Reasoning of the BSG ruling underlines the special position of legal therapeutic soloists

Rapiscan® was subjected to a benefit assessment by the German Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) in 2019 as a pharmacological stress trigger for the measurement of fractional flow reserve. This ultimately resulted in arbitration. The pharmaceutical manufacturer initially sued unsuccessfully before the Berlin-Brandenburg Regional Social Court, but the BSG has now upheld the claim in appeal proceedings. The hearing took place on February 22, 2023, and the reasons for the ruling were received on March 14, 2023.

The BSG's reasons for judgment now clarify and underline the court's view that a benefit assessment of a therapeutic soloist is unlawful - and thus all price negotiations and arbitrations based on it.
In the case of medicinal products, the approval ensures that they are per se and fundamentally appropriate for treatment in the indication. The G-BA can only evaluate the added benefit compared to therapy alternatives. However, such an added benefit assessment of a therapeutic soloist is unlawful and inadequate because the benefit assessment according to Section 35a SGB V is an added benefit assessment in comparison to an ACT determined by the G-BA. Since in the case of a therapeutic soloist no ACT can be determined, the added benefit assessment is not applicable.

Interestingly, the court explicitly mentions the legal exception of orphan drugs for rare diseases, for which an ACT does not have to be defined and the added benefit is considered proven by law on the basis of the approval. Whether the ruling consequently calls into question orphan procedures, in which the drug also represents a soloist, remains not entirely clarified and is left to legal interpretation.

In the case of Rapiscan®, the indication is not a rare condition, but nevertheless the drug is a therapeutic soloist. Only drugs in off-label use were defined as ACT. This is not adequate since their use is not appropriate under health insurance law. Only clearly defined off-label exceptions can be considered for prescribability according to the G-BA (Annex VI to K, AM-RL).

The ruling may have significant implications for some AMNOG procedures, up to and including the inadmissibility of the negotiated reimbursed prices - and thus pave the way back to the launch price.

• Directly affected by the court-confirmed special position of a soloist could potentially be all "non-orphan" procedures in which the ACT, due to the lack of approved therapeutic options, was defined as "Watchful Waiting", "Best Supportive Care" or "Therapy according to physicians' choice" solely based on therapeutic options without approval.
• Indirectly, however, the inadmissibility of a price negotiation under Section 130b SGB V could also significantly increase the price anchors for other drugs. The G-BA recognized this issue and published a corresponding statement: a legal adjustment that all reimbursable new active substances are to be subjected to an added benefit assessment according to § 35a SGB V is urgently needed.

SKC follows, analyzes and anticipates the political decisions and their respective implementations in detail and includes these directly in the day-to-day business with clients. Here, we generally recommend examining the individual situation and possible implications, and to actively take appropriate precautions.

Sources:

• Report of German Federal Social Court: BSG – B 3 KR 14/21 R
• Benefit assessment according to AMNOG
• Anlage VI zum Abschnitt K der Arzneimittel-Richtlinie zu Verordnungsfähigkeit von zugelassenen Arzneimitteln in nicht zugelassenen Anwendungsgebieten (sog. Off-Label-Use)
• Stellungnahme des G-BA
• SKC: German Federal Social Court: added benefit assessment for therapeutic soloists unlawful - The case of Rapiscan®
• MAIS database (combines AMNOG procedure and Lauer-Taxe)