The needs of patients with rare disease in Serbia. Why do we need National Strategy for rare disease?

The patients with rare diseases in Serbia face the difficulties in procurement of medications as the Health insurance fund does not cover reimbursement for some medications, they face difficulties in receiving proper diagnosis which makes their position specific and complex. In an attempt to provide more support for the patients with rare diseases, their families and caregivers the helpline for rare diseases was established in October 2015. The aim of this research was to identify, examine and systematise needs of helpline users and forms of assistance provided by the team from the free helpline. The research was designed as a cross-sectional study and was conducted between October 2015 and December 2016. The electronic database of National Organization for rare disease in Serbia helpline users was used as a data source. The user was the person who contacted helpline (patient, relative, friend, physician, etc). The "need" refers to the reasons for addressing the helpline. Helpline users had 549 needs in total; about healthcare-236 (42.98%), social care-113 (20.58%), psychological support-56 (10.20%) and other-144 (26.22%). Services were provided by the lawyer-130 (23.70%), social worker-71 (12.93%), Psychologist-56 (10.20%) and by all employees-292 (53.19%). The most common need for legal assistance among needs on healthcare was on legal aspects of access to and reimbursement of expenses for medications (32/74%-43.24%) and procedures for reimbursement of treatment abroad (11/74%-14.86%). The problems of patients with rare diseases and their families result primarily from the lack of relevant information and knowledge, as well as the non-recognition of rare diseases in the laws and regulations of health and social care. Some problems can simply be solved by legal changes and by a better organisation and do not require additional funding. Only by adopting and implementing the National Strategy and Action Plan for Rare Diseases, the greatest number of problems and needs of people with rare diseases can be systematised and solved.