Impact of orphan drugs on Latvian budget

Abstract

BackgroundNumber of orphan medicinal products on the market and number of rare disease patients, taking these usually expensive products, are increasing. As a result, budget impact of orphan drugs is growing. This factor, along with the cost-effectiveness of orphan drugs, is often considered in the reimbursement decisions, directly affecting accessibility of rare disease therapies. The current study aims to assess the budget impact of orphan drugs in Latvia.MethodsOur study covered a 5-year period, from 2010 to 2014. Impact of orphan drugs on Latvian budget was estimated from the National Health Service’s perspective. It was calculated in absolute values and relative to total pharmaceutical market and total drug reimbursement budget. A literature review was performed for comparison with other European countries.ResultsOrphan drug annual expenditure ranged between EUR 2.065 and 3.065 million, with total 5-year expenditure EUR 12.467 million. It constituted, on average, 0.84 % of total pharmaceutical market and 2.14 % of total drug reimbursement budget, respectively. Average annual per patient expenditures varied widely, from EUR 1 534 to EUR 580 952. The most costly treatment was enzyme replacement therapy (Elaprase) for MPS II. Glivec had the highest share (34 %) of the total orphan drug expenditure. Oncological drugs represented more than a half of the total orphan drug expenditure, followed by drugs for metabolic and endocrine conditions and medicines for cardiopulmonary diseases. Three indications: Ph+ CML, MPS II, and PAH accounted for nearly 90 % of the total orphan drug expenditure.ConclusionsBudget impact of orphan drugs in Latvia is very small. It increased slightly over a period of five years, due to the slight increase in the number of patients and the number of orphan drugs reimbursed. Current Latvian drug reimbursement system is not sufficient for most orphan drugs.

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

“…Share of orphan drug expenditure in total drug expenditure also showed a similar trend(Red line on Figure 2 1% of total drug expenditure over a period of eight years. Th is percentage is similar to that reported in 2004 in an early European study (0.7-1% per country) [60] and in one recently published Latvian study for 2010-2014 (0.70-1.04%) [18]. However, when comparing these results, it should be kept in mind that methods and data sources vary signifi cantly (sales data, national health care funds etc.).…”

“…Th e market for orphan drugs is diswww.hophonline.org tinguishable because of specially designed regulation [18]. Orphan drugs are highly expensive due to market exclusivity, lack of alternative therapy and small market size [16,18,19]. Legislation in essence creates small virtual monopolies for each orphan drug [15,20].…”

“…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].…”

“…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]. Oncology orphan drugs are associated with highest percentage of expenditures on orphan drugs [10,18,24,26,28]. In Czech Republic oncology orphan drugs generated up to 72.6% of the overall orphan drugs expenditures in 2013 [26].…”

“…In Czech Republic oncology orphan drugs generated up to 72.6% of the overall orphan drugs expenditures in 2013 [26]. In Latvia oncology drugs represented 52.99% of the total orphan drug expenditures [18]. Th ese uneven shares point out to the diff erent pace and nature of pharmaceutical spending evolution among the old EU-15 compared to Eastern European nations [30].…”