Issue Panels
Monday, May 18, 2015
11:00 AM - 12:00 PM
ISSUE PANELS - SESSION I
Health Policy Development Using Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon A, B, F (Level 5)

IP1: SHOULD THE NAME OF THE GAME BE MORE “SKIN IN THE GAME”? THE SCOPE & CONSEQUENCES OF RX COST SHIFTING FROM PAYERS TO PATIENTS (Invited Issue Panel)

Moderator:

Gerry Oster, PhD, Senior Economist, Policy Analysis Inc. (PAI) and Managing Co-Director, MINERVA Health Economics Network, Brookline, MA, USA bio

Panelists:

Elizabeth Hargrave, MPA, Principal Research Scientist, NORC, Bethesda, MD, USA bio

Elise Gould, PhD, Senior Economist & Director of Health Policy Research, Economic Policy Institute, Washington, DC, USA bio

A. Mark Fendrick, MD, Director, University of Michigan, Center for Value-Based Insurance Design, Ann Arbor, MI, USA bio

ISSUE:

In the US today, patients are being asked to pay an ever-increasing share of their health care costs. More “skin in the game” through higher deductibles, higher copays, and/or higher co-insurance rates, it has been argued, causes patients to become more prudent consumers. From this viewpoint, overly generous insurance coverage, which distorted prices and artificially increased demand, has been a major culprit behind health care cost inflation. Correct these distortions, so the argument goes, and patients will use health care more judiciously without compromising clinical outcomes. This line of argument depends critically on numerous assumptions, including the existence of treatment options that provide equivalent outcomes at lower cost, patients’ ability to make informed decisions about what health care to consume and what they can reasonably forgo, and the choices that consumers actually make when they face higher prices. How much more “skin” do patients have in the game today compared with the past? What do we know about the consequences of increased cost-sharing with respect to clinical outcomes, utilization, and costs? Should the name of the game be more “skin in the game”? The panel will consider these questions.

OVERVIEW:

Elizabeth Hargrave will provide an overview of important changes in pharmacy benefit design in both the public and private sectors in recent years, with an emphasis on patient cost-sharing.  Elise Gould will examine the conceptual foundations underlying increased cost-sharing and what we actually know about the impact of such policies on outcomes and costs. Mark Fendrick will examine problems with increased cost-sharing from a provider’s perspective and discuss an alternative, value-based paradigm for drug utilization and cost management.


11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon G (Level 5)

IP2: CAN WE AFFORD MEDICAL BREAKTHROUGHS FOR LARGE PREVALENCE DISEASES? LESSONS FROM HEPATITIS C

Moderator:

Dana P. Goldman, PhD, Leonard D. Schaeffer Chair & Director, Schaeffer Center for Health Policy & Economics, University of Southern California, Los Angeles, CA, USA

Panelists:

Ross Maclean, MD, Chief Operating Officer, Precision Health Economics, Los Angeles, CA, USA

Tomas J. Philipson, PhD, Daniel Levin Professor of Public Policy Studies, Irving B. Harris Graduate School of Public Policy Studies, University of Chicago, Chicago, IL, USA

Adrian Towse, MA, MPhil, Director, Office of Health Economics (OHE), London, UK bio

ISSUE:

Breakthrough medical innovation offers significant potential value to public and personal health. However, the financial burden of highly effective therapies for common diseases exerts strong budgetary pressure against generous reimbursement. Alternative financing models may be necessary to ensure the continued arrival and affordability of breakthrough innovations for high-prevalence diseases. Dana Goldman will moderate and provide an overview of the social costs and benefits of biomedical innovation. Tomas Philipson will represent the policy perspective and offer potential innovative financing models for breakthrough therapies. Ross Maclean will provide the industry perspective, and Adrian Towse will represent the health economics perspective.

OVERVIEW:

Continued advancement in the discovery of breakthrough therapies depends on sufficient rewards for the investment in drug development. A return on investment is typically accompanied by high prices potentially causing payers to curtail access to valuable new technologies. The balance between price and access is particularly problematic for treatments with long-term benefits, as demonstrated with recent breakthrough treatments for hepatitis C. While patients and payers face upfront costs of treatment, the benefits accrue over the course of a lifetime by preventing disease progression. This misalignment of the costs and benefits of a therapy results in perverse incentives for payers to limit access to patients in need. How can we better align the costs and benefits of medical breakthroughs? Can credit markets play a role in health care financing? What lessons can be learned from hepatitis C? This session will review the obstacles and potential solutions for financing current and future breakthrough therapies for large prevalence diseases.

Use of Real World Data Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon K-L (Level 5)

IP3: CHALLENGES AND OPPORTUNITIES FOR PRAGMATIC CLINICAL TRIALS – SHOULD EUROPE AND THE UNITED STATES APPROACH THEM DIFFERENTLY?

Moderator:

Rafael Alfonso-Cristancho, MD, PhD, MSc, Director, Value Evidence Analytics, Research and Development, GlaxoSmithKline (GSK), King of Prussia, PA, USA

Panelists:

Bryan R Luce, PhD, Chief Science Officer, Patient-Centered Outcomes Research Institute (PCORI), Washington, DC, USA

Tjeerd Van Staa, MD, PhD, MSc, Professor, Farr Institute, University of Manchester, Manchester, UK

ISSUE:

Pragmatic clinical trials are designed to evaluate the effectiveness of interventions in real-life routine practice conditions. Their results can be generalized and applied in routine practice settings. This is why they are so appealing to decision makers. Nevertheless, the design and implementation of Pragmatic clinical trials can be very challenging. These challenges may vary depending on the health care environment, the interventions to be compared, practice settings, and information systems available, among many others. These challenges are present in different levels in European countries and the US, mostly due to differences in the health care system settings and information systems available. Based on those specific circumstances, we will debate how European countries and the US should approach pragmatic trials, and if they should do so differently.

