Vertrouwen in intramurale geneesmiddelen: voortdurende af(g)ronding

Vertrouwen in intramurale geneesmiddelen: voortdurende af(g)ronding Martin van der Graaff PhD Secretaris WAR Zorginstituut

NVFG Nieuwjaarsbijeenkomst Buunderkamp, Wolfheze 20 januari 2015

Budget-Busters: The Shift to High-Priced Innovator Drugs in the USA

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New report analysing Top 100 drugs in the USA

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Median price of the Top 100 drugs has skyrocketed from $1,260 in 2010 to $9,400 in 2014, representing a sevenfold increase. 2

Zorginstituut Nederland Healthcare Insurance Board (CVZ) Cooperation with stakeholders Guidelines Protocols Best practices

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Healthcare Institute (ZIN)

Quality Institute

Package management

Risk-based package management with less detailed scrutiny of drugs

Initiative by industry, ZINL or insurers Notification or direct dossier demand Added value against ≥ 1 comparator

≥€

2,5 mill

CVZ demands Dossier (4M)

<€

Equal value against ≥ 1 comparator

2,5 mill

≥€

2,5 mill

<€

2,5 mill

No assessment but monitoring Possibly assessment later on via risk based package management

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Timing depends on agenda setting ZINL

jaaragenda

inbreng partijen ACP

Pakketvraag

Scoping vooraf 5

Conceptrapport Zorginstituut

WAR

Besluit RvB

Advies Zorginstituut

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Area’s to “handle with care” instead of ceilings 80 000

20 000

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Industrie 8

Zorginstituut

Earliest recommendations of NHCU

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infliximab Remicade® in CU & psoriasis

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ranibizumab Lucentis® in WMD

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omalizumab Xolair® in severe asthma

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the Pompe and Fabry issue

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Some take-home’s from earlier (re)-assessments

everolimus Afinitor® bij gevorderde borstkanker i.c.m. exemestaan:

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SW&P Ruime onzekerheid rondom schatting KE ICER waarsch. > € 100.000 Uitkomstenonderzoek zal niet helpen, dus geen verzoek tot uitvoering daarvan ACP: behandelduur handhaven, niet te lang wachten met herbeoordeling

natalizumab Tysabri® bij MS:

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SW&P KE onvoldoende onderbouwd, opzet uitkomstenonderzoek ontoereikend, geen goede vergelijking met alternatieven Goede richtlijn beroepsgroep met aandacht KE noodzakelijk Mogelijk niet-gepast gebruik. Ronde tafel moet garanties voor betaalbaarheid MS zorg opleveren en beeld van relatieve plaats interventies Risico’s op alle vier de pakketcriteria

trastuzumab Herceptin® bij borstkanker

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SW&P ICER ~€15.000/QALY; hoewel onzekerheden, voldoende onderbouwd “Zorg met lage uitvoeringsrisico’s”, ook weinig risico’s op niet-gepast gebruik.

voriconazol Vfend® bij invasieve aspergillose

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SW&P KE analyse voldoende kwal.: gunstig tov L-Amb Duidelijke database SWAB Weinig kans niet-gepast gebruik

Trends in the reimbursement environment

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Personalized medicine The domino effect The outcomes debate The scoping effort The negotiation initiative The registries avalanche

Population based medicine EVIDENCE Bewijs

Personalized medicine Passend Plausible bewijs effect

Afspraken/ Agreements Geen bewijs stakeholders

Role of government As evidence assessor Role interested parties Role Health Technology assessment

Role of political process 16

The domino effect

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FDA/EMA: indication-less approvals Payers to define breadth of indication and reimbursement Payers choose GRADE: nuanced recommendations Buck passed to doctors and insurers

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The outcomes debate

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Industry: the more outcomes, the more likely one will be significant Does value story address outcomes and start/stop? BECAUSE: Payers want outcomes defined and cost/outcome & cost/QALY Payers want start/stop criteria defined and enforced Reimbursement outcomes may differ from registration outcomes

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The scoping effort

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Bring all stakeholders together even before registration

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Useful, even if there a monopolistic product is involved What outcomes are relevant? What extent of outcome is regarded as an improvement? Exercise in assuming co-responsibility Is individualized treatment possible? Does the dossier contain answers to the questions? So,dossier may no longer be leading in answering reimbursement question!

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The negotiation initiative

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National negotiation agency Confidential agreements No longer insight in cost/QALY Cost reductions

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Isolated agreements industry/insurers new trend

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Perceived inequality central/decentral agreements

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The RWE/registries avalanche

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RWE mostly collected for what industry thinks relevant

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Informs generally more about safety than effectiveness

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Use of registry for payers (1/2) Will the registry:  include an outcome parameter that is easily measured and correlate with detectable patient improvement?  dare we use modern technology?  dare we share on an ongoing basis?  help discuss acceptable cost/outcome upfront  show to what extent the use of a given intervention is warranted /necessary?  diminish risk of non-rational use? 24

Use of registry for payers (2/2)

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enable insurers to orchestrate the use of the product? contribute to a cost-effective deployment of products in the entire indication? promote reporting on (enforcement of) start/stop & dosing changes foster dialogue between patients, doctors and payers? yield actionable data within a year?

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Reimbursement debate of the future Interested parties discuss well before registration what outcomes will be considered a (clinically) relevant improvement (scoping upfront)  Desirability of intended effect  Extent of intended effect  Credibility of intended effect

Statistically significant will become irrelevant Discuss how we value:  Outcomes for the patient  Outcomes for others  Outcomes for society 27

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