Last week the German parliament passed a bill on the “Law for more safety in the supply of medicines (GSAV)”.  According to VFA, this legislative attempt by German’s Health Minister Jens Spahn raises two threats for orphan drugs (OD’s) in Germany:

1) Special position of OD’s in the AMNOG procedure to be confined by including hospital turnover in overall turnover limit.

2) Increased authority for Federal Joint Committee to impose data collection requirements and price reductions if registry data do not support added value.

In the current situation, OD-specific privileges apply to a turnover limit of EUR 50 million annually. If this limit is exceeded, the Federal Joint Committee conducts a completely new additional benefit assessment procedure (ODs were previously exempt from this).

Since a considerable part of the OD’s can (almost) exclusively be used in hospitals, the draft bill’s suggestion to include sales outside the statutory health insurance physicians care (i.e. hospital sales) comprises a significant change of the calculation basis.

With regard to data collection (registries), criticism among different health systems stakeholders has been voiced that the requirements for these registries and the data collection is intended to be defined on national instead of at least European level. Especially, since the draft bill’s suggestion is to agree deductions from the reimbursement amount if the Federal Joint Committee does not see any quantifiable additional benefit on the basis of the data collected additionally. Considering the low patient numbers in individual rare diseases, globally recruiting registries could increase the chance to retrieve statistically meaningful outcomes to quantify the additional benefit of orphan drugs and reduce administrative efforts for setting up several small-sized registries in parallel.