Orphan drug for multiple myeloma – G-BA must formally certify legally assumed additional benefit
17.10.2023 15:06
Due to legal requirements, the Federal Joint Committee (G-BA) has completed the re-evaluation process for the active ingredient belantamab mafodotin and had to certify an additional benefit, although confirmation of approval of the drug is currently uncertain. The European Medicines Agency (EMA) had already recommended not to extend the approval of the active ingredient, which was originally subject to certain conditions. However, the final decision from the European Commission is currently missing.
The G-BA therefore had to complete the procedure for reassessing the additional benefit and, for formal reasons, certified that the active ingredient had an unquantifiable additional benefit. The G-BA had previously carried out an initial assessment of the active ingredient in 2020 and extended it twice in order to be able to include the additional data from the manufacturer on aspects such as safety and effectiveness required by the EMA.
Belantamab mafodotin is a so-called orphan drug, i.e. a medicine used to treat a rare condition. For orphan drugs, the additional benefit is already considered proven upon approval; the G-BA only decides on the extent of the additional benefit. If this scope cannot be specified based on the documents submitted by the pharmaceutical manufacturer, it is considered “not quantifiable.” However, the G-BA is generally prohibited from determining that the orphan drug has no additional benefit or even harm.
Prof. Josef Hecken, impartial chairman of the G-BA: “We are dealing with a very special constellation here that cannot satisfy anyone: The G-BA had to evaluate an active ingredient for which special rules that facilitate the procedure apply as an orphan drug, although its general approval is unclear. Because the EMA - like the American Medicines Agency before it - unfortunately no longer sees a positive benefit-risk profile based on the data requested and provided by the manufacturer and is against regular approval based on current data on safety and effectiveness. Since the European Commission's final decision is still pending, the G-BA had to complete its assessment process and could only certify an unquantifiable additional benefit due to the legal fiction of an additional benefit for orphan drugs. The open question of approval is a very difficult situation for patients and their treating doctors.”
Belantamab mafodotin in multiple myeloma
Belantamab mafodotin is approved as monotherapy for the treatment of adults suffering from multiple myeloma - from the fifth line of therapy, i.e. in a late treatment setting. The orphan drug had only received so-called conditional approval. Associated with this was the pharmaceutical company's obligation to submit further comprehensive clinical data on safety and effectiveness. The study data submitted to the EMA also formed the basis for the G-BA's assessment.
Background: Benefit assessment of orphan drugs
For orphan drugs, according to Section 35a Paragraph 1 Sentence 11 SGB V, the additional medical benefit is already proven by the approval. Only the extent of the additional benefit is assessed by the G-BA. The basis for this is the approval studies. A benefit assessment compared to a comparative therapy is only possible if the drug's sales are over 30 million euros.