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

HEALTH CARE DECISION MAKING

Working Groups (WG)

AROUND THE WORLD

HTA & Evidence Based Decision Special Interest Group - Forum

z

HTA & Good Research Practices for Reimbursement Decisions

z

HTA of Emerging New Technologies

z

Global HTA Used in Healthcare Reimbursement

ISPOR Liaison

ISPOR 11th Annual European Congress NOVEMBER 2008

Nadia Naaman - Randa Eldessouki

11/19/2008

2

HEALTH CARE DECISION MAKING AROUND THE WORLD

HEALTH CARE DECISION MAKING AROUND THE WORLD

Speakers

Moderator

Karl Matuszewski MS, PharmD Chicago, IL, USA

Marcin Czech PhD, MD, MBA Warsaw, Poland

Angeliki Angeli MSc Athens, Greece

Kevin Mayo PhD Denville, NJ, USA

Bonnie Handke RN, MBA Minneapolis, MN, USA 11/19/2008

3

ISPOR Global Health Care Systems Road Map

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.

11/19/2008

dsjfl

Afdlsa;jfl;

11/19/2008

4

ISPOR Global Health Care Systems Road Map

5

11/19/2008

6

http://www.ispor.org/HTARoadMaps/Default.asp

1

HTA of Emerging New Technologies Working Group Co-Chairs: Karl Matuszewski MS, PharmD, Senior Director, Clinical Knowledge Service, University HealthSystem Consortium, Oak Brook, IL, USA Noreen Sullivan BS, President and CEO, Technology Assessment Evaluation Group LLC, Encinitas, CA Sheryl Szeinbach BS, MS, PhD, RPh, Professor, College of Pharmacy, Ohio State University, Columbus, OH-US

Country-Specific Decision-Making Models

Leadership Members: Amit Chhabra MD, MPH, Outcomes Research Reimbursement Manager, Medtronic, Europe Sarl, Tolochenaz, Switzerland Eric Faulkner, Sr. Director RTI Health Solutions, Research Triangle Park, NC, USA Mayvis Rebeira MBA, MA, Health Economist/Policy Adviser, Ministry of Health, Toronto, ON, Canada 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

HTA of Emerging New Technologies – Working Group

Additional contributors Anke-Peggy Holtorf, PhD, MBA, Business Director, BioBridge Strategies, Basel, Switzerland

11/19/2008

Country Specific Decision-Making Models z

Project Objective: To provide a comprehensive, validated decision model for each country* that defines decision-makers and the decision-making process for coverage and payment of new and emerging technologies in pharmaceuticals, medical devices and diagnostics

z

Project Goal: – First, to define the process at the highest level (i.e. 30,000 ft. view), to provide a baseline understanding of the primary factors which impact decisions, for users who may or may not be familiar with these processes. Validate model with decision-making body. (Level 1) –

Country Specific Decision-Making Models z

Level 1: – Name of Decision-Maker – Name of Evaluator/Advisory Organization – Primary factors included in the decision-making process. – A very brief description of the Decision-Maker should be included in the “Notes” section of this slide to include the coverage population or # covered lives (reflecting the size of our chosen Decision-Maker.

z

Level 2: (visual or narrative) Describe how the Decision-Makers function (i.e. key elements) – Leadership structure of Decision-Maker (decision-making organization) – Description/overview of Decision-Maker (person and/or organization) – History of Decision-Maker – Geographic coverage map – Tools required (i.e. HTA, health economic analysis, etc.) – Website address

Second, to provide additional detail on specific data requirements and tools needed to support these decisions, by country, to support the decision-making process. (Level 2)

*The countries being evaluated will be determined based upon resources available. We will begin with U.S., Australia, France, Germany, Sweden, Spain, Canada and the U.K. The long-term plan for the SIG is to develop a model for every country, in collaboration with the 2 other working groups, and post on the ISPOR website.

11/19/2008

*The countries being evaluated will be determined based upon resources available. We will begin with U.S., Australia, France, Germany, Sweden, Spain, Canada and the U.K. The long-term plan for the SIG is to develop a model for every country, in collaboration with the 2 other working groups, and post on the ISPOR website.

