[A23-64] Concept for routine practice data collection for valoctocogene roxaparvovec 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 (Version1.0) – Second addendum t
Last updated 21.09.2023
Project no.:
A23-64
Commission:
Commission awarded on 04.07.2023 by the Federal Joint Committee (G-BA).
Report type:
Addendum
Status:
Commission completed
Department/Division:
Drug Assessment
Topic:
Haematology
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.
Project no. | Title | Status |
---|---|---|
A22-20 | Concept for routine practice data collection for valoctocogene roxaparvovec acc. to the Law for More Safety in the Supply of Medicines (GSAV) | Commission completed |
Federal Joint Committee (G-BA)
2023-09-21 A G-BA decision was published.
G-BA documents on this decision (German version)