While the last day of February is known as the Rare Disease Day, the first days of February 2023 mark the start of changes for rare diseases at the G-BA in Germany.

On 2 February 2023, the G-BA decided on the provisory suspension of two ongoing benefit assessments for an orphan drug that exceeded the new legal threshold of €30 million. For each indication, the pharmaceutical company must submit evidence for a regular (sic!) assessment by IQWiG over an appropriate comparator no later than 1 July 2023.

On the same day, the G-BA fixed in another resolution the exact dates of the dossier submission for orphan drugs that exceeded the €30m threshold by 1 December 2022. Much work ahead …

On 4 February, a new ATMP Quality Assurance Directive entered into force. Following the Federal Ministry of Health’s approval and a publication in the Federal Gazette, this text defines the quality procedure steps for advanced therapy medicinal products.

Next expected changes may directly address the orphan drug procedure. Hopefully in a way that enlightens this special month.

Sunset in Sitges

Meriem Bouslouk-Marx in action. Photo by © Francisco Brasil