Health Technology Assessment and Evidence-Based … comparative analyses, and post-marketing surveillance Systematic review, cost/economic analyses, and comparative analyses Top Methodologies

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NAVIGATING HEALTH TECHNOLOGY ASSESSMENT

PROCESSES AROUND THE WORLD

ISPOR 13th Annual International MeetingToronto, Canada, May 2008

HTA – EBD Forum

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Health Technology Assessment and Evidence-Based Decisions (HTA-EBD) Special Interest Group (SIG)

Working Groups (WG)

HTA & Good Research Practices for Reimbursement Decisions

HTA of Emerging New Technologies

Global HTA Used in Healthcare Reimbursement

ISPOR LiaisonNadia Naaman - Randa Eldessouki

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Moderator

Jalpa A. Doshi, PhDPhiladelphia, PA, USA

NAVIGATING HEALTH TECHNOLOGY ASSESSMENT PROCESSES AROUND

THE WORLD

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Speakers

NAVIGATING HEALTH TECHNOLOGY ASSESSMENT PROCESSES AROUND

THE WORLD

Jennifer StephensBethesda, MD, USA

Karl Matuszewski Chicago, IL, USA

Neil PalmerOttawa, Canada

Annie Chicoye Paris - France

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Global Survey to Assess Methods Used in HTA & Reimbursement

DecisionsHealth Technology

Assessment & Good Research Practices

for Reimbursement Decisions WG

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Co-Chairs:Jalpa A. Doshi PhD, Research Assistant Professor of Medicine, General Internal MedicineUniversity of PennsylvaniaBonnie Handke RN, Director, Health Policy and Planning, Medtronic, Inc, Neuromodulation Jennifer Stephens PharmD, Clinical Director & Partner, Pharmerit North America LLC

Gisselle Gallego PhD, Research Fellow, Centre for Health Economics Research andEvaluation (CHERE) University of Sydney, AustraliaWim Goettsch PhD, Consultant Pharmacoeconomics Dutch Health Insurance BoardSeema Haider MSc, Director, Pfizer Inc, Global HE/ORFranz Hessel MD, MPH, Senior Manager, Sanofi-Aventis, Managed Markets / HEOR Tove Holm-Larsen MSc, University of DenmarkDeborah Lubeck PhD, Vice President Icon Clinical ResearchJohn O’Donnell PhD, Regional DirectorI, Astra-ZenecaDan Ollendorf MPH, Chief Review Officer, Institute for Clinical and Economic ReviewCarlos Gouveia Pinto PhD, Associate Professor of Health Economics, Technical University of Lisbon

Other Key Contributors:Isaac Foley, Pharmerit North America LLC; Howard Tag, Tag & Associates

HTA & Good Research Practices for Reimbursement Decisions WG

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Objective: To assess current methods used globally in HTA and health care reimbursementOn-line survey of 48 items

– Primary methodologies, importance of attributes – Breakdown by drugs, medical devices, other technologies

30 respondents to date with mix of regions/countries– Australia - 3– Canada - 2– Europe - 17– Latin America - 2 – United States – 6 (mostly government perspective; only 2

respondents from the private payers)

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Overview

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Type of agency/organization:– 58% HTA only – 7% reimbursement only – 17% both HTA & reimbursement – 17% other (eg, 3rd party payers)

Role of HTA:– Coverage & reimbursement decisions (79%), – Clinical guidance (72%)– Pricing decisions (35%)

HTA funding: 62% receive government funding at some levelHTA work performed most commonly by:

– In-house HTA staff (28%), – Combined HTA staff and outsourcing to professionals (66%) – Academia (38%)


Characteristics of Survey Respondents

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Evolutionary stage when a new drug, device, or other technology (e.g., surgical procedure) is selected for assessment

– New (drug-76%, device-86%, other-66%) – Established (45%, 69%, 55%)– Emerging (38%, 48%, 45%)– Declining (10%, 10%, 7%)

Why are technologies selected?– Top 3 reasons:

Perceived impact on patient outcomesPotential costPrevalence of condition

– Other reasons:Selected new technologyTechnology identified by external stakeholdersPerceived interest by public, academia, health professionals


When and Why Selected for Assessment?

