Etranacogene dezaparvovec (haemophilia B): Review of the study protocol (Version 4.0) and the statistical analysis plan (Version 4.0) – Fourth addendum to Project A22-83 (RPDC concept)

Report documents

1. Addendum

   Etranacogene dezaparvovec (haemophilia B): Review of the study protocol (Version 4.0) and the statistical analysis plan (Version 4.0) – Fourth addendum to Project A22-83 (RPDC concept)

   published on

   03.07.2026

   Document type

   PDF document, 281 kB, DE, not accessible

   DOI-Link

   https://doi.org/10.60584/A26-21

   Download file : Addendum (PDF document, 281 kB, DE, not accessible)

Additional information

Project details

Project no.

A26-21

Commission

Commission awarded on 02.03.2026 by the Federal Joint Committee (GBA).

Report type

Addendum

Status

Commission completed

Department/Division

Drug Assessment

Topic

Haematology

Note

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 GBA 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 GBA.

DOI

https://doi.org/10.60584/A26-21

Connected Projects

1. RPDC concept

   Concept for routine practice data collection for etranacogene dezaparvovec acc. to the Law for More Safety in the Supply of Medicines (GSAV)

   Project no.

   A22-83

   Last updated

   03.07.2026

   Status: Commission completed