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CONTRIBUTED WORKSHOPS - SESSION I
Sunday, November 6, 15:30-16:30
CLINICAL STUDY METHODOLOGY ISSUES
W1: EXPOSURE PROPENSITY SCORES: CONCEPTS, APPLICATIONS,
AND PRACTICAL CONSIDERATIONS
DISCUSSION LEADERS: Almut G Winterstein PhD, Assistant
Professor, Pharmacy Health Care Administration,
University of Florida, Gainesville, FL, USA; Abraham G
Hartzema MSPH, PhD, Eminent Scholar, Dpt. of Pharmacy
Healthcare Administration, University of Florida,
Gainesville, FL,USA; Tobias Gerhard BS, Pharmacy Health Care Administration, University of Florida, Gainesville, FL, USA
Workshop Purpose: The purpose of this workshop is to
understand, evaluate, and conduct analyses of
observational data using Exposure Propensity Score (EPS)
based methods. Participants will learn how to choose
between EPS-based and conventional regression methods
depending on study objective and data structure.
Workshop Description: EPS-based methods have been
frequently suggested to control for confounding by
indication in observational studies. It has been shown
that EPS-based methods are more powerful and robust than
logistic regression when there are less than 8 events
per confounder. We will familiarize participants with
the concept of EPS (conditional probability of exposure
given measured covariates), demonstrate how to calculate
the EPS in SAS and SPSS, and introduce standard
applications of EPS, specifically matching,
stratification and multivariate adjustment.
Additionally, we will discuss more advanced issues, such
as selection of variables for the EPS, model
construction, and choice of analysis method (both
between different EPS-based methods and between
EPS-based and conventional regression methods). We will
conclude with a discussion on limitations of EPS-based
methods regarding clinical utility when magnitude and/or
direction of the treatment effect are highly variable
between subgroups (strata) of the study population. A
clinical dataset from a recently completed study will be
used to practically demonstrate the calculation and
application of EPS in a real life dataset. Results from
different analysis approaches of this dataset will be
available and used to discuss the impact of method
selection on results given data structure and study
objectives.
ADHERENCE/COMPLIANCE ISSUES
W2: METHODS FOR MEDICATION COMPLIANCE STUDIES: PATIENT
COMPLIANCE AS A PREDICTOR OF CLINICAL AND ECONOMIC
OUTCOMES
DISCUSSION LEADERS: Michael Nichol PhD, Associate
Professor & Chair, University of Southern California,
Dept of Pharm Econ & Policy, Los Angeles, CA, USA;
Femida Gwadry-Sridhar PharmD, PhD, Researcher, McMaster
University, London, ON, Canada; JB Benner PharmD, ScD,
Principal, ValueMedics Research, Falls Church, VA, USA
Workshop Purpose: This workshop will 1) review the
methodological issues that must be considered when using
patient compliance measures as independent or predictor
variables of clinical and economic outcomes; 2) discuss
appropriate interpretation of such studies; and 3)
discuss implications for future research in this field.
Workshop Description: Compliance with medications has
become an increasingly important area of research as
decision makers have recognized the extent of
noncompliance and questioned the effect of this problem
on important patient outcomes. In an increasing number
of studies, researchers have used patient compliance
data as an explanatory or independent variable. This
workshop will identify research design, analysis, and
interpretation issues associated with the use of various
measures of compliance as independent variables. The
faculty will illustrate common pitfalls, recommended
methods and interpretations of prospective and
retrospective study findings, and highlight several key
areas for future research. Examples will be drawn from
published and ongoing projects measuring patient
compliance and outcomes in a variety of medications and
diseases. Workshop participants will be encouraged to
offer their perspectives and methodological
recommendations regarding future studies of medication
compliance as a predictor of clinical and economic
outcomes.
COST STUDY METHODOLOGY ISSUES
W3: METHODS FOR PRESENTING PROBABILISTIC SENSITIVITY
ANALYSES FOR EFFECTIVE COMMUNICATION OF FINDINGS
DISCUSSION LEADERS: Douglas CA. Taylor MBA, Associate
Director, Health Economics & Outcomes Research, Innovus
Research (U.S.) Inc, Medford, MA, USA; Lisa J. McGarry
MPH, Associate Director, Health Economics & Outcomes
Research, Innovus Research (U.S.) Inc, Medford, MA, USA;
David Thompson PhD, Managing Director, U.S. Operations,
Health Economics & Outcomes Research, Innovus Research
(U.S.) Inc, Medford, MA, USA
Workshop Purpose: To share tips and techniques for
effectively communicating results of probabilistic
sensitivity analyses (PSA) in pharmacoeconomic models.
Workshop should be of interest to applied researchers
involved in the construction and estimation of
pharmacoeconomic models and managers responsible for the
critical appraisal of such models.
Workshop Description: Pharmacoeconomic models
increasingly are used to help decision-makers assess the
implications of adopting therapies into clinical
practice. Use of PSA to test the robustness of results
with regard to uncertainty in input parameters has
become de rigueur. PSA presented in a visually appealing
and intuitive fashion can greatly enhance the
credibility of model results; however, communicating
results of PSA can be challenging, both conceptually and
technically. Common methods for displaying PSA results
include confidence ellipses, cost-effectiveness scatter
plots, cost-effectiveness acceptability curves, and
confidence interval plots. Microsoft Excel has built-in
tools such as statistical functions, charting tools, and
Visual Basic for Applications (VBA) that can be used to
implement PSA and create visual displays in a fairly
straightforward manner. In this workshop we will discuss
the theoretical and practical advantages and
disadvantages of common techniques for communicating PSA
results to decision-makers and explore alternative
methods of communicating uncertainty in model findings,
including analyses of incremental net health benefit and
methods incorporating alternative analytic time-frames.
In addition, the workshop will familiarize participants
with less commonly-used features of Excel, including the
Excel macro recorder, through which VBA can be accessed
quickly and easily by users with no programming
experience and without the need for external add-in
tools (e.g., Crystal Ball®). In true workshop fashion,
participants will be asked to share their experiences in
presenting and interpreting results of PSA, and a “hands
on” illustration of a working model will be distributed
and discussed. Participants should have a working
knowledge of Excel formulas and charting.
HEALTH CARE COVERAGE AND REIMBURSEMENT ISSUES
W4: CASE STUDIES IN PERSONALIZED MEDICINE: VALUE
CREATION AND REWARDS--INCENTIVES AND IMPLICATIONS
DISCUSSION LEADERS: Louis Garrison PhD, Professor,
Department of Pharmacy, University of Washington,
Seattle, WA, USA; Adrian Towse MA, Director, Office of
Health Economics, London, UK; David L Veenstra MD,
University of Washington, Department of Pharmacy,
Seattle, WA, USA
Workshop Purpose: The purpose of this workshop is to
introduce participants to a value-based approach to
analyzing the impact of innovative
diagnostic-therapeutic combinations and to promote a
discussion of the issues this raises for both economic
evaluation and incentives for innovation
Workshop Description: Personalized medicine is the
latest buzzphrase for the idea of combining a diagnostic
with a pharmaceutical to target a more responsive
subgroup of patients, particularly based on their
genetic information. To date, there are only a limited
number of successful examples. Using three case studies,
this workshop will illustrate the economic and policy
challenges facing this type of linked drug-device
innovation. A standard cost-utility framework provides a
useful starting point for thinking about how the value
is created by a targeted treatment and how value is
redistributed among stakeholders. The three case studies
are: pegylated interferons for hepatitis C, the new
genetic test for predicting breast cancer recurrence,
and EGFR inhibitors for non-small cell lung cancer. Each
case will (1) assess the economic value created (in
terms of reductions in costs, morbidity, mortality, or
uncertainty) and (2) identify who captures what
share—the diagnostic or therapeutic manufacturer, the
payer, the patient, or the provider? This will
illustrate how economic incentives to develop innovative
targeted therapies are driven by several inter-related
factors: (1) reimbursement and pricing rules, (2)
regulatory constraints and costs, and (3) intellectual
property rights. From a policy perspective, the
implications for pricing and reimbursement will be
explored, and in particular the need for more
value-based, flexible pricing—for both diagnostics and
pharmaceuticals—if personalized medicine is to realize
its promise. Following brief presentations of the three
case studies, the last quarter of the session will be
devoted to an interactive discussion with participants
of the important economic evaluation and policy issues.
