SESSION I


Quality of Life Study Methodology Issues

W1: EXPLORING ALTERNATIVE USES OF QUALITY OF LIFE DATA COLLECTED FOR ROUTINE SURGICAL AUDIT

Lloyd A1, Whatling JM2, Wilson JL2,Vallance-Owen A3, Hutton J4, 1MEDTAP International, London, United Kingdom; 2Outcome Technologies, London, United Kingdom; 3BUPA, London, United Kingdom; 4MEDTAP International, London, United Kingdom

Learning Objectives: To describe the benefits and potential uses of routinely collected HRQL data.
Who Would Benefit?: Researchers and clinicians with an interest in the application of routinely collected quality of life data.
Workshop Description: We will describe the benefits and potential uses of routinely collected HRQL data. The workshop will draw upon data from a large database of over 80,000 patients who have undergone a surgical or diagnostic procedure in different UK hospitals. This database has known limitations, perhaps most importantly a lack of diagnostic information. The workshop will be designed to explore the potential uses of such a database despite its limitations. We will draw on examples of specific procedures and sub-groups of patients. All patients undergoing surgery within a large UK private not-for-profit health care system have been routinely audited for several years. All patients were asked to complete the Short form-36 (SF-36) health survey prior to their operation and at three months after surgery. The data was collected for routine audit purposes. Such a large dataset can be used in many different ways to address different issues. Despite limitations the database still presents important information on a large number of surgical procedures where conventional RCT data is missing. The purpose of the workshop is to explore the potential uses of this type of database. We will encourage discussion of how the limitations of the data can be overcome. We will discuss:
• Is it possible to assess the benefits of surgical procedures without diagnosis data?
• Can this database address the problems caused by a lack of properly conducted RCTs in surgery?
• Can this data be used to benchmark future studies of interventions?
• Could it be used to support NICE assessment of interventional procedures?
• Can large N overcome data limitations?

Health Policy - Reimbursement Issues

W2: SYNCRONISING DIFFERENT APPROACHES IN COVERAGE POLICY AND REIMBURSEMENT IN THE HUNGARIAN HEALTH CARE SYSTEM

Dózsa C, Bidló J, National Health Insurance Fund, Budapest, Hungary

Learning Objectives: To demonstrate the conflict between the short-term policy budgetary objective to control public spending on health technology and the
long-term objective to increase the efficiency of the allocation of health care
expenditures in Hungary.
Who Would Benefit?: Policy makers, researchers interested in silo budgeting and incorporating economic evaluation in the decision-making process.
Workshop Description: Due to the development of new technologies, health insurance is faced with challenges in the field of financing of health technologies. In Hungary, the nationwide statutory health insurance fund, in a monopole situation is faced with a number of separate, constrained budgets (e.g. inpatient, outpatient, pharmaceutical budget). The increasing demand for health technologies results in exceeding the planned yearly budgets. This financial pressure can be eased by an appropriate coverage policy. However at the present there is room for improvement in the field of transparency, evidence-based decision-making and providing an efficient long-term or short-term resource allocation. The current legal framework was recently established. Concerning pharmaceutical reimbursement to tackle the problems two strategies are proposed. One, introducing reference pricing in therapeutical groups, the other the introduction of health technology assessment into the coverage policy. The objective is to increase transparency in the reimbursement system. The reference pricing system aims to contain the fast growing pharmaceutical budget, by setting one reimbursement level at certain therapeutical groups. The health technology assessment program is based on the principles of requirements of the European Union’s transparency Directive (89/105/CEE) examining effectiveness, cost-effectiveness and equity taking into consideration the budget constraint. The objective is to analyze if the two above approaches fulfill the principles they are aiming for, the policy goals set out, and if they are in conflict with each other.

W3: PHARMACEUTICAL PRICING AND REIMBURSEMENT POLICIES IN CHINA

Hu S, School of Public Health, Fu Dan University, Shanghai, P.R China

Learning Objectives: To evaluate the present pricing and reimbursement policies of pharmaceuticals in China and to discuss how to improve the policy-making on setting rational drug prices and reimbursements lists.
Who Would Benefit?: Individuals involved in pricing and reimbursement of drugs.
Workshop Description: In China, the share of pharmaceutical expenditures accounted for 47~56% in national health accounts during the past decade. The average annual growth rate of pharmaceutical expenditures was 11.2% higher than that of gross domestic product. During this workshop the changes of pricing policy, which has gradually shifted from fixed cost markup to social average pricing and bulk procurement through price bidding will be presented. As a result of changes in the pricing policy, some negative impacts have occurred in the process of pooled purchasing. Although multiple approaches have been adopted, a significant savings in pharmaceutical expenditure has not occurred. Alternative drug policy reimbursement plans will be discussed such as reference-based pricing schemes and price negotiation with pharmaceutical companies as a future direction to health care cost containment. Workshop participants will be asked to share experiences with drug reimbursement policies in their country.