11/19/2008

9

Country Specific Decision-Making Models

10

Country Specific Decision-Making Models Model Description and Key:

Definitions: z

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

z

Evaluator: Defined as a person or organization who provides input into the decision-making process via HTA development but does not make final decisions for coverage and payment.

z

z

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 for coverage and payment.

11/19/2008

The ultimate decision-maker is listed at the top of the model. Therefore, decision-making flow runs from bottom to top.

Symbols:

z

z

8

z z z

11

Boxes: Decision-making bodies. Circles: Data requirements, tools, etc. which impact process. Solid Arrows: Required step in decision-making process. Broken Arrows: May or may not impact decision. Notes Section: Provides narrative to explain the flow of the model and the factors involved by both decision-maker and process.

11/19/2008

12

2

United States (U.S.)

United States (U.S.) Center for Medicare & Medicaid Services (CMS) State Medicaid Depts

#37 WHO Ranking for Health System Performance, 2000

Medicare Intermediaries

Agency for Healthcare  Research and Quality  (AHRQ)

Developing  Evidence to  Inform Decisions  about  Effectiveness (DeCIDE)

Council on Technology & Innovation

Evidenced‐ Based  Practice  Centers  (EPC)

Medicare  Evidence  Development &  Coverage  Advisory  Committee (MedCAC)

United States  Preventive  Services Task  Force (USPSTF)

NHS??? 11/19/2008

11/19/2008

13

14

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

Level 1

United States (U.S.)

Level 2

United States (U.S.)

WellPoint, Inc. - Blue Cross Blue Shield licensee WellPoint, Inc. - Blue Cross Blue Shield licensee WellPoint Pharmacy Management Director, Clinical Affairs

Pharmaceuticals

National Pharmacy & Therapeutics (NP&T) Committee

WellPoint Pharmacy Management Data Request Made to Manufacturer

National Pharmacy & Therapeutics (NP&T) Committee

WellPoint Outcomes Based FormularySM

Formulary Review & Benefit Design (FR&BD) Committee

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

No Further Review 11/19/2008

15

WellPoint Outcomes Based FormularySM

•Determine Safety, Efficacy, Effectiveness •Assign WellPoint NP&T Designation

Further Review Required

Formulary Review & Benefits Design (FR&BD) Committee

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

11/19/2008

16

Australia - Pharmaceuticals

Australia

Australian Government / Minister for Health and Aging / Department of Health and Aging (DoHA)

#32

Pharmaceutical Benefits Pricing Authority (PBPA)

Pharmaceutical Benefits Schedule

Pharmaceutical Benefits Advisory Committee (PBAC)

Australian Drug Evaluation Committee (ADEC)

Therapeutic Goods Administration (TGA)

Australian Register Of Therapeutic Goods (ARTG)

NHS??? 11/19/2008

17

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

11/19/2008

18

3

Australia – Medical Devices

France

Australian Government / Minister for Health and Aging / Department of Health and Aging (DoHA) Medicare Benefits Schedule

Prostheses List

Consultative Committees: Pathology Services Table Committee (PSTC) Medical Benefits Consultative Committee (MBCC) Diagnostic Imaging Committees / representatives From DoHA serve on these committees

Prostheses and Devices Committee (PDC)

Australian Register Of Therapeutic Goods (ARTG)

Therapeutic Goods Administration (TGA)

Medical Services Advisory Committee (MSAC)

Medical Devices Evaluation Committee

(MDEC)

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

HealthPACT (sub-committee of MSAC) Oversees Australia and New Zealand Horizon Scanning Network (ANZHSN)

Level 1

#1

Australian Health Ministers’ Advisory Council (AHMAC)

Nationally Funded Centers

11/19/2008

NHS??? 11/19/2008

19

Level 2

France

France Ministry of Health

Ministry of Health

Comité Economique des produits de Santé (CEPS)

20

Official Journal: LDMI (Liste des Dispositifs Medicaux Implantables)