HEALTH CARE POLICY DEVELOPMENT ISSUES
W5: MULTI-NATIONAL ASSESSMENT OF OUTCOMES VIA
RETROSPECTIVE DATABASES
DISCUSSION LEADERS:
Vittorio Maio PharmD, MSPH, Assistant Professor,
Department of Health Policy, Jefferson Medical College,
Thomas Jefferson University, Philadelphia, PA, USA;
Elaine J. Yuen PhD, MBA, Research Assistant Professor,
Center for Research in Medical Education and Health Care,
Jefferson Medical College, Thomas Jefferson University,
Philadelphia, PA, USA; Diana Brixner PhD, Associate
Professor, Pharmacotherapy Outcomes Research Center,
University of Utah College of Pharmacy, Salt Lake City,
UT, USA; Gary M Oderda PharmD, MPH, Professor,
Pharmacotherapy Outcomes Research Center, University of
Utah College of Pharmacy, Salt Lake City, UT, USA;
Carl Asche MBA, MSc, PhD, Associate Professor,
University of Utah, Salt Lake City, UT, USA; Kenneth D.
Smith, PhD, Research Assistant Professor, Department
of Health Policy, Jefferson Medical College, Thomas
Jefferson University, Philadelphia, PA, USA; Steve
Morgan, PhD, Assistant Professor, Centre for Health
Services & Policy Research, University of British
Columbia, Vancouver, BC, Canada
Workshop Purpose: The goal is to assess the challenges
of conducting multi-national retrospective outcomes
research through the presentation of datasets from
Canada, Italy and the United States. We will discuss the
potential application of such database research towards
the assessment of global health policy issues.
Workshop Description: The technology and sophistication
of health care utilization databases have expanded over
the last decade to include results of lab tests, vital
signs, and other clinical information. The richness and
depth of information on “real world” use of health
services for large population-based patient cohorts at
relatively low cost render such databases an attractive
resource for retrospective outcomes research. These data
may be used to explore cross-national variation in
health care provision and assess the impact of variation
on clinical and economic outcomes. However, the extent
to which individual countries have access to this data
and, therefore, the potential to evaluate outcomes vary.
In addition, little is known about the extent to which
information in these databases may be combined to
conduct outcomes research on healthcare-related issues
of global significance.
In this workshop, three databases from Canada, Italy and
the United States will be described including
reimbursement claims, patient records and electronic
medical records. The similarities and differences of
these databases will be shown with examples of
presenters' research studies. Using an ongoing case
study assessing the adherence to Antihypertensive and
Lipid-Lowering Treatment to Prevent Heart Attack Trial
(ALLHAT) guidelines before and after their publication,
methodological and analytical challenges to conduct
multi-national retrospective outcomes evaluations as
well as strategies and techniques to overcome them will
be illustrated. Workshop participants will be asked to
1) assess available data in their respective countries;
2) reflect how this data could answer cross-national
policy related questions; and 3) share information and
ideas for collaboration with presenters' research
agenda.
W6: A NEW APPROACH TO HTA - A COMPUTERIZED MODEL
DISCUSSION LEADERS: Ifat Abadi-Korek PhD,
Pharmacoeconomics Researcher, Israeli Center for
Technology Assessment in Health Care (ICTAHC), The
Gertner Institute for Epidemiology & Health Policy
research, Ramat Gan, Israel; Orly Tamir MSc, Health
Technology Assessment & Research, Israeli Center for
Technology Assessment in Health Care (ICTAHC), The
Gertner Institute for Epidemiology & Health Policy
research, Ramat Gan, Israel; Ishay Ostfeld MD,
Israeli Center for Technology Assessment in Health Care (ICTAHC),
The Gertner Institute for Epidemiology & Health Policy
research, Ramat Gan, Israel
Workshop Purpose: The objectives of HTA vary between
different interested parties. Since the rate of new
technologies is increasing rapidly and HTA become more
complicated and time consuming, we suggest a new elegant
computerized technology scoring model. The model would
be based on general objective parameters and result in
weight score for the technology. We would like to confer
with professionals in the field the procedure by which
appropriate weight for each variable should be
determined
Workshop Description: Participants will be introduced to
an innovative approach to HTA that simplifies and
accelerates the HTA process by assigning a weighted
score to any health technology. A discussion will ensue
that will aim to determine the next steps in arriving at
appropriate weights. HTA is a multidisciplinary activity
with aims that may vary according to the different
interests and needs of various parties. Therefore, we
would like to propose an innovative model, which could
provide the wide spectrum of HTA doers with core,
objective information to begin their decision process.
By using objective parameters and transforming the
assessment results into a weighted score, this model
would be independent of end-point goals. The suggested
model consists of a computerized HTA protocol that
includes 8 major categories related to any technology:
classification, categorization, purpose, target
population, major added value, maturity, implementation,
and costs. The overall score of the technology would
represent the weighted categories scoring, based on
consensus and norms. Defining the objective variables of
this model is of minor concern since they are derived
from the common characteristics of all HTA and the
technical characteristics of the technology. However,
the weight to be assigned to each variable is not
obvious, as the process of achieving consensus is
subject to disagreement and criticism. Because
determining the appropriate weights of the variables is
of superior importance, and due to the challenge it
imposes, we believe that professionals from the field
should confront the issue in order to determine the
right process for their establishment. Since the rate of
new and more complicated health technologies is
increasing rapidly and the HTA is becoming more time
consuming, a new elegant and quicker approach is
suggested in order for HTA to be relevant.
PREFERENCE-BASED STUDIES
W7: DISCRETE CHOICE EXPERIMENTS IN HEALTH POLICY AND
DECISION MAKING
DISCUSSION LEADERS: Mandy Ryan MSc, PhD, Professor of
Health Economics, Health Economics Research Unit,
Aberdeen University, University Medical
Building,Aberdeen, UK; Mark Sculpher PhD, Professor of
Health Economics, Centre for Health Economics,
University of York, Heslington, York, UK; Lorenzo G
Mantovani EconD, MSc, DSc, Head of Research, Center of
Pharmacoeconomics, University of Milan, Milan, Italy
Workshop Purpose: The aim of this workshop is to
illustrate discrete choice experiments (DCE) as a
technique for evaluating health care technologies from a
patient's perspective, and to understand the rationale
and implications of using this method in health care
decision-making processes.
Workshop Description: DCE are an attribute-based measure
of benefit that are based on two assumptions: firstly,
health care interventions, services or policies can be
described by their characteristics or attributes and,
secondly, an individual's valuation depends on the
levels of these characteristics. DCE were introduced
into health economics as a technique to go beyond the
traditional quality-adjusted life-year (QALY) paradigm.
Users were concerned with many aspects of health care
beyond health outcomes. Such factors included waiting
time, location of treatment, type of care (for example
surgical or medical), and staff providing care
(consultant or specialist nurse) and were referred to as
process attributes. Discrete choice experiments allow
investigation of the trade-offs between such process and
health outcomes attributes. In this workshop some recent
experiences of the use of this technique will be
presented and its strengths and limits will be
discussed. Participants will be asked to complete and
comment on a DCE questionnaire. The key point for the
discussion will be the implications of patient-centred
care for decision-making agencies such as the National
Institute for Health and Clinical Excellence (NICE) in
the UK.
QOL/PRO METHODOLOGY ISSUES
W8: WHAT REALLY ARE PATIENT-REPORTED OUTCOMES?
DISCUSSION LEADERS: Judith Barr MEd, ScD, Associate
Professsor and Director, Northeastern University,
National Education and Research Center for Outcomes
Assessment, Boston, MA, USA; Laurie Burke RPh, MPH,
Director, Study Endpoints and Label Development, Food
and Drug Administration, Rockville, MD, US; Pennifer
Erickson PhD, Co-founder, O.L.G.A, State College, PA,
USA
Workshop Purpose: Following a presentation providing
a conceptual foundation of characteristics and types of
patient-reported outcomes (PROs), workshop participants
will apply these principles to determine whether outcome
measures used in selected May 2005 ISPOR posters and
other sources really are patient-reported outcomes.
