Rare diseases and orphan drugs: Latvian story

Abstract

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.

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

“…In 2009 the European Council issued the recommendation for EU member states to create and adopt a plan focused on rare disorders by the end of 2013, with the goal to have an overall Community strategy for "ensuring effective and efficient recognition, prevention, diagnosis, treatment, care, and research for rare diseases in Europe" (46). For this purpose, the European Project for Rare Diseases National Plans Development (EUROPLAN) was introduced to promote and help EU members with the construction and implementation of their national plans 15,16 . NL, DE, UK, LV have created a national plan within the timelines defined by European Commission but in most of these EU countries the implementation is in progress (16, 29) 17,18,19,20 .…”

“…The reimbursement status of orphan drugs in Eastern Europe has been described by several authors recently (11)(12)(13)(14)(15). There have been multiple publications describing OMP policies in Central and Eastern Europe in single countries (16)(17)(18) or covering a larger number of countries in Europe (5,(19)(20)(21)(22). Pejcic et al focused on HTA and pricing as well as rare disease policies in 14 Eastern European countries (23).…”

“…Description of National Plan for Rare Diseases per country(16,29) 4 , (54-64)25,26 , (65) 27 , (66) 28,29 .CountryCharacteristics of the national plansARNo NP or special legislation for rare diseases.DENP developed and implemented: 7 focus areas, 52 proposed solutions, implemented in 2013. Twenty-eight rare disease institutions have been working together under the name NAMSE since 2009.…”

“…Comparison of reimbursement systems of orphan drugs and rare diseases policies(15,16,29, 70-72) 4, 24 , (68)30, 31 , (11, 69) 32 ,(73)(74)(75) …”

Policies and availability of orphan medicines in outpatient care in 24 European countries

“…Even though the challenges facing orphan medicines have been internationally recognised, research on the availability and reimbursement policies of orphan medicines is limited [5,11]. Publications and reports are often focused on a single country [14][15][16][17] or a limited number [11,[18][19][20][21][22][23] of countries. The latest comprehensive reports on multiple European countries, which suggest considerable variation in the availability and policies of orphan medicines between countries, were published in 2007 and 2011 [24][25].…”