Comparative Effectiveness Research

 CER Definitions

In their interviews about what impact trials like the CATT study could have on policies and innovation, experts from around the globe shared their definitions of CER.

Louis Jacques, MD: "I would define it as a paradigm for a member of a defined population, with a particular outcome in mind. That research is being used to identify management strategies that would be more or less likely to be successful in that population. And while some people essentially explicitly consider cost in that, cost does not have to be necessarily included in a comparative or comparative effectiveness research paradigm."

David O. Meltzer, MD, PhD: "CER is research that compares alternatives, and those alternatives can be viewed through a variety of perspectives. One perspective is the patient’s perspective. It’s the easiest to understand from the standpoint of a single patient – “What’s best for me?” But, often we have to make decisions that affect many groups of patients. Or we have to make decisions for individuals that are based on information that isn’t ideally personalized for them, but puts them within a class of people that are like them. That can often make it difficult to define that patient perspective. But that’s just the start, CER can be considered in terms of policies that effect populations of people. It can be considered in terms of decision by healthcare systems and providers who may have complicated objective functions who certainly include patients, but may include providers and cost, or even profits. Also, from a societal perspective there are costs and benefits to many, many parties as a result of medical decisions, and good social policy requires thinking about those. To me those all include the benefits of treatments, as well as their cost.” 

Kalipso Chalkidou, MD, PhD: “Comparative effectiveness research is basically a means of informing decisions based on evidence...It’s about informing consumers, and informing those that pay for care and those that offer care, about what the most appropriate treatment pathway is given their individual circumstances...Right now, the alternative to CER is basically having very little or no information. Comparative effectiveness can offer valuable information on what works and for whom."

Kevin Frick, PhD, MA: “Comparative effectiveness research is a relatively new buzz word. It has some important distinctions from earlier ways of making comparisons between alternative treatments. First and foremost, and probably the biggest one, is that we’re always aiming to compare multiple, viable treatment options; rather than comparing something with a placebo, or comparing something with nothing—unless nothing is a treatment option that’s really viable—we always want compare two viable options. And we also want to compare them in as close to a real-world setting as possible. We want to dig down and figure out what does it do for specific sub-populations if that’s possible...So all of those things are important to consider. And one thing that’s not included in say the U.S. Federal Government definitions of comparative effectiveness, but I think goes hand-in-hand with comparative effectiveness, is also looking at cost in order to say what kind of value are we getting for what we are spending, and not just comparing two different treatments against each other.”

Chris Henshall, PhD:“I think the most important thing about comparative effectiveness research, or relative effectiveness research as we tend to call it in Europe, is that it’s looking at effectiveness in the real world, in the setting in which treatments are actually given, and trying to compare effectiveness between real treatment options."

William W. Li, MD: “I actually think that the Institute of Medicine has given one of the best definitions for CER, or Comparative Effectiveness Research, and their definition is quite effective. And that definition is that CER is the generation and synthesis of evidence that compares benefits and harms of alternative medicine to prevent, diagnose, treat, and monitor diseases or to improve the delivery of care. And according to the Institute of Medicine, CER’s stated purpose is to assist consumers, clinicians, purchasers, and policymakers to make informed decisions that will improve healthcare both on the individual level, as well as the population level. In other words, CER is an approach to health research that helps us compare different methods for treating the same disease.”

Maureen G. Maguire, PhD: “I would define CER as comparison of two or more approaches to treatment in broad populations under very general conditions. A little bit differently than the typical clinical trial used to establish efficacy of treatment.”

Barbara McLaughlan: "I think the definition is in some ways quite straight forward because it is comparative effectiveness research—so it really does what it says…which means that it’s supposed to provide robust evidence to allow clinicians and patients to choose between treatments. But obviously, there’s also a major issue in that it also gives an evidence base to payers to decide on the effective use of their limited resources."

Dan Ollendorf, MPH, ARM: “I believe that CER in its best light should be considered an aid to decision-making. Because while it involves at times some sophisticated methodology to try to synthesize the available evidence, at its heart, what it attempts to do is to summarize that evidence in a way that will make cognitive comparisons of disparate interventions for a given condition feasible.”

Alexander Ommaya, DSc, MA: “I think that the Institute of Medicine definition which was put out in their report last year is sort-of straightforward and gets to the point, which is that evidence that compares the benefits and harms of alternative methods to prevent, diagnose, and treat, and monitor clinical condition or to improve the delivery of care, describes what comparative effectiveness research is trying to do pretty well.”

