Instead of the required 19 million euros, patients with rare diseases have received round zero this year for expanding treatment options, according to the informational report of the Ministry of Health “On ensuring the improvement of the availability of compensable medicines for 2024 and the following years”, approved by the Cabinet of Ministers.
The Alliance of Rare Diseases and the Association of Rare Disease Specialists of Latvia (RSSA) agree that the decision not to include medicines for the treatment of newly diagnosed rare diseases in the list of compensable medicines this year means that many patients will be deprived of their only opportunity to start state-paid treatment.
The informational report of the Ministry of Health (MoH) predicts that the list of compensable drugs will be supplemented with innovative drugs for patients of other disease groups, while the needs of people with rare diseases will be ignored, although there is still an acute shortage of state-paid drug therapy for rare diseases.
“For patients with rare diseases, the information report of the Ministry of Health has zero value, because this year the treatment options for our patients will not be expanded and a large number of people will be without therapy to ensure life functions. Currently, most of the drugs on the R list of compensable drugs have been authorized by the European Medicines Agency in the period from 1997. until 2013. There are 16 medicines older than 10 years on the R list, while 9 medicines have been authorized in Europe in the period from 2014 to 2024.
Only two diagnoses on the R list have drugs registered in the last couple of years. So, it’s not about absolutely innovative things waiting for their turn to be R-listed. We are still fighting for what has been available elsewhere in Europe for several years. I believe that our country must provide effective treatment and include drugs that have already been evaluated and recognized as cost-effective in the R list, so that in the end both children and adults with rare diseases are provided with adequate health care,” says Ieva Plūme, a member of the board of the Alliance of Rare Diseases.
RSSA board chairman Andris Skride also expresses concern about the informational report of the Ministry of Health, stating that it is a bad signal both for doctors who see an opportunity to help patients with the development of treatment options in the world, and for patients who are limited in receiving appropriate treatment.
“The reality is that if there are no compensable drugs available in the country for the specific rare disease, the treatment does not take place. The patient only has access to supportive therapy with the aim of preventing the discomfort caused by specific symptoms. It is hard to see that medical possibilities are developing, but we have not made any progress this year, which means that treatment options for patients with rare diseases have been frozen,” comments prof. A. Skride.
“Discrimination of patients with rare diseases is unacceptable, and the right to treatment is also available to patients with those diagnoses of rare diseases that are not included in the R list and for which drugs are available and registered in the EU. It is necessary to be aware that there are diagnoses where the specific drug is not an alternative, but the only choice. According to the data of the National Health Service, 19 million euros are needed for the treatment of new diagnoses of eight rare diseases this year, and we hope that the Ministry of Health will receive support in the funding request in order to find an opportunity to expand treatment for rare disease patients this year as well,” explains I. Plume.