Workshop Description: The incorporation of PROs into
pharmacoeconomic research is an important step in the
evolution of this field of study. However, there is a
need to clarify that not all Quality of Life (QoL)
outcome studies are PROs, and conversely, not all PROs
studies incorporate QoL measures. In this workshop, we
will present a conceptual overview of patient-reported
outcome measures based on the 1995 Wilson/Cleary causal
chain model linking clinical, symptom, functional
status, health perceptions, and quality of life
indicators of health and disease. We will expand the
Wilson/Cleary model to include the dimension of “source
of measurement”, suggest examples of PRO and non-PRO
outcome measures for each of these categories, and
contrast and compare the information content and
perspective of such measures. We will further develop
concepts and definitions of QoL and Health-related
Quality of Life (HRQL) to include general and
disease-specific HRQL measures as well as methods of
utility assessment. Additional PRO measures such as
patient satisfaction with treatment and
adherence/compliance will be linked to our expansion of
the Wilson/Cleary model. The place of clinician and
proxy measures in our model also will be examined. In
the last third of the workshop, participants will work
in small groups to examine cases based on
abstracts/posters from ISPOR and other sources,
determine whether or not the measures used really are
patient-reported outcomes, and identify the measurement
category within our expanded outcomes measurement model
for each study's outcomes. We will conclude the workshop
with a discussion of issues raised in these cases.
CONTRIBUTED WORKSHOPS - SESSION II
Monday, November 7, 10:45-11:45
CLINICAL STUDY METHODOLOGY ISSUES
W9: META-ANALYSIS OF LONGITUDINAL DATA FOR ECONOMIC
MODELS: AN APPLICATION OF MULTI-OUTCOME MODELS
DISCUSSION LEADERS: Khajak J Ishak MSc, Senior
Statistician, Caro Research Institute, Montreal, QC,
Canada; Robert W. Platt PhD, Associate Professor,
Department of Pediatrics & Epidemiology and
Biostatistics McGill University, Montreal, QC, Canada;
J. Jaime Caro MD, President & Scientific Director, Caro
Research Institute, Concord, MA, USA
Workshop Purpose: The objectives of this workshop are:
1) to illustrate the need and use of multi-outcome
models for the meta-analysis of longitudinal studies
where the effect has been measured at various times; 2)
to show how these models can be used to provide
equations for economic models.
Workshop Description: For economic models, a frequent
requirement is projection of the effects of disease and
treatment beyond the period of known data. A good source
for such projections are longitudinal studies where the
effects were measured at various times. Meta-analyses of
such studies commonly analyze each time point
separately. This does not allow for proper understanding
of the time-dependence of the effects. Pooling data from
all time points will yield biased results since it
ignores correlations between observations. A joint
analysis that overcomes these limitations can be
performed using special multi-outcome models that
properly incorporate the correlated structure of the
data and allow the inclusion of patient and study
characteristics that may explain between-study
heterogeneity. We will describe how to formulate and
estimate the parameters of these models with standard
statistical software and how to translate these to
equations that can be used to make projections in
economic models. Data from a meta-analysis of the effect
of deep brain stimulation (DBS) will be used to
illustrate the methods. The meta-analysis examined the
effect of DBS on the motor skills of patients with
Parkinson's disease, measured at 3, 6, 12 months or
later. Findings from the standard analyses will be
contrasted to those from the multi-outcome approach to
highlight the advantages of the latter. The broader
application of these techniques to economic models will
also be discussed with other examples.
ADHERENCE/COMPLIANCE ISSUES
W10: INCORPORATING MEASURES OF COMPLIANCE AND
PERSISTENCE IN PHARMACOECONOMIC EVALUATIONS
DISCUSSION LEADERS: Dyfrig Hughes PhD, Senior Research
Fellow in Pharmacoeconomics, Centre for the Economics of
Health, University of Wales, Bangor, UK; Warren Cowell
MSc, Health Economist, Roche Products Ltd, Welwyn Garden
City, Hertfordshire, UK; Tamas Andras Koncz MD, OR
Manager, Outcomes Research and Evidence Based Medicine,
Pfizer Ltd, Tadworth, UK
Workshop Purpose: Participants will learn about: 1) the
importance of differentiating between efficacy and
effectiveness; 2) current evidence on the health
economic impact of partial compliance and
non-persistence; 3) appropriate modelling strategies to
consider partial compliance and non-persistence in
pharmacoeconomic evaluations.
Workshop Description: Partial compliance (day-to-day
variation in dosing), and non-persistence (early
discontinuation of treatments) often lead to increased
resource utilisation due to reduction in effectiveness
and associated increase in the risk of therapeutic
failure. Despite this, economic evaluations rarely
incorporate compliance or persistence data to allow for
the differences observed in controlled clinical trials
and in routine clinical practice. The workshop will
first review current evidence for differences between
efficacy and effectiveness, in order to assess how
compliance and persistence are significant contributory
factors. Second, we will appraise the economic impact of
medication compliance and persistence by examining their
impact on the cost-effectiveness of pharmaceuticals.
Third, we will explore the scope and methods for
incorporating medication compliance and persistence data
into cost-effectiveness modelling by categorising drug /
disease combinations into those for which consideration
of those issues is important and those for which it is
not, and by reviewing current modelling techniques. We
will also explain the specific type of compliance
/persistence data that is appropriate for modelling and
review the sources of such data. Participants will be
encouraged throughout all three sections to comment on
presented methods, data and interpretation. We will
conclude with an interactive discussion on future
development of guidelines for economic evaluations that
would allow for inclusion of compliance and persistence
data.
COST STUDY METHODOLOGY ISSUES
W11: THE ADDED VALUE OF BAYESIAN BELIEF NETWORKS FOR
DECISION MAKING
DISCUSSION LEADERS: G Baio PhD, Post Doc, Department of
Statistical Science, University College London, London,
UK; JP Jansen PhD, Project Manager, MAPI VALUES, Houten,
Netherlands
Workshop Purpose: Participants will be introduced to
Decision Theoretic Networks. Particular focus is on the
applications of such extended decision models for
pharmacoeconomic evaluation and decision-making. The
workshop is intended to provide insight in the
advantages of the use of such models in terms of
understanding the problem, simplicity of representation,
and power of analysis.
Workshop Description: In this workshop Bayesian Networks
and Influence Diagrams are introduced as an alternative
to classical decision making tools. These Decision
Theoretic Networks consist of a graphical structure and
a set of conditional probability distributions. The
graphic side of Decision Theoretic Networks provides an
intuitively appealing interface by which
highly-interacting sets of variables can be modeled
making the problem under study explicit for decision
makers. Moreover, Decision Theoretic Networks have the
advantage of allowing a more compact representation than
Decision Trees. Another advantage is that decision
makers can combine their expert prior opinion or
information with empirical evidence in a very direct
way. The workshop examines the versatility of the
instrument as an extremely powerful modeling tool by
presenting basic theory and applications in different
settings, including clinical practice and economic
evaluation. Participants will be able to access expert
opinion on Bayesian Networks methodology and will be
encouraged to share their own assessment of its
practical utility.
HEALTH CARE COVERAGE AND REIMBURSEMENT ISSUES
W12: MIXED TREATMENT COMPARISONS
DISCUSSION LEADERS: Neil Stephen Hawkins MSc, Research
Fellow, Centre for Health Economics, University of York,
Heslington, York, UK; Mark J Sculpher MSc, PhD,
Professor, Centre for Health Economics, University of
York; Heslington, York, UK; Steve Palmer MSc, Centre for
Health Economics, University of York, Heslington, York,
UK
Workshop Purpose: Cost-effectiveness analyses of new
health technologies must include all relevant
comparators. This often requires comparisons with
treatments with which the new technology has not been
directly compared in ‘head-to-head’ trials. We will
demonstrate mixed treatment comparison methods that
allow direct comparisons to be made between treatments
based on a systematic analysis of all available clinical
trial data.
