The Changing face of ERM: The Insurance Company’s Perspective
Patient Groups as Research Partners: a changing perspective
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Patient Groups as Research Partners: a changing perspective
Dr. Cees Smit, VSOP/EGAN
Orphan Café, Utrecht
27 juni 2013
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Unmet medical needs
Unmet medical needs
are the driving force
for patient groups
to become active in medical research
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Age-distribution severe haemophilia (n=338)
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
1975 1985 1995 2000 2009
Year
Pro
po
rtio
n o
f p
ati
en
ts
>40 years
30-40 years
20-30 years
10-20 years
0-10 years
Mauser et al, Van Creveldclinic, Utrecht, 2010, personal communication
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Eerste boek over Nederlandse patiëntenbeweging
Harderwijk, 24 februari 2012 – Patiëntenorganisaties zouden meer betrokken moeten zijn bij medisch-
wetenschappelijk onderzoek. Zo ontstaat voor alle partijen – patiënten en
wetenschap – een win-winsituatie. Dit stelt Cees Smit in ‘Een nieuwe horizon: de
toekomst van de patiëntenbeweging in Nederland’ dat morgen verschijnt. Nooit
eerder is dit onderwerp in boekvorm beschreven.
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Dutch patient groups
• Micro About 400 – 500 disease-specific
patient groups
• Meso Large disease-specific umbrella groups
(diabetes, cancer, rheuma & arthritis, heart &
vascular diseases, COPD, neuromusculair diseases and genetic/rare diseases)
• Macro The 4 (NPCF, CG-Raad, VG, GGZ, & PGOSupp.)
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Worldwide patientmovement
• Similar patterns
• EPF European Patients’ Forum
• Eurordis Rare diseases
• EGAN/VSOP Genetic/rare diseases
• IAPO International Alliance of Patient Organizations
• Important Eur. Commission, EU Parlement contacts: EMA & EFPIA TT (4/year), WHO
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The patient in 40 years
Patiënt 1.0 Information, education Peer support Lobbying for their interests Patiënt 2.0 Internet, social media, Wiki’s, Patientslikeme, SWAN UK Patiënt 3.0 Cooperation researchers, patients and companies Driving force: ‘Unmet medical needs’
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Process of the drug development process
1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20
fundamental
pre-clinical
research
Ph. 1
Phase 2
Phase 3
Phase 4
Registration
clinical research
post marketing research
effective patent-period
Patent request
launch
product
End of patent
Years Ref: Jan Raaijmakers (2005, UU)
JR 2005
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‘Duw’ or ‘Push’ model
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‘Trek’ or ‘Pull’ model
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Participationladder from Arnstein
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Participationladder vlgs. PatientPartner
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Prader Willisyndroom
• Genetic disease (extreme obese)
• Observation mother (N-Zl), her son (39) symptoms: elderly with dementia
• Swaab, biobank research: yes, more cases
• Since that time worldwide more cases also General question:
• Will extreme obesity lead to an earlier explosion of dementia/Alzheimer
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Patients as driving force (1)
• Marshall Smith Syndrome, n = 3 in NL
• Parents of one of these patients started five years ago through the internet a search to other, all patients worldwide
• When they found 17 patients, they could identify one of the genes responsible for MMS (AMC, London)
• Now, ≥ 30 patients who have met each other and have regular contact by skype/webcam
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Patients as driving force (2)
• Peter Kapitein (leukemia) & his friends started in 2007 with the organization of a cycle tour to Alpe d’Huez in France
• (6 times up, 6 times down)
• Revenue in 2007: 370.000 Euro
• Revenue in 2012: 32 million Euro (2013: 25 mln.)
