Advertisement

PharmacoEconomics

, Volume 29, Issue 10, pp 883–893 | Cite as

Critical Assessment of Belgian Reimbursement Dossiers of Orphan Drugs

  • Alain Denis
  • Lut Mergaert
  • Christel Fostier
  • Irina Cleemput
  • Frank Hulstaert
  • Steven Simoens
Original Research Article

Abstract

Background: Orphan medicinal products are designed to diagnose or treat rare diseases that are serious, life threatening or chronically debilitating and that affect 50 or fewer people in every 100 000 in the EU. In Belgium, the Drug Reimbursement Committee (DRC) evaluates reimbursement requests for orphan drugs based on multiple criteria: the therapeutic value, price and proposed reimbursement tariff; the importance of the drug in clinical practice; and the budget impact of the drug.

Objectives: This study aimed to assess reimbursement dossiers of orphan drugs in Belgium and to compare them with the clinical evidence submitted to the European Medicines Agency (EMA).

Methods: A qualitative analysis examined all reimbursement dossiers of orphan drugs that were submitted in Belgium between January 2002 and June 2008. The following information was extracted from each dossier: description of the orphan drug; indication; reimbursement status; therapeutic value and needs; budget impact; and number of registered indications. For selected orphan drugs, an in-depth analysis extracted and compared information about the clinical trials, their primary endpoints and results from EMA documents (i.e. the marketing authorization application file, European public assessment report and summary of product characteristics) and the Belgian reimbursement dossiers.

Results: Reimbursement was awarded to the majority of orphan drugs. In addition to the official criteria, other negotiable factors, such as price adjustments, employment incentives, patient population restrictions and funding of diagnostic tests by the company, seemed to play a role in the reimbursement decision. Despite the low number of patients, randomized controlled trials were conducted for many orphan drugs. Budget-impact analyses were simplistic and did not consider the impact across multiple indications. Some differences were also observed between the clinical evidence submitted to the EMA and that submitted to the Belgian DRC.Conclusions: In addition to the official criteria, other negotiable factors, such as price adjustments and employment incentives, may play a role in Belgian reimbursement decisions of orphan drugs. Some differences have also been noted between the clinical evidence reported in EMA documents and the evidence included in Belgian reimbursement dossiers of orphan drugs. There appears to be a need for further standardization of Belgian reimbursement applications and for European cooperation in sharing clinical evidence of orphan drugs.

Keywords

Pulmonary Arterial Hypertension European Medicine Agency Orphan Drug Budget Impact Agalsidase Beta 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

Notes

Acknowledgements

Financial support for this research was received from the Belgian Health Care Knowledge Centre, a state-funded research institution. The Belgian Health Care Knowledge Centre was involved in the design and supervision of the study, but it was the responsibility of the authors to collect, analyse and interpret the data and write the report. The authors have no conflicts of interest that are directly relevant to the content of this study.

References

  1. 1.
    European Commission. Regulation (EC) No 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products. Official J European Communities 2000; L 18/1 [online]. Available from URL: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF [Accessed 2011 Jun 6]Google Scholar
  2. 2.
    European Commission. Register of designated orphan medicinal products. European Commission 2010 [online]. Available from URL: http://ec.europa.eu/health/documents/community-register/html/orphreg.htm [Accessed 2010 Mar 19]Google Scholar
  3. 3.
    Denis A, Mergaert L, Fostier C, et al. A comparative study of European rare disease and orphan drug markets. Health Policy 2010 Oct; 97 (2-3): 173–9PubMedCrossRefGoogle Scholar
  4. 4.
    de Varax A, Letellier M, Börtlein G. Study on orphan drugs. Phase I: overview of the conditions for marketing orphan drugs in Europe. Paris: Alcimed, 2006 [online]. Available from URL: http://ec.europa.eu/health/files/orphanmp/doc/pricestudy/final_final_report_part_1_web_en.pdf [Accessed 2011 Jun 6]Google Scholar
  5. 5.
    Cleemput I, Van Wilder P, Vrijens F, et al. Guidelines for pharmaco-economic evaluation in Belgium. Brussels: Belgian Health Care Knowledge Centre, 2008. Report No. 78AGoogle Scholar
  6. 6.
    Royal Decree governing the procedures, terms and conditions for the reimbursement of the public insurance for health care and for pharmaceutical specialties [in Dutch and French]. Brussels: Belgisch Staatsblad, 2001 Dec 29Google Scholar
  7. 7.
    WHO Collaborating Centre for Drug Statistics Methodology. Anatomical Therapeutic Chemical classification system for drugs. 2009 [online]. Available from URL: http://www.whocc.no/atc_ddd_index/ [Accessed 2011 Jun 6]Google Scholar
  8. 8.
    Centre for Reviews and Dissemination. Undertaking systematic reviews of research on effectiveness. York: Centre for Reviews and Dissemination, 2001. Report No. 4Google Scholar
  9. 9.
    Moher D, Schulz KF, Altman DG. The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomised trials. Lancet 2001 Apr 14; 357 (9263): 1191–4PubMedCrossRefGoogle Scholar
  10. 10.
    European Medicines Agency, Committee for Medicinal Products for Human Use (CHMP). Guideline on clinical trials in small populations. London: EMA, 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003615.pdf [Accessed 2011 Jun 6]Google Scholar
  11. 11.
    Mauskopf JA, Sullivan SD, Annemans L, et al. Principles of good practice for budget impact analysis: report of the ISPOR Task Force on good research practices — budget impact analysis. Value Health 2007 Sep-Oct; 10 (5): 336–47PubMedCrossRefGoogle Scholar
  12. 12.
    European Organisation for Rare Diseases. EURORDIS position paper on the “Centralised procedure for the scientific assessment of the therapeutic added value of orphan drugs”. Paris: EURORDIS, 2008 [online]. Available from URL: http://www.eurordis.org/IMG/pdf/position-paper-EURORDIS-therapeutic-added-value-ODFeb08.pdf [Accessed 2011 JuGoogle Scholar
  13. 13.
    The Pharmaceutical Forum. Improving access to orphan medicines for all affected EU citizens. 2008 [online]. Available from URL: http://ec.europa.eu/pharmaforum/docs/pricing_orphans_en.pdf [Accessed 2011 Jun 6]Google Scholar

Copyright information

© Adis Data Information BV 2011

Authors and Affiliations

  • Alain Denis
    • 1
  • Lut Mergaert
    • 1
  • Christel Fostier
    • 1
  • Irina Cleemput
    • 2
  • Frank Hulstaert
    • 2
  • Steven Simoens
    • 3
  1. 1.Yellow Window Management ConsultantsAntwerpBelgium
  2. 2.Belgian Health Care Knowledge CentreBrusselsBelgium
  3. 3.Research Centre for Pharmaceutical Care and Pharmaco-economicsCatholic University LeuvenLeuvenBelgium

Personalised recommendations