10

0.00%

10.00%

20.00%

30.00%

40.00%

50.00%

60.00%

70.00%

N/A

Clinica

l Tria

ls

Epi a

nd othe

r obs

erva

tiona

l ana

lys

Cost/

Econ

ana

lyses

Compa

rativ

e ana

lyses

Post-

marke

ting s

urve

illanc

Modell

ing

Expe

rt Opin

ion

Group

judg

emen

t

Benc

h-testi

ng

Syste

matic R

eview

Meta-an

alysis

Other

EmergingNewEstablishedWide/Decline


Common Methodologies for Drugs by Stage

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0.00%

10.00%

20.00%

30.00%

40.00%

50.00%

60.00%

70.00%

N/A

Clinica

l Tria

ls

Epi a

nd ot

her o

bserva

tiona

l ana

lyse

Cost/E

con a

nalys

es

Compa

rativ

e ana

lyses

Post-

marke

ting s

urve

illanc

e

Modell

ing

Expe

rt Opin

ion

Group

judg

emen

t

Benc

h-tes

ting

Syste

matic R

eview

Meta-ana

lysis

Other

EmergingNewEstablishedWide/Decline


Common Methodologies for Medical Devices by Stage

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The most frequent methodologies for HTA are:

– systematic reviews,

– meta-analyses– economic

analysis– modeling

These methods are most commonly used for new and established drugs and medical devices

0.00%

10.00%

20.00%

30.00%

40.00%

50.00%

60.00%

70.00%

Emer

ging D

rugs

New D

rugs

Establi

shed

Drug

s

Wide

spre

ad/D

eclin

ing D

rugs

Emergin

g Med

ical D

evice

s

New M

edica

l Dev

ices

Establi

shed

Med

ical D

evice

s

Wide

spre

ad/D

eclin

ing M

edica

l Dev

ices

Systematic ReviewMeta-AnalysesModelling


Comparison of Top Methodologies for Drugs and Medical Devices by Stage

3

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Systematic reviews and cost/economic analyses (>50%)

Systematic reviews and meta-analyses

Systematic reviews, meta-analyses, cost/economic analyses, comparative analyses, modeling, and expert opinion

Systematic reviews, meta-analyses, modeling, and expert opinion

Clinical trials, comparative analyses, and post-marketing surveillance

Cost/economic analyses, systematic reviews, clinical trials, modeling, and comparative analyses

Top Methodologies for New Drugs

Systematic reviews and meta-analyses(>40%)All Regions

Systematic reviews, meta-analyses, and clinical trialsSouth America

Same as ‘New’ + group judgment, and epidemiological and observational studiesCanada

<none>Australia

Same as ‘New’ + Systematic reviews, meta-analyses, and epidemiological/observational studies

USA

Systematic reviews, meta-analyses, and cost/economic analysesEurope

Top Methodologies for Established Drugs

Region


Common Methodologies for New and Established Drugs by Region

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Systematic reviews, meta-analyses, cost/economic analyses, and comparative analyses (>50%)

Systematic reviews and meta-analyses

NR

Systematic reviews, meta-analyses, and expert opinion

Epidemiological and observational studies, comparative analyses, and post-marketing surveillance

Systematic review, cost/economic analyses, and comparative analyses

Top Methodologies for New Medical Devices

Systematic reviews, meta-analyses, cost/economic analyses, and comparative analyses (>40%)

All Regions

Systematic reviews, meta-analyses, and clinical trialsSouth America

NRCanada

Systematic reviews, meta-analyses, cost/economic analyses, and comparative analyses

Australia

Epidemiological and observational studies, comparative analyses, post-marketing surveillance, clinical trials, systematic reviews, and meta-analyses

USA

Systematic reviews, meta-analyses, and cost/economic analysesEurope

Top Methodologies for Established Medical Devices

Region


Common Methodologies for New and Established Devices by Region

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The most common method of synthesizing evidence is with both systematic reviews and meta-analyses; primarily performed for new and established drugs and technologiesSystematic reviews alone are most commonly used to synthesize evidence for emerging technologies

New Drugs

0.00%

10.00%

20.00%

30.00%

40.00%

50.00%

60.00%

70.00%

80.00%

90.00%

100.00%

N/A Only systematic review Only meta-analysis Both sytematic reviewand meta-analysis

Other

AllEuropeUSAAustraliaCanadaSouth America


Methods for Synthesis of Evidence

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Effectiveness is a key attribute across technologies, and more commonly considered than efficacyCost-effectiveness is not as frequently used for medical devices (MD) as it is for drug therapies (Rx)More frequent use of safety and quality of life attributes differentiate the US

0

0.1

0.2

0.3

0.4

0.5

0.6

0.7

0.8

0.9

1

Rx Europe Rx USA MD Europe MD USA

EfficacyEffectivenessSafetyCostsCost-effectivenessQuality-of-lifeBudget Impact AnalysisEquityBurden of Illness


Common Attributes Evaluated for Drugs and Medical Devices

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Efficacy, effectiveness, costs, and cost-effectiveness (≥70%)

Efficacy, effectiveness, safety, costs, and cost-effectiveness (=100%)

100% for all except equity

33% for all except equity

Quality-of-life (>80%), efficacy, effectiveness, safety, and costs (>60%)

