Orphan drugs: nearly one third of all German new approvals were granted for rare disease drugs
About the author
Prof. Matthias P. Schönermark, M.D., Ph.D.
Founder and Managing Director
Founder and Managing Director
Fon: +49 511 64 68 14 – 0
Fax: +49 511 64 68 14 18
Fax: +49 511 64 68 14 18
A disease is defined as rare in the European Union (EU), if no more than 5 of 10.000 people are affected by it. More than 8.000 different rare diseases are counted worldwide. The path to the right diagnosis is often long and circumstantially for patients. It usually starts with a visit to a general practitioner, and then leads to medical specialists and further to university hospitals or specialized centers before the patient is finally treated by a designated expert. Nevertheless, the medical awareness for rare diseases did improve considerably, says Professor Hubert Wirtz from the university hospital in Leipzig.
Beyond that, the number of newly approved drugs with a new active substance shows, that rare diseases are becoming more relevant to the pharmaceutical industry: in Germany, between 9 and 13 orphan drugs were approved annually over the last four years. This number represents about one third of all new approvals in total. The ratio is expected to stay constant for 2018, too.
SKC will discuss the strategic challenges for the market access of orphan drugs in Europe on the 24th of September, 2018 in Cambridge, MA, USA. Please register for our complimentary seminar. We look forward to meeting you there.
BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Karolin Priese, MBA
Sources (German only):
Ärztezeitung: Wenn keine Diagnose gestellt werden kann
BMG: Seltene Erkrankungen
vfa: Ausblick auf 2018: Neue Medikamente können die Patientenversorgung verbessern
vfa: Neueinführungen und Zulassungserweiterungen seit 2003