Workshop Description: In the absence of head-to-head
trial data on alternative interventions, simply taking
values from individual trial arms does not make use of
all the available data, breaks the randomisation that
ensures that subjects are comparable within trials, and
makes the strong assumption that the absolute outcome
values are comparable between trials. Mixed treatment
comparison methods are a generalisation of standard
pairwise meta-analysis that allow the evidence from of a
group of trials including different sets of treatments
to be compared. These methods allow the direct
comparison of treatments, e.g. A, B & C, based on
indirect clinical trial results either through a common
comparator, e.g. trials comparing A vs B and A vs C, or
through a chain of evidence where there is not a common
comparator, e.g. trials comparing A vs B , B vs C. Mixed
treatment comparisons can be implemented as Bayesian
hierarchical models.
We will start by discussing a simple indirect comparison
where a common comparator exists and then more general
mixed comparison models using the WinBUGS software for
binary, ordinal and continuous outcome variables.
Various example analyses will be presented, including
several which have been part of NICE technology
assessment reviews. We will discuss the assumptions
regarding exchangeability and transitivity underlying
these models and the estimation of absolute treatment
effects. A Microsoft Excel spreadsheet demonstrating a
simple method illustrating direct comparison between
treatments will be distributed. A laptop computer will
be required for this exercise.
HEALTH CARE POLICY DEVELOPMENT ISSUES
W13: PRICING POTENTIAL OF A NEW DRUG: APPLICATION OF THE
PRICING MATRIX MODEL
DISCUSSION LEADERS: Mark JC Nuijten PhD, MD, MBA,
Consultant, Erasmus University, Rotterdam, Netherlands
Workshop Purpose: The objective of this workshop is to
present the Pricing Matrix Model, which is a new concept
for the development of a more solid pricing and
reimbursement strategy for a new innovative drug. The
participants of this workshop will gain an understanding
of the underlying methodology and its applications.
Workshop Description: Pricing and reimbursement of new
pharmaceuticals have been based until recently on the
traditional clinical trial outcomes (efficacy, safety
and quality parameters) used for registration. Now we
can distinguish various extra data requirements, which
relate to the use of the drug in real daily practice.
The most important new data requirements are
effectiveness, cost-effectiveness and budgetary impact.
The Pricing Matrix Model is presented, which can be used
for an assessment of the pricing potential of a new drug
taken into consideration the various data requirements
in the pricing and reimbursement process. The concepts
are illustrated for a new hypothetical antidepressant
drug in The Netherlands. This methodology is based on a
concept, which measures decision makers' preferences for
the critical success factors. The participants will
learn how to distinguish various data requirements, and
quantify uncertainty in these impacts to build a solid
pricing strategy.
W14: KEY ISSUES IN MARKET ACCESS: FRANCE, SPAIN, AND
ITALY
DISCUSSION LEADERS: Xavier
Badia MD, PhD, Managing Director, Health Outcomes
Research Europe, Barcelona, Spain; Annie Chicoye PhD,
Aremis Consultants, Neuilly sur Seine, France; Regina
Múzquiz PhD, Director of Public Affairs,
Sanofi-Aventis, Madrid, Spain; Cristina Negrini BSc,
Director, Pbe Consulting, Milan, Italy
Workshop Purpose: The availability of needed medications
to patients under their local health care systems is
sometimes referred to as “market access.” Ensuring
market access for pharmaceuticals and biologicals is a
complex function involving market factors, health care
systems factors and typically negotiations with payers.
Outcomes research (OR) and pharmacoeconomic (PE)
analyses are now key components of these negotiations in
advanced industrial countries. Yet, health care systems
are a moving target, and tailoring OR and PE analyses to
health care system decision makers requires a close
monitoring of health care system changes and an
understanding of how these changes can impact the use of
OR and PE studies to support market access and market
access strategies in general.
Workshop Description: This workshop will outline the key
health care system changes that impact market access
decision making in three key European countries: France,
Spain, and Italy. Among these are: *Changes in the
structure of health care institutions and decision
making *Changes in the financing of health care services
*Changes in the way pharmaceuticals and biologicals are
reviewed and approved for marketing *Changes in the
requirements for value appraisals for pricing and market
access, and particularly for and use of, OR and PE
information *Changes in key externalities (e.g., country
economic conditions, parallel trade, aging population,
political changes) that impact health policy and
perceptions of the role of pharmaceuticals and
biologicals in health care
PREFERENCE-BASED STUDIES
W15: CONJOINT ANALYSIS: APPLICATION TO GLOBAL PRICING
AND REIMBURSEMENT STRATEGY
DISCUSSION LEADERS: F. Reed Johnson PhD, Senior Fellow
and Principal Economist, RTI Health Solutions, Research
Triangle Park, NC, USA; Deirdre M Mladsi BA, Global
Head, RTI Health Solutions, Research Triangle Park, NC,
USA; Keiron Sparrowhawk MSc, MBA, Principal,
PriceSpective Ltd, Buntingford, UK
Workshop Purpose: To educate health outcomes researchers
regarding the relevance of conjoint analysis (CA) in
developing global pricing and reimbursement (PR)
strategies.
Workshop Description: CA has a longstanding history in
market research and is an increasingly important tool in
quantifying health-outcome preferences. While
patient-based CA is often used to support PR strategies,
it is sometimes criticized as having limited relevance
in pharmaceutical markets driven primarily by payer and
physician concerns. However, the patient perspective is
increasingly important in therapy choices and design and
modification of health insurance plans. The context of
the preference elicitation and its intended uses in PR
decision making have important implications for the
development, administration, and analysis of CA surveys.
In this workshop, we start with an interactive component
consisting of an experiment that induces context effects
in a survey administered to participants. The following
discussion will highlight both the potential advantages
as well as pitfalls of CA studies. We then will explore
a series of hypothetical case studies to understand how
elicited preferences may change depending on whether
product price is included as an attribute; whether the
CA is conducted with patients, providers, or payers; and
the relevant country- or market-specific pricing and/or
reimbursement mechanism. Such contextual factors vary
widely across Europe. In France, for example, once a
product is deemed to be of good value (and hence,
reimbursed), there is little pricing pressure on
physicians or patients. Alternatively, in Germany,
physicians may be budget-holders at the local level.
Thus eliciting CA tradeoffs between price and
therapeutic outcomes must be skillfully crafted and the
results carefully interpreted to avoid misleading
conclusions. In most circumstances, it is essential to
augment CA results with auxiliary information on
system-specific institutional factors to provide a
complete and relevant basis for decision-making.
QOL/PRO METHODOLOGY ISSUES
W16: THE USE OF COMPOSITE ENDPOINTS: A REVIEW OF THE
REGULATORS PERSPECTIVE AND METHODOLOGICAL ISSUES
DISCUSSION LEADERS: Christine De la Loge MSc, Project
Manager, Mapi Values, Lyon, France; Benoit Arnould MSc,
MA, Project Manager, Mapi Values, Lyon, France
Workshop Purpose: The objective of the workshop is to
provide an overview of the use of composite endpoints in
PRO research : 1/ regulators expectations regarding
complexity, global judgment and composite endpoints 2/
availability of methods to score and validate composite
endpoints.
