The little girl was lying in serious condition in intensive care when her parents reached the €2 million threshold. There is an effective drug, Zolgensma, which has been registered in the USA since 2019. The. Maximising your Virtual Congress Experience. Millionenschwere Erlöse bei Zolgensma – trotz Datenmanipulation, Härtefall-Programm für Zolgensma startet in Deutschland. Mit 2,125 Millionen US-Dollar ist Zolgensma die derzeit teuerste Behandlung aller Zeiten. can be used in patients with inherited mutations affecting genes known as SMN1, who have either been diagnosed with SMA type 1 – the most severe type – or have up to 3 copies of another gene known as SMN2. Latest news, analysis and comment on migration in Europe and beyond. its high price is justified considering the. 2. Health insurers, university hospital doctors and the German federal health technology assessment body GBA wrote to the country’s health minister, Jens Spahn, earlier this month to ask for a law to address this question. By contrast, Portugal, France and Germany decided to pay under provisional schemes allowing patients access to therapies for life-threatening conditions, even if they're not approved in Europe. Dies teilte der Hersteller Audentes. Seite 2: Blaupause für weitere neue Arzneimittel? Zolgensma can be used in patients with inherited mutations affecting genes known as SMN1, who have either been diagnosed with SMA type 1 – the most severe type – or have up to 3 copies of another gene known as SMN2. The rights of trade mark owners are acknowledged. rebates, deferred payments and installment options. Who would get Regeneron's COVID-19 antibody treatment? drugs enjoy a year of free pricing in Germany, before the country’s AMNOG cost-effectiveness system assesses their benefits compared with standard care and sets a price. Patients would then receive the therapy free of charge. “We do believe that it's the government's responsibility to decide on how they're going to allocate their health care funding and how they want to cover kids, especially pre-approval,” he said. Zolgensma muss man nur ein Mal anwenden – dafür kostet es knapp zwei Millionen Euro. Zolgensma will compete against another highly expensive drug – Biogen’s Spinraza (nusinersen), while Roche is also developing a potential rival called risdiplam that is slated for a European filing later this year. Zolgensma (onasemnogene abeparvovec) is already considered to be the world’s most expensive drug. Latest news, analysis and comment on elections in Europe and beyond. She found out about Zolgensma, an innovative therapy authorized in the U.S. in May, which is the closest to a cure the world has seen so far. Redakteurin Hauptstadtbüro My question to the Commission concerns a rare congenital disease, spinal muscular atrophy, which affects children and causes muscle weakness and loss. Novartis rechnet im ersten Quartal 2020 mit einer positiven Entscheidung des CHMP bei der EMA. Unlike some of their fellow EU countries, Portugal, France and Germany decided to pay for Zolgensma under special schemes. “In countries such as France, AveXis is leveraging existing early access funding pathways. lifetime cost of treating the disease of between 2.5 to 4 million euros. Zolgensma is reputed to be the most expensive drug in the world, with one dose costing USD 2.5 million, or CZK 55 million. Allerdings ist Zolgensma® nicht die einzige Behandlungsmöglichkeit: Seit 2017 steht mit Nusinersen (Spinraza®) ein in Deutschland zugelassenes Medikament zur Verfügung, das regelmäßig verabreicht werden muss. Merck to pay almost $3B for VelosBio, broadening antibody cancer drug push. Und so erklärten die Unterzeichner sehr deutlich: Auszug aus dem Brief des AOK-BV, des BKK-Dachverbands, des vdek, der Knappschaft Bahn-See, des IKK e.V., der SVLFG, des GKV-Spitzenverbands, des G-BA und des Verbands der Universitätsklinika Deutschlands an Gesundheitsminister Jens Spahn. Crowdfunding campaigns like the one organized for Matilde have played out in recent months across Europe. die Behandlung – wohl auch wegen des medialen Drucks. The fear of opening the floodgates for more very expensive therapy requests was also what drove Estonia to decline financing baby Annabel’s treatment in the U.S. Baby Pia, in Belgium, finally got the therapy in Antwerp. Die SMA ist mit einer Inzidenz von etwa 1:10.000 Neugeborenen eine seltene Erkrankung, deren Krankheitsverlauf bei Säuglingen unbehandelt rasch fortschreitet und zu einer generalisierten Muskelschwäche und Lähmung der Atemmuskulatur mit Todesfolge führt. He said the company didn’t offer the therapy for compassionate use in countries with crowdfunding cases “because if we approve one child, how do we say no to the others?”. Weitere Informationen sowie die Möglichkeit zum Widerruf finden Sie in unserer Datenschutzerklärung, Gentherapeutikum für Kinder mit Spinaler Muskelatrophie gilt als das teuerste Arzneimittel der Welt.