Addendum
Review of the study protocol and statistical analysis plan on routine practice data collection for onasemnogene abeparvovec acc. to the Law for More Safety in the Supply of Medicines (GSAV) – Addendum to commission A20-61
Report documents
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Addendum
Review of the study protocol and statistical analysis plan on routine practice data collection for onasemnogene abeparvovec acc. to the Law for More Safety in the Supply of Medicines (GSAV) – Addendum to commission A20-61
Project details
- Project no.
- A21-107
- Commission
- Commission awarded on 16.08.2021 by the Federal Joint Committee (GBA).
- Report type
- Addendum
- Status
- Commission completed
- Department/Division
- Drug Assessment
- Topic
- Muscles, bones and joints
- 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.