Workshop Description: One of the most critical
challenges in clinical drug development is the endpoint
definition. A good clinical endpoint should combine high
validity, reliability, specificity and sensitivity to
change over time, in addition to being meaningful to
both clinicians and patients. Since one single endpoint
cannot combine all of these desired properties, multiple
endpoints are often selected to provide a more complete
picture of the patient's health status and thus, a more
relevant criteria to assess drug efficacy. However, the
handling of multiple endpoints is not straightforward
and has several drawbacks (necessity to elaborate on a
specific statistical procedure to control for
multiplicity, difficulty in the interpretation, …). An
alternative approach is to develop composite endpoints
aggregating different endpoints or perspectives,
allowing a global judgment. Combining outcomes such as
clinical and patient reported outcomes into a global
judgment is of particular interest as it offers
clinicians the opportunity to easily translate
evidence-based medicine into applied medical judgment in
daily practice. However, these outcomes may not be
(highly) correlated and combining them into a single
index or composite endpoint raises methodological
questions. An extensive review of the notes for guidance
from the EMEA has been made to identify suggested
composite endpoints and further enquire about how such
endpoints have been developed (creation or selection of
individual endpoints, creation of a scoring algorithm or
weighting system to obtain a single composite endpoint)
and validated. In parallel, a literature review has been
conducted to clarify methodological issues associated
with the creation and validation of such composite
endpoints and how these are being handled. This
communication will present results of this research and
will be illustrated through examples drawn from disease
areas where there is a high incentive for using
composite endpoints.
CONTRIBUTED WORKSHOPS - SESSION III
Tuesday, November 8, 13:30-14:30
CLINICAL STUDY METHODOLOGY ISSUES
W17: THE USE OF HISTORICAL CONTROLS TO ENABLE ACTUAL
PRACTICE COMPARISONS FOR PROSPECTIVE OBSERVATIONAL
STUDIES
DISCUSSION LEADERS: J. Jaime Caro MD, President &
Scientific Director, Caro Research Institute, Concord,
MA, USA; Zeba Khan PhD, Head Health Economics & Pricing
Strategy, Novartis Pharma AG Basel, Switzerland; Krista Payne MEd, HECON Study Design and Data Capture, Caro Research Institute Montreal, QC, Canada
Workshop Purpose: The objective of this workshop is to
discuss the use of historical controls to allow
comparisons with actual practice (eliminating the
Hawthorne effect). Participants will learn how and why
to use historical controls in this context to provide
comparisons for prospective, observational studies and
economic models.
Workshop Description: To be useful, economic models must
address results in actual practice and, thus, they
require data on the effects of disease and treatment in
the context of the real world. These data are, however,
difficult to obtain because the act of studying someone
tends to change their behaviour (Hawthorne effect).
Thus, a good source for such data are longitudinal
retrospective case series (“historical controls”), where
the effects were already recorded at various times in
the past when the Hawthorne effect was not operating.
Prospective comparative studies — particularly
randomized trials —do not allow for proper
quantification of the effects. While claims database
studies have become common in outcomes research, these
analyses do not allow for study of a new intervention. A
hybrid design with historical controls can overcome
these limitations and allow determination of the
incremental effect compared to actual practice.
In this workshop, we will describe how to properly
design these hybrid studies and how to carry them out
efficiently despite the large sample sizes required.
This will be presented in light of a review of the use
of historical controls in epidemiology and in outcomes
research. Details from a hybrid study of the effect of
compliance-enhancing measures in hypertension will be
used to illustrate the methods. We will highlight the
advantages of the approach but also examine in detail
the pitfalls and approaches to dealing with them.
Participants will also have the opportunity to create
their own design in break-out sessions to be later
presented to the group for discussion.
ADHERENCE/COMPLIANCE ISSUES
W18: NON-COMPLIANCE WITH DRUG DOSING REGIMENS: AN UNDERESTIMATED BIAS IN DRUG TRIALS
DISCUSSION LEADERS: Bernard Vrijens PhD, Chief Scientist, AARDEX Ltd, Zug CH & Vise, Belgium, and Professor, Department of Mathematical Statistics, Liege University, Liege, Belgium;
John Urquhart MD, Chief Scientist, Dept of Biopharmaceuitcal Sciences, AARDEX Ltd, Zug CH & Vise, Belgium, and Professor, University of California San Francisco Medical Ctr, San Francisco, CA, USA
Workshop Purpose: To demonstrate
the statistical properties of various measures of
patients’ deviations from prescribed drug dosing
regimens, and discuss their respective degrees of
clinical explanatory power and their implications for
future research.
Workshop Description: Compliance with protocol-specified drug dosing regimens is a critical factor in clinical trials. Electronic monitoring data provides support for evidence-based decisions about the nature and consequences of deviations from protocol-specified drug dosing regimens. Data will be presented documenting electronically compiled drug dosing histories of 15214 anonymized patients in 87 trials in 10 major fields of pharmacotherapy. Study durations ranged from 30-1400 days. Participants will learn that patterns of deviation from prescribed dosing regimens varied widely (largely skewed toward longer intervals between doses than prescribed) in each field of therapy. Similarly, treatment persistence will be shown to be less than prescribed for chronic medications. The data also can be used to define drug holidays (3 or more consecutive days without drug intake) that occurred at least once a year in 50% of patients. Discussion will focus on the ways in which underdosing, holidays, and early cessation of dosing are frequent sources of low and variable drug responses. Dosing errors during clinical trials are usually grossly under-estimated by counting returned, untaken dosage forms, or by relying on diary entries. Examples will be drawn from our large data archive, plus published and ongoing projects in which drug dosing history data is being compiled electronically. Workshop participants will be encouraged to offer their perspectives on needs for future studies in this field.
COST STUDY METHODOLOGY ISSUES
W19: CALCULATING STUDY POWER FOR ECONOMIC ANALYSES
ALONGSIDE CLINICAL TRIALS
DISCUSSION LEADERS: Elinor CG Chumney PhD, MSc, Asst
Professor, Department of Pharmacy and Clinical Science,
Medical University of South Carolina, Charleston, SC,
USA; Kit N Simpson DrPH, Chair and Professor, Health
Administration and Policy, Medical University of South
Carolina, College of Health Professions, Charleston, SC,
USA
Workshop Purpose: Using standard biostatistical
software, we will introduce participants to the process
of performing power analyses for various economic
endpoints when the sample size is limited by clinical or
programmatic constraints. We will describe the purposes
of power analyses, review conditions that affect power,
work through several examples, and illustrate specific
design and measurement effects on economic measures.
Workshop Description: Increasingly, economists are being
asked to perform power analyses to ensure that they will
be able to find meaningful and statistically significant
differences for economic outcome measures as part of a
clinical trial or proposal. These analyses are often
constrained to a maximum sample size, defined by either
the clinical sample size requirements or design
decisions related to the size of a demonstration
project. They may be further complicated by clinical
study exclusion criteria, skewed cost distributions,
various methods for transforming this cost data,
heterogeneity in the baseline economic risk factors, and
sample selection bias in resource use measures. In this
workshop, we will demonstrate the iterative process that
we have used in the design of economic evaluations in
clinical trials and in evaluations of programs to
improve outcomes for patients with complex chronic
conditions. Specifically, we will (1) describe the
reasons for conducting power analyses for economic
outcomes alongside clinical trials, (2) review
conditions that affect power, (3) work through examples
using data from pancreatitis and HIV clinical trials,
and (4) discuss specific design and measurement effects
on economic measures. We will illustrate the important
contributions that a formal power analysis can have on
the specification of economic hypotheses and on the
decision to use advanced econometric methods for the
analysis of the economic end-points.
HEALTH CARE COVERAGE AND REIMBURSEMENT ISSUES
W20: GLOBAL PRICING AND REIMBURSEMENT STRATEGY: AN
INTEGRATED ANALYTICAL FRAMEWORK
DISCUSSION LEADERS: Keiron Sparrowhawk MSc, MBA,
Principal, PriceSpective Ltd, Buntingford, UK;
Deirdre M
Mladsi BA, Global Head, Health Outcomes Strategy, RTI
Health Solutions, Research Triangle Park, NC, USA;
Nigel
Gregson BA, Principal, PriceSpective LLC, Bluebell, PA,
USA
Workshop Purpose: To give direction to health outcomes
researchers and pricing professionals in the development
of an integrated analytical framework for the
implementation of a global pricing and reimbursement
(PR) strategy.