Efficacy, effectiveness, and cost-effectiveness (>75%)

Most Common Attributes for New Drugs

Efficacy, effectiveness, safety, cost-effectiveness, and quality-of-life (>50%)All Regions

Efficacy, effectiveness, safety, costs, and cost-effectiveness (=50%)South

America

100% for allCanada

NRAustralia

Effectiveness, safety, and quality-of-life (>80%)USA

Efficacy, effectiveness, and cost-effectiveness (>55%)Europe

Most Common Attributes for Established Drugs

Region


Common Attributes Evaluated for New and Established Drugs by Region

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Efficacy, effectiveness, safety, costs, and cost-effectiveness (>70%)

Efficacy, effectiveness, safety, costs, and cost-effectiveness (=100%)

100% for all

Effectiveness, safety, costs, cost-effectiveness, and quality-of-life (>80%)

Safety and quality-of-life (>60%)

Efficacy, effectiveness, costs, and cost-effectiveness (>70%)

Most Common Attributes for New Medical Devices

Effectiveness, safety, costs, cost-effectiveness, and quality-of-life (>60%)All Regions

Efficacy, effectiveness, safety, costs, and cost-effectiveness (=50%)South

America

100% for allCanada

Effectiveness, safety, costs, cost-effectiveness, and quality-of-life (>60%)Australia


Effectiveness, safety, and cost-effectiveness (>60%)Europe

Most Common Attributes for Established Medical Devices

Region


Common Attributes Evaluated for New and Established Devices by Region

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19

0.00%

10.00%

20.00%

30.00%

40.00%

50.00%

60.00%

70.00%

80.00%

90.00%

Efficacy Effectiveness Safety Costs Cost-effectiveness

Quality-of-life Budget impactanalysis

Equity Burden of illness

DrugsMedical Devices


Most Important Attributes: Overall

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Effectiveness and safety (>60%)

Effectiveness and costs (100%)

Efficacy, effectiveness, costs, cost-effectiveness, and quality-of-life (100%)

Effectiveness and safety (100%)

Effectiveness, safety (>60%), and quality-of-life (50%)

Efficacy, effectiveness, and safety (>60%)

Most Important Attributes for Drugs

Effectiveness and safety (>70%)All Regions

Effectiveness and costs (100%)Latin America

Efficacy, effectiveness, costs, cost-effectiveness, quality-of-life, and burden of illness (=100%)

Canada

Efficacy, effectiveness, and cost-effectiveness (=33%)Australia


Efficacy, effectiveness, and safety (>70%)Europe

Most Important Attributes for Medical Devices

Region


Most Important Attributes by Region

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Common methods for economic evaluation :– Cost-effectiveness decision models (drug-69%, device-76%, other-59%)– Clinical trial based economic evaluations (59%, 62%, 52%)– Economic analyses of observational databases (55%, 66%, 59%)

Perspectives: societal #1, then third party payerCommon types of analyses allowed: cost-effectiveness, cost minimization, cost/cost-consequence, budget impact45% (drug) and 35% (device) of respondents evaluated the economic analysis for conformance with PE guidelinesInternational data for patient utilities/QoL is more likely to be accepted than international resource use data (>30% require country-specific resource use data)Only 10% of respondents report that thresholds are used to determine whether technology is cost-effectiveFor more than 50% of respondents, estimation of uncertainty around ICERs is mandatory


Economic Analysis in HTA

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100% of respondents consider HTA evaluations conducted by other organizationsOnly 28% repeat or update the assessment in regular internals89% report that a different organization has the responsibility to make the final decision on reimbursement

– These other organizations only partially rely on the assessmentMaturity level of methods in the field of HTA is considered to be mixed:

– Mature: 45%– Not mature: 48%

Quality level of the HTA reports rated as “excellent” by majority (62-79%) of reimbursement bodiesAt some level, stakeholders are involved:

– in assessments ~50% of time– in final decisions ~30% of time


Current HTA Process/Methods

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Europe:

Austria: need for observational studies/real life data (monitoring, registries, etc), development of “acceptable” thresholds & methods for resource allocationDenmark: lack of good studies/data as inputs to the assessmentsFrance: Early assessment of technologies with mechanism for conditional coverage, lack of evidence for emerging technologiesGermany: development/use of methodologies for health economic evaluationsItaly: HTA moving as a priority to regional health care agendasNetherlands: selection of comparators/study populations, model structure & assumptionsPortugal: selection of comparators, identification & quantification of costs, uncertainty analysisSweden: link between theory and practice in HTA, uniform analyses for comparative purposes, assessment of diagnostics, timeliness, selection of topics/comparatorsSpain: transparence, rigor, quality assessments, collaboration with other HTA agencies nationally & internationally, improved methods, training of new researchersSwitzerland: horizon scanning, implementation of regular re-assessments


