Critical Assessment of Belgian Reimbursement Dossiers of Orphan Drugs

Denis, A.; Mergaert, Lut; Fostier, Christel; Cleemput, Irina; Hulstaert, Frank; Simoens, Steven

doi:10.2165/11585980-000000000-00000

Cited by 27 publications

(34 citation statements)

References 3 publications

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“…Dakin et al found that the number of randomized controlled trials and the presence of a submission from a patient organization are predictors of a positive evaluation by NICE [20,28]. The study from Denis et al concluded that evidence of therapeutic value tended to be very limited and often derived from few clinical studies [13]. Dupont and Van Wilder found that the same quality of clinical evidence as required for medicines for common indications was not required for orphan drugs.…”

Section: Discussionmentioning

confidence: 99%

“…Semi-structured interviews with six participants were conducted between January 17 th and February 5 th 2014. A semi-structured interview guide (Additional file 1: Appendix 1) was drafted in the form of a table presenting a non-exhaustive list of potential factors influencing reimbursement derived from a literature review [10,13,15,20]. Respondents were asked to comment on that list, provide real-life examples and suggest changes or additions.…”

Section: Methodsmentioning

confidence: 99%

“…four representatives of the Ministry of Public Health, of Social Affairs, of Budget and of Economics Affairs, one representative of the NIHDI, and two members of Pharma.be (representing the pharmaceutical industry in Belgium), and one member of Febelgen (representing the generic pharmaceutical industry)). The DRC evaluates drug reimbursement requests based on the five criteria: therapeutic value, price, proposed reimbursement tariff, the importance of the drug in clinical practice, and the budgetary impact of the drug [13]. The DRC may decide to compose a group of experts to evaluate the application.…”

Section: Introductionmentioning

confidence: 99%

“…Dupont and Van Wilder performed a qualitative analysis of Belgian reimbursement dossiers of all orphan drugs with a specific focus on the evidence related to the five reimbursement criteria (therapeutic value, price, proposed reimbursement tariff, the importance of the drug in clinical practice, and the budgetary impact of the drug) [15]. They concluded that factors other than the official criteria seem to play a role in the decision-making process, thereby creating uncertainty [13]. …”

Section: Introductionmentioning

confidence: 99%

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

Picavet

Cassiman

Simoens

2014

Orphanet J Rare Dis

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BackgroundMost 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.MethodsSix 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.ResultsDifferent 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.DiscussionThe 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.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-014-0139-z) contains supplementary material, which is available to authorized users.

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Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

Picavet

Cassiman

Simoens

2014

Orphanet J Rare Dis

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“…The assessment of methodological quality of economic evaluations in Belgian drug reimbursement applications was carried out by one researcher and not by multiple independent researchers. This approach was chosen because the researcher was involved as an expert in the development of the KCE guidelines [3], has previously carried out assessments of orphan drug reimbursement applications in Belgium [8], and has contributed to the design and conduct of economic evaluations for the purpose of Belgian reimbursement applications. …”

Section: Discussionmentioning

confidence: 99%

Assessment of Methodological Quality of Economic Evaluations in Belgian Drug Reimbursement Applications

Simoens

2013

PLoS ONE

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ObjectivesThis paper aims to assess the methodological quality of economic evaluations included in Belgian reimbursement applications for Class 1 drugs.Materials and MethodsFor 19 reimbursement applications submitted during 2011 and Spring 2012, a descriptive analysis assessed the methodological quality of the economic evaluation, evaluated the assessment of that economic evaluation by the Drug Reimbursement Committee and the response to that assessment by the company. Compliance with methodological guidelines issued by the Belgian Healthcare Knowledge Centre was assessed using a detailed checklist of 23 methodological items. The rate of compliance was calculated based on the number of economic evaluations for which the item was applicable.ResultsEconomic evaluations tended to comply with guidelines regarding perspective, target population, subgroup analyses, comparator, use of comparative clinical data and final outcome measures, calculation of costs, incremental analysis, discounting and time horizon. However, more attention needs to be paid to the description of limitations of indirect comparisons, the choice of an appropriate analytic technique, the expression of unit costs in values for the current year, the estimation and valuation of outcomes, the presentation of results of sensitivity analyses, and testing the face validity of model inputs and outputs. Also, a large variation was observed in the scope and depth of the quality assessment by the Drug Reimbursement Committee. ConclusionsAlthough general guidelines exist, pharmaceutical companies and the Drug Reimbursement Committee would benefit from the existence of a more detailed checklist of methodological items that need to be reported in an economic evaluation.

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Pharm Med

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Critical Assessment of Belgian Reimbursement Dossiers of Orphan Drugs

Cited by 27 publications

References 3 publications

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

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

Assessment of Methodological Quality of Economic Evaluations in Belgian Drug Reimbursement Applications

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