Onasemnogene abeparvovec (spinal muscular atrophy): Review of the study protocol (Version 5.01) and statistical analysis plan (Version 5.01) – Sixth addendum to Project A20-61

Report documents

1. Addendum

   Onasemnogene abeparvovec (spinal muscular atrophy): Review of the study protocol (Version 5.01) and statistical analysis plan (Version 5.01) – Sixth addendum to Project A20-61

   published on

   04.12.2025

   Document type

   PDF-Dokument, 263 kB, DE, PDF

   DOI-Link

   https://doi.org/10.60584/A25-102

   Download file : Addendum (PDF-Dokument, 263 kB, DE, PDF)

Additional information

Project details

Project no.

A25-102

Commission

Commission awarded on 04.08.2025 by the Federal Joint Committee (GBA).

Report type

Addendum

Status

Commission completed

Department/Division

Drug Assessment

Topic

Head and nerves

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://doi.org/10.60584/A25-102

Connected Projects

1. Rapid report

   Development of a scientific concept for routine practice data collection acc. to the Law for More Safety in the Supply of Medicines (GSAV) and data analysis for onasemnogene abeparvovec to inform a decision acc. to §35a (3b) SGB V

   Project no.

   A20-61

   Last updated

   16.05.2024

   Status: Commission completed