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Orphan Drugs – Funding remains important!

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Orphan Drugs – Funding remains important!

Wednesday, February 28, 2024, 3:35 p.m

“Orphan drugs are often the only hope in the treatment of seriously ill patients. Their approval and marketing must not be made more difficult,” says BPI Managing Director Dr. med. Kai Joachimsen on the occasion of Rare Disease Day. “Unfortunately, we often hear voices in the political sphere saying that there are too many orphan drugs and that their development is privileged. The opposite is the case: There are 8,000 rare diseases and so far only a fraction of them can be treated. So there are not too many orphan drugs, but too few!”

“Everyone has the right to good health care – this applies to patients with common diseases as well as those with rare diseases. In the vast majority of cases, an orphan drug is the first drug therapy available. And if nothing existed before, it is obvious that a new therapy has an additional benefit and must be introduced into care without major hurdles. If there are already therapeutic approaches, the regulatory authority will only grant orphan drug status if a so-called significant clinical benefit is proven.”

“The special status of orphan drugs enshrined by the legislator in AMNOG is not a privilege, but a blessing for affected patients! It absolutely must be preserved. To date, almost all orphan drugs approved in the EU are actually available for patient care in Germany. This is extremely important – but is completely ignored by certain circles who are calling for further restrictions on the special status. The consequences would be devastating: If an additional benefit of orphan drugs is no longer recognized on a larger scale, this, together with the AMNOG changes that have recently come into force, will result in a dangerous situation. Reimbursement amounts would be reduced to a level that would preclude economic marketing for manufacturers. But pharmaceutical companies develop orphan drugs for very small numbers of patients. For this reason alone, higher prices are necessary.”

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Note: The use of the photo is free of charge if the source is BPI eV and in connection with the press release.

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