Key Trends & Issues

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Canada:Increased consistency in economic evaluations and reviews leading to recommendationsDisease management, class review methodsStandards for rapid HTA

USA:Need for convergence around grading bodies of evidence Indirect vs.. direct meta-analysisApplicability of trials vs. potential for bias/confounding in observational studiesDifferentiation of new technologies, esp. biotechLack of solid published, peer-reviewed clinical evidence as inputs to assessment process

– Need access to good data– Lack of good comparative data with possible exception of drug therapies


Key Trends & Issues

5

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Australia:Timeliness, rapid review methodologies, prioritizing topics for reviewLack of evidence for some new & emerging technologies; small patient groupsIncreasing the use of economic analysisAssessment of diagnostic tests & use of linked evidenceSurrogate outcome validityValidity of combined endpointsAssessment of public health programs

Latin America:Burden of disease Microsimulation methodsEstablishment and validation of methodological guidelines for economic evaluation and systematic reviews


Key Trends & Issues

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Most HTA occurs at “new” or “established” stageTop methods used in HTA include systematic reviews, meta-analysis, cost-effectiveness analysis via modelingAttributes assessed and the importance of the attributes differ by country/regionKey issues & trends in HTA include standardizing methods for economic evaluations and grading of evidence, lack of evidence and data for emerging new technologiesPerception of maturity level of HTA methods varies


Summary

Decision-Makers & Health Technology Assessment Decision Making process

Health Technology Assessment of Emerging

New Technologies WG

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HTA of Emerging New Technologies WG

Co-Chairs: Noreen Sullivan BS, President and CEO, Technology Assessment Evaluation Group LLC, Encinitas, CA, USASheryl Szeinbach BS, MS, PhD, RPh, Professor, College of Pharmacy, Ohio State University, Columbus, OH, USA

Amit Chhabra MD, MPH, Outcomes Research Reimbursement Manager, Medtronic, Europe Sarl, Tolochenaz, Switzerland Eric Faulkner, Sr. Director RTI Health Solutions, Director, Genomics Biotech Institute, National Association of Managed care Physicians, NC, USA Karl Matuszewski MS, PharmD, Senior Director, Clinical Knowledge Service, University HealthSystem Consortium, Oak Brook, IL, USA Dimitris Polygenis PharmD, Vice President, Consulting Clinical Services, McKesson Phase 4 Solutions, Toronto, ON, Canada Mayvis Rebeira MBA, MA, Health Economist/Policy Adviser, Ministry of Health, Toronto, ON, Canada Prabashni Reddy PharmD, MS, RPh, Director of the Center for Drug Policy Partners Healthcare, Charlestown, MA, USA Jose-Manuel Rodriguez Barrios PharmD, MPH, MSc, Health Economics Reimbursement Manager, Medtronic, Iberia, Madrid, Spain Enrique Seoane-Vazquez PhD, Assistant Professor Ohio State University, Columbus, OH, USA

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Overview

Countries Evaluated:Australia, Canada, Germany, Spain, Sweden, United Kingdom and United States

Decision-Maker (DM): Defined as the payer (person or organization) who makes final decisions for coverage and paymentof a product or technology.

Evaluator: Defined as a person or organization who provides inputinto the decision-making process via HTA development but does notmake final decision for coverage and payment.

Decision-Making Process: The HTA evaluation process, as defined in the public domain, for emerging new technologies (i.e. medical device, pharmaceutical, diagnostic) in consideration forcoverage and payment.

Decision-Makers & HTA Decision-Making Process

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Pharmaceutical BenefitsAdvisory

Committee (PBAC)

Medical Services Advisory Committee

(MSAC) (21 specialists)

Ministry for Healthand Ageing -

Department ofHealth & Ageing

Australian Health Minister’s

Advisory Council (AHMAC)

Health Policy Advisory Committee

on Technology (Health PACT)


Australia

6

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Medical Devices & Non-Pharmaceutical Technologies

Canada

Payers* Provincial Ministries of HealthMedically necessary services (hospital and out-patient care)

Private Insurance & CashNot medically necessary

3. Other Influencers

• Hospital P&T Committees and Hospital Clinical Program Chairs (hospital focus)

1. Provincial Government Agencies

• Ontario: OHTAC (Health Technology Assessment Committee)

• Quebec: AETMIS (Agence d'évaluation des technologies et des modes d'intervention en santé)

• Alberta: AHFMR (Alberta Heritage Foundation for Medical Research)

