Innovation location Germany at risk

Despite all this, there is still an "unmet need" of many patients affected by one of the more than 6,000 rare diseases. The passing of the GKV-FinStG has now created an additional hurdle that makes it more difficult to provide patients with innovative orphan drugs in Germany.

A drastic change in the law by the German "Financial Stabilisation of Statutory Health Insurance System Act" (GKV-FinStG) is the reduction of the annual sales threshold for orphan drugs from 50 million to 30 million €. On the one hand, some medicinal products that currently have an orphan status now exceed this threshold and a regular benefit assessment according to non-orphan conditions must take place. This assessment can lead to a drug losing its additional benefit status. The reasons for this are often methodological, for example a too small patient population in the study. On the other hand, pharmaceutical companies will be forced to carefully evaluate whether possible indication extensions for an orphan drug make sense from an economic perspective, should this indication extension raise them above the 30 million € annual sales threshold.

In addition, the GKV-FinStG will result in further changes that are detrimental to the financial situation of pharmaceutical companies, for example the introduction of the new AMNOG pricing rules in dependence of the HTA benefit assessment and the price of the comparator. For this reason, financial resources are likely to be used more cautiously, which may lead to fewer resources being made available for the research and development of new active substances. This development may ultimately have a negative impact on the provision of orphan drugs to patients.

The concern that the security of supply of patients with orphan drugs could be endangered under the influence of the GKV-FinStG was also expressed in the Conference of the German Health Ministers (Gesundheitsministerkonferenz, GMK). Above all, the 'evaluation period' of the law until the 31^st of December 2023 was questioned in its reasonableness. For an evaluation regarding "the security of supply with innovative and economical medicinal products, in particular also for patients with rare diseases" (§130b (11) SGB V), the time span of only one year seems to be too short to evaluate real effects on the supply. It was therefore unanimously decided at the GMK that the Federal Ministry of Health must monitor the effects of the GKV-FinStG beyond 2023 in order to be able to take countermeasures at an early stage if undesirable developments occur.

SKC monitors the changes that the GKV-FinStG will bring to the provision of orphan drugs, as we are dedicated to supporting the market access of innovative drugs for patients with previously missing or insufficient therapy options.

Sources:

• Orphan Drugs: Innovationsfeindlichkeit per Gesetz?
• Bundesgesetzblatt Jahrgang 2022 Teil I Nr. 42 (11. November 2022)
• Präsentation BPI – Update GKV-Finanzstabilisierungsgesetz (08.11.2022)
• Gesundheitsministerkonferenz sorgt sich um Orphan Drugs