Comité Economique des produits de Santé (CEPS) Price

Economic dossier

Transparency Commission (TC)

Transparency Commission (TC) Marketing Authorization

ASMR Therapeutic Benefit

Medical dossier

11/19/2008

Marketing Authorization

21

11/19/2008

22

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

Spain

Spain Regional Governments (Autonomous Communities)

Central Government

National Health System Interterritorial Council (Consejo Interterritorial del Sistema Nacional de Salud)

Ministry of Health (Ministerio de Sanidad y Consumo)

#7 #7

HTA Agency Instituto de Salud Carlos III Source: Law 16/2003, Real Decree 1030/2006

H

TA

Re

Regional Departments of Health

rt s po

Collaboration

Regional HTA Agencies: Andalusia, Catalonia, Basque Country, Galicia, Madrid

NHS??? 11/19/2008

11/19/2008

23

24

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

4

Germany

Germany

Federal Ministry of Health

Statutory Health Insurance (SHI)

#25

Federal Joint Committee – Gemeinsamer Bundesausschuss (G-BA)

Institute for Quality and Efficiency in Health Care (IQWiG

German Institute for Health Technology Assessment (DAHTA) NHS??? 11/19/2008

11/19/2008

25

26

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

Sweden

Sweden

TLV (Pharmaceutical Benefits Board) base the decision of reimbursement of prescription drugs on a HTA submission from the pharmaceutical company and external recommendations

18 County Councils (providers)

#23

Swedish Council on Technology Assessment in Health Care (SBU)

Medical Products Agency (Läkemedelsverket) The National Board of Health and Welfare (Socialstyrelsen)

External clinical consultants National Corporation of Swedish Pharmacies (Apoteket AB) provide statistics

NHS??? 11/19/2008

11/19/2008

27

28

More details @ http://www.ispor.org/HTARoadMaps/Default.asp

We will diagram the major health systems of the world… one country at a time.

NHS??? 11/19/2008

29

11/19/2008

30

5

Global Health Technology Assessment used in Health Care Reimbursement WG Co-Chairs: Stefan Holmstrom BSc, MSc, Director of Project Management, HE&OR, NicOx SA, Antipolis cedex, France

Kevin Mayo PhD, Vice President Market Access Solutions, Bridgehead International Consulting, NJ,

ISPOR Global Health Care Systems Road Map

USA

Leadership Members Annie Chicoye, Senior Principal Director Pricing & Market Access IMS Consulting, Puteaux-Cedex

France

Hans Middelhoven PhD, F. Hoffmann – La Roche Ltd., Basel, Switzerland Mark Nuijten PhD, MD, MBA Eramus University, Rotterdam, The Netherlands W. Neil Palmer, Vice-President, Pricing and Reimbursement, RTI Health Solutions, Ottawa, Canada Carolyn Steeds BSc, Independent Consultant, Carolyn Steeds Consulting, Motherwell, Scotland Christoph Vauth MSc, PhD University of Hannover / IVBL, Hanover, Germany Feng Zeng PhD, Health Economist, MedImpact Healthcare Systems, San Diego, CA

Global HTA used in Health Care Reimbursement - Working Group

Additional contributors Angeliki Angeli MSc, Institutional Relations and Communication Director, Sanofi Aventis, Athens,

Greece

Christie Niziol, Associate Director- Global Services, ,Abacus International, UK Gulacsi Laszlo PhD, Health Economics and Technology Assessment Research Centre, Corvinus University of Budapest, Budapest, Hungary

Marcin Czech PhD, MD, MBA, Past President, ISPOR Poland Local Chapter & Adjunct, Dept of Pharmacoeconomics, Faculty of Pharmacy, Medical University of Warsaw, Warsaw, Poland

Rebecca Townsend BSc, Project Director, Evidence Research Unit, Macclesfield, UK Tony, Yen-Huei Tarn MS., PhD. (譚延輝), Director, Division of Health Technology Assessment

Center for Drug Evaluation Taipei, Taiwan

11/19/2008

Global Health Technology Assessment used in Health Care Reimbursement WG z

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.

