Zagreb, Croatia 2019.
On February 28, as part of the International Day of Rare Diseases, I participated as a member of the Parliamentary Committee for Health and Social Policy, in a panel discussion on rare diseases. With the presence of patients, their families, and the interested public, I discussed, together with the representatives of all the relevant bodies, about the possibilities of improved treatment of rare diseases in Croatia.
In Europe, rare diseases affect about 6-7% of the total population – approximately 36 million people. It is estimated that in Croatia there are about 250,000 people with a rare disease. Rare illnesses are predominantly chronic and progressive, some of which are deadly and represent a heavy burden not only on the affected patients as well as their families due to inherent specific social needs. In orthopedics and traumatology diseases such as algodistrophy and avascular necrosis of the head of the thigh bone belong to such group of diseases.
The health care of these patients is mostly inadequate, and due to their low prevalence and low impact on morbidity and mortality, these patients are often neglected in the field of research, especially in examining treatment options.
Because of the inequality in the availability of treatment and health care for this group of patients, also known as Orphan drugs, the EU wants to improve the present situation with its intervention. As such, the patients’ access to appropriate and timely diagnostics, information and care is more effective when operating at a European level than at a national level.
The European Network of Rare Diseases is part of the public health system with the participation of health ministers, patients, the pharmaceutical sector, doctors, researchers, as well as lobbyists who pressure for policy changes.
European reference networks have a strategic role to play in harmonizing care and improving patient care across the EU. National centers in the network are voluntary members, recommended by national authorities, with the option of becoming member or collaborators.
The long-term plan is for travel from one center to another within the same network for the purpose of confirming the diagnosis, seek a different opinion, or do a medical procedure (surgical operation, transplantation, invasive medical intervention) to become commonplace. In 2015, the Republic of Croatia has adopted its National Program for Rare Diseases 2015-2020.