Nov 30, 2012
Mom always told you that you were a unique and special person, right? But, can being unique put you at risk for not getting the medical care that’s best for you? On November 30, health care stakeholders came together to break down the "myth" of the average patient and explored how health policy decisions can impact patient care.
NPC President Dan Leonard kicked off the conference by thanking sponsors National Health Council and WellPoint, along with 17 cosponsors representing diverse health care organizations.
Leonard also outlined the main goals of the conference, which included:
Understanding how biology, genetics, demographics and/or individual preferences may lead to clinically important differences among patients.
Exploring the extent to which these variations exist and should be considered in developing treatment decisions, practice guidelines, and/or coverage and reimbursement policies.
Understanding the benefits and limitations of subgroup analyses to predict which treatments are best for individual patients.
Assessing how failure to individualize care may contribute to poor clinical outcomes and health disparities.
- Considering how to engage, educate and train your constituents in support of individualized patient care.
To view speaker presentations (PDF format), please click on the speaker's name. Presentations are not available for all speakers.
7:30 a.m. Registration Open and Continental Breakfast
8:00 a.m. Welcome and Introduction
- Dan Leonard, MA, President, National Pharmaceutical Council
- Marcus Wilson, PharmD, President, Healthcore, Inc.
8:15 a.m. Patient Perspective
- Myrl Weinberg, FASAE, CAE, President, National Health Council
8:45 a.m. Keynote Presentation on Patient Centeredness
- Joe V. Selby, MD, MPH, Executive Director, Patient-Centered Outcomes Research Institute (PCORI)
9:30 a.m. Framing Presentation
- Robert (Bobby) W. Dubois, MD, PhD, Chief Science Officer, National Pharmaceutical Council
10:00 a.m. One of These Folks Is Not Like the Other: Variability Among Patients and Study Findings
(Moderator: Myrl Weinberg, FASAE, CAE, President, National Health Council)
- Getting It Right the First Time: Can We Predict Who is Likely to Respond?
- David M. Kent, MD, MSc, Associate Professor of Medicine, Neurology, Clinical and Translational Science (CTS), Tufts Medical Center; Director of the CTS MS/PhD Program, Sackler School of Graduate Biomedical Sciences, Tufts University
- Patient Differences: Biological and Non-Biological Factors?
- C. Daniel Mullins, PhD, Professor, Pharmacoeconomics, Pharmaceutical Health Services Research Department; Associate Director, Center on Drugs and Public Policy; University of Maryland School of Pharmacy
- Heterogeneity and Health Disparities: How Patient Preferences Impact Outcomes
- David Meltzer, MD, PhD, Associate Professor of Medicine and Economics, and Public Policy; Director, Center for Health and the Social Sciences, University of Chicago
11:30 a.m. Luncheon With Keynote Presentation
- Patrick H. Conway, MD, MSc, Chief Medical Officer, Centers for Medicare and Medicaid Services; Director, Office of Clinical Standards and Quality
1:00 p.m. Deciphering Heterogeneity for Health Care Decision Making
(Moderator: Marcus Wilson, PharmD, President, Healthcore, Inc.)
- Jennifer S. Graff, PharmD, Director for Comparative Effectiveness Research, National Pharmaceutical Council
- Daniel C. Malone, RPh, PhD, Professor of Pharmacy and Director of Pharmaceutical Policy, Center for Health Outcomes and PharmacoEconomic Research, University of Arizona College of Pharmacy
- Paul Martino, Senior Vice President of Clinical Strategy and Innovation, WellPoint, Inc.
