© 2011 main assoc. of austrian social security institutionsicium 2011 page 1 a ssessment, p ricing,...

© 2011 Main Assoc. of Austrian Social Security Institutions ICIUM 2011

ASSESSMENT, PRICING, AND REIMBURSEMENT OF NEW HEALTH

TECHNOLOGIES:

HEALTH TECHNOLOGY ASSESSMENT POLICIES AND PRACTICES IN EUROPE

Anna Bucsics

Department of Pharmaceutical Affairs

Main Association of Austrian Social Insurance Institutions


Disclaimer

The contents presented here reflect my personal opinion.

They are not necessarily identical with those of the Department, the Federation of Austrian Social Security Institutions, its Advisory Committees or its management.


Overview

Health Care Systems in Europe EUNET HTA

Introduction – the Pharmaceutical Forum Joint Action Work Package 5

Access to Medicines Orphan Drugs Biosimilars


HEALTH CARE SYSTEMS IN EUROPE

http://evolution.berkeley.edu/evolibrary/article/analogy_04


Basic Classification of Healthcare Systems

Thomas BURKHARDT, 8-9/11/2011 © EDQM, Council of Europe,

All rights reserved

Bismarck systems

Social health insurance

Contributions/Payroll

Independent administration

Compulsory insurance

Beveridge systems

Nat. health service

Taxes

Administration by government

Inhabitants


The European Picture

Thomas BURKHARDT, 8-9/11/2011 © EDQM, Council of Europe, All rights reserved

Scandinavian

(generous/universal/tax)

Anglo-Saxon

(orientation on need/tax)

Continental

(encompassing

Payroll Contributions/

living standards)

Mediterranean

(insider protection, no safety net,

familialism)

New member states

(mixed Beveridgean and

Bismarckian catch-up)

Taken from: PPRI Report, S.Vogler, 2008

http://ppri.oebig.at/


Country-by-Country Bismarckian (social

health insurance)

AT BE BGCZ

DE EE FRHU

LT LU NLPL

SI SK

Beveridgean (national health service)

DK EL FIIE

IT LV MTPT

SE UK

Thomas BURKHARDT, 8-9/11/2011 © EDQM, Council of Europe, All rights reserved

CY

• Purely privately

organised systems

Ø

Taken from: PPRI Report, S.Vogler, 2008

http://ppri.oebig.at/


ASSESSMENT FOR REIMBURSEMENT

In most countries in Europe, medicines are assessed for reimbursement

Transparency guideline: 90 days (additional 90 days for price negotiations)

Pharmaco-economic evaluations

Capacity for evaluations is limited


Marketing Authorization Evaluation for Reimbursement

Quality What are the available alternatives?

Efficacy Is the new drug better?

Safety Is the price worth the difference?


THE EUROPEAN NETWORK FOR HEALTH TECHNOLOGY

ASSESSMENT

www.eunethta.net


PROLOGUE – THE PHARMACEUTICAL FORUM (2005-2008)

Conclusions:

Decisions on pricing and reimbursement lie with the national competent authorities

But members of the Forum agreed on: The importance of exchanging information The added value of common working definitions and

good practice principles The added value of information on data available,

needs and methodologies used High Level Pharmaceutical Forum

2005 - 2008


RELATIVE EFFECTIVENESS

Agreed working definitions of the Pharmaceutical Forum: Efficacy: is the extent to which an intervention does more good than

harm under ideal circumstances.

Relative efficacy: can be defined as the extent to which an intervention does more good than harm, under ideal circumstances, compared to one or more alternative interventions.

Effectiveness is the extent to which an intervention does more good than harm when provided under the usual circumstances of health care practice.

Relative effectiveness can be defined as the extent to which an intervention does more good than harm compared to one or more intervention alternatives for achieving the desired results when provided under the usual circumstances of health care practice.

High Level Pharmaceutical Forum

2005 - 2008


SURVEY ON THE AVAILABILITY OF DATA TO CONDUCT RELATIVE EFFECTIVENESS ASSESSMENTS

There is no clear consensus as to whether clinical trials yield efficacy or effectiveness information

When “more or better relative effectiveness data” are demanded, the word “relative” refers to trials that have the best possible alternative drug as a comparator. If this is not the case comparisons are indirect and have to be made either through value judgment or by modelling.


