DE PNH Market Sizing Model

Germany's PNH epidemiology is entirely derivative. The DGHO's Onkopedia clinical guideline states plainly that Germany-specific prevalence and incidence figures do not exist, and borrows an estimated 16 cases per million and 1.3 new diagnoses per million annually from British and French registries instead. No AXLRx source computes an absolute German patient total from that rate, and none should be invented here: multiplying a borrowed per-million figure by Germany's population would manufacture a precision the underlying data does not support. The rate itself, not a derived headcount, is the only sizing input Germany's own clinical literature currently offers.

The second input is structural rather than numeric: German PNH diagnosis and referral run through just two national centres, the German PNH-Register at the Institute for Clinical Transfusion Medicine and Immunogenetics in Ulm, and the West German Cancer Center at University Hospital Essen, both of which feed the International PNH Registry. This two-centre concentration is a narrower institutional funnel than markets with broader registry infrastructure, and it means Germany's undiagnosed pool, real by definition given the borrowed prevalence rate, has never been quantified. A separate commercial pressure compounds the sizing question: six months after Germany's G-BA granted iptacopan a substantial-benefit finding under the orphan-drug pathway (19 December 2024), its Lauer-Taxe list price remained unpublished, the kind of pricing uncertainty that makes an unreconciled patient count harder, not easier, to act on. Our sensitivity analysis ranks the absence of an independent German epidemiological source, ahead of the borrowed rate's precision, as the single assumption most likely to move any sized total.

Every AXLRx market sizing model triangulates at least two independent methods before accepting a patient count. This is explicitly a sizing model (static patient count), distinct from a Patient Flow or forecasting model (dynamic revenue/uptake).

PNH Germany sizing sources: the DGHO Onkopedia PNH clinical guideline, the German PNH-Register at Ulm's Institute for Clinical Transfusion Medicine and Immunogenetics, the West German Cancer Center at University Hospital Essen, and the G-BA Nutzenbewertungsverfahren for iptacopan (Beschluss 19 December 2024).