OVERVIEW:

After the initial background setting the stage, as described above in the issues, Bryan Luce will highlight the specific challenges and opportunities in designing and implementing pragmatic clinical trials in the US. In particular, the lessons learned through PCORI’s effort and their support in the development of pragmatic trials across health care networks in the US and highlighting specific examples. Following this presentation, Prof. Tjeerd Van Staa, will focus on the European experience with pragmatic trials; mostly focused on recent examples of studies developed in Sweden, England and Scotland. Highlighting the differences in these presentations will allow the moderator and the audience to focus on key points for the discussion with active participation from the audience.  Rafael Alfonso-Cristancho will summarize the arguments and present additional questions allowing each panelist to present their views and examples, and inviting the audience to advocate for different perspectives on the design and implementation of pragmatic trials in US and European settings. This debate can help to clarify ideal setting for specific types of studies, anticipating and overcoming common barriers.

Economic Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon H (Level 5)

IP4: WHAT IS THE VALUE OF BIG DATA IN COMPARATIVE EFFECTIVENESS RESEARCH AND CLINICAL DECISION MAKING?

Moderator:

William H. Crown, PhD, Chief Scientific Officer, Optum Labs, Cambridge, MA, USA

Panelists:

Miguel Hernan, MD, DrPH, Professor of Epidemiology and Biostatistics, Harvard T. H. Chan School of Public Health, Boston, MA, USA

Sarah Greene, MPH, Associate Director, CER Methods and Infrastructure Program, Patient-Centered Outcomes Research Institute (PCORI), Washington, DC, USA bio

Milton C Weinstein, PhD, Henry J. Kaiser Professor of Health Policy and Management, Harvard T. H. Chan School of Public Health, Boston, MA, USA

ISSUE:

How useful will Big Data be for medical decision making and health policy? Patients and physicians require causally valid evidence of patient-centered comparative effectiveness and safety to choose the best clinical strategies. Do analytic methods exist to obtain such evidence from Big Data?  If so, do we still need models, populated with information outside “Big Data”, to incorporate patient-specific factors including preferences?

OVERVIEW:

The term “Big Data” refers to massive data sets compiled from electronically-recorded transactions. Data mining approaches, when applied to consumer information, focus on prediction and not causality. When estimating treatment effects from insurance claims, clinical records, and patient/provider information, a myriad of issues introduce bias and present challenges.  One problem is that the choice of treatment may be influenced by variables that are affected by the intervention of interest.  Another is that missing data introduce bias if data are not missing randomly.  Econometric methods such as instrumental variables, statistical methods such as propensity scores, and novel causal inference methods such as the parametric G-formula and inverse probability weighting of marginal structural models, have been used to overcome bias in large observational data sets. The panel will discuss potential uses and limitations of Big Data on comparative effectiveness research, and analytic strategies that are needed to make meaningful use of it.  Dr. Crown oversees analytics for the largest health “Big Data” set in the U.S. and has applied econometric methods to reduce bias in inferences from observational data.  Dr. Hernán is an epidemiologist whose research focuses on methods to emulate randomized trials using observational data. Ms. Greene directs the development of PCORnet, envisioned as a national research data network, for the Patient-Centered Outcomes Research Institute.  Dr. Weinstein is a decision scientist and modeler who co-chaired the U.S. Panel on Cost-Effectiveness in Health and Medicine.

Patient-Reported Outcomes & Patient Preference Research Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon C, D, E (Level 5)

IP5: PROS AND BEYOND: ARE THEY FIT FOR PURPOSE TO IMPROVE HEALTH CARE POLICY AND PRACTICE?

Moderator:

Corinna Sorenson, MPH, MHSA, PhD, Senior Manager, Avalere Health, Washington, DC, USA

Panelists:

Eleanor M Perfetto, PhD, MS, Professor, Pharmaceutical Health Services Research, University of Maryland, School of Pharmacy, Baltimore, MD, USA

Kristi Mitchell, MPH, Senior Vice President, Avalere Health, Washington, DC, USA

Sachin Kamal-Bahl, PhD, Vice President & Head, Global Health & Value Innovation Center, Pfizer, Philadelphia, PA, USA

ISSUE:

Patient-reported outcomes (PROs) provide patients' perspectives on treatment benefit. As health care becomes more "patient-centered", PROs are assuming a growing role in different aspects of care. An Avalere Evidence-Based Medicine (EBM) Navigator analysis shows that from 2007 to 2014, focus on quality of life in health technology assessments grew by 34 percent. As use of PROs expands, it is important to consider whether current PRO methods and processes are "fit for purpose" and effectively inform policy and practice.

OVERVIEW:

The overarching aim of this issue panel is to explore different perspectives on the effectiveness and utility of using PROs across the health care system to date. Representatives from industry and the health policy and medical community will discuss and debate the role and use of PROs in clinical and economics studies, performance measurement, and point-of-care decision making. In particular, panelists will address the following issues: 1) challenges and opportunities of integrating PROs into different areas of health care practice and policy; 2) which PRO endpoints are most important and useful to different stakeholders; 3) levers and incentives for using PRO data in decision making; 4) collaboration between stakeholders involved in the development and use of PROs; and, 5) methodological and tactical strategies for enhancing the practical implementation of PROs in real world settings through electronic medical records, clinical decision support, social media, and/or patient registries. All presenters will participate in a dialogue facilitated by the moderator and the panel will actively engage the audience in the discussion. The panel will ideally provide insights into ways PROs can most successfully support effective, efficient, and patient-centered health care.

Tuesday, May 19, 2015
11:00 AM - 12:00 PM
ISSUE PANELS - SESSION II
Health Policy Development Using Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon A, B, F (Level 5)

IP6: HOW SHOULD THE FDA REGULATE THE COMMUNICATION OF HEALTH ECONOMIC DATA BY PHARMACEUTICAL COMPANIES TO PAYERS?