Compliance Issues

W4: IMPROVING PHYSICIAN ADHERENCE TO EVIDENCE-BASED CARE:
PERFORMANCE-BASED REIMBURSEMENT MODELS

Legorreta AP, Health Benchmarks, Inc, Woodland Hills, CA, USA

Learning Objectives: The primary learning objectives are to gain awareness of the high demand for performance-based reimbursement and to discuss examples of programs designed for several health plans. Health Benchmarks, Inc. has the unique perspective of having worked with various health care organizations in developing performance-based provider-reimbursement models. This case study workshop provides elucidation of: development of evidence-based clinical indicators based on administrative data, appropriate case-mix adjustment, and population-level outcomes.
Who Would Benefit?: Health care decision-makers, health services and health policy researchers, and others interested in improving the quality of health care.
Workshop Description: Performance-based reimbursement models are receiving widespread attention from health plans and policymakers. Health Benchmarks, Inc. (HBI) has a strong history of developing pay-for-performance programs with health plans and large employers across the nation. HBI has developed over 70 clinical indicators for more than 50 physician specialties covering a broad range of issues including appropriate use of pharmaceuticals, chronic disease management, prevention, over-utilization, and follow-up care. Furthermore, HBI has developed customized mixed-effect regression models to account for both patient demographic characteristics and also comorbid conditions and complications in a comprehensive case-mix adjustment approach. Organizations using HBI methodologies have observed longitudinal improvement in population-level outcomes associated with pay-for-performance programs. This rise in physician adherence to evidence-based guideline is likely to result in long-term improvements in patient health and reductions in associated health care costs. Increased adoption and more standardized evaluation of these programs will benefit payers and providers, and ultimately help drive a performance-driven health care system that improves the quality of health care for consumers.

Clinical Study Methodology Issues

W5: PATIENT REGISTRIES AS A SOURCE FOR OUTCOMES AND SAFETY DATA: DESIGNING PROGRAMS TO MEET BROAD ORGANIZATIONAL OBJECTIVES

Larson L1, Gordon M1, Waller H2, Knapp R1, Trotter JP1, 1Ovation Research Group, Highland Park, IL, USA; 2Galt Associates, Inc, Sterling, VA, USA

Learning Objectives: Participants will gain an understanding of: 1) international trends in regulatory requirements for post-marketing safety-surveillance registries; and 2) the increasing importance and value of integrating outcomes measures within these programs.
Who Would Benefit?: Outcomes researchers interested in exploring the design and application of patient registries to meet outcomes, commercial, and safety-surveillance objectives. Those with responsibility for implementing peri-approval programs to meet the growing demand for integrated risk management/outcomes data would also benefit.
Workshop Description: Worldwide, the requirement for post-marketing outcomes and safety data is rapidly increasing. The growing demands from regulators, including the EMEA and the FDA, as well as from global commercial markets, are accelerating the need to better document the real-world effects of new and marketed products, contributing to the necessity for – and proliferation of – patient registries. When designed appropriately, these registries represent a valuable new tool allowing outcomes researchers to collaboratively and efficiently meet a broad range of organizational objectives. While the responsibility for initiating a patient registry has traditionally fallen to clinical, marketing, or medical affairs, global health outcomes groups are increasingly involved in these programs. Patient registries, particularly multinational safety-surveillance programs, offer outcomes researchers an opportunity to create and access unique datasets. On a broader scale, the uses of "safety" patient registries may range from developing a competitive differentiation strategy based on safety and effectiveness, to meeting post-launch regulatory commitments (including risk-management demands), to providing protection against pre-mature withdrawal of newly launched medications. The proliferation of these programs will only expand this opportunity for outcomes researchers worldwide. This interactive workshop will review registry strategy and design, presenting several case studies considering the developing trend toward integrated safety/outcomes registries. Finally, global regulatory and risk-management trends will be discussed, highlighting the potential value of these programs throughout the sponsor organization.

Cost Study Methodology Issues

W6: THE SEARCH FOR THE HOLY GRAIL: ESTABLISHING A VALID METHODOLOGY FOR MEASURING WORK PRODUCTIVITY

Evans CJ, Mapi Values USA LLC, Boston, MA, USA

Learning Objectives: In this workshop we examine the measurement of lost time for paid work activities. Issues in the content validity of questionnaires, recall periods, the importance of risk adjustment, treated vs. untreated populations and international cultural adaptations will be explored.
Who Would Benefit?: This session is directed at individuals who are interested in the measurement (as distinct from valuation) of indirect costs in pharmacoeconomic studies. In particular, individuals who are interested in improving formulary pull through or reimbursement acceptance based on an indirect cost data would benefit.
Workshop Description: The assessment of productivity changes is one of the most challenging areas of outcomes research. There exist several methods for assessing productivity changes in paid employment: homemade questions, generic questionnaires, general productivity/absence measures and disease specific productivity/ absence measures. The collection of information that may be used in providing estimates of indirect costs is problematic for a number of reasons: recall errors, self-presentation bias, inconsistent use of employment categories, the presence of unusual work habits and a lack of accessible validating data. In this workshop we examine the measurement of lost time for paid work activities. Issues in the content validity of questionnaires, recall periods, the importance of risk adjustment, treated vs. untreated populations and international cultural adaptations will be explored. Particular emphasis will be placed on determining how best to establish criterion validity by examining the presence (or absence) of archival data in three well-developed questionnaires: The Health Performance Questionnaire, the Work Limitations Questionnaire and the Work and Health Interview Survey. In addition, we will assess the appropriateness of other methods (such as the WPAI or the Endicott Work Productivity Scale) of measuring changes in productivity.