Sean Tunis, MD, MSc: “I think of CER as a way of looking at clinical and health services research that is more heavily weighted to addressing questions of patients, clinicians, and payers. So, my view is that traditionally researchers are somewhat informed about and conscious of what are the critical gaps and knowledge from the perspective of what decision-makers actually deal with…[T]he view is that if decisions makers were more involved upfront in deciding which questions to answer and the method by which those questions would be answered, there would be fewer gaps in knowledge and so a better body of evidence-informed decisions. It so happens that the kinds of questions that decision makers are the most interested in are often comparative questions, i.e. what are the plausible alternative ways of treating or diagnosing a condition. That’s why you frequently will see them as comparative questions. And the effectiveness piece refers to studies that are done in the real-world settings that are likely to be relevant to what people actually encounter in their own clinical situations and policy situations.”

Cynthia Bens: "I consider CER to be research that compares the effectiveness of two or more types of interventions to determine which provides better care outcomes for certain patients. These interventions can be pharmacologic, non-pharmacologic, or interventions that improve systems of care.  I also like to include elements of the Patient Centered Outcomes Research Institute’s definition of PCOR into the way I view CER. I think one of the most important elements of the PCOR definition is the recognition of the need 'to incorporate a wide variety settings and a diversity of participants in research to address individual differences.' This is a critical point that will allow us to focus on groups of patients who have historically been left out in research for various reasons. Ultimately I think the PCOR definition allows patients to really play an active role in defining research that will help achieve health outcomes that are important to them."

Gail Hunt: "CER is health care research that compares different options that a patient and caregiver could have in regard to multiple treatments. For example: deciding if you need knee-replacement shots and comparing the alternatives; steroid shots, physical therapy, knee-replacement, and comparing those and saying what are the rates of successful outcomes. I also think that CER is not necessary just limited to comparisons of the effectiveness of treatments (medication, surgical procedures, etc), but also includes comparisons of alternative ways of involving patients in shared decision making, alternative ways of reaching out to hard to reach communities with information about healthcare, or comparing alternative ways of organizing the healthcare delivery system so that it’s in tune with what patients and caregivers need."

 Expert Views

These interviews from experts from around the globe explore comparative effectiveness research, the CATT trials, and what impact these and future trials could have on policies, innovation, and patient access.

Louis Jacques, MD, joined CMS in 2003 and has been director of the Division of Items and Devices in the Coverage and Analysis group since 2004.  Read his full bio here. 

Note that this discussion represents the views of Dr. Louis Jacques, MD, and does note necessarily represent the views of CMS or the U.S. government.  No statement should be construed as an official position of CMS.

"I think CATT showed the value of public investment in an important research question that was probably not going to be addressed in the private sector."

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Hon. Tony Coelho, is a former United States congressman from California, and primary author and sponsor of the Americans with Disabilities Act.  Read his full bio here.

"Medical decisions should never be determined by clinical trials by the government or insurers, I think they should be determined by the doctor with the patient."

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Kalipso Chalkidou, MD, PhD, is the director of the policy consulting arm of NICE (National Institutes for Health and Clinical Excellence), responsible for managing and developing consulting services for international clients drawing on the Institute's experience of using evidence to inform health policy and practice.  Read her full bio here.

"The question is not whether you have perfectly personalized information, or whether you have less perfectly personalized information.  The question is really whether you have some information or have no information.  Right now, the alternative to CER is basically having very little or no information."

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Kevin Frick, PhD, MA, is a health economist who has been on the faculty of the Johns Hopkins Bloomberg School of Public Health for nearly 15 years.  He is a professor in the Department of Health Policy and Management.  He has taught about and performed cost-effectiveness research and other research comparing costs and outcomes of care or interventions for most of his career.  Read his full bio here.

"If we can get the same main impact with no worse side effect profile and spend two orders of magnitude less on it, by all means, I think everyone would agree that would be a better buy...I think the main thing people are waiting to see is can we spend a lot less money without having to give up on clinical outcomes, or are we really back in the same situation that we oftenare, which is thinking about whether or not to make trade-offs and how to make trade-offs as we spend more money to get what would presumably be a better clinical outcome from the most expensive product."

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James Goodwin, PhD, is the Head of Research at Age UK, the new organization which resulted from the merger of Age Concern England and Help the Aged.  The mission of Age UK is to improve the lives of older people.  James holds a visiting professorship at Loughborough University in the Department of Human Sciences.  Read his full bio here.

"It is my personal opinion that the decisions I see made by NICE, would be met with stupefaction by people in the United States."

