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  • Zoltn KalProfessor of Health Economics

    ISPOR 18th Annual European Congress, Milan10 November 2015


  • 7-11 November 2015

    Milano Congressi, Milan, Italy

    Congress featured 2,450 presentations

    Over 5220 attendees from 90 countries

  • Hegazy A, George M, Kalo Z, Abaza S, Abbas YM, Atef M. Challenges Along the Road to Universal Health Coverage in Egypt, an HIO Perspective. Value Health. 2015 Nov;18(7):A567-8.

    Ragab S, Diaa M, Hassan R, Abou Shady R, El-Sisi G, Kalo Z, Abaza S, Shaheen M, Abbas Y. Pricing Policy for Pharmaceuticals in Egypt: Challenges and Opportunities. Value Health. 2015 Nov;18(7):A523.

  • Payers, health technology assessment (HTA) and regulatory leaders, patient and industry representatives, and key decision makers discussed the New Medical Device Regulation in Europe as well as the work underway within the Adaptive Pathways to Patients initiative for drugs.

    The panel presented candid views on adaptive pathways as the preferred approach to developing, licensing, assessing, appraising, and paying for new medicines and treatments.

    This session also examined the European Commission's HTA network plans following the transition of the successful EUnetHTA program, which built strength and dialogue both across Europe and globally.

  • In the last four decades, the assessment of outcomes has been moving from the mere ground of research into daily practice.

    The session provided researchers and policy makers with an update on current practices, challenges, opportunities, and future perspectives on the assessment of outcomes in different fields of health care:

    reimbursement of drugs and devices,

    evaluation of public health interventions,

    validation of new technologies

    financing of complex health services.

  • efficacy

    effectiveness (adherence and persistence)

    relative effectiveness to competitor technologies

    risks (e.g. rare serious adverse events)

    surrogate outcomes hard endpoints


    number of patients (eligible patients + market penetration)

    risk of off-label use (i.e. inappropriate targeting; longer therapy; etc.)

  • efficacy

    outcome guarantee


    Manufacturer-funded treatment initiation probation period

    conditional treatment continuation

    relative effectiveness

    coverage only with research - patient registry funded by manufacturers

    rare serious adverse events

    coverage only with research - patient registry funded by manufacturer

  • surrogate outcomes

    coverage only in research


    revision of previous policy decisions (coverage only with research)

    number of patients (eligible patients + market penetration)

    mandatory budget impact analysis

    price-volume agreement

    risk of off-label use (i.e. inappropriate targeting; longer therapy; etc.)

    financial risk-sharing based on market share

    utilization cap

  • A Managed Entry Agreement is an arrangement between a manufacturer and payer/provider that enables access to (coverage/ reimbursement of) a health technology subject to specified conditions.

    These arrangements can use a variety of mechanisms to address uncertainty about the performance of technologies or to manage the adoption of technologies in order to maximize effective their use, or limit

    their budget impact.1

    1 Klemp M. et al. 2011 What principles should govern the use of managed entry agreements?2 Simon-Kucher & Partners Healthcare Insights Winter 2014 - Volume 7, Issue 3

  • Source: UW PBRSA Database

  • Total Schemes Implemented 68

    Active 53

    Revised 9










    1997 1999 2001 2003 2005 2007 2009 2011 2013 2015


    By Year Cummulative Garrison et al., ISPOR, 2015

  • Managed enty agreement forces cinicians to focus on patient outcomes

  • 29

    Source: International experience with innovative price scheme Specific case studies - M. Marchetti November 25th, 2013

    Note: Setting of the price will be in line with applicable laws and regulations

  • Italian reimbursement landscape

    AIFA Registry


    The AIFA Registry was set up on

    December 2005 and published on

    line on April 2006

    The registration of the patients in

    the AIFA Registry is mandatory to

    get the reimbursement of the drug

    by NHS

    The first drug with MEA included in

    the Registry was Tarceva (erlotinib)

    Monitoring appropriate use of drugs according to approved therapeutic


    Purpose of the Registry

    Assessing and tracking patient eligibility

    Evaluating utilisation in clinical practice

    1 2 3

    Collecting epidemiological data including safety profile


  • 31










    Oncology indications

    Oncology indications with MEAs

    Oncology indications without MEAs

    Off-label indicationsby law 648/96

    Most drugs are

    monitored within

    multiple indications

    Half of the

    indications account

    for Oncology

    Registries are also

    used to monitor

    appropriate usage

    Note: The AIFA Registry doesn't allow the off label use of a drug (with the exception of the indications approved by AIFA for an off label use accordingly to

    the Law 648/96). The physicians can prescribe the drug for an off label indication without reimbursement by NHS after receiving the approval from the

    hospital and the informed consent from the patient. NO DATA AVAILABLE TO COMPARE THE APPROPRIATE USE OF DRUG BEFORE AND FATER


    SOURCE: adapted from: Lista aggiornata dei nuovi accessed October 2nd 2014

  • "you cannot manage ... what you do not measure"

    "in God we trust, all others must show data"

  • Multi-criteria decision analysis (MCDA) is an emerging new practice using a broad set of methodological approaches to assist in decision making, especially in an era of expensive but valuable technologies trading multiple criteria.

    The ISPOR MCDA Task Force Report discussed different approaches for conducting MCDA.

    Panelists presented emerging good practice recommendations presented in the Task Force report and identified remaining areas of controversy.

  • Belton and Stewart

    An umbrella term to describe a collection of formal approaches which seek to take explicit account of multiple criteria in helping individuals or groups explore decisions that matter

    Keeney and Raiffa

    An extension of decision theory that covers any decision with multiple objectives. A methodology for appraising options on individual, often conflicting criteria, and combining them into one overall appraisal


  • MCDA: comprehensive approach to improve the evidence base and transparency of policy decisions related to health technologies

    Budget impact is only one aspect which should be taken into account in decision making

    MCDA implementation helps

    policymakers objective and verifiable criteria for policy decisions

    society and patients less resources are sacrificed for inapproriate health care services and technologies

    health care manufacturers clear criteria for market access

  • IQWiG: 2 types of MCDA can

    contribute to determining the

    most important outcomes for

    patients as part of economic


    British Columbia:

    The HTA Committee

    uses MCDA to

    assess non-drug

    health technologies

    EMA: MCDA is

    valuable, providing

    clarity, particularly

    where the benefit-

    risk balance is


    Thailand: MCDA used

    to inform coverage

    decisions for HIV/ AIDS


    Hungary: MCDA has

    been used to evaluate

    new hospital medical

    technologies since


    Italy: Lombardy introduced

    MCDA in 2008 to decide on

    the introduction and delisting

    of health technologies

  • Step Description

    Decision problemProblem structuring to establish the decision problem i.e. identify

    objectives, alternatives and decision makers

    Identify criteria Identify value criteria relevant to the decision problem

    Measure performance Gather evidence on the performance of the alternatives on the criteria

    Performance scoringConvert performance measures into scores that describe the desirability

    of achieving different levels of performance for each criterion

    Weight criteriaElicit the opinions of the stakeholders on the relative importance of

    different criteria or their preferences for criteria.

    AggregationCombine or aggregate criteria scores and weights to estimate the

    overall value of an option

    Supporting decision

    makingUse the outputs from the MCDA exercise to support decision making

  • Objective


    Measure performance





    How these are done differentiates the MCDA methods


  • Selection of criteria

    Weighting of each criterion

    Scaling of each criterion

    One-off or reusable model

    Rule vs. Tool

    Questions regarding development of MCDA

    Application of MCDA

  • Criteria Definition



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