Moderator:

Peter J. Neumann, ScD, Professor & Director, Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies,Tufts Medical Center, Boston, MA, USA bio

Panelists:

Laurie Burke, MPH, RPh, Founder, LORA Group, Royal Oak, MD, USA

Joseph Jackson, PhD, Principal, Health Outcomes Insights LLC, Lavallette, NJ, USA

Albert F. Cacozza, JD, Partner, Ropes & Gray, Washington, DC, USA

ISSUE:

Section 114 of the Food and Drug Modernization Act of 1997 (FDAMA) established United States (US) regulations for the promotion of health economic information by pharmaceutical companies to formulary committees. Components of the Section have been criticized as being vague, and to date the FDA has not published any guidance, although it has indicated that a draft guidance may be published in 2015. Panelists will debate how current policy applies to pharmaceutical companies’ communications with health plans about health economic information and possible and needed policy changes from the perspectives of industry (Joseph Jackson), the FDA (Laurie Burke), and policy makers (Alan Bennett). If the agency issues new draft guidance on the matter before the ISPOR conference, panelists will debate its implications and merits.

OVERVIEW:

Although Congress passed FDAMA Section 114 nearly eighteen years ago, the FDA has yet to provide guidance on promotion of health economic claims by pharmaceutical companies to payers and other organizations that manage expenditures (e.g., Accountable Care Organizations). Neumann et. al. (2011, 2013) have shown that individuals in pharmaceutical companies’ health economics and outcomes research departments find the law confusing and hesitate to make promotional submissions under the Section. Meanwhile, an FDA official has cautioned against loosening restrictions on evidence required to support a claim. In the US and around the world, payers increasingly demand evidence of value and cost-effectiveness. The panelists will debate how which types of health care economic evidence, such as observational studies and real world data, are permissible claims under Section 114. Furthermore, the panelists will debate if and how the law should be clarified through guidance or altered through legislation. The moderator, an academic researcher who has written extensively on the topic, will facilitate dialogue among panelists and questioning from audience members.


11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon H (Level 5)

IP7: NEW MODELS OF CANCER REIMBURSEMENT: ARE THERE CONSEQUENCES FOR SPENDING AND PATIENT HEALTH?

Moderator:

Amitabh Chandra, PhD, Director of Health Policy Research, John F. Kennedy School of Government, Harvard University, Cambridge, MA, USA

Panelists:

Andrew Briggs, DPhil, MSc, William R. Lindsay Professor of Health Economics, Health Economics & Health Technology Assessment, Institute of Health & Wellbeing, University of Glasgow, Glasgow, UK

Darius N. Lakdawalla, PhD, Quintiles Chair in Pharmaceutical Development and Regulatory Innovation, School of Pharmacy, University of Southern California, Los Angeles, CA, USA

Jennifer Malin, MD, PhD, Medical Director for Oncology, Anthem, Woodland Hills, CA, USA

ISSUE:

Will alternative reimbursement models for oncology care strike the right or wrong balance between cost, quality, and patient outcomes? Dr. Chandra will moderate the discussion and provide an overview of the pressing policy issues. Dr. Briggs will provide the European perspective and contrast reimbursement for oncology care in the UK with the US fee-for-service model.  Dr. Lakdawalla will represent the innovator’s perspective, and Dr. Malin will provide the payer perspective.

OVERVIEW:

Under the existing fee-for-service (FFS) payment model for health care reimbursement, physicians are paid for the specific services they provide, without consideration of outcomes. On the other hand, measuring outcomes can be quite difficult, particularly in a disease like cancer where rates of treatment failure and disease-progression remain high, even when care is delivered optimally.  The challenge is to mitigate or eliminate incentives for overuse of expensive, ineffective treatment in an environment where outcomes are difficult to measure. In light of this challenge, organizations like the American Society of Clinical Oncology (ASCO) have recommended exploring alternative payment models to address the limitations of FFS in concert with other initiatives, including the establishment of accountable care organizations and delivery of personalized medicine. Several payers, including Anthem, have taken up this challenge by pilot-testing bundled payment or episode payment schemes that encourage providers to seek the most efficient mix of therapies. This session will compare and contrast the advantages and risks of various alternative payment models for cancer care. Specifically, bundled payment and episode payment schemes that UnitedHealth and Anthem have recently tested will be discussed, along with other hypothetical strategies for improving efficiency. Particular attention will be paid to reviewing common arguments used to support or refute different models of reimbursement for cancer care, identifying which claims can be substantiated by available evidence and which cannot.

Use of Real World Data Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon G (Level 5)

IP8: ARE WE COMFORTABLE APPLYING EXISTING QUALITY-DRIVEN ADHERENCE MEASUREMENT METHODOLOGIES TO SPECIALTY PHARMACEUTICAL PRODUCTS AND WHAT ARE THE RISKS, ESPECIALLY IF USING ADMINISTRATIVE CLAIMS DATABASES?

Moderator:

Craig Schilling, PharmD, Chair, Pharmacy Quality Alliance (PQA) Adherence Committee and Vice President, Patient Programs, Optum, Eden Prairie, MN, USA

Panelists:

Richard Faris, PhD, MSc, RPh, Co-Chair, Pharmacy Quality Alliance (PQA) Specialty Pharmacy Products Task Force and Director, HECOR, UCB, Inc., Smyrna, GA, USA

Michael Ingham, MSc, Director, HECOR, Janssen Scientific Affairs, LLC, Horsham, PA, USA

Stephen M Lund, RPh, Executive Vice President, Strategic Initiatives, Senderra Rx Specialty Pharmacy, Richardson, TX, USA

ISSUE:

Adherence measures are incorporated into CMS STARS rating programs, and have received endorsement from the National Quality Forum.  Such measures often take the traditional form of “Proportion of Days Covered” (PDC). PDC relies heavily on access to the “Days’ Supply” variable during analysis, and incorporates a component of persistence.  Specialty biologic products present an interesting challenge in measuring adherence when using claims related data, especially those products with unusual dosing regimens, multi-use dosage forms, or predominantly reimbursed as a medical benefit, where Days’ Supply is not readily available. PDC-like measures may mask very different non-adherence patient profiles, even when demonstrating similar adherence scores. This complicates understanding of the clinical importance of non-adherence, as well as the assessment of interventions to improve adherence.  So while traditional adherence measurement may not suffice when applied to specialty pharmaceuticals, there is value in standardizing and endorsing measures. Alternative methods may be necessary.