z

Achievements: – The ISPOR Global Health Care Systems Road Map

11/19/2008

32

ISPOR Global Health Care Systems Road Map

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. 33

11/19/2008

34

http://www.ispor.org/HTARoadMaps/Default.asp

ISPOR Global Health Care Systems Road Map

Pricing & Reimbursement System Greece

ISPOR Global Health Care Systems Road Map

11/19/2008

35

6

ISPOR Global Health Care Systems Road Map - Greece

ISPOR Global Health Care Systems Road Map - Greece

The Greek Health Care System

Health Insurance

Mixed system of public-private funding and provision of health care services. Constitutes of:

z

– – –

The National Health System (ESY) (Public hospitals, Health Centers and the National Centre of Emergency Care) The Social Insurance Funds The Private sector (diagnostic centers, private clinics, laboratories, infirmaries etc.)

z

Health Insurance in Greece is compulsory

z

Assignment to a Fund depends on the occupation of the insured and not on his/her income level

z

Insurance Funds are mainly funded through insured and their employers’ premiums as well as by the governmental budget, through social levies and subsidization of deficits.

Health care is funded by the governmental budget (general taxation), the social insurance (insured premiums) and private expenditure

z

11/19/2008

11/19/2008

37

ISPOR Global Health Care Systems Road Map - Greece

ISPOR Global Health Care Systems Road Map - Greece

The Pharmaceutical Policy z

38

Pricing Policy

Fragmented in 3 Ministries: z

–Pricing

Strict control Prescription

policy Î Ministry of Development 11-member Pricing Committee

OTC

policy Î Ministry of Health &

z

FundsÎ Ministry of Employment & Social Protection

z

–Reimbursement

Social Solidarity

exception of areas where low VAT rate applies)

–Sick

11/19/2008

Uniform retail price across the country (with the Generics priced at 80% of branded

11/19/2008

39

ISPOR Global Health Care Systems Road Map - Greece

ISPOR Global Health Care Systems Road Map - Greece

Prices

Retail Price Structure 100%

z

40

Pharmacy Purchase Price (PPP)

8.2% 23.8%

80%

Ex-factory price + wholesaler profit

5.3% 60%

z

Retail price (RP)

40% 62.7%

PPP + pharmacist’s profit + VAT z

20%

0%

Hospital price (HP)

Manuf act urer

PPP - 13%

Wholesaler

Pharmacist

VAT

Retail price= 100% 11/19/2008

41

11/19/2008

42

7

ISPOR Global Health Care Systems Road Map - Greece

ISPOR Global Health Care Systems Road Map - Greece

Reimbursement System

Pricing System Market Decree 6/12.12.2005 z

z

All marketed prescription medicines are reimbursed by Social Insurance (Law 3457/2006)

z

Reimbursement rates

Average of the 3 lowest prices in Europe (2+1) –

2 are selected from the15 EU original member states + Switzerland and one from the 10 countries joining the EU on1/5/2004

z

Once a price has been set, a 4-year monitoring period begins, during which the Ministry of Development, investigates prices every year. If the verification price has changed Î price readjustment

z

Price bulletins are issued every 90 days

z

Domestically produced medicines Î to be priced as the imported

z

Patent expiry Î Price of original to be reduced by 20% when generic reaches 5% of the market

11/19/2008

The standard reimbursement rate is 75% 90% reimbursement for treatments for chronic conditions (such as osteoporosis, Parkinson’s disease, coronary heart disease, etc.) 100% reimbursement for treatments for severe, debilitating or lifethreatening disease (such as cancer, multiple sclerosis, hormone deficiency, etc.)

