Addendum
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
Report documents
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Addendum
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 addendum to Commission A22-83
Additional information
Federal Joint Committee (G-BA)
2024-02-01 A G-BA decision was published.
Project details
- Project no.
- A23-99
- Commission
- Commission awarded on 13.10.2023 by the Federal Joint Committee (GBA).
- Report type
- Addendum
- Status
- Commission completed
- Department/Division
- Drug Assessment
- Topic
- Haematology
- Note
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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://dx.doi.org/10.60584/A23-99
Connected Projects
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RPDC concept
Concept for routine practice data collection for etranacogene dezaparvovec acc. to the Law for More Safety in the Supply of Medicines (GSAV)
Status: Commission completed