OVERVIEW:

Craig Schilling will set context and clarify definitions for the panel presentations.  Richard Faris will present PQA’s perspective on why PDC is generally their endorsed measure for adherence as laid out in the white paper by Dr. David P. Nau, and will summarize recent adherence measure endorsement within the Specialty Rx field. Michael Ingham will discuss risks and issues encountered when applying traditional measures to claims databases for specialty pharmaceuticals, including challenges to differing assumptions on day’s supply, and how to potentially improve the clinical relevance of adherence measurement of Specialty Rx.  Steve Lund will present an alternative view to capturing adherence data - directly from patients. Issues include recall accuracy, validation,  and subsequent targeting of interventions Craig Schilling will summarize the positions, reflect on the potential impact on adherence risk assessment tools, and invite the audience to advocate for different perspectives on how to standardize adherence measurement of specialty pharmaceuticals.

Clinical Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon K-L (Level 5)

IP9: CONTINUOUS PATIENT ENGAGEMENT: HOW DO WE PARTNER WITH PATIENTS THROUGHOUT THE RESEARCH LIFE CYCLE?

Moderator:

C. Daniel Mullins, PhD, Professor & Chair, Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, Baltimore, MD, USA

Panelists:

Emil Chiauzzi, PhD, Research Director, PatientsLikeMe, Cambridge, MA, USA

Daniel Frye, JD, Executive Director, NJ Commission for the Blind and Visually Impaired, Newark, NJ, USA

Marvin Mack, Chief Executive Officer, Center for Mind & Esteem Development, Baltimore, MD, USA

ISSUE:

Despite the desire for real world evidence in health outcomes research, there continue to be significant challenges in engaging diverse populations in clinical trials and comparative clinical effectiveness studies. Researchers should establish ongoing relationships with participants throughout and between research studies, but instead often limit participant involvement to the intervention and data collection stages.  We propose that meaningful patient engagement throughout the research continuum is the best way to assure that research studies authentically address patient-centered research questions. Key issues to be addressed include: (1) practical barriers in implementing such a process; (2) challenges with different research environments and patient populations; and (3) the implications of treating patients as research collaborators.

OVERVIEW:

As a framework for discussion, Dr. Mullins will present his 10-step framework for continuous patient engagement published in JAMA in 2012. Each panelist then will provide their perspective on challenges and opportunities for holistic patient engagement in clinical research. Dr. Chiauzzi will discuss the challenges and engagement strategies addressed in online research at PatientsLikeMe.  Mr. Mack will describe a holistic approach to patient engagement, drawing upon his experiences over a decade with public housing clients.  Mr. Frye will discuss how to include patients from impairment communities, drawing upon his advocacy experiences with the blind and vision-impaired community.  The moderator will summarize key similarities and differences in perspectives, highlight the implications of these perspectives, and solicit questions from the audience that address the potential for continuous patient engagement in research and partnership.

Economic Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Grand Ballroom, Salon C, D, E (Level 5)

IP10: EARLY ACCESS TO MEDICINES: WHAT IS IN IT FOR PAYERS?

Moderator:

Adam Heathfield, PhD, Senior Director, Global Health and Value Innovation Center, Pfizer, Surrey, UK

Panelists:

Adrian Towse, MA, MPhil, Director, Office of Health Economics (OHE), London, UK bio

Mark Trusheim, MS, Visiting Scientist & Executive in Residence, Sloan School of Management, Massachusetts Institute of Technology, Cambridge, MA, USA

Dan Ollendorf, PhD, Chief Review Officer, Institute for Clinical and Economic Review, Boston, MA, USA

ISSUE:

Regulatory initiatives to provide early access to medicines are proliferating across health care systems. Many different models are being developed, but earlier access is broadly supported by patients, clinicians and pharmaceutical companies. Many HTA agencies / P&T Committees acting on behalf of payers and reimbursement bodies have expressed concerns about funding new medicines on the basis of early data. For example, US payers have been critical of the FDA’s breakthrough therapies pathway. Without payer “buy in” and hence drug listing and reimbursement, companies do not have a viable commercial model for early access and patients do not get earlier access to medicines. Policy debate on early access needs to understand payer and other perspectives on the contrasting approaches embodied in initiatives such as FDA breakthrough therapy designation and EMA adaptive licensing pilots.

OVERVIEW:

Modelling to date (for example by Baird et al.) has focussed on regulator benefit risk and commercial potential. Adrian Towse will set out new modelling work showing the potential benefits (and costs) to payers of early access schemes. Mark Trusheim will explain how the MIT NEWDIGS JANUS program facilitates creative approaches acceptable to payers and others through simultaneous, multi-stakeholder impact simulations. He will also discuss the progress of the EMA Adaptive License pilots to engage payers. Dan Ollendorf will draw on ICERs extensive evidence review work for payers and its value initiative with payers to set out the concerns of payers with early access and how they could be addressed.