– – –

z

Average co-payment rate: 15%

z

Public hospitals dispense medicinal products to the poor at no charge 11/19/2008

43

ISPOR Global Health Care Systems Road Map - Greece

ISPOR Global Health Care Systems Road Map - Greece

The New Reimbursement System Law 3697/2008 Implemented from 1/1/2009 onwards z

44

Conclusions z

Social Insurance Funds will reimburse the Reference Price (excluding co-payment)

z

Reference Price = Retail Price – 3%

z

The difference between the Reference Price and the Retail Price will be incurred by the pharmaceutical industry

11/19/2008

Recent reforms of pricing and reimbursement systems have: – –

Improved access to new medicines Controlled parallel exports

z

Reforms in Greece are implemented very slowly

z

Fragmented regulatory framework

z

No incentives to conduct R&D

z

Economic evaluation: not included in decision making 11/19/2008

45

46

ISPOR Global Health Care Systems Road Map - Poland

Health Care System

Pricing & Reimbursement System Poland

z z

z

ISPOR Global Health Care Systems Road Map z

Insurance-budgetary model Services provided free at the point of service in case of sickness, injury, pregnancy, child-birth and confinement, as well as in prevention of diseases and health promotion The National Health Fund (NHF) – a non-profit body provided with legal personality - collects funds from premiums paid by citizens ( with a total annual budget at the level of 15,3 bil.€ in 2008) NHF manages the funds and contracts providers to render health services

©2008 ISPOR HTA- EBD SIG

11/19/2008

48

8

ISPOR Global Health Care Systems Road Map - Poland

ISPOR Global Health Care Systems Road Map - Poland

Health Care System z

z z z z

z

Reimbursement Approval Process and Time Frame

Majority of resources are allocated for inpatient treatment (hospitals), followed by reimbursement of medications (high, ~20%), outpatient general care and specialized outpatient care GPs are paid on per capita basis Referral to specialists is needed with an exclusion of selected specialists and in case of certain diseases Dentistry coverage from public funds is limited, patients usually pay out-of-pocket In hospitals, where referral is also needed (apart from emergency cases), a DRG system has been recently introduced Private sector is in place with an annual turnover of ~200 mln € and market evolution of 20% per year 11/19/2008

©2008 ISPOR HTA- EBD SIG

z

z z

Registration of medicinal product is granted either by the European Medicines Agency (EMEA) or the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Reimbursement approval process is performed by the Ministry of Health Reimbursement criteria are the following: – – – – – –

49

Necessity to provide health care for the society Making medicines accessible Safety Importance of a drug in a treatment of conditions associated with high epidemiological threat Influence of a drug on direct medical costs Affordability for the public payer obliged to finance healthcare services

ISPOR Global Health Care Systems Road Map - Poland

z

Pricing Approval Process • Prices for non-reimbursed drugs are free

There are 4 levels of reimbursement: 0% (nonreimbursed), 50%, 70% and 100% with price limits within international names groups and therapeutic groups Health Technology Assessment Agency (AOTM) was established as an advisory body to the Ministry of Health –

50

ISPOR Global Health Care Systems Road Map - Poland

Reimbursement Approval Process and Time Frame z

11/19/2008

©2008 ISPOR HTA- EBD SIG

• Price for the product submitted for reimbursement is set through a negotiation with a Drug Management Team of the Ministry of Health • The following criteria are taken into account:

• Production cost (provided by the manufacturer) • Cost of daily treatment • Cost of standardized therapy • Risk-benefit ratio compared to alternative pharmaceuticals for that indication • Therapy costs per day in comparison to products with the same efficacy • Evaluation of the economic impact on the national health system • Estimated sales of the new pharmaceutical product • Prices in countries with similar GDP

HTA reports prepared according to official HTA guidelines

• Pricing & reimbursement decisions are made on the national

level and published in the official journal

11/19/2008

©2008 ISPOR HTA- EBD SIG

51

ISPOR Global Health Care Systems Road Map - Poland

Key Players in the System z

Reference pricing system

z z

- Within the group with the same international name (INN) - Within therapeutic groups based on the same indication, comparable efficacy, the same way of administration and similar adverse effects

z

z z

©2008 ISPOR HTA- EBD SIG

52

ISPOR Global Health Care Systems Road Map - Poland

Pricing Approval Process •

11/19/2008

©2008 ISPOR HTA- EBD SIG

11/19/2008

53

National Health Fund http://www.nfz.gov.pl HTA Agency http://www.aotm.gov.pl Ministry of Health http://www.mz.gov.pl Office for Registration of Medicinal Products, Medical Devices and Biocidal Products http://www.urpl.gov.pl/ Main Pharmaceutical Inspectorate http://www.gif.gov.pl National Institute of Public Health www.gif.gov.pl

