According to an article published earlier this year by the SINTEF Group, the largest independent research organisation in Scandinavia, there exist some 30,000 people in Norway with rare hereditary or congenital disorders. With a total population of around five million in Norway, a rare disease is defined as affecting less than one person in ten thousand, which means that to be considered rare, a disorder will affect less than 500 of the country’s population.
Norway’s specialised healthcare is divided into four regions. There are 16 different state-financed resource centres established for the country’s rare disease patients. In order for a service to be established for a disorder at one of the resource centres, the condition must meet the criteria of being “congenital and complex/compound and there must be a need for multidisciplinary and cross-institutional services”. To date, services have been established for over 300 diseases, resulting in (in 2006) about 16,000 registered users at the resource centres. The Norwegian Directorate of Health, Helsedirektoratet, the country’s competent authority, responsible for technical functions as well as certain administrative roles, coordinates and monitors the services for rare disease patients in Norway. The centres contribute to the process of enabling patients to access the same work, educational and social opportunities as all Norwegians. The centres take up the needs for various forms of support unmet by standard services available. The centres also facilitate the development and dissemination of expertise. Monies for the centres are established via the state budget.
Helsedirektoratet maintains a free help-line for rare conditions available to patients, family members and professionals. Since the establishment of this service in 1999, enquiries have been received concerning over 700 rare disorders. Their website offers publications available to the public that directly and/or indirectly address rare disorders. One such publication, entitled Children and young people with disabilities – what are the family’s rights? discusses health care, education, housing, and service resources for persons with disabilities. Specific resources for rare diseases are outlined. Caregivers, for example, of patients with rare disabilities are permitted a training allowance in Norway. These are designated for parents to attend courses at a resource centre for rare disabilities, and include travel expenses. Furthermore, for families of children with rare diseases, support may be available to cover the cost of taking part in courses abroad, conditional on there being no resource centre or expert group for the rare disease in Norway. Another document available on the Helsedirektoratet website entitled Having children when the parents are blood relatives specifically discusses the risk for rare inherited disorders in consanguineous marriage. The Directorate of Health also contributes to international efforts. Collaboration with Rarelink, a Nordic website that offers a compilation of electronic links furnishing rare disease information and contact details compiled by organisations commissioned by the governments of Norway, Sweden, Denmark and Finland, is an example of cooperation at the international level.
At present, neonatal screening is routinely performed for PKU and congenital hypothyroidism. A working group established by the Directorate of Health in 2008 has recommended expanding the existing screening programme to include 23 disorders. Genetic counselling is offered free of charge as part of the public health services.
The Norwegian Medicines Agency (NOMA) determines which medicines can be reimbursed by the government from a predefined list. There are several criteria to be fulfilled before NOMA is allowed to add a medicine to the list. If a medicinal product does not have Norwegian Marketing Authorization, it cannot be placed on this predefined list. In such situations, the physician must apply for individually-based reimbursement. Officially, of the 58 orphan products that have received marketing authorisation from the European Medicines Agency, only one is systematically reimbursed in Norway. Inovelon, indicated for Lennox-Gastaut, a rare and severe form of epilepsy, has received approval for full reimbursement in Norway. For all other orphan drug usage, the treating physician must go through the process of applying for individual reimbursement for the use of a specific medicine for a particular patient. In the case of patients who are hospitalised with rare disorders, the hospital itself is financially responsible for the administration of medicines. In certain cases, home treatment is considered to be a prolongation of a hospital treatment, and the hospital will continue to assume the financial responsibility after a patient is released from hospital. Thus, rare disease medicines can be reimbursed via the official, public reimbursement system, either by meeting the criteria of the predefined list of medicines, by the individual application for a specific patient, or through hospital funding.
The funding of assistive devices has two sources. Short term allotment (less than 2 years) is financed by the municipalities, whereas long term allotment (more than 2 years) is financed by the state (through the assistive devices agency in each county). The allotment of assistive devices is governed by an agreement between the state and the municipalities.
The government contributes financially to many patients organisations. There must be 250 members in an organisation to qualify for government funding. Organisations with less than 250 members must merge with others in order to attain the sufficient number.
In 2008, the regional health authorities undertook a revision of the current cross-regional, national, and over-national resource functions (including the resource centres for rare disorders). The aim of this process is to identify from a five year perspective which centres of excellence/ resource centres are needed and where within the specialist health services they should be placed. The work will be finished in 2010, and will result in expanded services for several rare disorders.
As a user of long-term, coordinated health and/or social services, patients are entitled to an Individual Plan. Particularly relevant to rare diseases patients, and not conditional on any specific diagnosis or age, this plan will contain an outline of objectives, resources and the services the patient requires.
Despite the various mechanisms in place in Norway for rare disease patients, the SINTEF study reveals that, as is the case for every country in the world today, more specialist knowledge is needed, along with an “integrated approach” to health care. Stein Are Aksnes, country coordinator for Orphanet Norway and senior adviser for the Norwegian Directorate of Health, Department for Rehabilitation and Rare Disorders, commented that "Our system, with the national resource centres, has its definite advantages, and we try to expand this to as many users as possible. Still, we must not forget that the resource centres do not cover all rare disorders. About 14,000 people live in Norway with a rare disorder, but with no defined resource centre. Our biggest challenge is to assure the same rights and services to these persons as to the rest of the population”. Consult the SINTEF group studies on rare disorders (in Norwegian)