Workshop Description: Health outcomes researchers
working within the pharmaceutical industry are
increasingly being held accountable for product
reimbursement. In many markets, price and reimbursement
are inextricably linked. Health outcomes research
relates directly to a manufacturer's ability to garner
commercially desirable prices and levels of
reimbursement. Although the “value” in “value-based
pricing” resonates with health outcomes researchers, the
process and methods of developing and implementing a
global P&R strategy seem complex. In this workshop, a
process for developing a global PR strategy and support
will be presented, specifying the place of health
outcomes research in the P&R context. The process will
briefly explain the following key steps: determine
product placement scenarios (1st vs 2nd line, add-on or
stand-alone, within and across indications); conduct
competitive and environmental analyses (competitor
patent status, special indication limits, and
reimbursement status in major markets); perform purchase
decision analysis (all steps and parties involved,
extent of authority vs influence); prepare product value
analysis (economic modeling, payer and physician
qualitative research, preference studies, value
hypothesis generation and testing); and conduct
cross-market impact modeling (parallel trade, reference
pricing, market and indication launch sequencing).
Participants will be presented with historical case
studies of drug launches and asked to critique them.
Case studies will vary according to level of therapeutic
innovation offered by the new drug, placement of the new
product in the treatment pathway, strength and
persuasiveness of economic arguments in favor of the new
product; and country- and market-specific mechanisms for
prescription drug funding.
HEALTH CARE POLICY DEVELOPMENT ISSUES
W21: PHARMACOECONOMICS AND MARKET ACCESS IN EUROPE: CASE
STUDIES IN SCOTLAND AND THE NETHERLANDS
DISCUSSION LEADERS: KH Tolley MPhil, Market Access
Director, Mapi Values, Adelphi Mill, Bollington, UK;
Maarten J Postma Dr, Associate Professor, Groningen
University Institute for Drug Exploration, Groningen,
Netherlands
Workshop Purpose: Workshop Purpose: Based on personal
experience of the presenters, the workshop will provide
participants with a clear understanding of the
pharmacoeconomic evidence needs and impact on market
access of two influential European reimbursement
agencies that appraise new drugs at launch: the Scottish
Medicines Consortium in the UK and the Committee for
Pharmaceutical Help in the Netherlands.
Workshop Description: In many countries in Europe there
is now a need to submit evidence on the clinical and
cost-effectiveness of a new drug in order to attain
reimbursement or market access. NICE is a well known
agency that requires such evidence, but its remit is
wider than pharmaceuticals and it typically evaluates
only selected and established products. In contrast, the
Scottish Medicines Consortium and the Dutch Committee
for Pharmaceutical Help are agencies that evaluate
clinical and cost-effectiveness of all new
pharmaceuticals at launch. Hence, in many cases these
are the first agencies in Europe to make judgements
about cost-effectiveness of new drugs for reimbursement
guidance. After a brief comparison of the process of
decision making involved with each agency, the primary
aim of the workshop is to provide participants with a
clear understanding of the critical pharmacoeconomic
evidence needs of these agencies, with a focus on the
specific problems of the choice of the appropriate
comparator, the measurement of health outcomes and the
time horizon of analysis. The workshop will contain an
interactive exercise to review case study examples of
products that have been appraised (and for which
information is publicly available).The impact of each
agency on new product reimbursement and market access
within UK and Netherlands and in Europe will be
discussed. Recommendations on best practice in
optimising company pharmacoeconomic submissions will be
provided. As a theme, the workshop will draw on the
presenters personal experience of membership on both
committees.
PATIENT REGISTRIES
W22: UNDERSTANDING INTERNATIONAL REQUIREMENTS WHEN
DEVELOPING AND IMPLEMENTING A GLOBAL PATIENT REGISTRY:
STRATEGIES TO MAXIMIZE SUCCESS
DISCUSSION LEADERS: Matthew J Gordon BA, Senior Manager,
Ovation Research Group, Highland Park, IL, USA;
Alison
Wetherfield JD, Partner, Employment Department,
McDermott Will & Emery, London, UK; Leanne Larson MPH,
Senior Vice President, Patient Registries, Ovation
Research Group, Highland Park, IL, USA
Workshop Purpose: Participants will gain an
understanding of registry marketplace trends, logistical
considerations unique to international programs,
including country-specific legal and regulatory trends,
design principles and strategic opportunities to
maximize success.
Workshop Description: Growing demands from drug
regulators, as well as the need for information to
support global commercial markets are contributing to
the necessity for international patient registries.
Registries provide an opportunity to document the
real-world effects of new and marketed products once
introduced into the community. There are numerous
logistical components (i.e., patient confidentiality,
data ownership and transfer, translation considerations,
etc.) unique to each country. From a project team /
project management perspective, effectively managing a
program with international scope requires considerable
coordination and structure from the earliest stages of
registry development. It is during this design stage
that a unique opportunity exists to determine the
registry's scope, pertinent measures (outcomes and
pharmacoeconomic) as well as strategic, global marketing
objectives. Those attending this interactive workshop
will hear international case studies and will leave with
a better understanding of how to undertake one of these
programs.
PREFERENCE-BASED STUDIES
W23: INTERNATIONAL ECONOMIC EVALUATIONS OF IMMUNISATION
POLICIES: COMPARABILITY OF RESULTS AND CONCLUSIONS
DISCUSSION LEADERS: Ursula M Kuehnel MSc, Head of Health
Economics, Health Economics, Berna Biotech, Bern,
Switzerland; Jeremy Chancellor MSc, Managing Director,
Innovus Research (UK) Ltd, High Wycombe,
Buckinghamshire, UK; Michael Drummond PhD, Vice
President, Innovus Research (UK) Ltd, High Wycombe, UK
Workshop Purpose: This workshop will focus on the
interpretation and significance of cost-effectiveness
and cost-utility evaluations (CEA / CUA) in models
designed to inform decisions or preferences across
countries. The interpretation of cost-effectiveness
ratios, related measures and how meaningfull they are,
will be discussed in the context of international public
health and economic considerations. Participants of the
workshop will benefit by exploring inter-country and,
intercontinental differences and hence the level of
comparability of CEA / CUA results and conclusions to be
drawn using a cases study in the field of immunisation
policies.
Workshop Description: The workshop will introduce the
problems of transferring and comparing health economic
evaluations between jurisdictions to inform health
program decisions. Given the prerequisite of a high
degree of methodological comparability and information
on the homogeneity of patient populations studied within
multicountry economic models, usability and
interpretation of the results is a particular challenge
for decision makers. Moreover, exogenous economic
factors are likely to influence individual countries'
willingness to pay for health gains. Participants of the
workshop will be encouraged to explore inter-country
and, intercontinental differences and hence the level of
comparability of CEA / CUA results and conclusions to be
drawn. This will be exemplified by a case study
involving quantitative analysis of modifying routine
influenza vaccination policy for defined key countries.
Interactive sub-group discussions of the case study will
be stimulated by a prepared agenda of possible issues,
with the opportunity for participants to challenge,
respond and add to these.
QOL/PRO METHODOLOGY ISSUES
W24: RESCUING CLINICAL TRIAL DATA FOR ECONOMIC
EVALUATION
DISCUSSION LEADERS: Frank De
Charro, Executive Director, EuroQoL Group, Erasmus
University Rotterdam, Centre for Health Policy and Law,
Rotterdam, Netherlands; Xavier Badia MD, PhD, Managing
Director, Health Outcomes Research Europe, Barcelona,
Spain; Wolfgang Greiner MSc, Professor, Fakultaet fuer
Gesundheitswissenschaften, Bielefeld University,
Bielefeld, Germany; Paul Kind, Principal Investigator,
Centre for Health Economics, Outcomes Research Group,
University of York, York, UK
Workshop Purpose: The need to interpret clinical trial
data in economic evaluation is a major challenge facing
many researchers and regulatory authorities.
Observations used to measure outcomes in clinical
studies are often condition-specific and/or lack the
necessary requirements for use in economic evaluation in
general and in cost-utility analysis in particular. This
workshop will demonstrate methods for rectifying these
measurement failures. Participants will given insights
to the problem in general and to the specific remedial
heuristics derived by researchers from the EuroQoL Group
Workshop Description: The need to interpret clinical
trial data in economic evaluation is a major challenge
facing many researchers and regulatory authorities.
