Rare diseases and orphan drugs: Latvian story

Orphanet Journal of Rare Diseases

Santa Purvina¹  Dainis Krievins²  Konstantins Logviss¹ 

[1] Department of Internal Diseases, Division of Pharmacology, Riga Stradins University, 13 Pilsonu Street, Riga LV-1002, Latvia;University of Latvia, 19 Raina Boulevard, Riga LV-1586, Latvia

关键词: Latvia;    Accessibility;    Availability;    Orphan drugs;    Rare diseases;   

Others  :  1149884
DOI  :  10.1186/s13023-014-0147-z

 received in 2014-07-07, accepted in 2014-09-09,  发布年份 2014

【 摘 要 】

Background

Ten 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.

Methods

We 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.

Results

National 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.

Conclusions

Majority 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.

【 授权许可】

   
2014 Logviss et al.; licensee BioMed Central Ltd.

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