©2008 ISPOR HTA- EBD SIG

11/19/2008

54

9

HTA & Good Research Practices for Reimbursement Decisions WG Co-Chairs: Jalpa A. Doshi, PhD, Research Assistant Professor of Medicine, General Internal Medicine University of Pennsylvania Bonnie Handke, RN, MBA, Director, Health Policy and Planning, Medtronic, Inc, Neuromodulation, Shoreview, MN, USA Jennifer Stephens, PharmD, Clinical Director & Partner, Pharmerit North America LLC

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Health Technology Assessment &

Good Research Practices for Reimbursement Decisions WG

Gisselle Gallego, PhD, Research Fellow, Centre for Health Economics Research and Evaluation (CHERE) University of Sydney, Australia Wim Goettsch, PhD, Consultant, Pharmacoeconomics, Dutch Health Insurance Board Seema Haider, MSc, Director, Pfizer Inc, Global HE/OR Franz Hessel, MD, MPH, Senior Manager, Sanofi-Aventis, Managed Markets / HEOR Tove Holm-Larsen, MSc, University of Denmark Deborah Lubeck, PhD, Vice President, Icon Clinical Research John O’Donnell, PhD, Regional Director, Astra-Zeneca Dan Ollendorf, MPH, Chief Review Officer, Institute for Clinical and Economic Review Carlos 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

,

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

z

–

z

Characteristics of Survey Respondents

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

z

– – – –

Type of agency/organization: – – –

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

–

z

–

Australia - 3 Canada - 2 Europe - 17 Latin America - 2 United States - 6

–

Coverage & reimbursement decisions (79%), Clinical guidance (72%) Pricing decisions (35%)

z

HTA funding: 62% receive government funding at some level

z

HTA work performed most commonly by: – – –

11/19/2008

58% HTA only 7% reimbursement only 17% both HTA & reimbursement 17% other (e.g., 3rd party payers)

Role of HTA: –

30 respondents to date with mix of regions/countries –

56

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Overview z

11/19/2008

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

11/19/2008

57

58

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions International Journal of Technology Assessment in Health Care, 24:3 (2008), 244–258. Copyright c_ 2008 Cambridge University Press. Printed in the U.S.A. doi:10.1017/S0266462308080343

Application of Drummond et al Principles to Survey Respondents

Key principles for the improved conduct of health technology assessments for resource allocation decisions Michael F. Drummond

z

University of York

J. Sanford Schwartz University of Pennsylvania

Bengt J ¨onsson Stockholm School of Economics

Bryan R. Luce United BioSource Corporation

Peter J. Neumann Tufts University

Uwe Siebert

Proposed principles to be used for assessment of existing or establishment of new HTA programs –

University of Health Sciences

Sean D. Sullivan University of Washington Health technology assessment (HTA) is a dynamic, rapidly evolving process, embracing different types of assessments that inform real-world decisions about the value (i.e., benefits, risks, and costs) of new and existing technologies. Historically, most HTA agencies have focused on producing high quality assessment reports that can be used by a range of decision makers. However, increasingly organizations are undertaking or commissioning HTAs to inform a particular resource allocation decision, such as listing a drug on a national or local formulary, defining the range of coverage under insurance plans, or issuing mandatory guidance on the use of health technologies in a particular healthcare system. A set of fifteen principles that can be used in assessing existing or establishing new HTA activities is proposed, providing examples from existing HTA programs. The principal focus is on those HTA activities that are linked to, or include, a particular resource allocation decision. In these HTAs, the consideration of both costs and benefits, in an economic evaluation, is critical. It is also important to consider the link between the HTA and the decision that will follow. The principles are organized into four sections: (i) “Structure” of HTA programs; (ii) “Methods” of HTA; (iii) “Processes for Conduct” of HTA; and (iv) “Use of HTAs in Decision Making.”