2:15 PM - 3:15 PM
ISSUE PANELS - SESSION III
Health Policy Development Using Outcomes Research Issues

2:15 PM - 3:15 PM
Room: Grand Ballroom, Salon A, B, F (Level 5)

IP11: THE $2.6 BILLION QUESTION: WHY ARE DRUG DEVELOPMENT COSTS RISING AND CAN WE AFFORD IT? (Invited Issue Panel)

Moderator:

Lou Garrison, PhD, Professor, Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA bio

Panelists:

Joseph A. DiMasi, PhD, Director of Economic Analysis, Tufts Center for the Study of Drug Development, Tufts University, Boston, MA, USA bio

Patricia M. Danzon, PhD, Celia Moh Professor, The Wharton School, University of Pennsylvania, Philadelphia, PA, USA bio

Adrian Towse, MA, MPhil, Director, Office of Health Economics (OHE), London, UK bio

ISSUE:

Joseph DiMasi has recently updated his landmark 2003 costs of drug development study, and now estimates an average cost of $2.6 billion per approved new compound based on a sample of new medicines first tested in humans between 1995 and 2007.  Over time, clinical success rates by development phase have declined significantly, and total capitalized cost per approved new compound grew at an annual compound rate of 8.5%.  Some industry critics are citing this as prima facie evidence that the pharmaceutical industry is unproductive and providing poor clinical value for money, while making record profits.  Yet, new medicines are still being covered and reimbursed, including some that are called “cures.”   Considering revenues as well as costs, Dr. Ernst Berndt and colleagues have recently estimated that the rate of return for new prescription drugs has fallen dramatically since 2004.  And the Office on Health Economics in UK and the Center for Medical Technology Policy in the U.S. have collaborated with a small consortium of leading pharmaceutical manufacturers to address what elements would be needed for a new, more efficient drug development paradigm.

OVERVIEW:

Joseph DiMasi will first present the methods and results from his new study, and comment on the implications for drug development.  Patricia Danzon will provide a broader economic perspective on these latest results, commenting on the theoretical basis, the empirical methodology, the data challenges, and limitations, and also discussing implications for further research and policy.   Adrian Towse will comment on his research on the need for a new drug development paradigm and the implications for R&D costs, revenues, and returns.


2:15 PM - 3:15 PM
Room: Grand Ballroom, Salon H (Level 5)

IP12: PATIENT-FOCUSED DRUG DEVELOPMENT: ARE POLICY MAKERS LISTENING?

Moderator:

John F P Bridges, PhD, Associate Professor, Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA

Panelists:

Mark Walderhaug, PhD, Associate Director for Risk Assessment, Center for Biologics Evaluation & Research, Office of Biostatistics & Epidemiology, U.S. Food & Drug Administration, Silver Spring, MD, USA

Holly Peay, PhD, Senior Vice President, Community Research, Parent Project Muscular Dystrophy, Hackensack, NJ, USA

Frank W Rockhold, PhD, Senior Vice President, Global Clinical Safety and Pharmacovigilance, GlaxoSmithKline, Cary, NC, USA

ISSUE:

 In recent years, the U.S. Food and Drug Administration (FDA) and other regulators have received mandates to account for the perspectives of patients and caregivers in decision making. In response to PDUFA V requirements, FDA has focused primarily on inviting patients, caregivers and advocacy groups to provide testimony in 20 disease-specific meetings, with the aim of increased understanding of disease severity. The path forward on how patient-focused drug development will evolve and what role qualitative and quantitative data on patient and caregiver preferences will play in drug development and in regulatory benefit-risk evaluations is yet to be determined. 

OVERVIEW:

In a facilitated discussion, panelists from three different perspectives will evaluate the strengths and weakness of current patient-focused drug development initiatives at the FDA and debate the appropriate form and influence patient and caregiver preferences should have in regulatory decision making. Mark Walderhaug will present a regulator’s perspective on engaging patients and caregivers. He will describe challenges and benefits of FDA’s current patient-engagement program and other efforts by FDA to understand and incorporate patient and caregiver perspectives. Holly Peay will provide the perspective of a patient/caregiver advocacy group and will evaluate the strengths and weaknesses of the current approach, as well as the capacity of advocacy groups to engage in preference research. Frank Rockhold will provide a perspective from industry by detailing efforts to incorporate patient and caregivers in drug development and the practical challenges for incorporating patient and caregiver preferences into the regulatory process. John Bridges will moderate the session, facilitate debate on the appropriateness and likely acceptability of patient and caregiver preferences for regulatory decisions, and invite audience participation in the discussion.


2:15 PM - 3:15 PM
Room: Grand Ballroom, Salon C, D, E (Level 5)

IP13: CAN THE EUNETHTA HTA CORE MODEL© HELP TO ENSURE AN ALIGNED CONCEPT OF VALUE BETWEEN HTA, DECISION MAKERS/PAYERS, AND PHARMA INDUSTRY?

Moderator:

Finn Boerlum Kristensen, MD, PhD, Secretariat Director, Danish Health and Medicines Authority, Copenhagen, Denmark bio

Panelists:

Melvin Olson, PhD, Head of HEOR Excellence, Novartis Pharma, Basel, Switzerland

David Shum, PharmD, MBA, Head, Market Access, F. Hoffmann La Roche, Mississauga, ON, Canada

Chander Sehgal, MD, MBA, Director, Common Drug Review (CDR) & Optimal Use of Drugs, Canadian Agency for Drugs and Technologies in Health (CADTH), Toronto, ON, Canada

ISSUE:

Payers utilize different approaches to define and assess the value of a pharmaceutical product. Currently, the concept of value is sometimes restricted to clinical elements, thereby excluding other potentially important components of value that a pharmaceutical product might bring, including organisational, social and ethical aspects. The HTA Core Model© represents a broad and comprehensive concept of value. The HTA Core Model© could enable a common “language” to discuss elements of value between manufacturers, HTA and payers/decision makers. Adopting the HTA Core Model as a “value assessment framework” could help improve the dialogue and increase understanding of what value, in a broader sense, pharmaceutical products could bring to patients as well as society. A common value assessment framework could help ensure efficiency and consistency across products under assessment by public payers and decision makers

OVERVIEW:

David Shum will outline how an adoption of the HTA Core Model© within Roche could facilitate easier internal HTA evidence and submission sharing and promotes a broader perspective of value. Melvin “Skip” Olson, will discuss the need for a broader value concept for pharmaceutical products from a manufacturer’s perspective.  Chander Sehgal will share his opinion on the HTA Core Model as a value assessment framework. Finn Boerlum Kristensen will chair the panel, summarize the arguments and invite the audience to discuss different perspectives on the usefulness of a common and broad framework for value assessment.

