Research on drugs for rare diseases in Latvia

On 5 October at 15:00 in an open session of Rīga Stradiņš University Pharmacy Promotion Council (Hippocrates Lecture Theatre, 16 Dzirciema iela, Rīga) Mr Konstantīns Logviss will defend his thesis titled "Pharmacoepidemiologic and Pharmacoeconomic Latvian Study of Rare Diseases and Orphan Drugs".

Rare or orphan diseases are life-threatening or chronically debilitating conditions of differing origin. The majority of them are genetic disorders, others are rare malignant cancers, congenital malformations, autoimmune, toxic and infectious diseases. Orphan diseases have been recognised as a priority area for the European Community action in the public health system, though there are significant differences in the national healthcare services for rare disease patients among the EU States.

The aim of the study was to determine the situation in the area of rare diseases in Latvia, assess the impact of orphan drugs on the state budget and compare the findings with data from other European countries. The following sources of information were used in the study: National Plan for Rare Diseases, EUCERD reports, Orphanet data, laws and regulations of the Cabinet of Ministers and the European Union, publicly available data of institutions and registers of Latvia and the EU (State Agency of Medicines of the Republic of Latvia, The National Health Service, European Medicines Agency, European Community Register of Medicines and EU Clinical Trials Register), as well as direct communication with drug manufacturers and wholesalers. The impact of orphan medicines was analysed from the perspective of the National Health Service and covered the period from the beginning of 2010 to the end of 2014. A literature review was conducted to compare the results with other European countries.

Development and approval of two national plans for rare diseases (2013–2015 and 2017–2020) is an important step towards improving the situation in the area of rare diseases in Latvia. However, there are neither any official rare diseases expertise centres nor a unified rare diseases patient register in Latvia. A total of 51 interventional clinical trials relating to rare diseases which were conducted in Latvia were identified through the EU Clinical Trials Register. All the above-mentioned trials were multi-centre international studies. Besides, it was discovered that rare disease trials differ from non-rare disease trials. Significant differences were traced in randomisation (ensuring randomness), blinding, the number of patients involved and the use of active control, whereas the author found no significant difference in trial duration and the use of overall survival (as a primary endpoint).

Compared to other European countries, the budgetary impact of orphan drugs in Latvia is very small. Orphan drug annual expenditure ranged between 2 065 and 3 065 million EUR constituting, on average, 0.84 % of the total pharmaceutical market and 2.14 % of the total drug reimbursement annual budget. Total 5-year expenditure was 12.467 million EUR. Nearly 90 % of the total orphan drug expenditure covered three indications: Ph+ CML, MPS II, and PAH. The majority of orphan drugs authorised in the EU are not available in Latvia. Moreover, those drugs that are available are often not accessible because they are insufficiently reimbursed by the state, and are too expensive for patients to be able to afford them.