• Other provincial-based HTA agencies

2. Research Agencies

• (CADTH) Canadian Agency for Drugs & Technologies in Health (National focus)

• (ICES) Institute for Clinical Evaluative Sciences (Ontario focus)

Influencers

•Canada’s public health care system (known as the Canada Health Act) is provincially administered and funded in part by Federal Transfer Payments; hospitals are funded via annual operating budgets approved by Ministry of Health (Provincial); Pharmaceuticals are not part of the Canada Health Act and are either funded by the province, third party insurance or out-of-pocket (varies by province)


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Pharmaceuticals

Canada

Payers

Provincial Ministry of HealthHospitals & IV drugs

Private Insurance &CashOut-Patient care (Non-

Eligible)

Provincial:1. Provincial Drug Plan Advisory

Committees2. Specialty Agencies (i.e.,

Cancer)

Federal/National:1. (CDR) Common Drug Review

(makes recommendations to Provincial Drug Plan Advisory Committees)

2. Joint Oncology Drug Review (oncology arm of CDR)

• All hospitalized patients receive all pharmaceuticals involved in their treatment at no cost; paid for from hospital budget (budget approved by Ministry of Health)

Influencers

Provincial Ministry of HealthOut-Patient (Provincial Drug Plan)

• Only pays for pharmaceuticals not reimbursed under public healthcare

Local:1. Hospital P&T Committee2. Hospital Clinical Program

Chairs

Provincial:1. Ministry of Health2. Provincial Specialty

Agencies (i.e., Cancer)3. Provincial Drug Plan

(indirectly)

1. Coverage influenced by each plan via internal advisory committee

2. Outsourced third-party clinical evaluation group

3. Benefits influenced by employer and/or Pharmacy Benefit Manager

4. Provincial Drug Plan

• Each province has defined eligibility criteria for it’s drug plan; plan usually covers seniors, social assistance and disabled but some provide universal coverage to all


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Pharmaceuticals

Pharmaceuticals are paid by provincial ministries of health (public) and insurance companies (private).

The clinical and health-economic evaluations for pharma products are conducted by the Common Drug Review (CDR), part of CADTH as well as by provincial ministries of health. CDR does not fund any drugs.

Provincial bodies and insurance firms often conduct separate analysis and not bound to the recommendations of CDR.

All recommendations by national and provincial bodies are listed on public websites.

Canada


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Statutory Health Insurance (SHI funds)

Central Federation Association of Health Insurance Funds

Institute for Quality and Efficiency in Health Care

(IQWiG)

Federal Ministry of Health

Germany

Decision-Makers & HTA Decision-Making Process (proposed)

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Central Government

Ministry of Health(Ministerio de

Sanidad y Consumo)

Regional Governments

(Autonomous Communities)

Regional HTA Agencies:Andalusia, Catalonia,

Basque Country,Galicia, Madrid

National Health System Interterritorial Council

(Consejo Interterritorial del Sitemap Nacional de Salud)

HTA Agency Instituto de

Salud Carlos III

# Regional Departments

of Health

Source: Law 16/2003, Real Decree 1030/2006

Spain

HTA & Decision-Making Process

36

18County Councils

Swedish Council on Technology Assessment in Health Care (SBU)

Ministry of Health & Social Services

Medical Products Agency

Pharmaceutical Benefits Board

National Corporation of Swedish Pharmacies

Swedish Parliament

Sweden


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National Institute for Health and Clinical Excellence (NICE) –

Centre for Public Health Excellence, Centre for Health Technology

Evaluation, and Centre for Clinical Practice

HTA from academic centers- Universities from Birmingham,

Southampton, York

National Health Service (NHS)

National Collaborating Centers (NCC)

e.g., Acute Care, Cancer, Chronic Conditions, Mental

Health, etc.

United Kingdom


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Private/Commercial Decision-Maker

• Blue Cross Blue Shield Association- 39 BCBS Companies

• Large MCOs (Managed Care Organizations)

- Aetna- Cigna- Humana- United Healthcare- WellPoint *

• Regional/smaller MCOs• Large self-insured employers

Evaluator- BCBSA TEC

Public/GovernmentalDecision-Maker•CMS:Centers for Medicare & MedicaidServices•DOD: Department of Defense- VA Health SystemEvaluators– AHRQ– ECRI– UHC Clinical Knowledge Service– Hayes Inc.– SG2– Health Advisory Board– Many other public/private,

profit/non-profit organizations*WellPoint is a BCBSA member and licensee in 14 states

United States (US)


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Agency for Healthcare Research and Quality

(AHRQ)

Medicare Coverage Advisory Committee

(MCAC)

Developing Evidence to

Inform Decisions about

Effectiveness(DeCIDE)

Reference: www.nlm.nih.gov/nichsr/hta101/hta101012html

Evidenced-Based

Practice Centers (EPC)

United States Preventive Task Force (USPTF)

Medicare MC Intermediaries

Center for Medicare & Medicaid Services (CMS)

Medicaid50 States + U.S.

