Due to the small number of cases in each case, the clinical studies required for the approval of a drug are so complex and expensive that the usual market mechanisms do not work. The sad truth: Rare diseases are not worthwhile for pharmaceutical companies.
Until 1999, there was virtually no market for orphan drugs in Germany. An important milestone for patients was the EU Regulation on Orphan Medicinal Products in 2000. It allowed for a simplified approval process for orphan drugs and granted the manufacturers exclusive rights for a limited period.
Rising registration figures - financial hurdles
Zeitenwende in der Therapie
Today, researchers are increasingly succeeding in deciphering the pathological mechanisms of disease development. They are thus providing starting points for the development of targeted drugs and antibodies that can significantly reduce the symptoms of affected people.
Encouraging treatment successes with gene and cell therapy methods herald a real turning point in medicine. In particular, those affected by rare diseases with a monogenetic cause may hope for a cure if promising research approaches are now consistently pursued.
To advance the development of promising treatment approaches, Alliance4Rare initiated by our foundation, bundles the expertise of research-strong university children's hospitals along a cross-site research strategy for rare diseases.
Encouraging treatment successes show that gene replacement therapies are in principle capable of restoring the functionality of diseased organ systems.
They give reason to hope that rare diseases, in particular which have a monogenic cause, can be cured in the future if existing approaches are consistently developed further.