期刊论文详细信息
Orphanet Journal of Rare Diseases
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe
Warren Cowell3  Adam Hutchings4  Julien Patris2  Laura Gutierrez1 
[1] Celgene International Sàrl, Route de Perreux 1, Boudry, Switzerland;Celgene bvba/sarl Parc de l’Alliance, Boulevard de France 9, Braine L’Alleud, Belgium;Celgene Ltd, 1 Longwalk Road, Stockley Park, Uxbridge, London, UK;Dolon Ltd, 17-185 Gray’s Inn Road, London, UK
关键词: Value assessment;    Rare diseases;    Orphan medicinal products;   
Others  :  1211848
DOI  :  10.1186/s13023-015-0269-y
 received in 2015-02-18, accepted in 2015-04-15,  发布年份 2015
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【 摘 要 】

The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues for policymakers and decisions regarding their pricing and funding are highly complex. There is currently no European consensus on how OMP value should be assessed and inequalities of access to OMPs have previously been observed. Against this background, policy makers in many countries are considering reforms to improve access to OMPs. This paper proposes ten principles to be considered when undertaking such reforms, from the perspective of an OMP manufacturer. We recommend the continued prioritisation of rare diseases by policymakers, an increased alignment between payer and regulatory frameworks, pricing centred on OMP value, and mechanisms to ensure long-term financial sustainability allowing a continuous and virtuous development of OMPs. Our recommendations support the development of more consistent frameworks and encourage collaboration between all stakeholders, including research-based industry, payers, clinicians, and patients.

【 授权许可】

   
2015 Gutierrez et al.; licensee BioMed Central.

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【 参考文献 】
  • [1]Euoropean Commission. Pharmaceutical comittee 17 March 2015. http://ec.europa.eu/health/files/committee/74meeting/pharm684.pdf. Accessed 10 April 2015.
  • [2]European Commission. Regulation (EC) No 141/2000 of the European parliament and of the council of 16 December 1999 on orphan medicinal products. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_141_cons-2009-07/reg_2000_141_cons-2009-07_en.pdf. Accessed 13 Feb 2015
  • [3]Aymé S, Rodwell C, eds. 2013 report on the state of the art of rare disease activities in Europe. 2013. http://www.eucerd.eu/upload/file/Reports/2013ReportStateofArtRDActivities.pdf. Accessed 13 Feb 2015.
  • [4]European Commission. Policy, Rare diseases – what are they? http://ec.europa.eu/health/rare_diseases/policy/index_en.htm. Accessed 13 Feb 2015.
  • [5]Simoens S, Picavet E, Dooms M, Cassiman D, Morel T. Cost-effectiveness assessment of orphan drugs: a scientific and political conundrum. Appl Health Econ Health Policy. 2013; 11(1):1-3.
  • [6]Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007; 23(1):36-42.
  • [7]Hughes-Wilson W, Palma A, Schuurman A, Simoens S. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012; 7:74. BioMed Central Full Text
  • [8]Roos JC, Hyry HI, Cox TM. Orphan drug pricing may warrant a competition law investigation. BMJ. 2010; 341:c6471.
  • [9]Fellows GK, Hollis A. Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach. Orphanet J Rare Dis. 2013; 8:180. BioMed Central Full Text
  • [10]Le Cam Y. Inventory of Access and Prices of orphan drugs across Europe: A collaborative work between National Alliances on Rare diseases & Eurordis. Presentation, Paris. 2010.
  • [11]Habl C, Bachner F. EMINET. Initial investigation to assess the feasibility of a coordinated system to access orphan medicines. Vienna, May 2011. Updated Final Report. Commissioned by the European Commission, Directorate-General Enterprise and Industry. 2011. http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/access_orphans_initialinvest_052011_en.pdf. Accessed 13 Feb 2015.
  • [12]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(2–3):173-9.
  • [13]Consolidated versions of the Treaty on European Union and the Treaty on the Functioning of the European Union - Consolidated version of the Treaty on the Functioning of the European Union - Protocols - Annexes - Declarations annexed to the Final Act of the Intergovernmental Conference which adopted the Treaty of Lisbon, signed on 13 December 2007 - Tables of equivalences. http://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:12012E/TXT&from=en. Accessed 10 April 2015.
