Presentation (CTI)

OBJECTIVES: Price determination in Germany is based on a formalized negotiation methodology. It depends i. a. on the extent of additional benefit (AB), the appropriate comparative therapy (ACT) and its corresponding costs. However, for Orphan Drugs (OD) the Federal Joint Committee (G-BA) does not define an ACT and other reference might be used. Now, for OD with prior JCA, could the JCA report (JCAR) provide guidance for the price negotiation by defining treatment alternatives (TA) on a European level?
METHODS: The conducted case study compares two scenarios for price negotiations of OD considering case 1) without JCA and case 2) with JCA to discuss whether the JCAR will impact the price determination.
RESULTS: The JCAR provides information on epidemiology, TA and their relative effectiveness (RE). This information can serve as additional insights and impact national P&R. In case 1) the challenge is that clinical trials often do not allow for a direct comparison to standard of care. Therefore, price determination could be challenging for the negotiating parties. Usually, analogues are used to approximate the willingness to pay. In case 2) comparators from the PICO scoping included in the JCAR can inform about potential TA, providing guidance to this negotiation criterion. In addition, RE allows to justify the selection of comparators. Valued with prices in Germany, a hierarchy of RE of TA can be used to inform the price determination.
CONCLUSIONS: The conducted case study indicates that JCAR could have significant impact on national P&R once they become available; even though the national responsibility is not interfered with. The previous challenge of price determination might now be addressed on a broader information basis. Of course, it remains to be seen how impactful the JCAR for price negotiations in Germany will be in the specific individual case.