Rare diseases: Various medications for additional benefits…

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Written By Kampretz Bianca

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Berlin – The Federal Joint Committee (G-BA) assessed the additional benefit of several medicines for rare diseases (orphan medicines) because they exceeded the legal health insurance sales limit of 30 million euros per year. The assessment is very different for different active ingredients.

Based on an ongoing, randomized, multicenter study, G-BA confirms considerable additional benefit for the active ingredient tebentafusp in the treatment of adults with certain inoperable or metastatic uveal melanoma.

The situation is different for the medicine Patisiran due to medical progress. Here G-BA sees less benefit compared to a new comparative therapy.

Patisiran has been on the market in Germany since 2018 for the treatment of adults with hereditary transthyretin amyloidosis with stage 1 or 2 polyneuropathy – based on the placebo comparison presented at the time, G-BA found a considerable additional benefit.

Patisiran should now be compared with the active ingredient vutrisiran, which has been available since 2022, as an appropriate comparator therapy. As there were clear differences in the disadvantage of patisiran in the side effects category, the G-BA classified the additional benefit as a “minor benefit” compared to vutrisiran.

G-BA has also downgraded cannabidiol as add-on therapy for seizures related to Lennox-Gastaut syndrome or clobazam-related Dravet syndrome in patients two years of age and older. In 2020 he found a considerable additional benefit.

The problem with this therapy is that the manufacturer compared the cannabiol therapy with placebo and continued the antiepileptic treatment unchanged instead of the appropriate comparator therapy determined by G-BA – a patient-specific therapy using a selection of different active ingredients. In this context, the G-BA classified the additional benefit as “not proven”. © hil/aerzteblatt.de

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