Virgin Islands and Puerto Rico

United States (US)


40

Blue Cross Blue Shield AssociationPrivate/Commercial Payers

39 Blue Cross Blue Shield Individual Member Companies*

Decision-Maker

* HTA processes for coverage and payment is determined by each individual member plan. Scope and nature of process will vary by plan.**CMS may request that BCBS TEC conduct a HTA as part of the Evidence-based Practice Center program or in response for a request for an NCD.

United States (US)


Blue Cross and Blue Shield Association - Technology Evaluation Center (TEC))

Evaluator

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Private/Commercial PayersLarge MCOsAetna

Cigna Healthcare

U.S. Healthcare

Humana

Coverage policy developed

P&T committee meeting

Coverage policy implemented

Medical Device

Diagnostic Products

Pharmaceuticals

Small/Regional MCOs

Self-Funded Employers

United States (US)


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Large MCO Corporate Headquarters

Plan becomes aware of emerging technology

Internal HTA/coverage analysis group evaluates

evidence and makes recommendations



P&T committee meeting

Coverage policy implemented and reviewed annually

Key Decision Inputs:Systematic literature review

AMCP dossierExternal support (e.g., Hayes, ECRI)

Medical professional inputReview other payer policies

Other

Horizon scanningManufacturer communication

Payer-to-payer communicationOther

Coverage policy implemented

Feedback

Regional Plan/Business Unit

*

*

*Primary opportunities for manufacturer input

United States (US)


8

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Conclusion & Next Steps

To gain a better understanding of the HTA decision-makers and evaluators and the process used to determine coverage and payment for emerging new technologies.

Next Steps for Emerging New Technologies Special Interest Group:– Conduct 1-on-1 interviews with Decision-Makers

to further refine decision-making processes;– Publish results, i.e. manuscript(s) in Value in

Health, ISPOR website;– Timeline: Q3/Q4, 2008


ISPOR Global Health care Systems Road Map

Global Health Technology Assessment used in

Health Care Reimbursement WG

45

Co-Chairs: Stefan Holmstrom BSc, MSc, Director of Project Management, HE&OR, NicOx SA, Antipolis - cedex, France Kevin Mayo PhD, Director Health Outcomes Research, Daiichi Sankyo, Inc., Parsippany, NJ, USA

Lieven Annemans PhD, MSc, Ghent University, Faculty of Medicine, Meise, BelgiumAnnie Chicoye, Senior Principal Director Pricing & Market Access IMS Consulting, FranceShanlian Hu MD, MSc, Professor Training Center for Health Management School of PublicHealth Fu Dan UniversityChristine Huttin PhD, Health Scientist & Research Professor, ENDEPUSresearch and ENDEPResearch Group, Cambridge MA USAIsao Kamae MD, DrPH, Professor of Pharmacoeconomics, JPMA Research and EducationProject, Keio University of Tokyo, Fujisawa-City, Kanazawa, JapanProfessor Shu Chen Li, Chair, Discipline of Pharmacy , The University of Newcastle, Newcastle, AustraliaHans Middelhoven PhD, F. Hoffmann – La Roche Ltd., Basel, SwitzerlandMark Nuijten PhD, MD, MBA Eramus University, Rotterdam, The NetherlandsW. Neil Palmer, Vice-President, Pricing and Reimbursement, RTI Health Solutions, Ottawa, CanadaCarolyn Steeds BSc, Independent Consultant, Carolyn Steeds Consulting, Motherwell, ScotlandRebecca Townsend BSc, Project Director, Evidence Research Unit, Macclesfield, UKChristoph Vauth MSc PhD Student, University of Hannover / IVBL, Hanover, GermanyKonrad RB Wallerstein, Adelphi Focus, Doylestown, PA, USAFeng Zeng PhD, Health Economist, MedImpact Healthcare Systems, San Diego, CA

Global Health Technology Assessment used in Health Care Reimbursement WG

46

Objectives:– To develop a global review of available health

technology assessment information – To develop a roadmap of health technology

assessment information at the ISPOR website for health care decision-makers and payers.

Achievements:– The ISPOR Global Health Care Systems Road

Map

Global Health Technology Assessment used in Health Care Reimbursement WG

47

The Road Map provides an overview of country-specific health care delivery systems, reimbursement and pricing approval processes, reimbursement terminology and contact information for the most relevant HTA and other government organizations.


