The approval covers most infants and children with SMA who weigh up to 46 pounds, a label that would include almost all patients under the age of five, Novartis, Between 550 and 600 babies are born with SMA every year in Europe, Novartis said. Maximising your Virtual Congress Experience. The signatories also warned that if Zolgensma is reimbursed as a result of public pressure, drug companies could use it as a blueprint to get new drugs onto the market before authorization. Campaigning, lobbying and political influence in the U.K. Latest news, analysis and comment from POLITICO’s editors and guest writers in Europe. The little girl was lying in serious condition in intensive care when her parents reached the €2 million threshold. More than 600 patients have been treated with Zolgensma, including clinical … With regulators, drugmakers and payers pulling in similar directions, real-world evidence is likely to become a more central feature of drug development despite its many shortcomings. Der G-BA hat für Spinraza® für die fragliche Patientengruppe einen „erheblichen Zusatznutzen“ festgestellt. Matilde’s parents managed the impossible. In such cases they have to pay 10% VAT on top of that price, so the total cost of the drug in the Czech Republic amounts to roughly CZK 62 million (1) . Novartis, however, has justified the price by comparing it with how much the existing therapy approved in Europe, Spinraza, would cost to treat children with SMA. 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The availability of three vastly different, cutting-edge medicines for the rare disease has put patients, families and doctors in an unfamiliar position. Explore the live extension of our journalism, The wonk's survival guide to the EU Green Deal. “I hope the debate will go forward and something fundamentally changes about how these prices are set,” Ellen De Meyer, Pia’s mother, told POLITICO. Telefax: +49 (0) 30 246267 - 20 Could the Commission introduce a tax or VAT exemption for Zolgensma? The one-time treatment, priced in the U.S. at $2.1 million, brought in sales of $170 million for Novartis in the first quarter, slightly lower than the $186 million posted in the fourth quarter. Schwerpunkt des Redaktionsprogramms ist die Vermittlung aller Informationen und Nachrichten aus den Bereichen Wissenschaft, Arzneimitteltherapie, Praxis, Berufs- und Gesundheitspolitik sowie Recht, die für pharmazeutische Berufe von Interesse sind. “Does it help to reduce the price of a product if a 'crowd' thinks a therapy is worth a donation?” he wondered. Lumis International GmbH spins off its consulting business to Lumis Life Science Consulting ... WCG Appoints Two New Independent Directors to its Board, From no drugs to 3: Patients with spinal muscular atrophy now face hard choices, Pfizer, with vaccine results positive, readies plans to ship coronavirus shot, Finding the right mix: Digital transformation offers opportunities for improved HCP engagement, Novartis rare disease drug, repurposed for COVID-19, fails in late-stage study. Das Problem: Diese eine Spritze kostet rund 2 Millionen Euro – und während die amerikanische Arzneimittelbehörde FDA dem Gentherapeutikum im vergangenen Mai die Zulassung erteilte, ist die Europäische Arzneimittel-Agentur (EMA) noch nicht so weit. 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. Topics covered: Pharma, biotech, FDA, gene therapy, clinical trials, drug pricing and much more. Zusatzinformationen, Literaturverzeichnisse. The European Commission granted a conditional marketing, weighing up to 21 kg with the most severe form of the, atients in Germany are likely to get the drug first, as drug pricing is decided by each member state. “I hope the debate will go forward and something fundamentally changes about how these prices are set” — Ellen De Meyer, baby Pia's mother. This risks creating a toxic environment, warned Martin Seychell, DG SANTE's deputy director general, speaking at a pharma event in Brussels in early November. Most children die after a couple of years. Discover announcements from companies in your industry. That’s obviously bringing a lot of pressure along” — Belgian Health Minister Maggie De Block. The Swiss drugmaker argues that the hefty price tag for the medicine provides value for insurers and health-care systems. GmbH, Seite 1: Zolgensma: Kassen fordern Härtefallprogramm auf Herstellerkosten. ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Whilst in other countries, is already in advanced discussions to agree on terms to enable immediate access.”, Elsewhere in Europe the company hopes to offer flexible pricing options including. Sie starteten Aktionen mit der Presse und im Internet, sammelten Spenden und viel Aufmerksamkeit. Unlike some of their fellow EU countries, Portugal, France and Germany decided to pay for Zolgensma under special schemes. Spinale Muskelatrophie: Millionenteures Medikament Zolgensma... Wieviel ist ein Menschenleben wert? Belgium, along with Estonia, Hungary, Greece and Cyprus, has so far resisted the pressure to pay. In Europe, for instance, Novartis claims the cost of care per patient can exceed $4 million over the first 10 years of life, if infants survive what is typically a deadly disease in the most severe form. Zolgensma® von Novartis macht seit einiger Zeit auch in Deutschland Schlagzeilen: Das Gentherapeutikum für Kinder mit Spinaler Muskelatrophie gilt als das teuerste Arzneimittel der Welt. In-depth reporting, data and actionable intelligence for policy professionals – all in one place. 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. “We are confident that we shall reach an agreement so that families can access our gene therapy. In der EU ist das neue Gentherapeutikum noch nicht zugelassen, doch die Kassen sind bereits alarmiert. It also says that the drugmaker should provide it free of charge. Set up like a lottery scheme, the program randomly allocates doses of Zolgensma for participating patients under the age of two with genetically confirmed spinal muscular atrophy. This was a decision the Belgian government needed to take, “which they abdicated, which was unfortunate,” he added. Dazu gehören insbesondere aktuelle Mitteilungen und Informationen über Arzneimittel, Übersichtsarbeiten von anerkannten Fachautoren aus Wissenschaft und Praxis, Tagungs- und Kongressberichte, Neuigkeiten über Arzneimittel und Arzneimitteltherapien, Kommentare und Meinungen. Mit 2,125 Millionen US-Dollar ist Zolgensma die derzeit teuerste Behandlung aller Zeiten. is leveraging existing early access funding pathways. Latest news, analysis and comment on security in Europe and beyond. Zolgensma was approved by the U.S. Food and Drug Administration in May 2019 and represents the first approved therapeutic in Novartis Gene Therapies’ proprietary platform to treat rare, monogenic diseases using gene therapy. This article is part of POLITICO’s premium policy service: Pro Health Care. Fraser added that in the UK, AveXis already began talks with NICE in 2018 and the drug is being assessed using its Highly Specialised Technology pathway, but said that the review had been “impacted somewhat” by the COVID-19 emergency. "It feels like they say [the children] will die soon, so it’s not worth spending too much money [on] them.". Telefon: +49 (0) 30 246267 - 0 and negotiations with payers in other major European countries are ongoing. Around 550-600 babies are born with the disease in Europe each year, which causes rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. SUBSCRIBE free here. The 10th Annual RNA Therapeutics Returns to London this Feb! Zolgensma muss man nur ein Mal anwenden – dafür kostet es knapp zwei Millionen Euro. Spinraza has to be administered throughout a patient's life, and its price is in the hundreds of thousands of euros per patient per year. Elsewhere in Europe the company hopes to offer flexible pricing options including early access rebates, deferred payments and installment options, and outcomes-based rebates. The medicine also has approval in Japan. 1. However, right now there are children for whom this deadline does not give hope, as Zolgensma must be administered by the age of two at the latest. Indem Sie fortfahren, unsere Seite zu nutzen, akzeptieren Sie die Verwendung von Cookies und stimmen den Analysemaßnahmen zu. Over two weeks last summer, they fund-raised €2.5 million to get the toddler a life-saving medicine that's not yet authorized in Europe. Dennoch zahlen erste Kassen die Behandlung – wohl auch wegen des medialen Drucks. The. In der EU ist das neue Gentherapeutikum noch nicht zugelassen, doch die Kassen sind bereits alarmiert. In the end, the money her family raised paid for it instead. The company is also engaged in private discussions with payers in other European countries in the build-up to other national launches, the company told pharmaphorum. That fate was something Matilde’s mom couldn’t accept. Geschlechtsangleichende Hormontherapie bei... Fehlende Standards zur Bauchdeckenentlastung nach... 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