Personalized medicine as orphanization: legal and ethical questions

Definition of the problem The development of personalized medicine is currently a popular topic. Particularly the personalized pharmacotherapy is a matter of relative importance due to pharmacological and molecular-genetic developments. For this reason it is required to examine the implications of personalized pharmacotherapy to statutory health insurance and health care of patients. Arguments In this context the question of orphanization arises: If medical drugs of personalized medicine could be regularly indicated as orphan drugs, i.e., as drugs to medicate orphan diseases, it would not be necessary according to the "Arzneimittelneuordnungsgesetz" (AMNOG) to prove an auxiliary value within the assessment of the "Gemeinsamer Bundesausschuss" (GBA). Crucial to the drug's indication as an orphan drug is among other things the rate of prevalence of the patient group, whose disease should be medicated with the drug. In this connection, there is importance attached to the stratification of patient groups within personalized medicine. Conclusion An orphanization causes consequences in terms of prices to the statutory health insurance due to the lack of knowledge about an auxiliary value. For patients, the implications must be considered in a more differentiated manner.