Reimbursement of drugs for ultra-rare diseases in Lithuania

Abstract

Introduction and objectives: Drugs for ultra-rare diseases in Lithuania are reimbursed on a named-patient basis. The hospital in which patient is being treated can request to receive a non-reimbursed or off-label treatment if the disease’s incidence is 1 per 200,000 people in Lithuania. Other criteria for reimbursement include the drug having orphan drug designation by European Medicines Agency (EMA), authorized indication in European, Lithuanian or any other European country’s national registry and clinical evidence. All this information is presented by the hospital and is evaluated by the State Medicines Control Agency Health Technology Assessment (HTA) unit, and then the deliberation is made at the Committee for reimbursement of ultra-rare diseases (Committee). The Committee makes the final decision if a drug should be reimbursed. Our main objective of this study was to analyse submission evaluation and decision-making processes. Methods: This cross-sectional survey was conducted during the sixmonth period (August 1st, 2020 till January 31st, 2021). HTA unit was responsible for evaluation of the drug according to the criteria (see above) and assessment of the clinical data (shown as direct endpoints). HTA unit’s evaluation does not involve cost and budget impact assessment. Additionally, we checked what the decision of the Committee was. The results are presented as descriptive statistics. Results: During the six-month period, HTA unit evaluated 33 drugs for ultra-rare diseases. 12 out of the 33 of the requested drugs were intended for haematological or oncological diseases. 7 out of the 33 drugs had orphan designation by the EMA. 19 out of the 33 drugs had authorized indication in European, Lithuanian or any other European country’s national registry, other drugs sought to be reimbursed off-label. 21 out of the 33 drugs were evaluated in clinical trials, clinical cases or guidelines using direct [...].