Lessons learnt from national initiatives. Presentation of the Bulgarian National Plan
Rumen Stefanov
Information Centre for Rare Diseases and Orphan Drugs, Bulgaria
THE START
2004 Nov 2004 Dec 2006 Feb – First letter to the Ministry of health for a national plan for RD – Article published about importance of national plans in a leading national public health journal – Second letter to Ministry of health, supported by:
RDTF, ORPHANET, CNMR (Italy), EURORDIS National medical consultants Bulgarian patient organizations
2006 May 2006 Nov 2006-2008 2008 Feb 2008 Sept 2008 Nov
– A working group established at the Bulgarian Ministry of health to draft a national plan – First draft of the National plan on rare diseases submitted to MoH – 4 working groups, working on the draft of the plan – RD in the agenda of the Parliamentary healthcare commission – Final version of national plan, approved by the Minister of health – Awaiting budget approval by the Council of ministers
STATE OF THE ART
National Plan for Rare Diseases 2009-2013 (genetic, birth defects and non-inherited diseases)
To create an adequate institutional framework and mechanisms, ensuring timely prevention, diagnosis, optimal treatment and rehabilitation of patients with rare diseases (genetic, birth defects and non-inherited diseases) in Bulgaria.
Patient associations Awareness Research Neonatal screening Integration Medical training Genetic services EU collaboration
Budget: 11.3 M €
Epidemiological surveillance
LESSONS LEARNT
EU good practices and support Strong patient organizations or alliance
Political goodwill
RD PLAN
Medical community support
Public support and sympathy
FUTURE
RD plan will:
Adapt healthcare system for rare diseases Increase knowledge on epidemiology of RD Improve neonatal screening and genetic services Feasibility study on Reference centres for RD Increase public awareness, medical education European “added value” for other MS
Problems to solve:
Registration of new orphan drugs (small country) Access to orphan drugs and optimal dosage (small budget) Transparency of reimbursement procedures Social services to rare disease patients and families