German Ethics Council discusses challenges in medical care for rare diseases
About the author
Prof. Matthias P. Schönermark, M.D., Ph.D.
Founder and Managing Director
Founder and Managing Director
Fon: +49 511 64 68 14 – 0
Fax: +49 511 64 68 14 18
Fax: +49 511 64 68 14 18
Antje Behring from the Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) emphasized that in Germany the approval process for medicines for rare diseases (orphan drugs) is eased compared to “regular” drugs. She also introduced a novel treatment offer for affected patients: the outpatient specialist treatment (ambulante spezialfachärztliche Versorgung, ASV). The lack of price limits which has led to a dramatic increase of the yearly therapy costs for orphan drugs, was identified as a challenge which requires substantial improvement in the future. In addition, for orphan drugs with low patient numbers and low evidence, a regulated-by-law participation in clinical registries was described as desirable in order to collect data on patient relevant endpoints in the context of the German health care system. The introduction of a potential new category of added value “by virtue of legal fiction” is also currently being debated for orphan drugs with low or non-existing evidence.
Daniel Strech from the Hannover Medical School elaborated on ethical challenges associated with rare diseases such as the fair distribution of financial resources for treatment offers and research programs. During the following discussion, the panel collected ideas for the resolution of the challenges in dealing with rare diseases, such as: higher transparency through new infrastructures to educate affected patients, stronger involvement of self-aid groups in the development of concepts of care, expansion of clinical trials and promotion of registries for rare diseases.
SKC is your experienced partner in the early benefit assessment of innovative orphan drugs according to the AMNOG in Germany. Please find comprehensive information in our white paper "Orphan Drugs in Germany – lessons learned from AMNOG, best and worst practices and strategic implications" or contact us for further questions.
BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Lydia Gibson, M.Sc. psychology
Sources:
Deutscher Ethikrat