Drugs to treat several rare and severe diseases added to state-funded list
The list of medications covered by the Estonian Health Insurance Fund was expanded in January, with new additions including treatments for serious and rare diseases.
Getter Hark, head of the Estonian Health Insurance Fund's (Tervisekassa) medicines reimbursement service, said that the new medications added in January will amount to nearly €4 million from the overall medicines budget in their first year of coverage.
The additions include treatments for various cancers, such as colorectal, cervical, endometrial and lung cancer, lymphoblastic leukemia and multiple myeloma — as well as three serious chronic diseases: systemic lupus erythematosus, polymyalgia rheumatica and transthyretin amyloidosis. Medications were also added for three rare conditions: paroxysmal nocturnal hemoglobinuria, progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome.
One of the new substances added to the Fund's list of reimbursable treatments is atezolizumab, used for treating lung cancer. Small-cell lung cancer is the most aggressive form of the disease, accounting for roughly 10 to 15 percent of all lung cancer cases.
The disease is usually discovered only after it has spread or reached an advanced stage and, until now, with existing treatment options, the average five-year survival rate for patients with stage III or IV cancer was about 5 percent.
"Until now, patients have only had access to chemotherapy. The addition of atezolizumab to the Health Insurance Fund's reimbursement list offers, according to clinical studies, an average life expectancy increase of two months. However, there is a small subset of patients — about 10 to 12 percent — who derive long-term benefit from the treatment, with the disease remaining under control for several years. This raises the previous five-year survival rate from 5 to 12 percent," Hark explained.
According to her, this change will affect around 64 people annually.
About a third of cervical cancer cases are diagnosed at an advanced stage. In such cases, the first-line treatment is pembrolizumab, but only for patients whose tumors have a specific biomarker (PD-L1).
"For other patients, treatment must begin with platinum-based chemotherapy. Starting in January, we've made cemiplimab available with a reimbursement as a second-line treatment for these patients. Previously, they only had access to further chemotherapy after first-line treatment," Hark said.
She noted that the challenge is that tumors may stop responding to chemotherapy in the second-line setting and switching or combining different chemotherapy drugs no longer yields results.
Studies show that patients undergoing chemotherapy in such cases have an average life expectancy of up to nine months. With the addition of cemiplimab to the reimbursement list, it is now possible to significantly improve quality of life and extend survival by an average of 3.5 months.
Tervisekassa is also now covering pembrolizumab for a new indication: first-line treatment of advanced endometrial cancer, regardless of whether the patient has mismatch repair-deficient or proficient genes. Around 240 cases of the disease are diagnosed in Estonia each year.
"Unlike several other cancers, the incidence of endometrial cancer is rising. We've decided to fund pembrolizumab, which delays disease progression by about four months," Hark said, noting that the new treatment option will benefit about 30 patients.
Colorectal cancer is currently treated primarily with chemotherapy in both first and second-line settings. According to Hark, there is a significant need for newer therapies, especially for patients with a specific mutation (BRAF) whose average survival after first-line chemotherapy is under six months.
"Starting this year, a combination of encorafenib and cetuximab is available to patients, which extends survival by 3.4 months. About 19 patients per year require this treatment," said Hark.
Tervisekassa also improved treatment options for people with polymyalgia rheumatica (PMR), a condition characterized by rapidly developing severe pain in the shoulders, pelvic area and thighs, which worsens over days or weeks.
The pain is inflammatory in nature and especially severe at night and in the early morning. A hallmark of the condition is pronounced stiffness in the limbs — so intense that patients may be unable to lift their arms or perform everyday tasks such as washing, dressing or combing their hair. Fatigue, fever and weight loss often accompany the condition.
"This disease has a severe impact on quality of life — mobility is restricted and it can lead to partial or total work incapacity. Older patients may lose their independence and require daily assistance or care," Hark explained.
Previously, patients were treated with glucocorticoids, which help control inflammation. However, long-term use (60 days or more) comes with significant side effects: infections, osteoporosis, high blood pressure, diabetes, gastrointestinal ulcers, cataracts, skin changes and psychological issues.
The introduction of tocilizumab now offers patients a treatment that controls inflammation and reduces the need for glucocorticoids, thereby lowering the risk of serious side effects. More than 50 patients per year are expected to need this treatment.
As of this year, the drug teclistamab is available for treating multiple myeloma in patients whose disease has progressed after treatment with at least three previous drugs. These patients previously had no effective options and the new drug extends life expectancy by 10 months. About 15 patients per year will benefit from this treatment.
In early January, treatment options were also added for two very rare diseases: progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome.
These conditions are marked by uncontrollable itching, bile flow disorders, growth delays and the potential need for a liver transplant. The active substance odevixibat, now added to the list of reimbursed medications, lowers bile acid levels and reduces itching, significantly improving quality of life.
"In Estonia, a child with intrahepatic cholestasis (PFIC) is born once every four to eight years and a child with Alagille syndrome once every three to five years. Should a treatment need arise, the necessary medications are now already included in Tervisekassa's list," said Hark.
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Editor: Karin Koppel, Marcus Turovski
