Impact of orphan drugs on Latvian budget

Konstantins Logviss,Santa Purvina,Dainis Krievins

doi:10.1186/s13023-016-0434-y

Konstantins Logviss, Santa Purvina + Show 1 more

Open Access

https://doi.org/10.1186/s13023-016-0434-y

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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.

Highlights

Number of orphan medicinal products on the market and number of rare disease patients, taking these usually expensive products, are increasing
Four drugs were provided through multiple reimbursement mechanisms: Sprycel and Wilzin were provided individually prior to inclusion in the reimbursement list; Glivec was simultaneously reimbursed individually and through the reimbursement list; Cystadane was provided through the individual reimbursement and the Clinical University Hospital (CCUH) program
Therapeutic areas of orphan drugs Oncological drugs represented more than a half of the total orphan drug expenditure, followed by the drugs for metabolic and endocrine conditions and the medicines for cardiopulmonary diseases

Summary

Introduction

Number of orphan medicinal products on the market and number of rare disease patients, taking these usually expensive products, are increasing. 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. Orphan drugs are medicinal products intended for the treatment, diagnosis or prevention of life-threatening or seriously debilitating rare conditions affecting less than one person in 2 000 individuals across the European Union [1]. Orphan medicinal products are often highly expensive [8] Factors, such as costs of research and development, marketing exclusivity, lack of alternative therapies, disease severity, and small market size can affect orphan drug prices. Orphan drugs for treatment of diseases with lower prevalence generally have higher costs than drugs indicated to treat more common conditions [3, 9]. Standard cost-effectiveness criteria are often not applicable to orphan drugs [4, 8], considering the high costs of these medicines and often modest health gains

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