–

Evaluated adherence to principles across all sections Focus on Methods (principles 5-9)

The authors are members of The International Group for HTA Advancement

11/19/2008

59

11/19/2008

60

10

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Principle 5: HTA should incorporate appropriate methods for assessing costs and benefits

Principle 5: HTA should incorporate appropriate methods for assessing costs and benefits z

Development and consistent implementations of rigorous analytical methods is required to engender stakeholder and public trust in the process and its findings. This requires clarity of HTA process and methods, as well as access to experts with appropriate clinical and multidisciplinary methodological training.

11/19/2008

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Majority of respondents specified methods that are used for assessing cost and benefits Meta-analyses (37.5%) and comparative analyses (34.17%) are the most common for drugs Meta-analyses (45%), comparative analyses (35.83%), and post-marketing surveillance (37.5%) are the most common for medical devices

– –

z

European agencies have very clear national guidelines, while great variation exists in the US

11/19/2008

61

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Methods and Analyses for Evaluation of Cost-Effectiveness Information for Drugs in Europe Methods of Evaluation of CE Country

Trial-based HE analyses

CE models

Retrospective Database Analyses

Cost

CE

√

√

√

√

√

Denmark

√

√

√

√

France

√ (50%)

√ (50%)

√ (50%)

√ (50%)

√

√

Hungary

√

√

Italy

√

Netherlands

√

Portugal

√

Spain

√ (25%)

√ (50%)

Cost consequence

Cost minimization

√

√

Budget impact

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√ (25%)

√

√

√ (50%)

√

√ (50%)

√ (50%)

√ (50%)

Sweden

√

√

√

√ (67%)

√

√ (67%)

√ (33%)

Switzerland

√

√

√

√

√

√

√

United Kingdom

√

HTAs require use of data from experimental, quasiexperimental, observational, and qualitative studies, integrations of both endpoint and validated surrogate data, and assessment of the incremental impact of and trade-offs among multiple clinical, economic and social outcomes in clinically relevant populations.

√ (50%)

√

√ (50%)

Principle 6: HTAs should consider a wide range of evidence and outcomes

Types of Analyses for CE Evaluation

Austria

Germany

√ (25%)

√

√

√

11/19/2008

11/19/2008

63

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

RCTs (for clinical evidence, efficacy and causal inferences) alone are generally not sufficient for the conduct of HTAs

80.00% 70.00%

√

√

√ (50%)

√ (50%)

√ (50%)

√ (50%)

√ (50%)

√

√

√

Hungary

√

√

Italy

√

√

Netherlands

√

√

Portugal

√

√

Spain

√ (50%)

√ (50%)

Sweden

√

Switzerland

√

th er

√ √ (50%)

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√

√ (50%)

√ (50%)

√ (50%)

√ (50%)

√ (25%)

√ (25%)

√

√ (33%)

√

√

√

√ (33%)

√ (67%)

√ (67%)

√

√

√

√

√

√

√

√

√

√ √ (50%)

O

y s

u it

es

Eq

√ (50%)

√

√ (50%)

Other

√ (33%)

en

of

il ln

√

√

rd

im

Burden of Illness

√

dg

et

Equity

√

Bu

fe f- li

li ty -o

pa

ct a

na ly s is

s es s ua

Q

Budget Impact

√

Bu

ty

Co st Co

st

-e f

fe

cti ve n

s

fe

es

Sa

cti ve n

A N/

QoL

√

√ (50%)

0.00% fi c ac y

CE

√

√

10.00%

Ef

Costs

√

Germany

30.00% 20.00%

fe

Safety

France*

South America

40.00%

Effectiveness

√

Denmark

Canada

50.00%

Efficacy

√

USA Australia

Ef

Effectiveness, safety, costs, and cost-effectiveness are the most common attributes evaluated across technologies and evolutionary stages (>40% across technologies and evolutionary stage)

Country Austria

Europe

100.00%

60.00%

z

Primary Attributes Evaluated for New Drugs

Primary attributes evaluated for new drugs

90.00%

64

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Principle 6: HTAs should consider a wide range of evidence and outcomes z

62

* Medical Devices (Drugs NR)

11/19/2008

65

11/19/2008

66

11

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions Principle 7: A full societal perspective should be considered when undertaking HTAs

Principle 7: A full societal perspective should be considered when undertaking HTAs

Perspective used for CE assessment in Europe

HTAs should adopt a broad societal perspective to optimize efficiency and societal benefit and to avoid and identify potentially distorted clinical decisions and health policies resulting from adoption of narrower perspectives used by various healthcare system stakeholders.

