[A23-99] Routine practice data collection for etranacogene dezaparvovec (haemophilia B) acc. to the Law for More Safety in the Supply of Medicines (GSAV): Evaluation of the study protocol (Version 1.0) and the statistical analysis plan (Version 1.0) – Second addend

Routine practice data collection (RPDC): Review of the study protocol and statistical analysis plan

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 a rare disease, the study data at the time of marketing authorization are usually not very informative for the early benefit assessment. As in the present case, the G-BA can require the pharmaceutical company to collect routine practice data, including the associated analysis, for the benefit assessment. The pharmaceutical company has to submit a study protocol and a statistical analysis plan for the RPDC. IQWiG reviews these documents on behalf of the G-BA.