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Monitor Weesgeneesmiddelen in De Praktijk 2019 > Retouradres Postbus 320, 1110 AH Diemen Zorginstituut Nederland Aan de minister voor Medische Zorg en Sport Zorg I Oncologie Postbus 20350 Willem Dudokhof 1 2500 EJ Den Haag 1112 ZA Diemen Postbus 320 1110 AH Diemen www.zorginstituutnederland.nl [email protected] 2019053395 T +31 (0)20 797 85 55 Onze referentie 2019053395 Datum 20 november 2019 Betreft Monitor Weesgeneesmiddelen in de praktijk 2019 Geachte heer Bruins, Hierbij zenden wij u de vastgestelde Monitor Weesgeneesmiddelen in de praktijk 2019 toe. Dit is de derde monitor van de jaarlijks terugkerende reeks. De monitor is aangekondigd in het rapport pakketbeheer weesgeneesmiddelen in 2015. Het doel van de monitor is om op basis van interviews en declaratiedata inzicht te geven in de inzet van niet-oncologische weesgeneesmiddelen in de Nederlandse zorgpraktijk. De monitor biedt enerzijds een inhoudelijk inzicht in de zorgverlening en anderzijds een inzicht in de kosten. In deze monitor besteden we onder andere aandacht aan de praktijk rondom de toepassing van de weesgeneesmiddelen voor de ziekte van Gaucher. De centralisatie van zorg in één expertisecentrum maakt het makkelijk om start- en stopcriteria toe te passen voor de indicatie Gaucher. Het belangrijkste probleem rondom de weesgeneesmiddelen voor Gaucher is de hoge prijs (macro en per patiënt per jaar). Het middel imiglucerase is al sinds 1997 op de markt en is nauwelijks gezakt in prijs, ook niet nadat de weesstatus is verlopen of er een vergelijkbaar middel op de markt is genomen. Zorgverzekeraars, het expertisecentrum en uw ministerie zijn mede om die reden in gesprek om de Gaucher-middelen over te hevelen van het GVS naar de medisch-specialistische zorg per 2021. Er zijn plannen voor het uitschrijven van een tender voor twee middelen die vergelijkbaar zijn. Deze aanpak ondersteunen wij. Daarnaast worden in deze monitor de eerste resultaten besproken van het onderzoek naar de toegankelijkheid van weesgeneesmiddelen. Een voorlopige conclusie over de toegankelijkheid van niet-oncologische weesgeneesmiddelen geregistreerd in de periode 2012-2017, is dat de doorlooptijden van de geanalyseerde middelen sterk uiteenlopen. Redenen hiervoor zijn nog niet geheel duidelijk, de vervolgfase van het onderzoek moet hier meer duidelijkheid over geven. Daarnaast laat de monitor zien dat er wederom een stijging heeft plaatsgevonden van het totaal vergoede bedrag. Tussen 2013 en 2017 heeft er een stijging plaatsgevonden van €195 naar €272 miljoen. Dit is een stijging van 39% over vijf jaar met een gemiddelde stijging van 9% jaarlijks. Over deze periode steeg ook het aantal patiënten in vergelijkbare mate, namelijk 38%. De verwachting is dat Pagina 1 van 2 de kosten de komende jaren zullen blijven stijgen aangezien de Horizonscan de Zorginstituut Nederland komende jaren 57 nieuwe niet-oncologische weesgeneesmiddelen of indicatie- Zorg I Oncologie uitbreidingen van bestaande weesgeneesmiddelen verwacht. Deze cijfers onderstrepen het belang van gepast gebruik van de niet-oncologische Datum weesgeneesmiddelen en de monitoring hiervan. 20 november 2019 Onze referentie We vertrouwen erop u hiermee voldoende te hebben geïnformeerd. 