In Hungary the need for a special approach to rare disease was recognised as early as 2000, when the first Hungarian Rare Disease Division at Debrecen University was set up both for out and inpatient care. The initiative quickly received the support of different stakeholders: policy makers, health authorities, opinion leaders, health care providers (HCP), university medical schools and rare diseases patient associations. It is worthwhile to mention that 55 Hungarian patient associations form a strong and effective umbrella association called the Hungarian Federation of People with Rare and Congenital Diseases (HUFERDIS). The present 2014-2020 National Plan on Rare Diseases reflects well the opinion and interest of each of the stakeholders mentioned above. It should also be noted that important help was from the outset provided by French rare disease associations and experts, such as Ms Ségolene Aymé (Research Director, at that time, at INSERM (French national institute for health and medical research), specialised in the field of genetics and rare diseases and at the origin of Orphanet, reference website in the field of rare diseases), who personally visited the country.

In 2017 five Centres of Expertise (CoE) were set up and clear patient pathways put in place to guide patients from their General Practitioners to these centres or to other specialised HCPs. The CoEs have an important coordinating role for clinical care of patients affected by rare diseases: once a patient with a suspected rare or complex (undiagnosed) disease has been seen, his or her case will be reported to the appropriate member of the “virtual clinic” built up inside the “real clinic”. The “virtual clinic” is made of specialised units within the CoEs (university clinics, the highest level care), where a specialist is named and is responsible for the RD-patients sent to that special ward. In this way, the expertise located in different CoEs works in a network modality. The five Centres of Expertise are taking part in the ERNs through their different specialised units (total of 14), ensuring that national expertise is linked to that at European level. Five additional affiliated partners have also been designated and will soon be enabled to collaborate with additional ERNs at European level. Each CoE regularly organises both graduate and postgraduate courses on rare diseases focussing on case reports to improve clinical awareness and clarification of ideal patient pathways. The Hungarian members of ERNs play a pivotal role in this and from time-to-time a guest lecturer of an ERN-member HCP from abroad is also invited.

An Electronic Health Records (EHRs) system has also been launched in Hungary. Specific efforts are being made to fully integrate Orpha codes in the patient set of health data, enabling health professionals to have direct access to the patients’ rare disease information.

National Clinical Practice Guidelines and Clinical Decision Support Tools (CPG, CDST) are currently under revision, and the new versions are being revised in particular to be aligned with ERN expectations.

Orphan drug availability in Hungary is secured for each patient. The system works electronically and based on guidelines and real-time collection of data on the patient's health status. Results are evaluated by an Expert Committee working as an advisory group, and decision are made by the National Health Insurance Fund of Hungary (Hungarian acronym: NEAK). A special ward inside NEAK is responsible for rare patients who require expensive orphan drug care and reimbursement. The members of the decision-preparing board are medical experts, representatives of governmental institutions and patient organisations.