2005 - 2008


EUnetHTA Joint Action 2010-12

The overarching objective of the EUnetHTA Joint Action (JA) is to put into practice an effective and sustainable HTA collaboration in Europe that brings added value at the European, national and regional level.

www.eunethta.net


Health technology is the application of scientific knowledge in health care and prevention.

Health technology assessment (HTA) is a multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to inform the formulation of safe, effective, health policies that are patient focused and seek to achieve best value.

Despite its policy goals, HTA must always be firmly rooted in research and the scientific method.

http://www.eunethta.net/Public/About_EUnetHTA/HTA/


EUNETHTA‘S CORE MODEL

A structured manner of creating and presenting HTA information as elements to support European collaboration: Domains – wide framework

Topics – specific area– Issues - questions that can be answered

through answering one or more research questions

www.eunethta.net


DOMAINS OF THE CORE MODEL

1. Health Problem and Current Use of the Technology

2. Description and technical characteristics of technology

3. Description and technical characteristics of the technology

4. Safety

5. Clinical Effectiveness

6. Costs and economic evaluation

7. Ethical analysis

8. Organisational aspects

9. Social aspects

10. Legal aspects

www.eunethta.net


WP 5 – RELATIVE EFFECTIVENESS ASSESSMENT OF PHARMACEUTICALS

Objective: Development of HTA tools and methods for

assessing the Relative Effectiveness of Pharmaceuticals, based on the Core Model

Application and field testing of developed tools and methods

Deliverables: Background Review Guidelines on Methodology “test run” Publication of Results

Lead Partner: CVZ, NetherlandsCo-Lead Partner: HAS, France

www.eunethta.net


PHARMACOLOGICAL/TECHNICAL CONSIDERATRIONS

Mechanism of Action, Pharmacodynamics

Pharmacokinetics

Technical Data, e.g. Number of Puffs for Inhalation? Scored Tablet? Drops/ml? Usability? Expiry Date

© 2011 Main Assoc. of Austrian Social Security Institutions ICIUM 2011 20

WHICH COMPARATORS SHOULD BE USED?

Pharmacologically most similar product? Standard treatment? Most commonly used treatment? Most expensive/cheapest treatment? Usual treatment, if there is no standard

treatment with proven effectiveness?

Which dose?

© 2011 Main Assoc. of Austrian Social Security Institutions ICIUM 2011 21

PROBLEMS ASSESSING RELATIVE EFFECTIVENESS

Unanswered questions

Indirect comparisons

Inefficient mechanisms for remedying decisions made on the basis of such unreliable data

Intransparency


INNOVATION AND OTHER ADVANTAGES

Innovation per se must not be confused with effectiveness

Innovation may lead to a drug which has other benefits: – greater applicability– easier to use– more appropriate

which may contribute to greater relative effectiveness

… and others which don’t:– greater ease of production, storage, handling – less environmental impact


ACCESS TO MEDICINES IN EUROPE1. Mechanism of coordinated access to orphan

medicinal products

2. Capacity building on managed entry agreements for innovative medicines

3. Facilitating the supply in small markets

4. Promoting a good governance for non- prescription drugs

5. Market access for biosimilars

The Directorate General for Enterprise and Industryhttp://ec.europa.eu/enterprise/sectors/healthcare/competitiveness/process_on_corporate_responsibility/platform_access/index_en.htm


MECHANISM OF COORDINATED ACCESS TO ORPHAN MEDICINAL PRODUCTS – PROBLEM STATEMENT

Prices of Orphan Medicines are extremely high

They are creating concerns about affordability and solidarity

Average Price per Package excl. VAT of all Drugs in Austria in

2009

Average Price per Package excl. VAT of Orphan Drugs in Austria

in 2009

€ 22,00 € 2.732,51

Neurologists and paediatricians call for action on “massive” rises in the prices of orphan drugs

Open letter to prime minister David Cameron and health secretary Andrew Lansley; BMJ 2010; 341:c6466

EU legislation offers an exclusive marketing period as an incentive for companies to develop drugs for rare diseases. But pricing for

orphan drugs hinders access and may warrant a competition law investigation

Jonathan C P Roos, Hanna I Hyry, Timothy M Cox, BMJ 2010; 341:c6471


MARKET ACCESS FOR BIOSIMILARS

Definition:

A biosimilar medicinal product is a medicinal product which is similar to a biological medicinal product that has already been authorised (the ‘biological reference medicinal product’). The active substance of a biosimilar medicinal product is similar to the one of the biological reference medicinal product.

