Hospital.Help

Berlin – The Federal Joint Committee (G-BA) has assessed the additional benefit of various drugs for rare diseases (orphan drugs) because they exceeded the statutory health insurance turnover limit of 30 million euros per year. The assessment varies greatly for the various active ingredients.

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

The situation is different for the drug Patisiran due to medical progress. Here the 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, the G-BA found a considerable additional benefit.

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

The G-BA also downgraded cannabidiol as additional therapy for seizures in connection with Lennox-Gastaut syndrome or Dravet syndrome in connection with clobazam in patients aged two and over. In 2020 he found a considerable additional benefit.

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