Estimating the budget impact of orphan drugs in Sweden and France 2013–2020

Orphanet Journal of Rare Diseases

Karolina Antonov⁴  Felix Achana³  Richard Dutton⁵  Carina Schey¹  Adam Hutchings² 

[1] GMAS Sàrl, Route de Buchillon 65, St-Prex 1162, Switzerland;Dolon Ltd, 175-185 Gray’s Inn Road, London WC1X 8UE, UK;Biostatistics Unit, University of Leicester, University Road, Leicester LE1 7RH, UK;LIF, Box 17608 Stockholm 118 92, Sweden;KeyFormula Ltd, Roma House, Charrington Place, St Albans, Hertfordshire AL1 3NX, UK

关键词: Europe;    Health economics;    Budget impact;    Orphan medicines;   

Others  :  863253
DOI  :  10.1186/1750-1172-9-22

 received in 2013-10-25, accepted in 2014-02-06,  发布年份 2014

【 摘 要 】

Background

The growth in expenditure on orphan medicinal products (OMP) across Europe has been identified as a concern. Estimates of future expenditure in Europe have suggested that OMPs could account for a significant proportion of total pharmaceutical expenditure in some countries, but few of these forecasts have been well validated. This analysis aims to establish a robust forecast of the future budget impact of OMPs on the healthcare systems in Sweden and France.

Methods

A dynamic forecasting model was created to estimate the budget impact of OMPs in Sweden and France between 2013 and 2020. The model used historical data on OMP designation and approval rates to predict the number of new OMPs coming to the market. Average OMP sales were estimated for each year post-launch by regression analysis of historical sales data. Total forecast sales were compared with expected sales of all pharmaceuticals in each country to quantify the relative budget impact.

Results

The model predicts that by 2020, 152 OMPs will have marketing authorization in Europe. The base case OMP budget impacts are forecast to grow from 2.7% in Sweden and 3.2% in France of total drug expenditure in 2013 to 4.1% in Sweden and 4.9% in France by 2020. The principal driver of expenditure growth is the number of new OMPs obtaining OMP designation. This is tempered by the slowing success rate for new approvals and the loss of intellectual property protection on existing orphan medicines. Given the forward-looking nature of the analysis, uncertainty exists around model parameters and sensitivity analysis found peak year budget impact varying between 2% and 11%.

Conclusion

The budget impact of OMPs in Sweden and France is likely to remain sustainable over time and a relatively small proportion of total pharmaceutical expenditure. This forecast could be affected by changes in the success rate for OMP approvals, average cost of OMPs, and the type of companies developing OMPs.

【 授权许可】

   
2014 Hutchings et al.; licensee BioMed Central Ltd.

【 图 表 】

【 参考文献 】

• [1]EURORDIS: About rare diseases. http://www.eurordis.org/about-rare-diseases webcite
• [2]Regulation (EC) No 141/2000 of the European parliament and of the council of 16 December 1999 on orphan medicinal products http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF webcite
• [3]Sharma A, Jacob A, Tandon M, Kumar D: Orphan drug: development trends and strategies. J Pharm Bioallied Sci 2010, 2:290-299.
• [4]European Commission: Community register of medicinal products. 2013. http://ec.europa.eu/health/documents/community-register/html/orphreg.htm webcite. Analyzed May 2013
• [5]Simoens S: Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis 2011, 6:42. BioMed Central Full Text
• [6]Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S: A comparative study of European rare disease and orphan drug markets. Health Policy 2010, 97:173-179.
• [7]Roos JC, Hyry HI, Cox TM: Orphan drug pricing may warrant a competition law investigation. BMJ 2010, 341:c6471.
• [8]Sheldon T: Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs. BMJ 2012, 345:e5461.
• [9]Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S: Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013. J Med Econ 2010, 13(2):295-301.
• [10]Orofino J, Soto J, Casado MA, Oyaguez I: Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy 2010, 8:301-315.
• [11]Picavet E, Annemans L, Cleemput I, Cassiman D, Simoens S: Market uptake of orphan drugs–a European analysis. J Clin Pharm Ther 2012, 37:664-667.
• [12]Bignami F: Eurordis survey on orphan drugs availability in Europe. 6th eurordis round table of companies workshop, Barcelona, 9 July 2007. 2007. http://www.eurordis.org/IMG/pdf/2007ODsurvey-eurordis.pdf webcite
• [13]Schey C, Milanova T, Hutchings A: Estimating the budget impact of orphan medicines in Europe: 2010–2020. Orphanet J Rare Dis 2011, 6:62. BioMed Central Full Text
• [14]Le Cam Y: Inventory of access and prices of orphan drugs across Europe. http://img.eurordis.org/newsletter/pdf/mar-2011/ERTC_13122010_YLeCam_Final.pdf webcite
• [15]Garau M, Mestre-Ferrandiz J: Access mechanisms for orphan drugs: a comparative study of selected European countries. OHE Briefing 2009, 52:1-24.
• [16]Habl C, Bachner F: EMINET. Initial investigation to assess the feasibility of a coordinated system to access orphan medicines. Vienna: Commissioned by the European Commission, Directorate-General Enterprise and Industry; 2011. [Updated Final Report]
• [17]Intercontinental Medical Statistics (IMS) Health database MIDAS; 2013. Data on file
• [18]Groupement pour l’Elaboration et la Réalisation de Statistiques (GERS) 2013. Data on file
• [19]Orphan drugs US food and drug administration 2013. http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm webcite] Data on file. Analyzed March 2013
• [20]US Food and Drug Administration: Developing Products for rare Diseases and Conditions. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm webcite
• [21]Lunn DJ, Thomas A, Best N, Spiegelhalter D: WinBUGS – a Bayesian modelling framework: concepts, structure, and extensibility. Stat Comput 2000, 10:325-337.
• [22]Patent and marketing exclusivity analysis 2012. Celgene Data on file
• [23]TLVFS: 4 Tandvårds- och läkemedelsförmånsverkets föreskrifter och allmänna råd om prissättning av utbytbara läkemedel och utbyte av läkemedel m.m. 2009. http://www.tlv.se/Upload/Lagar_och_foreskrifter/tlvfs-2011-4.pdf webcite
• [24]Comite Economique Des Produits De Sante: Rapport d’activitite 2012. 2013. http://www.sante.gouv.fr/les-activites-du-ceps.html webcite
• [25]Tandvårds- och läkemedelsförmånsverket (TLV) http://www.tlv.se/apotek/apotekets-marginaler/%5D webcite
• [26]Orphanet. Orphan Drugs. The number of orphan medicine designations passes the 1000 milestone in Europ 2012. http://www.orpha.net/actor/EuropaNews/2012/120620.html webcite
• [27]EURORDIS: Orphan drugs: rising to the challenge to ensure a better future for 30 million patients in Europe. 2009. http://www.eurordis.org/sites/default/files/publications/Statement_Future_of_Orphan_Drugs_14_October_09.pdf webcite
• [28]Rovira J, Espín J, García L, Olry de Labry A: The impact of biosimilars’ entryin the EU market. 2011. http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/biosimilars_market_012011_en.pdf webcite
• [29]European medicines agency http://www.ema.europa.eu/ema/index.jsp?curl=search.jsp&q=imatinib&btnG=Search&mid=WC0b01ac058001d124 webcite
• [30]European commission. Pharmaceutical sector inquiry. Preliminary report 2008. http://ec.europa.eu/competition/sectors/pharmaceuticals/inquiry/preliminary_report.pdf webcite