Use of Real World Data Issues

2:15 PM - 3:15 PM
Room: Grand Ballroom, Salon G (Level 5)

IP14: DISTINGUISHING BIOSIMILARITY – HOW CAN WE GENERATE REAL-WORLD EVIDENCE TO SUPPORT DECISION-MAKING?

Moderator:

Nancy A. Dreyer, PhD, MPH, Senior Vice President, Global Chief of Scientific Affairs, Real-World & Late Phase Research, Quintiles, Cambridge, MA, USA

Panelists:

Robert W Dubois, MD, PhD, Chief Science Officer, National Pharmaceutical Council, Washington, DC, USA

Jaclyn L Bosco, PhD, MPH, Director of Epidemiology, Real-World & Late Phase Research, Quintiles, Cambridge, MA, USA

Jeff White, PharmD, MS, Director, Drug Evaluation and Clinical Analytics, Clinical Pharmacy Policy, Anthem, Inc., Costa Mesa, CA, USA

ISSUE:

Biosimilars as a class are approved for use by showing that these products are equivalent to approved biologics, except for the active biotechnological substance and manufacturing process. Many patents for biologics are expiring soon and it is expected that the availability of biosimilars and/or copy biologics will increase across the globe. However, the different manufacturing processes for these biosimilars creates uncertainty around their safety and effectiveness. Patient registries and observational research methods provide important tools to evaluate the value of biosimilars, but there is considerable debate and uncertainty about what evidence will be needed and how to determine the value of evidence. The issues that will be addressed include interchangeability, International Nonproprietary Naming, biosimilarity of different delivery mechanisms, extrapolation of indications, and changes in reimbursement.

OVERVIEW:

Dr. Nancy Dreyer will introduce the panel and provide a brief introduction to the current landscape of biosimilars, discuss stakeholder perspectives, and highlight key regulatory decisions. TBD will represent the panel from the payer perspective and discuss the clinical and economic evidence to support recommendations on the appropriate use of biosimilars, including considerations about interchangeability. Dr. Robert Dubois will represent the panel from the pharmaceutical industry perspective and discuss the impact of these biosimilar issues on health policy and the biopharmaceutical industry. Dr. Jaclyn Bosco will represent the research perspective and discuss biosimilar issues in the context of methodological challenges in using patient registries and observational research methods to generate evidence to support stakeholder decision-making. Methodological approaches to study design options, comparison group identification, confounding, and data acquisition will also be considered. Dr. Nancy Dreyer will summarize the view points and invite the audience to advocate for different perspectives on the issues of biosimilars in generating real-world evidence.

Economic Outcomes Research Issues

2:15 PM - 3:15 PM
Room: Grand Ballroom, Salon K-L (Level 5)

IP15: THE CONTROVERSIAL ROLE OF COST-EFFECTIVENESS ANALYSES (CEA) AND INCREMENTAL COST-EFFECTIVENESS RATIO (ICER) THRESHOLDS IN VALUE-BASED ASSESSMENTS (VBA) OF HEALTH TECHNOLOGIES: WHAT ARE THE FUTURE CHALLENGES?

Moderator:

Zeba M. Khan, RPh, PhD, Vice President, Celgene Corporation, Summit, NJ, USA

Panelists:

Michael Schlander, MD, PhD, MBA, Chairman & Scientific Director, Institute for Innovation & Valuation in Health Care, University of Heidelberg, Wiesbaden, Germany

Ron Akehurst, PhD, Professor Emeritus of Health Economics, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

John Proach, MBA, Executive Vice President, Pricing and Market Access, Market Access Solutions LLC, Raritan, NJ, USA

ISSUE:

In some jurisdictions, including the UK, Canada, and Australia, CEA is the prevailing methodology used to establish the “value for money” offered by medical technologies, and thereby guiding reimbursement decisions. However, in CEA, contextual factors such as the burden of the disease, medical need, innovativeness of the therapy, and broader societal benefits remain largely unaccounted. In Germany, such factors may be considered by the G-BA in establishing the benefit rating assigned. In the UK, the value-based assessment (VBA) methodology has been challenged. Should additional factors, other than clinical efficacy and cost, be considered? The panel will address the current role and future challenges of VBA and ICER thresholds in the context of paying for “value” from the societal perspectives and country-level health-policy perspectives in the UK, Germany, and Switzerland.

OVERVIEW:

In the UK, CEA assesses incremental cost per quality-adjusted life year gained and fixed ICER thresholds of £20,000-£30,000/QALY are applied in reimbursement decisions. In Germany, IQWiG/G-BA bases reimbursement decisions on assessment of additional therapeutic benefit over a comparator for price negotiations. The ECHOUTCOME survey of 1,300 respondents suggested that NICE’s decision based on QALYs alone may not comprehensively appraise the societal value of medical technologies. Similarly, attendees of 2014 EU ISPOR supported the use of economic evaluation with increased and varying ICER thresholds by therapeutic areas and clinical unmet needs. Additionally, outcomes from a recent EU-5 HTA expert  workshop concluded that new technology evaluation should include the degree of unmet need, level of therapeutic innovation and wider societal benefits. Notwithstanding, in the ongoing pursuit and challenges of measuring “value” of medical technologies, it is important to discuss the shortfalls of current economic evaluations and propose alternative approaches and/or additional considerations  for health technology assessments. This panel will consider these issues with specific case-studies from the UK, Germany, and Switzerland.