W7: USE AND MISUSE OF THE CASE-CONTROL METHODOLOGY IN ECONOMIC ANALYSES

Caro JJ, Huybrechts KF, Caro Research Institute, Concord, MA, USA

Learning Objectives: To review the case-control methodology and to critically evaluate its proper use and its usefulness in economic evaluations.
Who Would Benefit?: Researchers involved in conducting health economic analyses, as well as those responsible for evaluating such analyses.
Workshop Description: A growing number of economic studies appearing in the literature are said to be based on case-control methodology. Upon closer examination, however, most of these are not case-control studies at all: they are in fact mislabeled cohort studies. In these studies, individuals with a given illness (incorrectly defined as "cases") are followed over time and the outcome of interest — often, total medical cost — is compared to that of those without the illness (incorrectly defined as "controls"). The net cost of the illness is estimated as the difference in cost. A proper case control study involves cases defined by the outcome of interest (e.g., patients who are admitted to a nursing home), while controls are a sample of the population from which the cases came (e.g., patients in the catchment area for those nursing homes) taken to provide estimates of the denominators for the rates of interest (e.g., patients with Alzheimer’s Disease and patients without this diagnosis). Although case control studies are typically more prone to bias than cohort studies, properly conducted they are enormously efficient, particularly when the outcomes sought are rare. During this workshop, we will present the case-control methodology and highlight the differences with cohort studies. We will also review the circumstances where this methodology can be useful in the context of economic evaluations. Throughout the workshop, the issues will be illustrated by means of interactive group exercises and views of the participants will be vigorously elicited.


SESSION II


Quality of Life Study Methodology Issues

W8: FROM PRACTICE TO POLICY: MEASURING HEALTH OUTCOMES USING EQ-5D

Badia X1, Busschbach J2, de Charro F2, Greiner W3, Kind P4, 1Health Outcome Research Europe, Barcelona, Spain; 2Erasmus University, Rotterdam, The Netherlands; 3University of Hannover, Hannover, Germany; 4University of York, York, United Kingdom

Learning Objectives: Participants will gain an understanding of the versatility of EQ-5D in its role as a single index and a generic measure of health outcomes. The workshop will provide a unique insight of the current status of EQ-5D in a wide range of settings including its use by regulatory bodies. Participants will be able to access expert opinion on EQ-5D and will be encouraged to share their own assessment of its practical utility.
Who Would Benefit?: Health services researchers in academia, industry or regulatory bodies. Decision-makers at all levels of clinical practice.
Workshop Description: The workshop is presented by senior members of the EuroQoL Group who will summarize EQ-5D applications in different settings including clinical practice and population health surveillance, as well as in clinical trials and economic evaluation. The workshop examines the versatility of EQ-5D in its role as a single index, generic measure of health outcomes. Examples are drawn from several different countries. The workshop will provide a unique insight of the current status of EQ-5D in a wide range of settings including its use by regulatory bodies. Participants will be able to access expert opinion on EQ-5D and will be encouraged to share their own assessment of its practical utility.


Health Policy Reimbursement Issues

W9: ECONOMIC EVALUATION OF PHARMACEUTICALS FOR FORMULARY LISTING AND REIMBURSEMENT IN U.S. MANAGED CARE

Sullivan SD1, Veenstra D1, Ramsey S2, 1University of Washington, Seattle, WA, USA; 2Fred Hutchinson Cancer Research Center, Seattle, WA, USA

Learning Objectives: The purpose of this session is to review the AMCP submission process to support pharmaceutical and biotechnology listing and reimbursement, as well as the prevalence and influence of the guidelines on managed care formulary committee decisions in the United States.
Who Would Benefit?: Health policy and pharmaceutical decision-makers, cost-effectiveness researchers, and the pharmaceutical industry.
Workshop Description: Introduced in 2000, the U.S. Academy of Managed Care Pharmacy (AMCP) guidelines for use of clinical and economic data to support drug formulary consideration have been adopted in health plans and pharmacy benefit management organizations that cover more than 100 million lives. These guidelines have been adapted partly from non-US models (Australia, Canada and the UK-NICE) and are intended to support and encourage the use of economic evaluations of pharmaceutical and biotechnology products for decision-making and resource allocation within U.S. managed care plans. Presentation Description: The purpose of this session is to review the AMCP submission process to support pharmaceutical and biotechnology listing and reimbursement, as well as the prevalence and influence of the guidelines on managed care formulary committee decisions in the United States. The first 45 minutes will include summary statistics and examples that illustrate the quality and relevance of cost-effectiveness information in the U.S. decision-making process. Barriers to effective implementation will also be reviewed. The remaining 15 minutes will be devoted to audience questions and panelist responses.

