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27-02-2026

Reimbursement for the treatment of very rare conditions: more decisions – more opportunities

Iliustracija

Funding for the treatment of very rare human health conditions is steadily increasing each year, and the number of patients receiving treatment is also growing, according to the analysis performed by the National Health Insurance Fund. Between 2020 and 2025, the assets from the Compulsory Health Insurance Fund, allocated to cover the costs of treating these conditions, increased 2.5 times, while the number of treated patients grew 1.7 times. This indicates that even for the rarest and most complex diseases, Lithuania is actively seeking solutions to cover the costs of extremely expensive medicines—through negotiations with manufacturers of medicines, assessment of clinical data, and ensuring sustainable financing.

Compensation for the treatment costs of very rare diseases in Lithuania is individualized—requests submitted by healthcare institutions are assessed based on each patient’s situation. Decisions regarding the funding of such treatment are made by the Commission for the Reimbursement of Treatment Costs for Very Rare Human Health Conditions. The National Health Insurance Fund (NHIF) coordinates the work of this commission and, based on its decisions, issues guarantee letters to healthcare institutions. These letters ensure that the institution will be reimbursed for the patient’s treatment costs, allowing the patient to receive the necessary care without additional financial burden.

Last year, more than EUR 22 million was allocated from the fund for the treatment of very rare conditions, and 469 patients received treatment. This shows that access to treatment is improving, but at the same time, the need for funding is increasing.

“Expenses for very rare conditions are growing faster than the number of patients, as innovative and extremely expensive medicines are increasingly used in treatment. When making decisions, we first assess their therapeutic benefit to ensure that funding is justified and that patients can start treatment as quickly as possible. In every case where reimbursement is requested for a medicine whose annual treatment cost per patient exceeds EUR 100,000, we refer the matter to the negotiation commission to reduce the treatment price,” says Irma Medžiaušaitė, Chair of the Commission for the Reimbursement of Treatment Costs for Very Rare Human Health Conditions.

According to her, treating these conditions requires consistent and responsible decision-making. Last year, the commission reviewed 311 applications—significantly more than in previous years. Over the past five years, the number of applications submitted for review has increased 1.8 times. This growth reflects both a greater need for treatment and more proactive requests from healthcare institutions for individual patient funding.

The largest portion of funds allocated for the treatment of very rare human health conditions each year is used to continue ongoing, extremely expensive treatments – for patients treated for very rare metabolic, oncological, genetic, or autoimmune diseases. However, a significant share of expenses also goes to patients who are just beginning treatment with very expensive medicines.

“Last year, we had 28 new patients for whom negotiations were conducted to reduce treatment costs. The average treatment cost varied widely, reaching as much as EUR 211,000 for some patients. Therefore, one of the main tasks is to ensure that the fund resources are sufficient for all patients with very rare diseases who require innovative and life-saving treatments,” says I. Medžiaušaitė.

In 2025, reimbursement was started for 13 new medicines for patients treated for very rare diseases. These included treatments for a very rare form of amyloidosis, where abnormal protein accumulation damages vital organs, as well as paroxysmal nocturnal hemoglobinuria, a rare blood disorder in which the immune system destroys the body’s own red blood cells. Favorable reimbursement decisions were also made last year for patients treated for severe, widespread forms of epilepsy, fungal granulomas (a rare form of skin lymphoma), certain inherited metabolic disorders, and others.

“The treatment of these diseases is often long-term and complex, and without specific medicines, the disease can progress rapidly, causing severe disability or even life-threatening complications. Therefore, the commission’s decisions on new medicine reimbursements provide patients with a real opportunity to begin treatment and prevent disease complications,” emphasizes I. Medžiaušaitė.

The National Health Insurance Fund reminds that a very rare human health condition is defined as one that is diagnosed in no more than one person per 200,000 residents per year. Although each very rare condition is extremely uncommon individually, together these diseases affect hundreds of residents.

The NHIF invites you:

Last updated: 25-03-2026

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