Observations used to measure outcomes in clinical
studies are often condition-specific and/or lack the
necessary requirements for use in economic evaluation in
general and in cost-utility analysis in particular.
EQ-5D is a generic measure of health-related quality of
life for which social preferences weights exist in many
countries. In some cases these weights have been
established using utility estimation procedures. EQ-5D
has been extensively used for more than a decade in
national surveys of population health and in clinical
studies in a wide range of therapeutic settings. A vast
array of data exists including normative population data
standardised by age/gender and self-reported morbidity
as well as data from observational and interventional
studies where EQ-5D has been fielded alongside
established clinical measures. In total, these data
support the conversion of clinical measures into a
corresponding EQ-5D metric. This “crosswalk” conversion
enables data from clinical trials to be expressed in
terms of a single index with the necessary 0-1 scale
properties required for economic evaluation. Examples of
this data conversion process will be presented, drawing
on the experience of researchers in several European
countries working in different therapeutic areas.
CONTRIBUTED WORKSHOPS - SESSION IV
Tuesday, November 8, 16:00-17:00
ADHERENCE/COMPLIANCE ISSUES
W25: CHALLENGES OF DESIGNING PROSPECTIVE AND
RETROSPECTIVE COMPLIANCE STUDIES
DISCUSSION LEADERS: Won Chan Lee PhD, HERQuLES,
Associate Director of Health Economics, Abt Associates,
Bethesda, MD, USA; Asli Memisoglu ScD, Lead
Pharmacoeconomist/Outcomes Scientist, HERQuLES, Abt
Associates, Lexington, MA, USA; Chris L Pashos PhD, Vice
President/Executive Director, HERQuLES, Abt Associates,
Lexington, MA, USA
Workshop Purpose: The real-world clinical effectiveness
and economic value of a drug depend, in part, on
compliance and adherence of patients to appropriate
regimens. Thus, studies aimed at understanding
compliance and developing effective interventions have
become more common. The goal of this workshop is to
provide participants with 1) a brief summary of
published studies dealing with compliance, adherence,
and persistency issues; 2) various methods typically
employed; 3) valid statistical tools and study designs
addressing methodological challenges; 4) critiques of
commonly used algorithms to describe switching and
discontinuation of therapy in a claims based study; 5)
desired data collection methods for these parameters in
a prospective study.
Workshop Description: Measures used to characterize drug
utilization patterns (including compliance and
adherence) vary widely, and include drug possession
ratio, proportion of days covered, and proportion of
patients discontinuing or switching treatment. More
recently, researchers have adopted complementary
measures such as time to discontinuation, time to switch
and time to failure using Kaplan Meier curves and Cox
proportional hazards models. This workshop will provide
a review of literature in this area, comparing and
contrasting key methods and their value. Throughout the
workshop, participants will be given opportunities to
study real-world examples, to identify methodological
challenges that each case presents, and statistical
tools or study design to overcome these challenges. Most
notably, this workshop is intended to highlight creative
solutions to address utilization and associated costs. A
portion will be devoted to presenting and discussing
data collection methods related to evaluation of
switching and discontinuation in prospective studies.
Overall, participants will be presented with a series of
analytical tools that will facilitate design and
implementation of a successful retrospective or
prospective study of drug utilization and compliance.
COST STUDY METHODOLOGY ISSUES
W26: DRG-BASED CASE COSTS: WHY THEY ARE NOT COMPARABLE
ACROSS BORDERS AND SHOULD BE USED WITH CAUTION
DISCUSSION LEADERS: Judith A. O'Brien RN, BSPA, Vice
President, Caro Research Institute, Concord, MA, USA
Workshop Purpose: This workshop’s objective is to
provide an understanding of why case costs established
from a system like U.S. Diagnosis Related Groups (DRG),
designed for prospective payment, do not represent the
same value as those calculated in a DRG-like system
designed primarily for prospective budgeting (e.g.,
Groupe Homogènes de Malades (GHM)/de Sèjours (GHS), Case
Mix Groupings (CMG). Participants should gain an
understanding of case assignment factors and how they
affect case-mix and case costs.
Workshop Description: As more countries adopt or adapt
DRG-type case classification systems, researchers
needing to estimate hospital costs for the same clinical
problem across different countries are becoming
increasingly reliant on reported case costs by DRG or
other case assignment categories. Yet, case costs from
one country do not necessarily reflect comparable costs
from another, even when the clinical condition or
procedure category appears similar. DRG-type systems are
not identical and are not utilized for the same purpose.
Some countries employ DRG-type systems to implement
prospective payment programs; others for prospective
budgeting, and while the use of the case cost within the
country is appropriate, it may not be correct to use it
comparatively with other countries. The purpose of why
the system was enacted can change the composition of the
case-mix within a given “DRG”. This workshop will focus
on some fundamental differences between case assignment
in prospective payment and prospective budget systems
and how that can affect case costs. Examples of how to
crosswalk cases from one system to another will be
presented and discussed. Specific cost estimate examples
will be used to illustrate why grouped data
classifications systems should not be thought of as the
utopian common denominator for estimating comparable
case costs across countries. Participants will be
encouraged to relate relevant experience with like
classification systems in their respective countries.
HEALTH CARE COVERAGE AND REIMBURSEMENT ISSUES
W27: METHODOLOGICAL CHALLENGES FOR COST-EFFECTIVENESS
STUDIES FOR MEDICAL DEVICES
DISCUSSION LEADERS: Mark JC Nuijten PhD, MD, MBA,
Consultant, Erasmus University, Rotterdam, Netherlands
Workshop Purpose: The participants of this workshop will
gain an understanding of the methodological challenges
faced in performing cost-effectiveness studies for
medical devices.
Workshop Description: From a scientific point of view,
there is no consensus on the methodologically
appropriate execution of cost-effectiveness studies for
medical devices. Important issues of debate are: the
choice of the comparator, perspective, handling of
depreciation and discounting, non-medical costs, and
extrapolation of short-term efficacy to long-term
effectiveness. We run into this question when we
performed a cost-effectiveness study for the NESS
handmaster, an innovative device in the rehabilitation
of patients with a paretic or paralysed upper limb due
to central nervous system disorders. This study is used
for purpose of illustration, but we will broaden the
scope to studies in medical devices in general. We will
present a statement for each research topic followed by
a group discussion.
HEALTH CARE POLICY DEVELOPMENT ISSUES
W28: KEY ISSUES IN MARKET ACCESS: UK, SWEDEN, AND
GERMANY
DISCUSSION LEADERS: Pippa Anderson MSc,
Director, Fourth Hurdle Consulting Limited, London, UK; Ulf Persson PhD,
Research Director, IHE, The Swedish Institute for Health Economics, Lund, Sweden; Bernd Brüggenjürgen MD, MPH,
Assistant Professor, Charité - Universitätsmedizin
Berlin, Institut für Sozialmedizin, Epidemiologie und
Gesundheitsökonomie, Berlin, Germany
Workshop Purpose: This workshop will outline the key
health care system changes that impact market access
decision making in three key European countries: UK,
Sweden, and Germany. Among these are: *Changes in the
structure of health care institutions and decision
making *Changes in the financing of health care services
*Changes in the way pharmaceuticals and biologicals are
reviewed and approved for marketing *Changes in the
requirements for, and use of, OR and economic
information for pricing and market access *Changes in
key externalities (e.g., country economic conditions,
parallel trade, aging population, political changes)
that impact health policy and perceptions of the role of
pharmaceuticals and biologicals in health care
Workshop Description: The reimbursement, funding and
uptake of health technologies to patients served by the
local health care systems is sometimes referred to as
“market access.” Ensuring market access for health
technologies is a complex function involving market
factors, health care systems factors and, typically,
negotiations with many levels of budget holders and
other decision makers. Outcomes research (OR) and
economic analyses are now key components of these
negotiations in advanced industrial countries. The
establishment of the National Institute for Health and
Clinical Excellence, the Scottish Medicines Consortium
and LFN and the stringent evidence requirements provides
substantial challenges to the outcomes researcher.
