In Germany, the Federal Joint Committee (G-BA) recently amended its rules of assessment procedures to consider new therapies and products without complete clinical data at the time of approval.

During the review of Zolgensma®, a gene therapy for spinal muscular atrophy, the G-BA determined for the first time, the type of data, and the process needed for assessing the long-term benefit when there is no comparative data on alternative therapies.

At the time of approval, some of these medicinal products for the treatment of rare diseases have limited or incomplete clinical data available to assess the additional benefit in the Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) benefit assessment, which until now, has been a standardized process. The aim should be to close existing gaps in clinical evidence and, at the same time, to make the relevant medicinal products available to patients in the German statutory health insurance (SHI).

In the new process, information about the possible evidence gaps from the approval process of the drug is used in addition to information from study reports to assess whether, and what additional data collection is required. The G-BA obtains this information in exchange with The Federal Institute for Drugs and Medical Devices (BfArM) and the Paul Ehrlich Institute (PEI) to avoid duplication with current and planned data collection already requested by regulatory authorities. The G-BA or the Institute for Quality and Efficiency in Health Care (IQWiG), on behalf of the G-BA, will prepare a draft concept for the application-related data collection. The concept details the following requirements: type, duration scope, and methods of the data collection, and the patient-relevant endpoints to be considered as outcomes. The draft concept will be forwarded to the involved bodies including the BfArM and PEI, the medical scientific associations, the German Medical Association, the pharmaceutical company, and other experts, if necessary. All statements are incorporated into the final concept. Concept preparation generally will be completed within 6 months.

The final decision is made in the G-BA plenum, where the timelines for the review of the data and the submission of the dossier for the benefit assessment also is determined. At regular intervals, but at least every 18 months, the G-BA checks whether the data collection is or can no longer be carried out, whether it will provide sufficient evidence for a new benefit assessment or whether there is a need for adjustments to the requirements of the decision.

Orphan drug manufacturers and stakeholders now have more clarity about the patient registries, which were introduced for drugs for rare diseases with the GSAV (law for security in drug supply) in 2019. This change provides innovators of rare disease treatments improved opportunities to engage in the “coverage with evidence development” process to secure earlier reimbursement for these high-value therapies.  Please contact us to learn how we can leverage our extensive experience in helping companies navigate the complexities of Germany’s health system.