11/19/2008

Country

Societal

√

Armed with a broad assessment, individual decision makers can then identify those costs and benefits most important to them.

Austria Denmark

√

z

Cost-effectiveness perspective: – Primarily a societal perspective (~50% across technologies), followed by third party payer (~35% across technologies) – Europe uses a societal perspective (68.63% across technologies) while the US is more oriented to third party payers and other perspectives (22.22% and 33.33%, respectively) Are cost per QALY endpoints mandatory? – Not mandatory (>50% across technologies) – 2 out of 11 European countries reported mandatory cost/QALY

France

√ (50%)

√ (50%)

Germany

√

√

Hungary

√

√

z

Italy

√

Portugal

√

√

Spain

√ (50%)

√ (25%)

Sweden

√

√ (33%)

Switzerland

NR

NR

United Kingdom

Optional Extra (new NICE methods guide)

11/19/2008

68

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions Principle 8: HTAs should explicitly characterize uncertainty surrounding estimates

Principle 8: HTAs should explicitly characterize uncertainty surrounding estimates

Country z

All data are imperfect point estimates of underlying distributions that incorporate a variety of errors. All analytical methods are subject to biases and limitations. Thus, extensive sensitivity analyses are required to determine the robustness of HTA findings and conclusions. The limitations of the analysis should always be acknowledged.

z

All decisions are subject to uncertainty…Thus, extensive one-way and multi-way sensitivity analyses of input variables…and alternative structural representations are required to define the thresholds within which decisions are stable and beyond which decisions may change.

Is estimation of uncertainty (e.g. confidence intervals) around the costeffectiveness estimate mandatory? – Globally, uncertainty is primarily mandatory (50% for Rx and MD, 43.33% for OT) – For European countries, uncertainty is primarily mandatory the percentage is 64%

Uncertainty evaluation mandatory?

Austria Denmark

√

France

√ (50%)

Germany

√

Hungary

√

Italy Netherlands

√

Portugal

√

Spain Sweden

√ (67%)

Switzerland United Kingdom

√

11/19/2008

69

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

70

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions Principle 9: HTAs should consider and address issues of generalizability and transferability

Principle 9: HTAs should consider and address issues of generalizability and transferability

z

Examination of the generalizability and transferability of HTA findings across clinical populations and policy relevant perspectives is required, given the inherent variability of disease, intervention responses, and outcomes across patients, populations, providers, healthcare delivery sites and healthcare systems.

11/19/2008

√

Netherlands

67

Global Survey to Assess Methods Used in HTA & Reimbursement Decisions

11/19/2008

3rd party payer

z

71

Is international data (e.g., from multinational trials) on patient utilities, for instance, accepted in your country or do they require data specific to the country for the following items? – Generally, international patient utilities/QoL are accepted (~50% across technologies) – Resource utilization data is split between international and country-specific (~30% each, across technologies) – Unit cost/price weight data is country-specific (~45% across technologies) – Both Europe and the US follow this trend, though US (16.67%50%) responses were fewer than European responses (52.94%64.71%)

Country

Patient util/QoL data

Resource utilization data

Unit cost/price weight data

Austria

International

Country

Country

Denmark

International

Country

Country

Country (50%)

Country (50%)

France Germany

Country

International

Country

Hungary

Country

Country

Country

Italy

International

International

N/A

Netherlands

International

Country

Country

Portugal

International

Country

Country

Spain

International (50%)

Sweden

International

International

Country

Switzerland

International

International

International

United Kingdom

Country preferred

Country

Country

11/19/2008

72

12