- Lewis (Lew) G. Sandy, MD, Senior Vice President, Clinical Advancement, UnitedHealth Group
2:30 p.m. Break
2:45 p.m. Issues and Opportunities on the Journey to Individualized Care
(Moderator: Robert (Bobby) W. Dubois, MD, PhD, Chief Science Officer, National Pharmaceutical Council)
- Sharon Allison-Ottey, MD, Executive Director and Director of Health and Community Initiatives, The COSHAR Foundation
- C. Daniel Mullins, PhD, University of Maryland School of Pharmacy
- Ellen V. Sigal, PhD, Founder and Chairperson, Friends of Cancer Research
- Myrl Weinberg, FASAE, CAE, President, National Health Council
3:30 p.m. Summary and Adjournment
- Dan Leonard, MA, President, National Pharmaceutical Council
National Health Council President Myrl Weinberg, FASAE, CAE, outlined the importance of recognizing individual treatment effects when choosing among health care treatments. She explained that part of the challenge is that many patients do not understand what comparative effectiveness research is, which can make it difficult to engage patients in the development of research. Patients and caregivers who do learn about CER have concerns that findings could be used to block access to treatment options. Developing usability criteria to guide the research process will help ensure findings serve patients - the true focus of the health care system, Weinberg said.
Patients, Research and Access to Health Care
Myrl Weinberg, FASAE, CAE, president of the National Health Council, says that it's important to for individuals, especially those with chronic conditions, to have access to the right health care, which will result in better health outcomes. She also encourages patients to get involved early on in the research process.
Ms. Weinberg spoke at "The Myth of Average: Why Individual Patient Differences Matter," a conference focusing on issues related to individual treatment effects.
Keynote Presentation on Patient Centeredness
Dr. Joe Selby, executive director of the Patient-Centered Outcomes Research Institute (PCORI), said that the law creating his agency requires research to take individual treatment effects into account. Specifically, "research shall be designed, as appropriate the potential for differences in the effectiveness of health care treatments, services, and items as used with various subpopulations..."
A Conversation With PCORI's Executive Director Dr. Selby
National Pharmaceutical Council President Dan Leonard recently sat down with Dr. Joe Selby, executive director of the Patient-Centered Outcomes Research Institute to talk about some of the agency's recent activities. In this wide-ranging conversation, Dr. Selby details recent efforts to engage patients in crafting research questions, the agency's accomplishments in 2012, and takes a look at what to expect during the next year.
Joe Selby - PCORI methodology standards include requirement that study pops represent spectrum of pt population. #myth2012
Q&A: Spkr from Natl Genome Ctr at HowardU at the mic: Variation @ heart of the genome, opp to use large data sets to understand #myth2012
"As you increase the number of subgroups you analyze, you zero in on the individual." -@pcori president Joe V. Selby, MD #myth2012
NPC Chief Science Officer Dr. Robert Dubois debunks the myth of the "average" patient and explains the concept of heterogeneity, or individual treatment effects, and why researchers need to take it into account when conducting studies. Dr. Dubois says that researchers must consider the potential causes of heterogeneity; payers must consider how to handle it when making coverage decisions; and patients must consider how they can utilize these studies in making treatment decisions.
One of These Folks Is Not Like the Other: Variability Among Patients and Study Findings
Even when researchers pour over large numbers of studies, it can be difficult to parse out which subgroups will experience different treatment responses. A panel of researchers explored some of the challenges, including the variable quality of past studies, the variety of environmental and biological factors involved, the need for large numbers of research subjects, and the role of self selection in outcomes.
Factors Affecting Patient Responses to Therapies
Dr. C. Daniel Mullins, Professor, University of Maryland School of Pharmacy, explains some of the biologic and non-biologic factors that can affect patient responses to therapies, as well as how to determine which of those factors might be the main cause. He also discusses potential policy implications when considering how to address individual treatment effects.
Individual Treatment Effects and Randomized Controlled Trials
David M. Kent, MD, MSc, associate professor of Medicine, Neurology, and Clinical and Translational Science at Tufts Medical Center, describes the limitations of randomized control trials in identifying individual treatment effects. To offset these limitations, he recommends researchers routinely risk stratify their trial populations since risk modeling is a fundamental determinant of the opportunity for treatment benefit.
Luncheon With Keynote Presentation
As research defines exactly who is mostly likely to benefit from specific therapies, Dr. Patrick Conway, chief medical officer of the Centers for Medicare & Medicaid Services (CMS), said it is important to develop processes that ensure patients in a given population receive the treatment shown to be effective in that subgroup. Companion diagnostics offer one promising opportunity to help identify which patients are likely to have better outcomes with a specific treatment, he said.
