German pharma innovating to aid rare disease sufferers

Germany's pharmaceutical companies are currently working on 1500 projects to develop drugs to treat rare diseases – medicines that are also known as "orphan drugs." The German Pharmaceutical Industry Association (BPI) and the Association of Research-Based Pharmaceutical Companies (vfa) marked Rare Disease Day on February 29 with a review of their members' efforts to bring orphan drugs to patients who need them.

Inside a pharmacy
© gemphotography

The vfa reported that among the drugs being developed, many have already been tested in clinical trials with patients. It added that products for treating 30 rare diseases were expected to come onto the market by 2019. The deputy head of the BPI, Dr. Norbert Gerbsch described the challenges involved in developing orphan drugs. "BPI member companies doing R&D on drugs for rare diseases invest millions in these projects for periods that may be as long as 15 years before the drug is ready to go onto the market."

Some of the funding for this ambitious work comes from the European Union (EU). The vfa's chief executive Birgit Fischer said, "Even if their diseases are rare, patients want to be cured of their illnesses and get better. Since 2000, the EU has been fostering the development of orphan drugs with financing and research programs of its own. This has helped pharmaceutical companies expand their own research. Meanwhile, about a fifth of the new drugs approved annually are orphan drugs."

In addition, the vfa reported that there are already licensed orphan drugs for around 130 diseases. In the EU, an orphan drug is one that is used to treat an illness from which less than one in 2000 people suffer. Both the BPI and the vfa belong to Germany's National Action League for People with Rare Diseases (NAMSE), an organization that promotes providing patients with rare diseases rapid diagnoses and the best care possible. They are also members of Health – Made in Germany's Pharmaceuticals Working Group.