W10: IMPROVING THE USE OF COST EFFECTIVENESS MODELS BY REIMBURSEMENT AUTHORITIES: TRANSPARENCY AND QUALITY CONTROL

Hutton J1, Pang F2, Tolley K3, 1MEDTAP International, London, United Kingdom; 2Abbott Laboratories, LTD, Berkshire, United Kingdom; 3Ortho Biotech, Buckinghamshire, United Kingdom

Learning Objectives: Participants will learn: 1) how cost-effectiveness (CE) models are used by NICE and other national reimbursement authorities; 2) how CE models submitted by technology sponsors are reviewed; and 3) how CE models could be used to better advantage.
Who Would Benefit?: Applied researcher involved in CE modeling; technology sponsors submitting CE evidence to reimbursement authorities; those responsible for reviewing CE submission to NICE and similar bodies.
Workshop Description: Evidence of the CE of health care technologies is being demanded by an increasing number of countries at or near the time when new products are launched. This necessitates the use of modeling the present evidence of comparative CE. If several technologies are approached simultaneously, then separate submissions from a sponsor may use different models. In these circumstances, bodies such as NICE in England and Wales, use independent groups to review submissions, who may develop a further model on which the eventual decision is based. Access to such models by sponsors of technologies is limited, making effective contributions to the review process more difficult. Using NICE as a case study, and drawing contrasting examples from other countries, the workshop will present evidence of the way in which NICE has used sponsors’ CE models; how NICE reviews groups who have developed their models; and how the latter have been subjected to outside scrutiny. The second half of the workshop will be an interactive discussion asking for audience reaction to various proposals to improve the system for the production, review and application of modeled CE evidence used by NICE and other decision-making bodies. Ideas will be presented from the perspectives of the sponsors, analysts, reviewers and decision-makers; all of whom is hoped will be represented in the audience. The increased understanding of these needs and constraints may help influence the future development of procedures within these bodies.


Compliance Issues

W11: COMPLIANCE, ODD DAY DRUG HOLIDAY? NO, SABBATICALS! SOMETHING HAS TO BE DONE. IT’S WIN WIN WIN FOR PATIENT, PROVIDER AND PHARMA

Parkinson J, Davey P, MEMO, University of Dundee, Dundee, United Kingdom

Learning Objectives: Participants will learn that merely specifying drug compliance as a percentage of drugs that should have been taken hide many different patterns of drug holiday. That there are such variations in drug holidays is suggestive that solving non-compliance may require far greater understanding and co-operation amongst all those involved- Pharma, health care professional, provider, carriers and the patient.
Who Would Benefit?: Delegates from industry and health care providers as well as outcomes researchers will see during the workshop data, at the individual patient level, (anonymous of course) that will enable them to have a better understanding of the sheer complexity of non-compliance.
Workshop Description: That compliance is a real problem is not disputed. However despite many hundreds of research papers over the last 30 years the problem is getting worse as more patients get more medications and cost cutting measures such as 3 monthly prescriptions may only be exacerbating the problem. Additionally much of the research is contradictory as to the variables that affect compliance. The UK with its NHS (cradle to grave health care for all), Scotland with its 20-year-old unique patient identifier and Tayside a region within the Scottish NHS that has since 1993 generated a person specific file of all dispensed prescribing is uniquely able to analyze person specific behaviour. Drug holiday patterns will be shown for patients on varying numbers of medications and reasons discussed as to the possible causes of such patterns. For statin treatment the data will be linked to outcomes showing at what level patients get little or no benefit. Participants will be asked to form views on what MUST be done to alter the situation and whether or not efforts have to be co-ordinated to have any real effect.

Clinical Study Methodology Issues

W12: CLINICAL MEANINGFULNES; DEFINING, INCORPORATING AND INTERPRETING THE MCID

Brod M1, Friedman M2, Wyrwich KW3, 1The BROD Group, Mill Valley, CA, USA; 2Purdue Pharma, Stamford, CT, USA; 3Saint Louis University, St. Louis, MO, USA

Learning Objectives: The objective of the workshop is to review the current state of the art of how to calculate the MCID and provide guidelines for the incorporation and interpretation of the MCID in clinical trials.
Who Would Benefit?: Researchers who must address issues of clinical meaningfulness when using health outcomes measures as well as those who are involved in the design and interpretation of health outcomes endpoints in clinical trials should attend.
Workshop Description: Interpretability of clinical significance of PRO measures or how we assign meaning to the magnitude of change in our health outcomes is not a simple task. This workshop will review current methods for calculating the MCID and how to incorporate clinical significance into protocols and analysis plans. The role of the MCID in PRO measure development and the relationship between the patient, regulatory and physician perspective will be discussed. Unanswered questions regarding the future direction of the clinical meaningfulness debate will be raised.

W13: IN SEARCH OF AN UNBIASED ESTIMATE OF TREATMENT EFFECT USING OBSERVATIONAL DATA: A COMPARISON OF PROPENSITY SCORING AND HECKMAN TWO STAGE SAMPLE SELECTION MODELS

Martin BC1, Ganguly R2, Dorfman JH1, Rizzo JA3, 1University of Georgia, Athens, GA, USA; 2University of Mississippi, University, MS, USA; 3Cornell University, Ithaca, NY, USA

