[G23-26] Iptacopan (paroxysmal nocturnal haemoglobinuria); assessment according to §35a Social Code Book (SGB) V; estimation of patient numbers

Estimation of patient numbers:

In the case of new drugs that have entered the health care system via a special marketing authorization or as a drug for the treatment of an orphan condition, the study data at the time of marketing authorization are usually not very informative for the early benefit assessment. In these cases, the G-BA can require the pharmaceutical company to conduct a so-called “anwendungsbegleitende Datenerhebung”, i.e. the collection of routine practice data (RPD) acc. to the Law for More Safety in the Supply of Medicines (GSAV). This includes the associated analysis for the benefit assessment.

When assessing whether RPD collection is necessary for new drugs, the G-BA also examines the feasibility and appropriateness of data collection. In this context, IQWiG may be commissioned to estimate patient numbers for the relevant therapeutic indication.

The current version 1.1 of the Estimation of patient numbers replaces version 1.0 published on Aug 7, 2024.