• With ‘Live2inspire’, they now have an international action plan developed against cancer (support DNB, KNAW) & they have a say at KWF Kankerbestrijding
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Innovative power patients
• To bring together patients and their body material (MSS)
• Sharing of data for research and the organization of cooperation (CF/EuroBiobank)
• Fundraising for biobanks and research (gene therapy), also for the long term
(AFM, Généthon, DPP, Alpe du Hu’Zes)
(Ref.: Van der Valk & Smit, NTvG, mei 2011)
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Kwaliteit van de zorg en de patiënt: de botsing tussen
behandeling en QALY-grens
Dr. Cees Smit, VSOP/EGAN
Orphan Café, 27 juni 2013
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23
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NOS Journaal
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Besluit ACP/CvZ 21 september 2013
• Doorgaan met behandeling, apart fonds
• Minister van VWS: Ja, 2013 basispakket
• 2014: Nog géén beslissing
• Prijsonderhandelingen zijn gaande
• Zomer 2013 besluit voor 2014
• Uitkomst onderhandelingen geheim (NICE)
• Verdere beleidsvragen staan ook nog open
• Regeerakkoord: Kosteneffectiviteit in de wet
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Kort overzicht QALY-discussie
• De ‘Trechter van
Dunning’, 1991
• RvZ rapport, ‘Zinnige
en duurzame zorg’, 2006
QALY 80.000 euro/jaar
• CvZ-advies Pompe en
Fabry, zomer 2012
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Kosteneffectiviteit & QALY-grens
• Is ziektelast uit te drukken in een getal tussen 0 en 1 ?
• Weten we hoeveel zorg duurder is dan 80.000
Euro/jaar? (nierdialyse, stamceltransplantatie)
• Zitten alle kosten en baten in een QALY-calculatie?
• Juiste uitkomstmaten: 6 minuten looptest/rolstoel
• Bij stoppen behandeling:
niet behandelen vraagt ook zorg (achteruitgang)
• Verlies aan onderzoeksinvesteringen (30 jaar onderzoek naar de ziekte van Pompe in ErasmusMC)
• EU-beleid stimuleren ; NL groei zorg afremmen
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Off-spin discussie
Huub Schellekens, hoogleraar farmacie, UU
'Wat je nodig hebt voor het middel tegen de ziekte van Pompe is 20 gram eiwit per jaar. Zeg nou eens dat het 200 euro per gram kost. Dan heb je het in zijn zuiverste vorm. Dat is alles.‘ (Citaat uit: Volkskrant, 11 augustus 2012)
Hans Büller, RvB ErasmusMC
‘Ik zou heel graag willen dat we het op Europees niveau als een verantwoordelijkheid zien die geneesmiddelen te ontwikkelen’
(Citaat uit: NOS Journaal, 12 augustus 2012)
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Symposium AMC, 8 januari 2003
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QALY Hemofilie
Ong. 200.000 Euro/jaar voor ernstige vorm
Kan het goedkoper?
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Olieraffinage
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Bloedproducten vs plasmaproducten
Rode bloedcellen
Bloedplaatjes Bloedcomponenten
Vers bevroren plasma
Albumine
Immunoglobulinen
Stollingsfactoren Plasmaproducten/geneesmiddelen
C1-remmerconcentraat
Andere eiwitten
Samenvoeg
en
Sanquin Divisie
plasmaproducten
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Prijs olie / bloed
Vat olie (188 liter) Vat bloed
• 1998 13 $ 20.000 $ • Na raffinage 42 $ 67.000 $
• 1980: 67€ (250 E. FVIII) → 2013 207,50 € (250 E. FVIII)
• 2013 • NL behoefte Factor VIII = 5 gram • 1 gram = 20 à 25 miljoen (plasma/biologicals) • Iran: factor 10 goedkoper, 2 à 2,5 miljoen
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Kan het goedkoper ?
• In 40 jaar niet gebeurd
• Zowel in ‘profit’ sector als in ‘not for profit’
• Voortdurende productvernieuwing
• Noodzakelijk (virusveiligheid, hiv, hcv, BSE)
• Vervanging plasmaproducten door rDNA (biologicals, biosimilars op komst)
• Goedkopere technologie (wie betaalt dat?)
• Bijv. gentherapie, levertransplantatie
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Sanquin model
• Hybride structuur (‘profit’ en ‘not for profit’)
• Mogelijk wel interessant business-model voor ontwikkeling andere weesgeneesmiddelen in samenwerking met universiteiten en start-up biotech bedrijven vanuit universiteiten
• Wie pakt het op?
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Nieuw boek september 2013
‘Een zeldzame ziekte, je leeft ermee’
Hemofilie als voorbeeld:
Lessen uit het verleden,
Opties voor de toekomst
Annemarie de Knecht-van Eekelen & Cees Smit
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For more information
www.smitvisch.nl