Mandatory Registries for the German Market Access of Orphan Drugs

Tue, 2017 / 01 / 31

The German Federal Joint Committee (G-BA), representing the most influential decision making body in the AMNOG-based market access process for new drugs, has recently ordered in three cases that the pharmaceutical manufacturer has to set up a specific German patient registry which should collect all relevant patient data in the future. With the published decisions on the additional benefit of Santhera’s eye drug Raxone® and the two enzyme replacement therapies from Alexion, Strensiq® and Kanuma®, the G-BA argued that the submitted data base was not strong enough to come to a final and solid decision on the additional benefit of these drugs.

Therefore, the committee limited the validity period of its decision (all three orphan drugs achieved a „non quantifiable additional benefit“) to 30 months after which Santhera and Alexion will have to resubmit a value dossier to the German authorities. This new dossier will need to include data from a specific German patient registry which shall include all data from all treated patients in Germany on all relevant endpoints. The G-BA explicitly made clear that the European Medicine Agency’s (EMA) criteria for a patient registry would not be sufficient. Furthermore, the committee stated that neglecting this obligation could lead to a denial of an additional benefit in the future re-assessment, although there is currently no legal base for this.

In summary, the G-BA has demonstrated once again, that it will use additional instruments to regulate the market access of pricey orphan drugs, if the evidence base is perceived as being not strong enough. Anticipating this position and adjusting the strategy (specifically the outlay and setup of a registry) is a mandatory activity for pharmaceutical maufacturers, planning to launch in Germany.

In a recently published publication (PharmR 38/8:305-309; 2016), Prof. Christian Dierks, M.D., J.D. and Prof. Matthias P. Schönermark, M.D., Ph.D. discuss the role of patient registries in the German market access context with specific regard to the decision of the Federal Joint Committee (G-BA) that pledged two pharmaceutical maufacturers to set up a specific German patient registry as a pre-requiste for the future reassessment oft he respective drugs‘ benefit.

The article comments on the legal basis for the G-BA’s ruling and deduces strategic and tactical implications for the pharmaceutical companies, planning to launch orphan drugs in Germany. It can be downloaded on our website https://skc-beratung.de/references