2019053395 Hoogachtend, Sjaak Wijma Voorzitter Raad van Bestuur Pagina 2 van 2 Monitor Weesgeneesmiddelen in de praktijk 2019 12 NOVEMBER 2019 | DEFINITIEF | Van goede zorg verzekerd | Monitor weesgeneesmiddelen in de praktijk 2019 | 12 november 2019 | Definitief Zorginstituut Nederland en het evaluatieprogramma De missie van Zorginstituut Nederland is: van goede zorg verzekerd, niet meer dan nodig en niet minder dan noodzakelijk. Om de effecten van de producten van het Zorginstituut op de praktijk van de zorg in kaart te brengen, heeft het Zorginstituut een evaluatieprogramma ontwikkeld. Het beginpunt van een evaluatie of monitor is het beoogde effect van een (of meer) eerder door het Zorginstituut uitgebrachte product (standpunt, handreiking, advies, etc.). Met een evaluatie of monitor wil het Zorginstituut vooral inzicht krijgen in het effect van een product op de praktijk van de zorg. Het versterken van de doorwerking van een product speelt daarnaast ook een belangrijke rol. Meer informatie over de activiteiten van het Zorginstituut en het evaluatieprogramma vindt u op www.zorginstituutnederland.nl. 2 Monitor weesgeneesmiddelen in de praktijk 2019 | 12 november 2019 | Definitief Inhoud Samenvatting 5 1 Inleiding 7 2 Weesgeneesmiddelen bij de ziekte van Gaucher: toepassing in de praktijk 11 2.1 Inleiding 11 2.2 Omschrijving ziektebeeld 11 2.3 Geneesmiddelen bij Gaucher: toepassing in de praktijk 13 2.4 Het perspectief van een patiënt met de ziekte van Gaucher 14 2.5 Europese en internationale samenwerking 16 2.6 Vergoeding internationaal 16 2.7 Toekomstige geneesmiddelen 16 2.8 Conclusie 17 3. Weesgeneesmiddelen-arrangementen 18 3.1 Inleiding 18 3.2 Formele weesgeneesmiddelen-arrangementen (centraal afgesloten) 18 3.3 Decentrale weesgeneesmiddelen-arrangementen (afgesloten door partijen) 21 3.4 Conclusie 22 4 Toegankelijkheid van weesgeneesmiddelen in Nederland 23 4.1 Inleiding 23 4.2 Selectie van middelen 23 4.3 Bevindingen 23 4.4 Conclusie tussenrapportage 24 4.5 Vervolgonderzoek 24 5 Weesgeneesmiddelen in het basispakket 25 5.1 Inleiding 25 5.2 Ontwikkelingen in kosten en aantallen patiënten 25 5.3 Oncologische weesgeneesmiddelen 27 5.4 Prijsarrangementen VWS 27 5.5 Recente beoordelingen van niet-oncologische weesgeneesmiddelen door het Zorginstituut 28 5.6 Conclusie 29 6 Beleidsontwikkelingen rondom weesgeneesmiddelen 30 6.1 Weesgeneesmiddelen en de Horizonscan 30 6.2 Regie op registers 31 6.3 Rondetafel Weesgeneesmiddelen 32 6.4 Voorwaardelijke toelating weesgeneesmiddelen 32 7 ACP-advies 34 8 Conclusie 35 3 Monitor weesgeneesmiddelen in de praktijk 2019 | 12 november 2019 | Definitief Bijlagen 38 Bijlage 1 Protocol Gaucher 38 Bijlage 2 Resultaten jaarlijks verslag aHus 62 Bijlage 3 Resultaten verslag IPF 65 Bijlage 4 Protocol Sebelipase alfa 70 Bijlage 5 Protocol Velmanase alfa 77 Bijlage 6 Onderzoek toegankelijkheid weesgeneesmiddelen – fase 1 83 Bijlage 7 Kosten en volume niet-oncologische weesgeneesmiddelen 99 Bijlage 8 Kosten en volume oncologische weesgeneesmiddelen 103 Bijlage 9 Overzicht Weesgeneesmiddelen Horizonscan 106 Bijlage 10 Samenvatting reacties partijen incl. reacties Zorginstituut 109 Bijlage 11 Volledige reacties partijen 115 4 Monitor