  • [14]Michel M, Toumi M. Access to orphan drugs in Europe: current and future issues. Expert Rev Pharmacoecon Outcomes Res. 2012; 12(1):23-9.
  • [15]Garau M, Mestre-Ferrandiz J. Access mechanisms for orphan drugs: a comparative study of selected European countries. OHE Briefing. 2009; 52(October):1-24.
  • [16]Barham L. Orphan Medicines. Special treatment required? 2020 Health 2012. http://www.2020health.org/2020health/Publications/publications-2012/Orphan-Drugs.html. Accessed 13 Feb 2015.
  • [17]Nicod E, Kanavos P. Inter-country variability in coverage decisions for orphan drugs: criteria driving HTA recommendations in six countries. Presented at ISPOR 18th Annual International Meeting. New Orleans; 2013. http://www.valueinhealthjournal.com/article/S1098-3015(13)00090-9/fulltext Presentation.pdf. Accessed 13 Feb 2015.
  • [18]Davies JE, Neidle S, Taylor DG. Developing and paying for medicines for orphan indications in oncology: utilitarian regulation vs equitable care? Br J Cancer. 2012; 106(1):14-7.
  • [19]Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM. 2005; 98(11):829-36.
  • [20]Morel T, Arickx F, Befrits G, Siviero P, van der Meijden C, Xoxi E et al.. Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries. Orphanet J Rare Dis. 2013; 8:198. BioMed Central Full Text
  • [21]Timoney A. Scottish Medicines Consortium. SMC Modifiers used in Appraising New Medicines. 2012. https://www.scottishmedicines.org.uk/About_SMC/Policy_statements/SMC_Modifiers_used_in_Appraising_New_Medicines. Accessed 13 Feb 2015.
  • [22]All Wales Medicines Strategy Group. Report for the Minister of Health and Social Care on the Review of AWMSG’s policy for appraising Orphan and Ultra Orphan Medicines. 2014 http://www.awmsg.org/docs/awmsg/appraisaldocs/inforandforms/AWMSG%20policy%20relating%20to%20ultra-orphan%20medicine.pdf. Accessed 13 Feb 2015.
  • [23]Pearce F, Godfrey J. Evaluation of Highly Specialised Technologies for very rare diseases in England, National Institute for Health and Care Excellence. 2013. http://www.rare-diseases.eu/wpcontent/uploads/2013/08/139_t4.pdf. Accessed 13 Feb 2015.
  • [24]National Institute for Health and Care Excellence. NICE to assess high cost drugs for rare conditions. Assessed July 2012. http://www.nice.org.uk/News/Article/nice-to-assess-high-cost-drugs-for-rare-conditions. Accessed 13 Feb 2015.
  • [25]Eurordis. 2013. http://www.eurordis.org/about-rare-diseases. Accessed 13 Feb 2015.
  • [26]McCabe C, Edlin R, Round J. Economic considerations in the provision of treatments for rare diseases. Adv Exp Med Biol. 2010; 686:211-22.
  • [27]Drummond MF. Challenges in the economic evaluation of orphan drugs. Eurohealth. 2008; 14(2):16-7.
  • [28]Council of the European Union. Council recommendations on an action in the field of rare diseases. 2009. http://ec.europa.eu/health/ph_threats/non_com/docs/rare_rec2_en.pdf. Accessed 13 Feb 2015.
  • [29]Commission staff working documents on the experience acquired as a result of the application of Regulation (EC) No 141/2000 on orphan medicinal products and account of the public health benefits obtained. http://ec.europa.eu/health/files/orphanmp/doc/orphan_en_06-2006_en.pdf. Accessed 13 Feb 2015.
  • [30]European Comission Regualation (EC) No 847/2000. 27 April 2000. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_847/reg_2000_847_en.pdf. Accessed 13 Feb 2015.
  • [31]Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov. 2010; 9(12):921-9.
  • [32]Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011; 71(4):488-96.
