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

Orphanet Journal of Rare Diseases

Steven Simoens¹  David Cassiman²  Eline Picavet¹ 

[1] KU Leuven Department of Pharmaceutical and Pharmacological Sciences, Herestraat 49, Leuven 3000, Belgium;Department of Hepatology, University Hospital Leuven, Herestraat 49, Leuven 3000, Belgium

关键词: Orphan drugs;    Reimbursement;   

Others  :  1149892
DOI  :  10.1186/s13023-014-0139-z

 received in 2014-05-23, accepted in 2014-08-25,  发布年份 2014

【 摘 要 】

Background

Most orphan drugs do not meet traditional standards of cost-effectiveness. Yet, most orphan drugs are reimbursed, which implies that other factors are taken into account at the time of reimbursement. To increase accountability of decision-makers, there is a need for more transparency in the factors that play a role in reimbursement decisions of orphan drugs. Therefore, the aim of this study is to use a combination of qualitative research methods to examine which official and non-official factors influence reimbursement decisions for orphan drugs in Belgium.

Methods

Six semi-structured interviews with past or present members of the Drug Reimbursement Committee (DRC) were performed with a view to obtaining an overview of the potential factors influencing reimbursement. Additionally, these presence of these factors was assessed in the reimbursement dossiers of all orphan drugs (n?=?64) for which an application for reimbursement was submitted to the National Institute for Health and Disability Insurance in Belgium between January 2002 and July 2013.

Results

Different official (i.e. therapeutic value, budget impact, price and impact in clinical practice) and non-official factors (i.e. pricing and reimbursement in other countries, interference by patient organisations and experts, arguments related to quality of branded drug versus compounding, media attention, innovative character, economic importance, ethical arguments and the political climate) may have influenced past reimbursement decisions for orphan drugs in Belgium.

Discussion

The identification of factors influencing orphan drug reimbursement is a crucial step in the development of a transparent and consistent framework which will guide future decision-making for reimbursement of orphan drugs.

【 授权许可】

   
2014 Picavet et al.; licensee BioMed Central Ltd.

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