain rare diseases except for 2 rare diseases) |
Deticek A, Locatelli I, Kos M (2018) [35] | To estimate patient access to different medicines for rare diseases from the comprehensive Orphanet list in various European countries in the past decade | Secondary data analysis of IMS Health data | AT, BE, BG, CH, CZ, DE, EL, ES, FI, FR, HR, HU, IE, IT, NL, NO, PL, RO, SE, SK, SI, UK | 2014d | No, sales data | OP and IPe | AT: 4 mill. EUR/inh. BE: 11 mill. EUR/inh. BG: 4 mill. EUR/inh. CH: 12 mill. EUR/inh. CZ: 2 mill. EUR/inh. DE: 15 mill. EUR/inh. EL: 0.2 mill. EUR/inh. ES: 8 mill. EUR/inh. FI: 7 mill. EUR/inh. FR: 12 mill. EUR/inh. HR: 3 mill. EUR/inh. HU: 2 mill. EUR/inh. IE: 7 mill. EUR/inh. IT: 12 mill. EUR/inh. NL: 7 mill. EUR/inh. NO: 6 mill. EUR/inh. PL: 1 mill. EUR/inh. RO: 2 mill. EUR/inh. SE: 9 mill. EUR/inh. SI: 8 mill. EUR/inh. SK: 6 mill. EUR/inh. UK: 11 mill. EUR/inh. | - | - IMS Health data might not reflect the actual access to orphan medicines in the studied countries - Expenditures might be overestimated (products with more than one indication that are not for rare diseases) - Sales data only included if sales was continuous over a certain time - Number of patients in need of treatment might differ from country to country due to prevalence of diseases and potential prescribing restrictions |