  • [33]Gemeinsamer Bundesausschuss. Benefit assessment of pharmaceuticals. The special case of orphan drugs. http://www.english.g-ba.de/benefitassessment/information/faq/ - 4. Accessed May 2014.
  • [34]Mavris M, Le Cam Y. The European Medicines Agency and transparency: The rare disease patients’ perspective. Regulatory Rapporteur. 2014.
  • [35]Mavris M, Le Cam Y. Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe. Mol Syndromol. 2012; 3(5):237-43.
  • [36]Mentzakis E, Stefanowska P, Hurley J. A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study. Health Econ Policy Law. 2011; 6(3):405-33.
  • [37]Clarke JT. Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy. CMAJ. 2006; 174(2):189-90.
  • [38]Schlander M, Beck M. Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI. Curr Med Res Opin. 2009; 25(5):1285-93.
  • [39]Picavet E, Dooms M, Cassiman D, Simoens S. Orphan drugs for rare diseases: grounds for special status. Drug Dev Res. 2012; 73:115-9.
  • [40]Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011; 6:42. BioMed Central Full Text
  • [41]Devlin N, Sussex J. Incorporating multiple criteria in HTA: methods and processes. Office of Health Economics. 2011. https://www.ohe.org/publications/incorporating-multiple-criteria-hta-methods-and-processes. Accessed 13 Feb 2015.
  • [42]Sussex J, Rollet P, Garau M, Schmitt C, Kent A, Hutchings A. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value Health. 2013; 16(8):1163-9.
  • [43]Schlander M, Garattini S, Kolominsky-Rabas P, Nord E, Persson U, Postma M et al.. Incremental cost per Quality adjusted life years gained? The need for alternative methods to evaluate medical interventions for ultra rare disorders. Value Health. 2013; 16:A324.
  • [44]Process on Corporate Social Responsibility in the Field of Pharmaceuticals Platform on Access to Medicines in Europe Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA-OMP). http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/orphans_framework_en.pdf. Accessed 13 Feb 2015.
  • [45]Rémuzat C, Toumi M, Falissard B. New drug regulations in France: what are the impacts on market access? Part 2 - impacts on market access and impacts for the pharmaceutical industry. J Mark Access Health Policy. 2013; 1:20892.
  • [46]Simoens S. Market access of orphan drugs and the role of multi-criteria decision making. Orphanet J Rare Dis. 2012; 7 Suppl 2:A26.
  • [47]Irwin J, Peacock S. Multi-Criteria Decision Analysis: An emerging alternative for assessing the value of orphan medicinal products. Regulatory Rapporteur. 2015.
  • [48]Schey C, Connolly MP. Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs- Presented at the European conference on rare diseases and orphan products, 8–10 May 2014, Berlin. http://www.rare-diseases.eu/wp-content/uploads/2013/08/145_t5.pdf. Accessed 13 Feb 2015.
  • [49]Kanavos P, Nicod E. What is wrong with orphan drug policies? Suggestions for ways forward. Value Health. 2012; 15(8):1182-4.
  • [50]Eichler HG, Oye K, Baird LG, Abadie E, Brown J, Drum CL et al.. Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther. 2012; 91(3):426-37.
  • [51]National Institute for Health Care Management Research and Educational Foundation (NIHCM). Changing Patterns of Pharmaceutical Innovation. 2002. http://www.nihcm.org/pdf/innovations.pdf. Accessed 13 Feb 2015.
  • [52]Eurordis. Rare diseases: understanding this public health priority. 2005. http://www.eurordis.org/sites/default/files/publications/princeps_document-EN.pdf. Accessed 13 Feb 2015.
  • [53]Butlen-Ducuing F, Riviere F, Aarum S, Llinares-Garcia J. European Medicines Agency support mechanisms fostering orphan drug development. Drug News Perspect. 2010; 23(1):71-81.
  • [54]Rollet P, Lemoine A, Dunoyer M. Sustainable rare diseases business and drug access: no time for misconceptions. Orphanet J Rare Dis. 2013; 8(1):109. BioMed Central Full Text
  • [55]Simoens S, Cassiman D, Dooms M, Picavet E. Orphan drugs for rare diseases: is it time to revisit their special market access status? Drugs. 2012; 72(11):1437-43.
