Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013

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

doi:10.3111/13696998.2010.491427

Cited by 39 publications

(49 citation statements)

References 5 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…The relevance of budget impact considerations was discussed in 13 papers [12, 14, 18, 29, 30, 34, 37, 38, 50, 51, 58, 60, 63]. Several authors questioned the need to consider it at all since the budget impact of many orphan drugs is ‘modest’ due to small patient numbers [34].…”

Section: Resultsmentioning

confidence: 99%

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

et al. 2014

View full text Add to dashboard Cite

BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or ‘opportunity cost’-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-014-0235-x) contains supplementary material, which is available to authorized users.

show abstract

Section: Resultsmentioning

confidence: 99%

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

et al. 2014

View full text Add to dashboard Cite

show abstract

“…For instance, the annual price per patient of reimbursed orphan drugs in Belgium ranged from h4600 for busulfan to h376 000 for galsulfase in 2008. [3] The impact of the orphan drug budget amounted to h66.2 million (or 5% of the Belgian hospital drug budget) in 2008. The impact was projected to increase to h130-204 million by 2013.…”

Section: Introductionmentioning

confidence: 99%

Are some orphan drugs for rare diseases too expensive? A study of purchase versus compounding costs

Simoens

Cassiman

Picavet

et al. 2011

Drugs & Therapy Perspectives

Self Cite

View full text Add to dashboard Cite

show abstract

“…The share of the total European pharmaceutical market represented by orphan drugs was 3.3% in 2010, and it was predicted by Schey et al to increase to a peak of 4.6% in 2016. Another analysis estimated that orphan drugs constituted 1.9% of total drugs expenditure in Belgium in 2008, and predicted it to increase to about 4% in 2013 [37]. While the average budget impact of orphan drugs accounted for 1.7% of the total pharmaceutical expenditure across France, Germany, Italy, Spain and the UK in 2007 [38].…”

Section: Discussionmentioning

confidence: 99%

Rare diseases and orphan drugs: Latvian story

Logviss

Krieviņš

Purvina

2014

Orphanet J Rare Dis

View full text Add to dashboard Cite

BackgroundTen years have passed since Latvia became a Member State of the EU in 2004. As a result European regulations, including those related to rare diseases and orphan drugs, have been applied to Latvian legislative system. Orphan diseases have been recognized as a priority area for action in the public health system, though there are significant differences in the national healthcare services for rare diseases among the EU States. This study aims to determine situation in the field of rare diseases in Latvia and compare it with other European countries.MethodsWe used the national plan for rare diseases, EUCERD reports, Orphanet data, Latvian and European regulations, publicly available data from the state agencies, and directly contacted drug manufacturers and wholesalers.ResultsNational plan for rare diseases was developed and approved in 2013. Although there are no official designated centers of expertise as well as no specific register for rare diseases. Newborns are screened for only two disorders: phenylketonuria and congenital hypothyroidism. Currently 34 orphan drugs are available on Latvian market. Three medicines (8.8%) are included in the reimbursement drug list, all indicated for Ph + CML. 15 drugs (44.1%) were reimbursed within the framework of individual reimbursement system, and five drugs (14.7%) were provided within the program of medicinal treatment of rare diseases in children.ConclusionsMajority of orphan drugs authorized in the EU are not available in Latvia, moreover those drugs that are available are often not accessible because they are insufficiently reimbursed. Besides, approval of the national plan might be an important step towards improving situation in the field of rare diseases.

show abstract

Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013

Cited by 39 publications

References 5 publications

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

Are some orphan drugs for rare diseases too expensive? A study of purchase versus compounding costs

Rare diseases and orphan drugs: Latvian story

Contact Info

Product

Resources

About