G-BA brings clarity to registry requirements

Using the gene therapy Zolgensma as its first case, the Gemeinsame Bundesausschuss (G-BA, Federal Joint Committee) has set its rules for patient registries for orphan drugs.

Orphan drugs manufacturers and stakeholders have been curious about what form patient registries would take since the enactment of the Gesetz für mehr Sicherheit in der Arzneimittelversorgung (GSAV, law for more security in drug supply) in 2019. The law stated that drugs for rare diseases may be required to gather data in an ongoing manner, but had not provided detail on, for example, the types of data to be gathered, the responsibilities for gathering the data, or the responsibility of maintaining the data.

G-BA said:

“For reasons of patient safety, it is of immense importance, especially for medicinal products that have been approved under atypical circumstances despite the weak study situation, that data collection for further assessment of the benefits or harm begins as early as possible and that the use of the therapy is also linked to the treatment facilities who participate in the data collection. For Zolgensma, a recently approved gene therapy for spinal muscular atrophy, the G-BA determined for the first time the need for an accompanying data collection because no meaningful data for assessing the long-term benefit or harm of the treatment are available and there is no comparative data on alternative therapies.”

Read G-BA’s press release for more information.

Tagged with

Published on 17. July 2020 in News, News Germany