  • [56]Cheng MM, Ramsey SD, Devine EB, Garrison LP, Bresnahan BW, Veenstra DL. Systematic review of comparative effectiveness data for oncology orphan drugs. Am J Manag Care. 2012; 18(1):47-62.
  • [57]Burls A, Austin D, Moore D. Commissioning for rare diseases: view from the frontline. BMJ. 2005; 331(7523):1019-21.
  • [58]Hutchings A, Schey C, Dutton R, Achana F, Antonov K. Estimating the budget impact of orphan drugs in Sweden and France 2013–2020. Orphanet J Rare Dis. 2014; 9:22. BioMed Central Full Text
  • [59]McCabe C, Tsuchiya A, Claxton K, Raftery J. Orphan drugs revisited. QJM. 2006; 99(5):341-5.
  • [60]Simoens S, Dooms M. How much is the life of a cancer patient worth? A pharmaco-economic perspective. J Clin Pharm Ther. 2011; 36(3):249-56.
  • [61]Hughes D. Rationing of drugs for rare diseases. Pharmacoeconomics. 2006; 24(4):315-6.
  • [62]Connock M, Juarez-Garcia A, Frew E, Mans A, Dretzke J, Fry-Smith A et al.. A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry’s disease and mucopolysaccharidosis type 1. Health Technol Assess. 2006; 10(20):iii-iv.
  • [63]Farrugia A, O’Mahony B, Cassar J. Health technology assessment and haemophilia. Haemophilia. 2012; 18(2):152-7.
  • [64]Dunoyer M, Rollet P. Value and specificity of rare diseases business model-is the pursuit of this societal priority sustainable? Orphanet J Rare Dis. 2012; 7 Suppl 2:A23. BioMed Central Full Text
  • [65]Chabot I, Rocchi A. Oncology drug health technology assessment recommendations: Canadian versus UK experiences. Clinicoecon Outcomes Res. 2014; 6:357-67.
  • [66]NICE calls for a new approach to managing the entry of drugs into the NHS. 2014. http://www.nice.org.uk/news/press-and-media/nice-calls-for-a-new-approach-to-managing-the-entry-of-drugs-into-the-nhs. Accessed 13 Feb 2015.
  • [67]Picavet E, Annemans L, Cleemput I, Cassiman D, Simoens S. Market uptake of orphan drugs–a European analysis. J Clin Pharm Ther. 2012; 37(6):664-7.
  • [68]Owen A, Spinks J, Meehan A, Robb T, Hardy M, Kwasha D et al.. A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: the Bosentan Patient Registry. J Med Econ. 2008; 11(2):235-43.
  • [69]Bélorgey C. Temporary Authorisations for Use (ATU). Agence nationale de sécurité du médicament et des produits de santé. 2001. http://agence-tst.ansm.sante.fr/html/pdf/5/atu_eng.pdf. Accessed 13 Feb 2015.
  • [70]Agence nationale de sécurité du médicament et des produits de santé. Autorisations Temporaires d’Utilisation. http://ansm.sante.fr/Activites/Autorisations-Temporaires-d-Utilisation-ATU/Autorisations-Temporaires-d-Utilisation/%28offset%29/0. Accessed 13 Feb 2015.
  • [71]Bélorgey C. The compassionate use of mediacinal products. The French ATU system. Agence nationale de sécurité du médicament et des produits de santé. 2012. http://www.eurordis.org/content/eurordis-general-assembly-2012. Accessed 13 Feb 2015.
  • [72]Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S. Issues surrounding orphan disease and orphan drug policies in Europe. Appl Health Econ Health Policy. 2010; 8(5):343-50.
  • [73]Commission of the European Communities. Communication from the commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions - on rare diseases: Europe’s challenges. Brussels; 2008. http://ec.europa.eu/health/ph_threats/non_com/docs/rare_com_en.pdf. Accessed 17 Feb 2015.
  • [74]Rosenberg-Yunger ZR, Daar AS, Thorsteinsdottir H, Martin DK. Priority setting for orphan drugs: an international comparison. Health Policy. 2011; 100(1):25-34.
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