weesgeneesmiddelen in de praktijk 2019 | 12 november 2019 | Definitief Samenvatting Zorginstituut Nederland werkt aan het op peil houden van de kwaliteit, toegankelijkheid en betaal- baarheid van de gezondheidszorg. In deze derde ‘Monitor weesgeneesmiddelen in de praktijk’ zijn deze pijlers ook nadrukkelijk terug te vinden. Het doel van de monitor is inzicht te geven in de inzet van weesgeneesmiddelen in de dagelijkse Nederlandse praktijk. Dit doet het Zorginstituut door te monitoren hoe weesgeneesmiddelen na toelating tot het basispakket worden toegepast. Daarbij richten we ons op het volgende: • In beeld brengen van de kosten en volumes van de inzet van weesgeneesmiddelen. • Inzoomen op de toepassing van één of meerdere weesgeneesmiddelen; dit jaar staan de middelen voor de ziekte van Gaucher centraal. • Monitoren van de lopende centrale en decentrale weesgeneesmiddelen-arrangementen. • In kaart brengen van de stand van zaken voor wat betreft de toegankelijkheid van weesgeneesmiddelen. • Volgen van de belangrijkste beleidsontwikkelingen op het terrein van deze geneesmiddelen. Belangrijkste bevindingen • Tussen 2013 en 2017 heeft een stijging van de uitgaven aan weesgeneesmiddelen plaatsgevonden van €195 naar €272 miljoen per jaar. Dit is een stijging van 39% over 5 jaar met een gemiddelde stijging van 9% jaarlijks. Over deze vijf jaar steeg ook het aantal patiënten dat met deze middelen behandeld wordt, van 5000 naar 6900. De stijging in het aantal patiënten is met 38% vergelijkbaar met de stijging van de kosten. • De verwachting is dat de kosten de komende jaren zullen blijven stijgen aangezien de Horizonscan de komende jaren 57 nieuwe niet-oncologische weesgeneesmiddelen of indicatie-uitbreidingen van bestaande weesgeneesmiddelen verwacht. • De ziekte van Gaucher is een zeldzame, erfelijke stofwisselingsziekte die onbehandeld een ernstige verloop kent. Door de komst van weesgeneesmiddelen is er voor de patiënten de afgelopen 25 jaar veel veranderd en kunnen patiënten een (vrijwel) normaal leven leiden. Er zijn op het moment vier geneesmiddelen op de markt voor deze ziekte. Door het toepassen van strikte startcriteria en door de toepassing van een lagere dosering is door het expertisecentrum miljoenen op jaarbasis bespaard. • Het belangrijkste probleem rondom de weesgeneesmiddelen voor Gaucher is de hoge prijs. Het middel imiglucerase is al sinds 1997 op de markt en is nauwelijks gezakt in prijs, ook niet nadat de weesstatus is verlopen of er een vergelijkbaar middel op de markt is genomen. Zorgverzekeraars, het expertisecentrum en het ministerie van VWS zijn mede om die reden in gesprek om de Gaucher- middelen over te hevelen van het GVS naar de medisch-specialistische zorg per 2021. Er zijn plannen voor het uitschrijven van een tender (een procedure met een aanbesteding) voor twee middelen die vergelijkbaar zijn. Deze aanpak ondersteunen wij. • Er zijn aanwijzingen dat een subgroep van Gaucherpatiënten wellicht met een nog lagere dosering effectief behandeld kan worden of zelfs tijdelijk kunnen stoppen met enzymtherapie totdat biomarkers laten zien dat stapeling weer is toegenomen. We adviseren de minister van Medische Zorg om onderzoeksbudget
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