48

France


9

49

Mandatory Health Insurance Funds(CNAMTS, MSA, CANAM)

Population

Complementary Insurance Funds

Dental & Optical Care

Pharmaceuticals

Doctors’ fees

HospitalsIncome based contribution

(60%)

Special Income taxation

(40%)

Salary/risk based

premiums

Co-payments

35%

60%

75%

92%

The French Health Care System


50

Prices for pharmaceuticalshave been regulated since1945OTC/non reimbursablemarket share is limited to 8 % Reimbursement issubmitted to enlisting on positive lists (4)

Retail reimbursableMarket

Hospitaldrugs

Hospital onlydrugs

Delivered to out-patients

Drugs chargedto HI in addition To DRGs tariffs

Hospitalformularies

What is the process for access to reimbursement ?


51

TransparencyCommittee (CT) Chairman : Pr. Bouvenot

Economic Comittee for Health care Products (CEPS)

Chairman : Mr. Renaudin

Members Medical & methodological experts(voting) Representatives of payers & Government Mission

Technical assessment for reimbursement decision

Haute Autorité de Santé(HAS : health high authority )

Health MinistryMembersHealth, Finance and industry MinistriesPayers (mandatory & complementary) MissionPer product price fixing Global regulation of pharma expenditures

Risk/benefitPharmaceutical quality

EMEA/ AFSSAPS

MA application

MAMA

«« SMR & ASMRSMR & ASMR »» ratingrating

Admission to Admission to reimbursmentreimbursment(Official Journal)(Official Journal)

3 to 6 months

3 to x (?) months



52

TransparencyCommittee (CT) Chairman : Pr. Bouvenot

Economic Comittee for Health care Products (CEPS)

Chairman : Mr. Renaudin

Members Medical & methodological experts(voting) Representatives of payers & Government Mission

Technical assessment for reimbursement decision

Haute Autorité de Santé(HAS : health high authority )

Health MinistryMembersHealth, Finance and industry MinistriesPayers (mandatory & complementary) MissionPer product price fixing Global regulation of pharma expenditures

Risk/benefitPharmaceutical quality

EMEA/ AFSSAPS

MA application

MAMA

«« SMR & ASMRSMR & ASMR »» ratingrating

Admission to Admission to reimbursementreimbursement(Official Journal)(Official Journal)

Every 5 years

New labelling ? (indication, administration

scheme..)

Class re-assessed ?

Product re-assessed



53

EpidemiologyPrevalence, incidence

Segmentation

Burden of Disease

Morbidity/mortalityQoL

Clinical dataCT opinion

Data supporting MA : EPAR/CHMP opinion Other clinical data :

on-going clinical trials, post-marketing surveillance

Risk management plan

Pharmaceutical Profile & mechanism

of action

Literature(epidem & PRO) RegistriesDatabasesAd hoc studiesExpert opinions ..

MedicalGuidelines


What are the data considered by the Transparency Committee ?

National (HAS, Scientificsocieties )

InternationalOther HTAs assessment

54

Should the product be reimbursed ? For which patients ? At which rate ?

« Service médical rendu» (SMR *)

Risk/benefit ratioSeverity of diseaseNumber of alternatives, if anyPosition in therapeutic strategy (first/second

line, sub group of patients) restrictedreimbursement

Benefit for the public health

Rating « Major » or « Important »« Moderate »« weak » ou « Insufficient »

* medicalservice rendered

What are the criteria considered by the Transparency Committee ?


10

55

Should the product be priced at a premium price ? « Amélioration du Service médical rendu» (ASMR *)

Clinical studies versus reference product :– Cheapest, most prescribed, most recently

enlisted– Usual therapy eventually

Direct or indirect comparisons (meta-analysis, lit review, experts opinions)

Determined for each indication / sub-group of population

RRating versus determinedcomparatorGrade I: MajorGrade II: Important Grade III: ModerateGrade IV: MinorGrade V: no ASMR

* Improvement of medicalservice rendered


What are the criteria considered by the Transparency Committee ?

56

SMR Rating - 2006

0

50

100

150

200

250

300

350

Major/important moderate weak insufficient

SMR grade

Num

ber o

f pro

duct

s

First enlisting new indication

ASMR - 2006

050

100150200250300350400

I II III IV V

Comments

ASMR Rate

Num

ber

of

prod

ucts

/indi

catio

n

First enlisting New indication

SMR 2006: 87 % rated major / important 3 % rated unsufficient

Source HAS – report 2006

ASMR 2006 : 11 % rated I to III 4 % rated IV 87 % rated V


Not discriminant for SMR, but highly for ASMR

57

Economic negotiation with CEPS Technical assesement by CT

Reimbursement rate (100%, 65% 35%)SMR rate

EU price corridor Premium price versus competitor (I to III)

ASMR rate

Sales / volumes price agreementTarget population, position in therapeutic strategy, duration of treatment ..

