Orphan drugs expenditure in the Netherlands in the period 2006–2012

Abstract

BackgroundThe relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of orphan drugs can still be substantial. In this study, we assess the uptake and budget impact of orphan drugs in the Netherlands.MethodsWe examined the number of orphan drugs, the number of patients and budget impact of orphan drugs in the Netherlands in the period 2006 to 2012, both for inpatient and outpatient orphan drugs. Budget impact was provided in absolute numbers and relative to total pharmaceutical spending.ResultsThe number of orphan drugs and patients treated increased substantially over the period studied. Overall, budget impact increased substantially over a period of six years, both in absolute terms (326% increase) as well as relative to total pharmaceutical spending (278% increase). Growth rates decreased over time. In 2012, 17% of available drugs had an individual budget impact of more than €10 million per year.ConclusionsIndividual budget impact of orphan drugs is often limited, although exceptions exist. However, in total, the budget impact of orphan drugs is considerable and has grown substantially over the years. This could potentially influence reimbursement decisions for orphan drugs in the future.

Addressing the challenge of high-priced prescription drugs in the era of precision medicine: A systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks

“…European regulatory bodies offer exclusive licensing for ten years, lower regulatory fees and scientific advice. [ 148 ; 150 ] Orphan drug legislation has worked, yielding at least 73 drugs for orphan indications in the European Union since the law passed in 2000[ 151 ] and 335 in the US since the FDA set regulations in 1983 and 2002[ 152 ].…”

“…The moral argument for orphan drug regulation is that society, and medical science in particular, has an obligation to pursue new therapies for everyone, including people who suffer from orphan diseases. [ 149 ; 152 ] The downside of this policy is the non-utilitarian outcome that money is being used for diseases that very few people have, and that the same money could have been used for more relevant research reaching a larger population[ 1 ; 145 ; 151 ; 152 ].…”

Budget impact analysis of medicines: updated systematic review and implications

“…Features of the health care system: 22 studies (24%) [12,13,15,16,18,19,26,33,35,48,51,54,77,81,83,84,86,88,92,93,100,102] described some type of feature of the health care system in which the analysis was performed. The most reported characteristics were universal health coverage (59%).…”

Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia

“…Th e share of orphan drugs in total pharmaceutical market in 2013 reached 3.6% in Czech Republic [26] and 3.2% in Poland [27]. [23]. Countries located in North America reported even higher shares.…”

“…Studies conducted in various countries reported that share of orphan drug expenditure had increased since introduction of orphan drug legislation [10,18,[23][24][25][26][27][28]. However, this share represented mainly low percentage (less than 8%) of overall drug expenditure [10,18,[23][24][25][26][27][28]. Picavet et al observed that principal determinants in the market uptake of orphan drugs across 23 countries in Europe were the gross domestic product and the availability of a formal technology assessment organization [29].…”