Reimbursement of orphan drugs in Belgium: What (else) matters?

Orphanet Journal of Rare Diseases

Volumn 9, Issue 1, 2014, Pages

  Picavet, Eline ^a   Cassiman, David ^b   Simoens, Steven ^a  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b UNIVERSITY HOSPITALS LEUVEN   (Belgium)

Author keywords

Orphan drugs; Reimbursement

Indexed keywords

ORPHAN DRUG;

ARTICLE; BELGIUM; CLINICAL PRACTICE; DRUG COST; DRUG QUALITY; ETHICS; REIMBURSEMENT; SEMI STRUCTURED INTERVIEW; COST BENEFIT ANALYSIS; DRUG INDUSTRY; DRUG MANUFACTURE; ECONOMICS; HUMAN; PROCEDURES; TRENDS;

BELGIUM; COST-BENEFIT ANALYSIS; DRUG INDUSTRY; HUMANS; INSURANCE, HEALTH, REIMBURSEMENT; ORPHAN DRUG PRODUCTION;

EID: 84908218130     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-014-0139-z     Document Type: Article

References (29)

1. European Commission: Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Official J Eur Commun 2000, L 18:1-5.
2. Orphanet; [http://www.orpha.net/consor/cgi-bin/index.php]
3. Alcimed: Study on orphan drugs. Phase I: overview of the conditions for marketing orphan drugs in Europe; [http://ec.europa.eu/enterprise/sectors/pharmaceuticals/files/orphanmp/doc/pricestudy/final-final-report-part-1-web-en.pdf]
4. 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.
5. Heemstra HE: Variations in access and use of orphan drugs among EU Member States. Eur J Hosp Pharm Pract 2010, 16:25-27.
6. Eurordis: Survey on orphan drugs availability in Europe; [http://ec.europa.eu/health/files/orphanmp/doc/pricestudy/final-final-report-part-1-web-en.pdf]
7. Blankart CR, Stargardt T, Schreyogg J: Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia. Pharmacoeconomics 2011, 29:63-82.
8. Drummond M, Towse A: Orphan drugs policies: a suitable case for treatment. Eur J Health Econ 2014, 15(4):335-340.
9. Rosenberg-Yunger ZR, Daar AS, Thorsteinsdottir H, Martin DK: Priority setting for orphan drugs: an international comparison. Health Policy 2011, 100:25-34.
10. Cerri KH, Knapp M, Fernandez JL: Decision making by NICE: examining the influences of evidence, process and context. Health Econ Policy Law 2014, 9:119-141.
11. 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, 16:1163-1169.
12. Denis A, Simoens S, Fostier C, Mergaert L, Cleemput I: Beleid voor zeldzame ziekten en Weesgeneesmiddelen. KCE reports 2010, 112A:
13. Denis A, Mergaert L, Fostier C, Cleemput I, Hulstaert F, Simoens S: Critical assessment of belgian reimbursement dossiers of orphan drugs. Pharmacoeconomics 2011, 29:883-893.
14. Royal Decree of December 21th, 2001 establishing the procedures, terms and conditions concerning the reimbursement of the compulsory insurance for health care and allowances in the cost of pharmaceutical specialities; [http://www.ejustice.just.fgov.be/cgi-loi/change-lg.pl?language=nl&la=N&cn=2001122138&table-name=wet]
15. Dupont AG, Van Wilder PB: Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol 2011, 71:488-496.
16. Hutton J, McGrath C, Frybourg JM, Tremblay M, Bramley-Harker E, Henshall C: Framework for describing and classifying decision-making systems using technology assessment to determine the reimbursement of health technologies (fourth hurdle systems). Int J Technol Assess Health Care 2006, 22:10-18.
17. Rogowski WH: An economic theory of the fourth hurdle. Health Econ 2013, 22:600-610.
18. Fischer KE, Leidl R, Rogowski WH: A structured tool to analyse coverage decisions: development and feasibility test in the field of cancer screening and prevention. Health Policy 2011, 101:290-299.
19. Fischer KE: A systematic review of coverage decision-making on health technologies-evidence from the real world. Health Policy 2012, 107:218-230.
20. Linley WG, Hughes DA: Reimbursement decisions of the All Wales Medicines Strategy Group: influence of policy and clinical and economic factors. Pharmacoeconomics 2012, 30:779-794.
21. Pope C, Mays N: Qualitative research in health care. Oxford: Blackwell Publishing; 2006.
22. QSR Nvivo 9 Software for Windows; [http://www.ejustice.just.fgov.be/cgi-loi/change-lg.pl?language=nl&la=N&cn=2001122138&table-name=wet]
23. Picavet E, Cassiman D, Hollak CE, Maertens JA, Simoens S: Clinical evidence for orphan medicinal products - a cause for concern? Orphanet J Rare Dis 2013, 8:164.
24. Morel T, Arickx F, Befrits G, Siviero P, van der Meijden C, Xoxi E, Simoens S: 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.
25. Boy R, Schwartz IV, Krug BC, Santana-da-Silva LC, Steiner CE, Acosta AX, Ribeiro EM, Galera MF, Leivas PG, Braz M: Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I. J Med Ethics 2011, 37:233-239.
26. Gericke CA, Riesberg A, Busse R: Ethical issues in funding orphan drug research and development. J Med Ethics 2005, 31:164-168.
27. McCabe C: Balancing economic, ethical and equity concerns in orphan drugs and rare diseases. Eur J Hosp Pharm Pract 2010, 16:22-25.
28. Dakin HA, Devlin NJ, Odeyemi IA: "Yes", "No" or "Yes, but"? Multinomial modelling of NICE decision-making. Health Policy 2006, 77:352-367.
29. 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.