Learning Objectives: Attendees would learn about the theoretical basis, uses, comparisons between approaches and how to apply the techniques used to adjust for selection bias in health services research.
Who Would Benefit?: Health services researchers and economists who conduct observational studies using administrative claims or survey databases.
Workshop Description: Many studies using observational databases attempt to estimate treatment effects by comparing a treated and an unexposed group. In these studies, the treatment is assigned without the benefit of randomization, which frequently results in differences between the two groups on several observed and unobserved confounding covariates. The propensity scoring technique is an approach to reduce selection bias by matching individuals based on their likelihood (propensity) to be in the treatment or comparison group. However, bias due to unobservable confounders may still exist, especially in the context of administrative databases that have limited direct disease measures. The Heckman two-stage estimation technique may potentially address this concern as it controls for selection bias due to observable confounders in the first stage and may also account for potential bias due to unobservable confounders that may continue to exist in the second stage. These two approaches will be illustrated through a case study of our research where we estimate the effect of using multiple vs. single antipsychotic therapy on health care cost in schizophrenia patients where we utilize propensity scoring and Heckman two-stage sample selection models. The workshop will be structured into four modules: 1) introduction: confounding inherent in observational studies and basic approaches used to adjust for it; 2) propensity scoring; 3) heckman two-stage sample selection models; and 4) comparison of propensity scoring and Heckman two-stage models including empirical comparisons. The presentation will include a theoretical background to each approach, uses in health services research, a reference bank of related articles, a simple step by step approach to applying the approaches illustrated through data from our research including sample SAS programs.

Cost Study Methodology Issues

W14: USE OF DATABASES FOR NATURALISTIC ECONOMIC EVALUATIONS OF NEW PRODUCTS: LINKING EFFICACY WITH EFFECTIVENESS

Jansen JP1, Crawford B2, 1Mapi Values, Houten, The Netherlands; 2Mapi Values, Boston, MA, USA

Learning Objectives: Clinical trials frequently provide the first opportunity for cost-effectiveness studies. However, trials provide efficacy information on a new product and therefore do not reflect what will happen during day-to-day practice. This workshop will introduce the modeling of the expected effectiveness of a new product based on its efficacy. It will also provide a link between efficacy and effectiveness of comparative products and the application to cost-effectiveness analyses.
Who Would Benefit?: This session is directed at individuals who are applied researchers, research sponsors, and decision makers interested in the cost-effectiveness of new products in daily practice.
Workshop Description: Often clinical trials form the starting point for cost-effectiveness evaluations of new products. Hence, the cost-effectiveness predictions of these evaluations are based on efficacy data, and not on naturalistic effectiveness data. The objective of this workshop is to show how efficacy data from clinical trials can be adjusted to obtain estimates for the expected effectiveness of the new product in daily practice. In addition, participants will learn that identifying predictors for the difference between efficacy and effectiveness are a valuable tool to predict effectiveness and medical resource consumption of new products in daily practice. In the workshop the focus is on the methodological concepts, instead of detailed formulas and statistical techniques. This allows for interactive discussion of the key issues to be tackled.


SESSION III


Quality of Life Study Methodology Issues

W15: UNDERSTANDING TREATMENT SATISFACTION: THE WHO, WHAT, WHERE WHEN AND HOW OF INCORPORATING TS AS A CONCEPTUALLY SOUND AND VALID PRO

Brod M1, Skovlund S2, Atkinson MJ3, 1The BROD Group, Mill Valley, CA, USA; 2Novo Nordisk A/S, Bagsvaerd, Denmark; 3Pfizer, Kalamazoo, MI, USA

Learning Objectives: Treatment Satisfaction is increasingly used in clinical trials and marketing studies to evaluate patient reported benefits and disadvantages of new pharmaceutical products. This workshop will review the history and controversy surrounding the use of TS as a legitimate PRO, present a conceptual model that should be used to develop TS measures, examine issues of generic vs. disease/treatment specific measures and provide a framework for psychometric validation of TS measures.
Who Would Benefit?: This workshop is directed at individuals from clinical, marketing, health outcomes, health care management and academic departments who are interested in understanding the appropriate incorporation of TS into the clinical evaluation process or developing new measures of TS.
Workshop Description: TS has been linked to improved clinical outcomes and as a result is increasingly being included as an endpoint in the evaluation of new treatment regimens. Further, TS has the potential to improve clinical decision making as well as increase the quality of information provided to patients and health care professionals. However, there is currently a lack of methodological and conceptual clarity in the assessment of TS, and often TS is assessed without a conceptual framework or psychometrically sound instruments. As a result, the use of TS as a valid PRO, meeting the scientific standards for regulatory consideration and publications, is controversial and has been limited. This workshop will review the current conceptual and methodological issues for assessing TS from the perspective of the PRO researcher and the pharmaceutical industry. Attendees will learn about the conceptual basis of TS, how to use TS as an important vehicle to increase understanding of specific benefits or drawbacks of new treatments and the instrument development process that should be used to create conceptually sound and psychometrically valid TS measures. Case studies linking TS and clinical outcomes will be presented and generic as well as treatment/disease specific measures will be identified.

