Concept for routine practice data collection – Valoctocogen roxaparvovec: review of the study protocol (version 2.0) and the statistical analysis plan (version 2.0) – 3. Addendum to project A22-20

Report documents

1. Addendum

   Concept for routine practice data collection – Valoctocogen roxaparvovec: review of the study protocol (version 2.0) and the statistical analysis plan (version 2.0) – 3. Addendum to project A22-20

   published on

   18.01.2024

   Document type

   PDF document, 402 kB, DE, not accessible

   DOI-Link

   https://doi.org/10.60584/A23-102

   Download file : Addendum (PDF document, 402 kB, DE, not accessible)

Additional information

Project details

Project no.

A23-102

Commission

Commission awarded on 20.10.2023 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://dx.doi.org/10.60584/A23-102

Connected Projects

1. Rapid report

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

   Project no.

   A22-20

   Last updated

   21.09.2023

   Status: Commission completed