Enzyme replacement therapies within the German HTA (AMNOG) process
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
In an analysis, first, the ERTs that were finally assessed by the German Federal Joint Committee (G-BA) and the German Institute for Quality and Efficiency in Health Care (IQWiG) were identified in a systematic database search. Subsequently, the relevant entries were analyzed in terms of indication, pivotal trials, added benefit granted by the G-BA, treatment costs at launch as well as the reimbursement rebates. Dr. Alexandra Kuhn and Prof. Matthias P. Schönermark, M.D., Ph.D. presented the corresponding analyzes and results with an accompanying poster at the ISPOR Europe 2018 in Barcelona. The investigation considered all completed AMNOG assessment procedures from 2011 to June 2018. Please download the full ISPOR poster here. The orphan drugs Asfotase alfa (Strensiq®), for the treatment of pediatric-onset hypophosphatasia, Cerliponase alfa (Brineura®), for the treatment of neuronal ceroid lipofuscinosis type 2, Elosulfase alfa (Vimizim®), for the treatment of Morquio syndrome A and Sebelipase alfa (Kanuma®), for the treatment of lysosomal acid lipase (LAL) deficiency were investigated.
In conclusion, the results suggest that orphan drugs as ERTs face different challenges in the market access process. The small number of study patients and the heterogeneity of the disease lead to a limited evidence to determine an additional benefit. To obtain more evidence, the G-BA linked the resolution with a request to set up a disease-specific registry. The current regulations according to §35 a, SGB V grant orphan drugs at least a none-quantifiable benefit. The G-BA therefore only determines the extent of the additional benefit for the various patient groups. This extent is the central criterion for reimbursement negotiations, which also direct the benefit assessment of orphan drugs. However, price negotiations can also be influenced by other factors, e. g. the launch price, precedents and the severity of the disease. Due to the sparse level of evidence, the collection of evidence in the form of registers will be required more frequently in the future. The individual market access strategy of specific products, including the establishment of a patient registry, should be adapted to the German HTA requirements early in the process.
As an established consulting company in the German healthcare sector with more than 13 years of experience, SKC consulting supports pharmaceutical entrepreneurs in the market access of medicines for rare diseases. One of our focuses is to attend our clients through the process of early benefit assessment according to § 35 a SGB V within the AMNOG. Therefore, we use our extensive knowledge in the presentation of patient registries.
BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Bianca Prelle, B. A. Health Management