Re-assessment of ASMR withprice/volume agreement consequences

Utilisation profile, real life effectiveness, new clinicalevidence

No pharmacoeconomics are required, although they can betaken into consideration


The CT assessessment is determinant for price negotiation

58


Canada

59

Federal Canada Health Act defines Canadian “Medicare”– Universal access – all Canadians covered– Publicly administered – Coverage is portable across provinces– Comprehensive – most medical necessary services are covered

(But not prescription drugs dispensed outside hospitals)– Publicly funded (funding shared between federal/provincial

governments)All provinces have established prescription drug benefit programs (no federal funding)

– Significant differences in coverage, eligibility between provincesFederal government has established drug benefit plans for:

– Veterans, Native Peoples, Federal Prisons, etc.


Canadian Heath Care System

60

Public (government funded) schemes– Over 65 years of age– Social assistance (welfare)– High drug costs to Income– Targeted diseases (e.g., HIV/AIDS, MS)– Hospital in-patients (covered by hospital “global” budget)

Private insurers provide coverage to working populations

– part of “extended health care benefits” offered by employers, employee groups or associations (and more recently to individuals)

No coverage– Uninsured (e.g., unemployed, self-employed, small

employers)– Non-reimbursed drugs (e.g., lifestyle drugs)

Total “out of pocket”– Individuals contribute 22% of total expenditures

no coverage + deductibles / co-payments

Government45%

Private Insurers

33%

Out of Pocket

22%


Who pays for prescription drugs in Canada?

11

61

Patented Medicine Prices Review Board (PMPRB)– Federal quasi-judicial agency with a mandate to ensure prices of

patented medicines are not excessive can order price reductions,repayment of excess revenues

– PMPRB limits prices of most new medicines to the range of prices in the same therapeutic class

Breakthrough / Substantial Improvement drugs may be allowed higher prices (international median price)PMPRB limits price increases to increases in CPIPrices of patented medicines can never exceed the range of international prices (among PMPRB reference countries)France, Germany, Italy, Sweden, Switzerland, UK, US

– PMPRB decisions have no direct role in reimbursement or decisions of drug plans to list (or not list) new drugs as benefits


Canada Price Controls

62

PMPRB Guidelines do not consider “cost-effectiveness” in determining whether prices are excessive

*if primary test is not possible or not appropriate

CPI Test: 3 year cumulative change in CPI

1 year “cap” (1.5 x CPI)Existing Medicines:

International Median

Therapeutic Class Comparison

3. Moderate / No Improvement

N/AHigher of

Therapeutic Class & Intl. Median

2. Breakthrough / Substantial

Improvement

Prices ofpatented

medicines cannot exceed

the International

MaximumPrice


Reasonable Relationship Test1. Line Extension

All PatentedMedicines

Secondary Test*Primary TestNew Medicine

Category

*if primary test is not possible or not appropriate

CPI Test: 3 year cumulative change in CPI

1 year “cap” (1.5 x CPI)Existing Medicines:

International Median


3. Moderate / No Improvement

N/AHigher of

Therapeutic Class & Intl. Median

2. Breakthrough / Substantial

Improvement

Prices ofpatented

medicines cannot exceed

the International

MaximumPrice


Reasonable Relationship Test1. Line Extension

All PatentedMedicines

Secondary Test*Primary TestNew Medicine

Category


PMPRB Price Review Tests

63

The CDR is a program of the Canadian Agency for Drugs and Technology in Health (CADTH)The CDR reviews new drugs (and new indications) provides formulary listing recommendations to all publicly-funded drug benefit plans in Canada (except Quebec)The CDR conducts clinical and health economic reviews but not budget impact

– Budget impact analyses need to be submitted to each of the drug plans and must be specific to each plan

Each drug plan independently advises manufacturer of its listingdecision and coverage status of the drug.

– Affordability / budget impact are often the key factors for the plans


Common Drug Review (CDR)

64

+ 9 weeks

+ 25 weeks

+ 0

Elapsed Time (weeks)

+ 28 weeks

+ 52 weeks


Common Drug Review Process

65

List: 4%

List with Conditions:

28%

Do not List: 54%

List as Similar: 14%

The majority of new drugs are refused by CDRThose with a positive recommendation usually have restrictions


Common Drug Review Process: CDR RecommendationsNAVIGATING

HEALTH TECHNOLOGY ASSESSMENT PROCESSES AROUND THE WORLD

THANK YOU MERCI

Documents

Health Technology Assessment and Evidence-Based … comparative analyses, and post-marketing surveillance Systematic review, cost/economic analyses, and comparative analyses Top Methodologies