Wednesday, May 20, 2015
8:30 AM - 9:30 AM
ISSUE PANELS - SESSION IV
Health Policy Development Using Outcomes Research Issues

8:30 AM - 9:30 AM
Room: Grand Ballroom, Salon G (Level 5)

IP16: DO STATE MEDICAID FORMULARIES WORSEN OUTCOMES FOR PATIENTS WITH MENTAL ILLNESS?

Moderator:

Seth A. Seabury, PhD, Associate Professor of Research, Department of Emergency Medicine, University of Southern California Keck School of Medicine, Los Angeles, CA, USA

Panelists:

Dana P. Goldman, PhD, Leonard D. Schaeffer Chair & Director, Schaeffer Center for Health Policy & Economics, University of Southern California, Los Angeles, CA, USA

Anupam B. Jena, MD, PhD, Assistant Professor of Health Care Policy and Medicine, Harvard Medical School, Boston, MA, USA

Darius N. Lakdawalla, PhD, Quintiles Chair in Pharmaceutical Development and Regulatory Innovation, School of Pharmacy, University of Southern California, Los Angeles, CA, USA

ISSUE:

In an effort to stem rising prescription drug costs, nearly all state Medicaid programs currently employ some form of utilization management, and psycho-therapeutic drugs have been a primary focus. While formulary restrictions are associated with decreased use of targeted medications, there is debate around whether they impose greater social costs by jeopardizing the health of patients with little social support. Dr. Seabury will moderate the panel and provide an overview of the public health issues raised by limiting access to treatments for mental illness. Dr. Goldman will represent the health policy perspective. ­­Dr. Jena will provide the physician’s perspective, and Dr. Lakdawalla will provide the health economics perspective.

OVERVIEW:

Patients with severe mental illness in the US are significantly more likely to be disabled and unable to hold a job and a large portion of them receive their health care through state Medicaid programs.  As a result, mental health drugs account for a large share of Medicaid’s formulary costs.  This has led many state programs to target these drugs with prior authorization, step therapy and other formulary restrictions in an effort to lower costs by directing patients away from brand drugs to cheaper generics. However, there is growing evidence that these policies lead to treatment disruptions that worsen outcomes for patients with mental illness, including greater health care utilization, worse disease control, greater reliance on public assistance and even higher rates of incarceration and imprisonment. On the other hand, states are under increasing budgetary pressure and are forced to find even short-term savings in response.  The panelists will offer alternative perspectives on state fiscal crises and their implications for Medicaid mental health policy.  In particular, the panelists will discuss whether formulary restrictions are necessary for cash-strapped states, or whether they lead to negative fiscal consequences on balance.


8:30 AM - 9:30 AM
Room: Grand Ballroom, Salon A-F (Level 5)

IP17: VALUE-BASED FORMULARY DESIGN (VBFD): IS PREMERA A VOICE CRYING IN THE WILDERNESS?

Moderator:

Kathleen E. Hughes, MBA, Vice President, Health Economics and Outcomes Research, Avalere Health LLC, Washington, DC, USA

Panelists:

Dan Danielson, MS, RPh, Pharmacy Manager, Clinical Services, Premera Blue Cross, Mountlake Terrace, WA, USA

Edmund J. Pazella, MD, MPH, National Medical Director, Pharmacy Management, Aetna, Inc., Hartford, CT, USA

John Graham, Pharm. D, Vice President & VEO, CVM-NS, Global Value Evidence and Outcomes - RD Projects Clinical Platforms & Sciences, GlaxoSmithKline, King of Prussia, PA, USA

ISSUE:

Payers in many other countries routinely use sophisticated value-based decision making principles when designing formularies.  However, few US payers employ this philosophy to the extent that it is being used by Premera BlueCross BlueShield.  Is Premera on the leading edge of a c-change in formulary development in the US, or is it an outlier, proceeding at a pace that will not be replicated?  Will more VBFD tools be used by payers in the US or not?  If so, what tools will these be, and how, when, and by whom will they be used?  What will be the implications for pharmaceutical manufacturers?  This panel explores trends in VBFD from multiple constituent perspectives.

OVERVIEW:

Panelist 1(moderator) will provide an overview of trends in VBFD in the US.  Panelist 2, a representative from Premera BCBS, will provide a detailed description of how his company has used the VBFD principles.  Panelist 3, a representative from a large national payer, will discuss the plans and actions of his company and attempt to summarize what other payers individually and collectively are prepared to do or are doing in the VBFD arena.   Panelist 4 will speak from the manufacturers’ perspective as to what is being done pro- and re-actively to prepare for or react to VBFD.


8:30 AM - 9:30 AM
Room: Grand Ballroom, Salon H (Level 5)

IP18: COMPETITIVE BIDDING FOR THERAPEUTICALLY-EQUIVALENT BRANDED PHARMACEUTICALS: PROMISE OR THREAT?

Moderator:

Josephine A. Mauskopf, PhD, MHA, MA, Vice President, Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA

Panelists:

David W. Miller, PhD, Senior Vice President, Global Market Access, Biogen Idec, Maidenhead, UK

Susan Hogue, PharmD, MPH, Senior Director, Value Insight and Access Strategy, Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA

ISSUE:

The introduction of a competitive bidding program resulting in an exclusive contract between a large purchaser of therapeutically-equivalent branded pharmaceuticals and the manufacturer has the goal of achieving lower prices for quality products but may also have negative impacts on society, the patient and the manufacturer.

OVERVIEW:

The pros and cons of competitive bidding and exclusive contracts for therapeutically-equivalent branded pharmaceuticals will be debated from the payer, manufacturer and economist perspectives. Examples of its use for branded pharmaceutical products in the US will be presented.  