Health Policy/Reimbursement Issues

W16: THE IMPACT OF PARALLEL TRADE (PT) IN THE GREEK PHARMACEUTICAL MARKET

Hatzikou M1, Kontozamanis V2, Liaropoulos L3, 1Boehringer Ingelheim Elliniko, Athens, Greece; 2Foundation for Economic and Industrial Research (IOBE), Athens, Greece; 3University of Athens, Ampelokipi, Athens, Greece

Learning Objectives: The status quo of Parallel Trade, its implications in the Greek market and the effects from the governmental and pharmaceutical industry perspective.
Who Would Benefit?: European governmental participants and managers from the pharmaceutical industry.
Workshop Description: Objectives: Parallel trade (PT) in pharmaceuticals exists because identical medicinal products have varying prices in different countries. Hence, parallel traders exploit the significant difference of prices between ‘low price countries’ and ‘high price countries’ and make a profit margin. Greece, sets prices according to the lowest ex-factory applied in Europe thus, it is a source country for parallel exports. The objective of this study is to point out the current situation of PT in Greece and assess the impact in the Greek market and public health. Methods: We reviewed sales, the regulatory framework and expenditure data from various sources as well as data regarding delays of patients’ access to medicines and shortages observed in the pharmacy sector. Based on the above, we assessed the impact of PT in the Greek market both from the industries and the public policy perspective. Results: In Greece, parallel exports have increased significantly the last years and accounted for 16% of total pharmacy market in 2001. Principal beneficiaries of parallel trade are the wholesalers, who benefit from the price differential and enjoy significant profit margins. The pharmaceutical industry is not able to forecast future domestic demand due to fluctuations in the orders for products. The disturbance of the proper supply of the Greek pharmaceutical market with medicinal products has negative effects on the access of citizens to pharmaceutical treatments due to observed shortages of major products from pharmacies. Conclusions: The current price setting system of medicinal products in Greece favors parallel exports. This situation affects negatively the pharmaceutical industry, the Greek patients and the operation of the health system. The growth of PT results in the delayed launch of new products and the shortage of important and necessary medicines to the Greek patient.
 

W17: HOW HEALTH CARE CAN BENEFIT FROM CHANGING THE MINDSET: FROM COST AND EFFECTIVENESS TO COST-EFFECTIVENESS

van Loon J1, Crawford B2, Monteban HC3, Evans CJ4, 1Mapi Values, Houten, The Netherlands; 2Mapi Values, Boston, MA, USA; 3Pfizer, Capelle a/d IJssel, The Netherlands; 4Mapi Values USA LLC, Boston, MA, USA

Learning Objectives: In this workshop, various examples from real life practice will illustrate the tools that can be applied to influence behaviour change by key stakeholders. Attendees will be encouraged to participate in an interactive discussion on how to get these groups interested and what tools are available to change their mindset.
Who Would Benefit?: This session is directed at individuals who are responsible for the dissemination of economic and outcomes studies. Individuals who are responsible for performing health economic and outcomes research will also benefit from this workshop.
Workshop Description: Payers are under tremendous pressure to spend more but they must also balance this with spending constraints. Cost-effective health policy is a method applied in many countries. Cost-effectiveness is of relevance for all pertinent stakeholders in health care, from decision-makers to GPs. Day-to day experience show that parties such as GPs, pharmacists, and health insurers claim to be interested; however, their behaviour shows otherwise. Instead of completing the bridge to cost-effectiveness, decision-makers and pharmacists tend to stick to the cost side of this claimed policy. Physicians on the other hand are primarily interested in the effectiveness. These parties do not see the relevance of an efficient health policy due to institutional constraints. In this workshop, various examples from real life practice will illustrate the tools that can be applied to influence behaviour change by key stakeholders. These include: specific outcomes detailing by pharmaceutical firms (e.g., dossiers, reimbursement kits/interactive models), training, workshops, symposia and advisory boards. Attendees will be encouraged to participate in an interactive discussion on how to get these groups interested and what tools are available to change their mindset.

Compliance Issues

W18: MEASURING THE EFFECT OF INCREASED COMPLIANCE TO TREATMENT ON PREVENTION OF ADVERSE OUTCOMES USING ADMINISTRATIVE DATA

Ishak KJ1, Caro JJ2, Huybrechts KF2, 1Caro Research, Dorval, Quebec, Canada; 2Caro Research Institute, Concord, MA, USA

Learning Objectives: Participants will learn: 1) methodology to measure compliance from administrative data; 2) intricacies involved in analyzing these data, including alternative definitions for "good compliance", appropriate modeling strategies, and efficient methods to estimate model parameters; and 3) advantages and limitations of this approach.
Who Would Benefit?: Health researchers and research sponsors interested in cost-efficient methods to assess compliance and the expected benefit from improving compliance on health outcomes.
Workshop Description: Administrative data offer a cost-efficient alternative to data collection and better reflect real-world practices compared to clinical trials and even observational studies. Such data are used regularly for a variety of objectives such as describing resource usage profiles and natural history of disease. We propose a novel usage, namely, to assess compliance and its effect on outcomes. Administrative data typically include inpatient and outpatient services and medication dispensing records. We describe an adaptable algorithm that uses dispensing records and usual prescribing patterns to derive each subject’s compliance profile over the course of the study period. The output from the algorithm may be in the form of compliant days or number of pills taken over regular time intervals; we discuss the advantages and disadvantages of these and other definitions of compliance. The occurrence and timing of adverse outcomes is assessed from inpatient and outpatient service records. Logistic regression analysis may be used to study acute outcomes, while proportional hazards models are generally better suited for chronic outcomes with long follow-up periods. It is imperative to ensure concurrent timing of compliance measures relative to the outcome; otherwise, biased or nonsensical effect estimates might arise. This may be done with the use of time-dependent variables in the proportional hazards model, at the cost of relatively long run-times to estimate parameters; we present more time-efficient modeling strategies. We conclude with a discussion of advantages and limitations of the method.