CMS' Conway on Individual Treatment Effects
National Pharmaceutical Council President Dan Leonard sat down with Dr. Patrick Conway, chief medical officer at the Centers for Medicare and Medicaid Services and director of the Office for Clinical Standards and Quality. During their conversation, Dr. Conway discusses how his agency is thinking about individual treatment effects when making coverage decisions, as well as the evidence they consider.
Deciphering Heterogeneity for Health Care Decision Making
Payers want to create nuanced policies that address varying patient responses to treatments, but that can be challenging in an environment where scientific evidence of these individual differences is still emerging. A panel of payers and comparative effectiveness research policy experts discussed strategies that are being developed to ensure patients and health care professionals have the flexibility to access needed treatment options and personalized care.
A Payer Tool for Considering Individual Treatment Effects
Dr. Jennifer Graff, director of Comparative Effectiveness Research at the National Pharmaceutical Council, explains that payers are often faced with making coverage decisions using research based on population averages rather than individual treatment effects. She describes a new tool developed by NPC and Precision Health Economics to help payers consider the need for coverage flexibility, along with whether the health plan accommodates what works best for an individual as well as the general population.
Payer Views of Individualized Health Care
Dr. Daniel Malone, professor at the University of Arizona School of Pharmacy, explains how payers view the issue of individualized therapy. He says that while payers have a good understanding of population-based analyses they need more tools to drill down to the patient level and tailor approaches to managing individual patient needs.
Payers and Individual Patient Needs
Paul Martino, SVP of Clinical Strategy and Innovation at WellPoint, Inc., explains that it’s important for payers and other health care stakeholders to understand individual patients’ health care needs. He says that WellPoint has been working on a variety of programs to address this issue, and that they have seen improvements in health care outcomes as a result of these programs.
Issues and Opportunities on the Journey to Individualized Care
Patients are hungry for more information about how they will likely respond to a given treatment and what potential side effects they might experience, according to a panel of patient advocacy experts. Although researchers are making progress in mapping some biomarkers, more evidence is still needed to help patients and health care professionals navigate the decision-making process. To address these needs, panelists agreed patients must remain at the forefront of the comparative effectiveness research conversation.
Cancer and the Importance of Individual Treatment Effects
Dr. Ellen Sigal, Chairperson and Founder of Friends of Cancer Research, explains the importance of individual treatment effects, especially for cancer patients. She says that ultimately, they need treatments that work for them the first time around.
We spoke with Dr. Sigal during "The Myth of Average: Why Individual Patient Differences Matter," a conference focusing on issues related to individual treatment effects.
Dr. Allison-Ottey on Individualized Patient Care
Dr. Sharon Allison-Ottey, executive director of the COSHAR Foundation, explains that when it comes to ensuring optimal care for patients, all of medicine must be individualized. It is also very important, she notes, to share health care information in a way that patients and their providers can understand and act upon it.
We spoke with Dr. Allison-Ottey during "The Myth of Average: Why Individual Patient Differences Matter," a conference focusing on issues related to individual treatment effects.
Summary and Adjournment
The event’s panels and presentations gave stakeholders new insights into both the potential and limitations of the research data that is available today. As it becomes easier to obtain large data sets, NPC President Dan Leonard said there will be new opportunities to address patients’ most pressing questions. Patients and caregivers are looking for ways they can be engaged in the research process and weigh in on the direction studies take.
@NPCNow President Dan Leonard wrapping up the panel: It takes big data to ask small questions. #Myth2012 #CER
NPC@npcnow TY to all who joined today’s tweeting on #Myth2012 @PCORI @CancerResrch @NHCouncil @MiriamETucker @ThinkWellPoint @IAmBiotech
Paul Martino of @WellPoint Lewis Sandy of @UHG_AARP_LA & Jennifer Graff of @npcnow at #Myth2012 http://pic.twitter.com/E0K1Skfx