The use of competitive bidding programs to reduce health care expenditures is already a reality both in the United States and in Europe. In the United States, competitive bidding has been recommended in recent years as one way to control unsustainable growth in health care costs. A competitive bidding program was implemented for durable medical equipment supplied to Medicare enrollees in 2013. Such programs are now being implemented for pharmaceutical products. For example, on January 1, 2015 a multi-year agreement was announced between AbbVie the manufacturer of a new treatment regimen for chronic hepatitis C and Express Scripts for exclusive placement of the new regimen on their National Preferred Formulary in return for an undisclosed discount.

Concerns that have been suggested with competitive bidding programs for pharmaceuticals include the following: 1) Quality of lower cost goods; 2) Supply limits and stock-outs from a single supplier; 3) Reduction in the number of suppliers over time resulting in reduced competition in the long run; 4) Comparability of benefits with therapeutic substitution; 5) Switching costs with choice of a sole source provider for those with chronic conditions; 6) Patient and physician concerns with limitations in their choice of treatment or required changes of treatment; 7) Long-term disincentives for research and development of new pharmaceutical products.

Economic Outcomes Research Issues

8:30 AM - 9:30 AM
Room: Grand Ballroom, Salon I (Level 5)

IP19: READY FOR RISK SHARING? CHALLENGES AND IMPLICATIONS FOR MANUFACTURERS

Moderator:

Kim White, MBA, Senior Consultant, Numerof & Associates, St. Louis, MO, USA

Panelists:

Bryan Gilpin, SM, Director, Customer Solutions & International Operations, Boston Scientific Corporation, Marlborough, MA, USA

Sherry Thornton, Vice President, Healthcare Alliances, Medtronic, Inc., Memphis, TN, USA

Don Liss, MD, Vice President, Clinical Programs and Policy, Independence Blue Cross, Philadelphia, PA, USA

ISSUE:

With spiraling costs and outcomes that consistently lag many other developed nations, the U.S. health care system is in the midst of fundamental change.  As part of this, payers have been exploring ways to shift payments (and risk) to increase accountability for cost and quality.  Thus far, most activity has occurred between payers and providers, through such mechanisms as value-based contracts and bundled pricing.  Consequently, providers are now increasingly adopting evidence-based care paths to better control their own costs and outcomes.  As pressure for “better outcomes at lower cost” continues to mount, manufacturers must understand that they face a future in which they may be asked – or even required – to take on more of the risks related to the performance and cost of their products.  However, before entering into risk sharing agreements, manufacturers must understand the challenges and implications of these agreements, including considerations for the collection of outcomes data.  In addition, manufacturers must be able to accurately assess when these arrangements are beneficial, and when they are not. 

OVERVIEW:

Kevin Conlin will highlight the current trends and future expectations of risk sharing.  In addition, Mr. Conlin will provide insight on how payers are approaching these types of agreements with manufacturers and providers.  Bryan Gilpin and Sherry Thornton will provide the audience with an interesting comparison of how different manufacturers – and different therapeutic areas – are approaching risk sharing agreements.  They will discuss the specific challenges they’re facing as well as the decision-making process for deciding whether to enter into a risk sharing agreement.  Mr. Gilpin and Ms. Thornton will also discuss the internal processes and capabilities needed to successfully execute these agreements.  Kim White will draw out unique insights from the panelists, providing the audience with rich perspectives on the key challenges and implications for manufacturers considering risk sharing agreements.

Patient-Reported Outcomes & Patient Preference Research Issues

8:30 AM - 9:30 AM
Room: Grand Ballroom, Salon K-L (Level 5)

IP20: APPLICATION OF PROMIS TOOLS IN PHARMACEUTICAL AND DEVICE INDUSTRY STUDIES: ARE WE MOVING THE WHEEL FORWARD?

Moderator:

Stacie Hudgens, MA (AbD), Managing Partner, Clinical Outcomes Solutions, Tucson, AZ, USA

Panelists:

David Cella, PhD, Professor, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

Ashley Slagle, PhD, Clinical Outcome Assessment Qualification Scientific Coordinator & Study Endpoints Reviewer, U.S. Food and Drug Administration, Silver Spring, MD, USA

Rajiv Mallick, PhD, Senior Director, Health Economics and Outcomes Research, BTG International Inc., West Conshohocken, PA, USA

ISSUE:

The NIH-sponsored PROMIS tools provide clinical researchers with instruments to assess symptoms and function applicable to a range of diseases. PROMIS tools are ‘domain-specific’, not disease-specific.  Standardization on a common domain-specific metric enables universal comparison of disease burden and treatments, and in relation to US population norms. PROMIS tools would thus appear to be uniquely suited to meet growing comparative effectiveness mandates in the US and supplement use of generic health state utility measures used globally in health technology assessments of drugs.  Yet, it is unclear how these potential advantages may be incorporated in industry setting where treatments are developed and regulated for specific indications and where there is an assumption that relevant outcomes are disease- and context-specific. Can domain-based tools be tested and incorporated in a program of research, providing the potential for more common ground in endpoint assessment across a range of diseases?

OVERVIEW:

Dr. Cella will present the IRT underpinnings of PROMIS domains and item banks, its basis in population health, and select condition-specific qualitative and quantitative research, current status of research efforts including approaches to augmentation of PROMIS measures with disease specific concepts and tools using both qualitative and IRT-based co-calibration methods. Dr. Slagle will provide a regulatory perspective on the disease state approach and how this may be accomplished with use of domain-based tools for labeling consistent with FDA PRO Guidance principles. Representing an industry perspective, Dr. Mallick will discuss creative approaches to integrating PROMIS measures in industry clinical studies, incorporating principles of ‘fitness for purpose’ for PRO tool development and use, and selective use of customized or PROMIS short forms that reflect known disease specific content and range of severity on the domain.

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