As defined by the European Agency for the Evaluation of Medicinal Products, “orphan drugs” are medicines developed for the treatment of rare life-threatening or chronically debilitating conditions.

There are very few orphan drugs, and they are often unavailable for lack of laboratories to ensure their production. Indeed, the process of developing a new molecule, from its discovery to its commercialization, is long, extremely expensive and unpredictable. Pharmaceutical companies are often reluctant to develop drugs whose number of sales, necessarily small, will not cover the cost of bringing new products on the market.

To address this problem in Europe, many governments and associations struggled in the ‘90s to encourage pharmaceutical laboratories to invest in the development of orphan drugs through economic incentives.

In 2000, the European Parliament adopted a regulation providing to orphan drugs tax advantages, assistance with the marketing protocol for the medicinal product and patent exclusivity for ten years. The European legislation is today offering to the industry the possibility of developing niche markets particularly interesting to small and medium-sized enterprises. These incentives are only partially successful as the number of orphan drugs remains very limited and the majority of rare diseases do not benefit of effective treatments.


At national level in Switzerland, the answer is an “Implementation Plan” that provides patients with equitable access to diagnosis, treatment and care. This plan includes psychosocial support for patients and their relatives, the presence of hospital and cantonal coordinators to support and guide them. The Swiss Implementation Plan also includes a component to encourage research and collection of epidemiological data, as well as an emphasis on training and dissemination of information.

The Swiss Federal Council adopted the National Rare Diseases Policy on 15th October 2014 and its Implementation Plan on 13th May 2015. The implementation of the national concept is build around four projects with 19 concrete measures.