The name, appearance and packaging of a biosimilar medicinal product may differ to those of the biological reference medicinal product. It may also contain different inactive ingredients1.

The upcoming emergence of biosimilars may create new market dynamics.

The objective of this project will be to define what the necessary conditions within the pharmaceutical environment are to ensure informed adequate uptake of biosimilars2.

1 http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/12/WC500099907.pdf

2 http://ec.europa.eu/enterprise/sectors/healthcare/competitiveness/process_on_corporate_responsibility/platform_access/index_en.htm


Thank You!


Additional Material


Work Packages of JA 2010-2012:

1. Coordination

2. Dissemination

3. Evaluation

4. Core HTA

5. Relative Effectiveness Assessment of Pharmaceuticals

6. Information Management System

7. New Technologies

8. Strategy and Business Model Developmentwww.eunethta.net


PREAMBLE TO THE GOOD PRINCIPLES OF RELATIVE EFFECTIVENESS ASSESSMENTS The aim of relative effectiveness (here-after RE) assessment is to compare healthcare

interventions in practice in order to classify them according to their practical therapeutic value(1). Differences between the objectives and priorities of different national healthcare systems may create differences in the way in which healthcare interventions will be valued relative to one another. In the EU context, this means that a relative effectiveness assessment is most likely to be meaningful at the national (local healthcare) level. However there is considerable(2) value of stimulating exchange of information, methodologies and experiences between the relevant national authorities.

The first step in assessing relative effectiveness is an assessment of relative efficacy. Working definitions of these terms have been developed in the course of the full Working Group’s deliberations (see here-after).

While the rules and processes within a given healthcare system should be established by discussion among the local stakeholders concerned, some classical principles of public administration are likely to be generally relevant. The working group suggest the following principles for the Pharmaceutical Forum to endorse, for non-binding use as appropriate in Member States.

(1) EFPIA quotes: "The aim of RE assessment is to compare healthcare interventions in practice in order to determine their practical therapeutic value".

(2) EFPIA suggests deleting the adjective "considerable"

29


2005 - 2008


1. PRINCIPLE OF RELATIVE EFFECTIVENESS ASSESSMENTS

Individual Member States may use RE assessments for different purposes. Decisions on the detailed operation of RE assessments, including methods and relevant stakeholders(3), are most appropriately made at a national level.

(3) Relevant stakeholders include patients and health

professional organisations, the pharmaceutical industry and social insurers. 8. RE assessment should include comparison with the most appropriate healthcare


2005 - 2008



RE assessment processes, selection of products to be assessed, working methodologies and quality assurance processes should be transparent to all parties and evidence-based.

21.1.2010 Diemen International Course on HTA 31


2005 - 2008


Relevant stakeholders should be able to contribute to the development of assessment methodologies. The purpose of RE assessment and the organisation(s) responsible for its conduct should be clearly identified.



2005 - 2008



RE assessment processes should remain separate from product market authorisation procedures (though this does not mean that they are necessarily performed by different organisations).



2005 - 2008




RE assessment processes should be time-framed, and should minimise or avoid causing unnecessary procedural delays consistent with any associated Transparency Directive requirements where applicable.



2005 - 2008



RE assessments should be capable of addressing transparently uncertainty in the evidence base, and the methodological challenge of translating evidence on relative efficacy and other appropriate available data into conclusions on relative effectiveness.



2005 - 2008



The sources of evidence which are to form the relevant RE input should be specifically discussed among the identified key stakeholders, who should each be able to submit evidence or argumentation for appraisal.



2005 - 2008



RE assessment should include comparison with the most appropriate healthcare interventions. Such comparison should build on the results of active controlled clinical trials, where available.



2005 - 2008



When concluded, outcomes should be communicated in a clear and timely manner to all interested parties. Communication by means of publishing the supporting evaluation on a publicly accessible website is strongly encouraged.



2005 - 2008



RE assessments should be capable of subsequent revision and updating as the evidence base develops.



2005 - 2008



RE assessments should aim to identify areas in which the evidence base on an intervention could most usefully be developed in the future.



2005 - 2008


CHECKLIST FOR USE OF THE PRINCIPLES

The purpose of the checklist is to provide Member States with a framework for evaluating their relative effectiveness assessment as well as their national relative effectiveness assessment systems, and especially how they fit in with the core principles agreed by the Member States.



2005 - 2008

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