Clinical Study Methodology Issues

W19: TO CASE-CONTROL OR COHORT: CAN WE CONTROL FOR CONFOUNDING BY MAKING THE RIGHT CHOICE?

Qizilbash N1, Kiri VA2, Boudiaf N2, Feudjo-Tepie MA3, 1GlaxoSmithKline, Harlow, Essex, United Kingdom; 2GlaxoSmithKline, Greenford, Middlesex, United Kingdom; 3London School of Hygiene and Tropical Medicine, London, United Kingdom

Learning Objectives: Participants will learn: 1) about nested case-control design vs cohort analysis through "real life" examples from pharmacoepidemiology; and 2) will be able to identify studies in which confounding can be encountered and decide accordingly which design to adopt.
Who Would Benefit?: Researchers using retrospective databases and/or real life data for predicting drug/disease risk/benefit.
Workshop Description: Confounding by indication/contra-indication is a major handicap in outcome research and could be leading to wrong interpretation. Review of published papers shows that the nested case-control design remains the preferred analysis of large-scale retrospective databases whereas cohort analysis should be more widely and efficiently used. The objective of this workshop is to introduce participants to the principles of cohort analysis and ways to tackle the lack of comparability of study groups. With practical examples of studies on UK General Practice Research Database and UK Mediplus database, we will demonstrate that nested case-control may exaggerate risk estimates and hence orientate the participants into making the right choices for their studies. Assessing treatment effectiveness is not an easy task therefore participants will be given the tools needed to decide which design fits best their research question and what precautions should be taken in interpreting results.

Cost Study Methodology Issues

W20: EARLY STAGE MODELLING: A FLEXIBLE TOOL TO AID DECISION MAKING FOR CLINICAL PROGRAMMES, PRICING AND REIMBURSEMENT

McGuire A1, Martin M2, 1London School of Economics, London, United Kingdom; 2Innovus Research, High Wycombe, United Kingdom

Learning Objectives: To highlight the issues and possible pitfalls in developing decision analytic models for products in early stage development, while providing practical health economic information to aid decision making for clinical trial programmes, pricing and reimbursement.
Who Would Benefit?: Pharmaceutical health economists and others involved in drug or product development interested in learning how early stage models can contribute to decisions in clinical programme designs and pricing and how best to address uncertainty surrounding model variables.
Workshop Description: Companies are increasingly under pressure to develop products that compare favourably to established drugs, in terms of efficacy and value-for-money. This challenge, combined with high drug development costs and short patent life, means that early stage modeling of the cost-effectiveness of new compounds in development has become increasingly necessary in order to provide information on the likely range of cost-effectiveness outcomes. However, this entails analyzing the impact of parameters, often using assumptions rather than evidence. Using real life examples, participants will learn how to address issues of uncertainty and how models can be developed that can evolve with the compound, incorporating new information as it becomes available. The workshop will explore examples of flexible model designs that can be used for the initial cost-effectiveness analysis of the compounds in development. It will also demonstrate, again using real life examples, how these models can aid planning and decisions regarding clinical trial programmes. Finally the workshop will address the issue of pricing and its relationship with cost-effectiveness. An outline of a typical early stage model will be developed using audience input regarding compound and variables characteristics.

W21: IS THERE A FUTURE FOR PIGGYBACK ECONOMIC EVALUATIONS?

Thompson D1, O'Sullivan A1, Drummond M2, 1Innovus Research (US) Inc, Medford, MA, USA; 2Innovus Research (UK) Ltd., and University of York, York, United Kingdom

Learning Objectives: To discuss issues surrounding the conduct of piggyback economic evaluations, including their advantages and disadvantages relative to alternative methods of economic evaluation and their future role in pharmacoeconomic research.
Who Would Benefit?: Researchers who want to gain an understanding of the relative strengths and weaknesses of piggyback evaluations, plus industry pharmacoeconomists responsible for collecting economic data alongside clinical trials.
Workshop Description: "Piggyback" evaluations, in which health-economic data are collected alongside an otherwise typical clinical trial, have certain advantages over other types of pharmacoeconomic studies. Randomization, blinding, and other elements of a trial’s experimental design can benefit the economic analysis, and it is often more practical to collect economic data alongside a trial rather than to fund a stand-alone costing study. However, the artificiality of the experimental setting and other well-documented problems, such as protocol-driven care, can undermine attempts to conduct economic evaluations alongside trials. Consequently, piggyback evaluations have been the target of attack from two camps—one claiming that economic evaluations should only be performed in appropriately designed "pragmatic" or "naturalistic" trials, the other eschewing trial-based economic evaluations completely in favor of modeling. Nevertheless, piggyback trials remain a commonly used approach in pharmacoeconomic evaluation. The lack of consensus on this issue gives rise to questions concerning the role of piggyback evaluations in future pharmacoeconomic research. This workshop will present the advantages and disadvantages of piggyback evaluations, highlight the key challenges that arise in their design and conduct, and discuss their future role. Workshop participants will initially be administered a survey designed to ascertain their preferences for and experiences with piggyback evaluations, the specific problems they have encountered, and ways in which they addressed them. The session will conclude with an interactive group discussion of survey results focused on identifying how piggyback evaluations can be performed with increased scientific rigor and relevance in the future.


