[A21-149] Review of the study protocol and statistical analysis plan on routine practice data collection for onasemnogene abeparvovec - Second addendum to Commission A20-61
Last updated 29.03.2022
Project no.:
A21-149
Commission:
Commission awarded on 24.11.2021 by the Federal Joint Committee (G-BA).
Report type:
Addendum
Status:
Commission completed
Department/Division:
Drug Assessment
Topic:
Muscles, bones and joints
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.