DE PNH Payer & HTA

Germany's AMNOG process (Arzneimittelmarktneuordnungsgesetz) ordinarily requires a manufacturer to submit a benefit dossier to IQWiG, which assesses additional benefit against a G-BA-defined comparator before the G-BA issues its Beschluss and GKV-Spitzenverband negotiates price. Iptacopan skipped that comparator fight entirely: as an orphan-designated therapy, its additional benefit counted as established through EMA approval alone under SGB V §35a Absatz 1 Satz 11, so IQWiG never built a head-to-head dossier against anti-C5 therapy. The G-BA's own review, decided 19 December 2024, went further, finding a substantial (beträchtlich) additional benefit specifically on quality of life for patients switching from anti-C5 therapy. In March 2025 the G-BA also declined to require accompanying data collection, judging a parallel registry disproportionate given the International PNH Registry already in place.

Ravulizumab's German HTA record is narrower and less favourable. Its only PNH-specific G-BA review to date, a March 2022 assessment of its pediatric indication against eculizumab, found no additional benefit. Crovalimab, the remaining pipeline entrant, submitted its benefit dossier on 12 September 2024 and remains under active IQWiG assessment, meaning its AMNOG outcome is not yet decided. For a payer or a competing manufacturer, the practical read is that Germany's HTA bar in PNH has effectively already been set at iptacopan's level, an orphan-pathway approval with a substantial QoL finding, not at the anti-C5 incumbents' more modest evidence base.

Every AXLRx assessment is built from primary regulatory sources — G-BA decisions, IQWiG dossier assessments, and SGB V statutory text, not secondary summaries. Findings are independently verified before inclusion.

PNH Germany Payer & HTA sources: G-BA Nutzenbewertungsverfahren for iptacopan (19 Dec 2024) and ravulizumab pediatric indication (18 Mar 2022); IQWiG Dossierbewertung G24-16; G-BA crovalimab dossier submission record.