Orphan diseases are rare diseases with a prevalence (frequency with which they occur) of less than 5 per 10,000 in the population. Compared with the large number of conditions that come under this heading, there are relatively few specific medicines, known as “orphan drugs”, available to treat them.
The authorities introduced incentives for the pharmaceutical industry to research and develop orphan drugs to make this a more attractive proposition. As long ago as 1983, the USA introduced a law to promote the development of medicinal products to treat rare diseases. The EU created a similar legal foundation in 1999 in the form of Regulation (EC) No 141/2000. It foresees various measures to promote the recognition and visibility of rare diseases and reinforces collaboration and coordination at EU level. It supports the creation of European reference networks and promotes the networking of specialist centres and experts in different countries. It has also created incentives for more research into rare diseases. Moreover, rare diseases are one of the priorities within the EU Framework Programme for Research and Innovation. These supportive measures have resulted in an increase in the number of authorised medicines for the treatment of rare diseases.
Orphan drugs in Switzerland
In Switzerland, Art. 14 of the Therapeutic Products Act (TPA) provides for a simplified authorisation procedure for important medicinal products for rare diseases. The implementing provisions have been incorporated into the Ordinance of the Swiss Agency for Therapeutic Products on the Simplified Licensing of Therapeutic Products and the Licensing of Therapeutic Products by the Notification Procedure (TPLO). A distinction is made in the Ordinance between recognition of the status as an important medicinal product for rare diseases (Art. 4 – 7 TPLO) and the authorisation of a medicinal product that has been granted Orphan Drug Status by Swissmedic (Art. 24 – 26 TPLO).
Orphan drugs are reimbursed either in accordance with the List of pharmaceutical specialities or in accordance with Art. 71b HIO under the same conditions as for off-label use. These conditions state that the cost of medicinal products that are not on the List of pharmaceutical specialities and used outside the registered indications will also be reimbursed. However, this is only the case if the disease may have a fatal outcome or lead to severe or chronic problems for the insured person, and there are no therapeutic alternatives.
This decision-making process is a complex one, and patients with rare diseases are frequently in a considerably worse position with respect to reimbursement than patients with more common disorders.