SESSION IV


Health Policy/Reimbursement Issues

W22: THE PHARMACEUTICAL PRICING AND REIMBURSEMENT ENVIRONMENT IN CANADA

Palmer WN, Neale S, Palmer D'Angelo Consulting Inc, Ottawa, ON, Canada

Learning Objectives: To provide workshop participants with an overview of Canadian pharmaceutical pricing and reimbursement policies, the role of pharmaco-economics and outcomes research, an assessment of proposed reforms, and the implications of parallel trade with the United States.
Who Would Benefit?: Decision makers with responsibilities for pharmaceutical pricing and reimbursement, policy makers, and researchers conducting studies for pricing and reimbursement purposes. Pharmaceutical companies and biotechnology firms interested in their products’ market potential in Canada.
Workshop Description: The Canadian health care system is similar in many respects to European systems of government-sponsored health insurance, however both the public and private sectors play an important role in pharmaceutical reimbursement. Moreover, patented medicines are subject to strict (federal) price controls and complex (provincial) reimbursement systems, which vary from one jurisdiction to another. This workshop is designed to assist decision makers understand Canada’s pricing and reimbursement environment. We will discuss the role of the Patented Medicine Prices Review Board (PMPRB), which regulates prices of patented medicines, illustrate pharmaceutical price trends and international price comparisons, and describe the impact of government policies on prices. We will explain provincial reimbursement programs and outline the role and importance of pharmaco-economics and outcomes research in the listing decisions of the public and private drug plans. The workshop will offer an assessment of the proposed reforms of the Canadian pricing and reimbursement environment. For example, we will provide an overview of the "Common Drug Review", a new national initiative designed to promote more uniform patterns of drug reimbursement across Canada. The emergence of parallel trade (internet pharmacies, re-importation legislation) with the United States has resulted in upward pressure on Canadian drug prices as well as new distribution arrangements targeted at limiting supplies to Canadian pharmacies. In light of these and related developments, the workshop will consider the outlook for parallel trade in North America and the implications on pricing and reimbursement in Canada.

W23: DISCRETE EVENT SIMULATION FOR ASSESSMENT OF BUDGET IMPACT

Caro JJ, Migliaccio-Walle K, Lee K, Huybrechts K, Caro Research Institute, Concord, MA, USA

Learning Objectives: To contrast traditional economic evaluation and budget impact analysis and present techniques for addressing the latter.
Who Would Benefit?: Health care decision makers, policy makers and researchers who are responsible for conducting, evaluating and utilizing health economic analyses.
Workshop Description: It is becoming increasingly necessary for payers to formally evaluate the affordability of new therapies and their impact on budgets at a detailed local level. Several countries have adopted guidelines that provide the context in which these issues ought to be addressed. Nevertheless, traditional economic evaluation (e.g., cost-effectiveness) has been the method commonly employed to estimate the budget impact of new therapies. This approach, however, only addresses the question of efficiency, and does not properly evaluate affordability because its very concepts (e.g., opportunity costs, lifetime horizons, aggregated ratio measures and modeling of hypothetical cohorts) go counter to the needs of local health authorities. The question of affordability requires its own methodology. In this workshop, simulations of the use of atypical antipsychotics for the management of mental illnesses (schizophrenia and bipolar disorder) will be employed as case studies to demonstrate the use of discrete event simulation as a novel methodology for budget impact modeling. We will illustrate how the needs of decision makers translate to technical requirements for the models and how these can be met with the new technique vis a vis traditional modeling approaches. The process for implementing these models will be described and software options will be shown. We will also present the data needs and the types of output that are possible. The different, yet complimentary, approaches of economic evaluation and budget impact analysis will be contrasted to establish the basis for introducing discrete event simulation as the best approach to modeling. Specific recommendations for budget impact analyses will be presented for discussion.

Clinical Study Methodology Issues

W24: OUTCOMES RESEARCH IN SPECIAL POPULATIONS: GERIATRICS

Noe LL1, Weishaar RE2, Larson L1, Trotter JP1, 1Ovation Research Group, Highland Park, IL, USA; 2Omnicare Clinical Research, Peri-Approval & Geriatric Research (PAGeR) Unit, King of Prussia, PA, USA

Learning Objectives: Participants will gain an understanding of: 1) clinical and pharmacoeconomic issues related to drug treatment in the elderly; and 2) opportunities for implementing outcomes research programs in this special population.
Who Would Benefit?: Outcomes researchers interested in conducting outcomes research in special populations like the elderly, and those with responsibility for implementing peri-approval research programs designed to collect additional outcomes data in special populations.
Workshop Description: Clinical trials often focus on homogeneous patient populations in order to demonstrate a drug's safety and efficacy, and cost-effectiveness analyses frequently use such trial data to demonstrate product value. Rarely are these drugs studied adequately in special populations, such as children or the elderly. The world’s population is growing older, and the need for efficacy, safety, and outcomes information specific to medical treatment in the elderly is becoming increasingly important. Common problems with drug use in the elderly include noncompliance, altered pharmacokinetics, adverse drug reactions, and drug interactions; alone or combined, these issues