, 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


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.


Pulmonary Arterial Hypertension European Medicine Agency Orphan Drug Budget Impact Agalsidase Beta 
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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.


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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

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