The lack of satisfactory treatments for many rare diseases is increasing the need for specialty pharmaceuticals.
There are some 7,000 described rare diseases, with a total number of sufferers amounting to some 30 million people in the EU and about 25 million in the US. Currently only some 400 of these rare diseases can be treated. Also, only about 600 drugs for treating rare diseases are currently being developed in the EU and less than 50 of these have been approved for use.
Existing legislation in the US and the EU supports and protects the development of orphan drugs. The EU’s new growth plan establishes health and healthcare as priority areas. In particular, there is now a clear focus on continuing to promote the development of and improve access to orphan drugs for patients with rare diseases.
Rare diseases are largely neglected due to inadequate diagnostics and many of them still lack satisfactory treatments. It is estimated that out of the 30 million rare disease patients in Europe, only two to three million are currently receiving treatment. The diseases are often life-threatening or cause chronic disability and therefore have a severe impact on patients and their families. As the population age and birth rates fall, it is becoming imperative to ensure that the working-age population is able to contribute economically. Treating patients with rare diseases and enabling them to work and earn a living is thus important, not only for the individuals concerned but also for society as a whole.
Despite the relatively small patient groups and limited number of prescriptions, rare disease drugs have significant market potential. Due to the high medical need and relatively few patients per disease, fewer resources are required to develop these drugs than drugs for the treatment of primary care diseases. Consequently, there is good reason for a company of our size and with our expertise to focus our business model on drugs aimed at rare diseases. We are convinced that we will be able to help patients suffering from rare diseases or diseases for which there is no satisfactory treatment by providing valuable medicines and thus enable patients to live a better life.