Orphan drugs - Interpharma

Putting the focus on patients

Orphan drugs

A more efficient reimbursement process is needed to ensure that patients in Switzerland can benefit sooner from medical innovation.

Overview The authorisation and reimbursement process Pricing Off-label use Orphan drugs Rationing

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.

Further information

About us

Interpharma, the association of Switzerland’s research-based pharmaceutical industry, was founded in Basel in 1933.

Interpharma informs the public about issues that are important to the research-based pharmaceutical industry in Switzerland, including the pharma market in Switzerland, healthcare and biomedical research.

Annual report

Information on our key figures and activities in the financial year 2020

read more

Board and Executive Management

Introducing Interpharma

read more

Vision and Mission

More about the tasks and overriding aims of Interpharma

read more

Contact

Contact us

read more

Media

Latest information and media contacts for media representatives

read more