Berlin – The European Commission is currently revising the EU drug legislation and is also focusing on drugs for rare diseases (orphan drugs). There must be no cuts in the incentive system for the development of orphan drugs, demands the Federal Association of the Pharmaceutical Industry eV (BPI) on the occasion of Rare Disease Day. There are currently around 200 medicinal products for around 8,000 known rare diseases. The need for research remains high.
“Research and development of orphan drugs must continue to be fully funded,” says BPI Managing Director Dr. Kai Joachimsen. “The incentives and the therapies that result from them are glimmers of hope for the patients affected. We must not make any compromises when it comes to finding therapies where adequate treatment options have so far been lacking.”
“Investment decisions are already being made difficult for pharmaceutical companies,” says Joachimsen. “The recently passed Statutory Health Insurance Fund Financial Stabilization Act counteracts the idea of promoting orphan drugs: Drugs only receive a special status in the AMNOG if they are below an annual turnover threshold that has been reduced from formerly 50 to 30 million euros. As the BPI, we have repeatedly pointed out that this is accompanied by a step backwards in the care of patients with rare diseases. It is now all the more important that the future EU drug legislation sticks to the proven funding instruments – especially with regard to the duration of market exclusivity rights,” emphasizes Joachimsen. “Currently, orphan drugs are quickly and comprehensively available to patients in Germany. This must not be jeopardized by changed framework conditions. The numbers prove that the European legal framework works: When the legal framework came into force, there were more and more orphan drugs. In the past five years (until 2021), around 16 registrations per year have taken place in Germany.”
Background: Rare diseases
At the national level, the Medicines Market Reorganization Act (AMNOG) recognizes that orphan drugs have an additional benefit as soon as they are approved. Either a therapy against the respective rare disease comes onto the market for the first time with a new active substance for which there was previously no adequate comparator therapy. Or the new drug offers a clinically relevant advantage over the previous therapy. But even in Germany, the dismantling of this special position in the AMNOG is discussed again and again. And this at a time when many patients are still urgently waiting for therapies.
Since these patient groups are small, pricing incentive structures must be set correctly so that pharmaceutical companies can also develop special therapies for just a few patients. Because they have the same right to good health care as people with frequent illnesses. Appropriate legislation at national and European level is therefore also an expression of the political will to provide adequate care for people with rare diseases.
Further information can be found in the BPI-Themenwelt Orphan Drugsim BPI-Themendienst Orphan Drugs as well as in the BPI-AMNOG-Daten 2022.
Note: The use of the photo is free of charge with the source ©BPI and in connection with the press release.