Tailoring OR and economic analyses to meet these
requirements as well as those of other health care
system decision makers requires a close monitoring of
health care system changes and an understanding of how
these changes can impact the use of OR and economic
studies to support market access and market access
strategies in general.
W29: INTRODUCTION AND DIFFUSION OF INNOVATIVE INPATIENT
DRUGS. COMBATING INEQUALITIES
DISCUSSION LEADERS: Elly Stolk PhD, Researcher,
Institute Medical Technology Assessment, Erasmus
University, Rotterdam, Netherlands; Floor Nooten van
PhD, Researcher, Institute for Medical Technology
Assessment, Erasmus University, Rotterdam, Netherlands;
Carin Uyl PhD, Director, Institute of medical Technology
Assessment, Erasmus University, Rotterdam, Netherlands
Workshop Purpose: Today, we see a large variation in
diffusion patterns of new drugs both within and between
countries. The aim of this workshop is to increase
understanding of the processes that affect the
introduction and diffusion of innovative drugs. People
who can benefit from the course are health outcomes
researchers, industry, and those involved in regulation
and listing of inpatient drugs.
Workshop Description: In an ideal world, there is no
variation in implementing innovative drugs in clinical
practice, and there is no interference of non-scientific
factors in this process. In real life, however, there
exists a considerable variation in the introduction and
the time associated with diffusion of new drugs in
different countries. For example, the introduction of
innovative drugs in the Netherlands and UK lag behind
with other countries. Moreover, also within countries
(e.g. Denmark, Sweden, Austria) we see asymmetrical
diffusion patterns. Because of resulting inequalities,
this is cause for concern and one should strive to
minimize the differences. Partially the differences may
depend on prescriber characteristics, which are
difficult to change. However, in a subtle way the
financing structure and regulatory and organisational
characteristics of health care systems also affect
uptake of new drugs in clinical practice. This becomes
apparent from a comparison of drug diffusion patterns
and health care characteristics of European countries.
The objective of this workshop is to map the
inequalities, and to explain their causes. In addition,
particular focus will be given to partial solutions to
the problem; e.g. Time&Motion studies, changes in the
financing structure, and measures to increase
transparency. A hands-on element is included in the form
of a brief questionnaire that elicits the participants'
experiences with obstacles in the uptake of innovative
drugs for inpatient use. This will also serve as the
basis for the plenary discussion.
PREFERENCE-BASED STUDIES
W30: THE VALUE OF CONJOINT ANALYSIS IN OUTCOMES RESEARCH
DISCUSSION LEADERS:JP Jansen PhD , Project Manager,
Health Economics, MAPI VALUES, Houten, Netherlands; KH
Tolley MPhil, Market Access Director, Mapi Values,
Adelphi Mill, Bollington, UK; Elyse Trudeau PhD, Unit
Director, Mapi Values, Lyon, France
Workshop Purpose: The workshop is intended to provide an
introduction to conjoint analysis and discrete choice
modeling with the focus on applications in outcomes
research. In particular the value of these methods for
patient preference studies will be discussed.
Workshop Description: In this workshop conjoint analysis
and discrete choice modeling are introduced as a
methodology to study patient preferences for various
treatment alternatives. Conjoint analysis alloys to
determine which individual treatment attributes are the
most important to patients and it is also possible to
model patient preferences for different profiles of
treatment This workshop examines the key issues in study
design, analysis, and interpretation of results.
Furthermore it will be shown how the information
obtained can be used in brand support and
direct-to-patient-activities, including patient
education and awareness. Participants will be encouraged
to share their own assessment of the value and practical
utility of these techniques in different therapeutic
areas. Participants who would benefit the most from this
workshop are health services researchers, health care
decision makers, and policy makers who are responsible
for conducting, evaluating and utilizing outcomes
research studies.
QOL/PRO METHODOLOGY ISSUES
W31: HEALTH STATUS AND QUALITY OF LIFE ASSESSMENT FOR
CRITICAL DECISION MAKERS -- CLINICIANS, ADMINISTRATORS
AND HEALTH ECONOMISTS
DISCUSSION LEADERS: Pennifer Erickson PhD, Co-founder,
O.L.G.A, State College, PA, USA; Nancy Kline Leidy PhD,
President and CEO, The MEDTAP Institute at UBC,
Bethesda, MD, USA; Margaret Rothman PhD, Executive
Director, HE&P, PGSM, Johnson & Johnson Pharmaceutical
Services, Raritan, NJ, USA
Workshop Purpose: The aim is to improve critical
decision makers' understanding of conceptual,
methodological and practical aspects of health status
and quality-of-life assessment. At the end of the
workshop, participants should be able to identify and
select health-related quality-of-life measures that are
relevant to stakeholders' concerns and to interpret the
findings appropriately to inform decision making.
Workshop Description: This workshop is an initiative of
the Concepts and Definitions Working Group of the
QoL-SIG and aims to provide ISPOR members and their
professional colleagues with principles of good
practices for understanding and communicating the
science of health status and quality-of-life measurement
to decision makers. As instruments for these concepts
are increasingly applied to measure patient outcomes,
interest in the optimal use of the findings has grown. A
clear understanding of outcomes measurement and
interpretation is an important part of decision making.
This workshop will identify major stakeholders and
discuss how their concerns relate to commonly used
measurement strategies. Instrument content will be
evaluated to assist participants in deciding which
instruments capture outcomes of greatest stakeholder
concern and in understanding the meaning they may (or
may not) ascribe to study results. Then, measurement
properties will be discussed in terms of their effect on
the magnitude of the observed effect. Methods for
interpreting results will be addressed, with particular
attention given to issues surrounding the the use of
minimal important difference (MID) . Each of these
aspects of patient reported outcomes will be discussed
within the context of the stakeholders and key decision
makers. Several interactive exercises are planned.
RISK ASSESSMENT/RISK MANAGEMENT ISSUES
W32: RISK ADJUSTMENT IN NON-COMPETITIVE MARKETS
DISCUSSION LEADERS: Kenneth D. Smith PhD, Asst
Professor, Department of Health Policy, Jefferson
Medical College, Thomas Jefferson University,
Philadelphia, PA, USA; Vittorio Maio PharmD, MSPH, Asst
Professor, Department of Health Policy, Jefferson
Medical College, Thomas Jefferson University,
Philadelphia, PA, USA; Elaine J. Yuen PhD, MBA, Research
Asst Professor, Center for Research in Medical Education
and Health Care, Jefferson Medical College, Thomas
Jefferson University, Philadelphia, PA, USA
Workshop Purpose: To demonstrate the utility of risk
adjustment in non-competitive markets, and to
demonstrate how risk adjustment tools may be used in
these markets. The workshop will include exercises on
how risk adjustment designs impact global budgets using
a non-market-based health system as a case study.
Workshop Description: Risk adjustment is a useful tool
for setting budgets, measuring performance, and
measuring quality of care in any healthcare system.
Methods developed in free enterprise systems like the
U.S. may be inappropriate in other kinds of healthcare
systems. In market-based systems, providers receive
risk-adjusted capitated payments as reimbursement for
services provided to individuals within its patient
base. If the sum of all capitated payments across the
patient base exceeds the actual cost of providing
services, then the provider receives a profit;
otherwise, it incurs a loss. The objective of risk
adjustment in market-based systems, then, is to design
the optimal mix of financial incentives to induce
providers to manage care effectively without stinting or
gaming. Design issues in non-competitive markets,
however, have not been explored adequately. For example,
when budgeting is a part of a negotiation process,
setting budgets using strict risk adjuster formulae may
be impractical. In such cases, risk adjusters can be
designed to provide incentives to improve the
negotiation process. For example, in developing risk
adjusters to set budgets for hospital and pharmacy
costs, we designed negotiable budget bands. These bands
define upper and lower limits of permissible budget
levels. Use of these budget bands in budget negotiations
can help ensure cost containment during the budget
negotiation process. This workshop will provide
researchers and managers with an overview of risk
adjustment designs and methodologies. Participants
should bring their own computer for exercises.
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