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Chris Henshall, PhD, is the Chair of the Health Technology Assessment international (HTAi) Policy Forum for the period of 2010 to 2013, and is an Associate Professor in the Health Economics Research Group at Brunel University and an Honorary Fellow in the Centre for Health Economics at the University of York in the UK.  Read his full bio here.

"If you want to be a successful innovator, you need to think to yourself very hard about what a health system is likely to want to pay for, and not assume that they're going to pay for everything that you want to sell them."

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William W. Li, MD, is the President, Medical Director, and Co-Founder of the Angiogenesis Foundation, and has been changing the face of modern medicine for 25 years, by tackling a common denominator of disease called angiogenesis, or new blood vessel growth.

"At the end of the day, CATT respresents one early milestone, and an important study that's going to be used as a case study for comparative effectiveness, or CER research.  And one of the more interesting policy questions is, since the NIH provided funding for the CATT study, should the NIH be funding even more CER studies?"

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Maureen G. Maguire, PhD, is the Carolyn F. Jones Professor of Opthalmology and Vice Chair for Clinical Research in the Department of Ophtalmology at the University of Pennsylvania.  She has served on and chaired severeal data and safety monitoring committees and is on the editorial board of the journal of Opthalmology and Cornea.  Read her full bio here.

"I think patients and the public need to weigh the differential costs of the drug, with the safety profile, with the number of injections that are required, and think about which treatment approach is best for them.  And what might be best for the public funders of healthcare."

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Barbara McLaughlan, is the Policy & Campaigns Manager at the Royal National Institute of Blind People (RNIB).  At RNIB, McLaughlan has played a critical role in the successful campaign to ensure that patients with wet age-related macular degeneration are given treatment with new anti-VEGF treatments on the NHS.  Ms. McLaughlan is also the Chair of Patients involved with NICE (PIN), an independent group made up of patient and voluntary organizations that work closely with nice.  Read her full bio here.

"From a patient's perspective, if I were a patient I would always say 'I want the licensed treatment because that's gone through all the trials and I can be absolutely sure about what I'm getting, plus there is monitoring of outcomes and possible adverse events following licensing and use in clinical practice.' So from a patient's perspective it's always better to get licensed treatments."

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Dan Ollendorf, MPH, ARM, is Chief Review Officer for the Institute of Clinical Economic Review (ICER.  In this capacity he is responsible for the conduct of systematic reviews of the comparative effectiveness of new or emerging health care technologies, as well as the coordination and oversight of the broader health technology assessmentprocess.  Read his full bio here.

"In terms of policymakers, I think the concern is...what sorts of other head-to-head trials should be conducted in similar situtations where there may be an older stand-by or a product that has kind of been subsumed by the process of innovation, but may actually in fact still have relatively high effectiveness versus a comparative treatment."

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Alexander Ommaya, DSc, MA, is the Director of Translational Research at the Department of Veterans Affairs.  In this role he is responsible for managing development of new research activities focusing on health systems improvements, genomic medicine, text analysis of the VA electronic health record, CER, traumatic brain inury, and PTSD.  Read his full bio here.

This discussion represents views of Alexander Ommaya, DSc, MA, and does not necessarily represent the views of the Department of VA or the US government.  No statement should be construed as an official position of the Department of VA.

"It's always difficult to chang existing practice patterns, and even when the data is published in high-profile scientific journals, like the New England Journal, it doesn't by itself necessarily change practice."

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Sean Tunis, MD, MSc, is the Founder and Director of the Center for Medical Technology Policy in Baltimore, MD.  CMTP's main objective is to improve the quality and relevance of clinical research by providing a neutral forum for collaboration amongst experts, stakeholders, and decisionmakers.  Read his full bio here.

"I think the results of the CATT trial raised a lot of nuances about what questions are  useful to study, how to study them, and how you might use the results."

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David O. Meltzer, MD, PhD, is the Chief of the Section of Hospital Medicine, Director of the Center for Health and the Social Sciences, and the Chair of the Committee on Clinical and Transational Science at the University of Chicago, where he is an associate professor in the Department of Medicine, Department of Economics, and the Harris School of Public Policy Studies.  Read his full bio here.

"Off label use is common, and I think we allow it from a societal perspective because, if we limit ourselves only to things for which strong evidence exists, we will miss opportunities to do benefit."

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Cynthia Bens, is the Vice President of Public Policy at the Alliance for Aging Research in Washington, DC.  In this capacity, Ms. Bens is responsible for guiding the organization's federal policy work.  Read her full bio here.

"...[i]f CER results are poorly communicated, the availability of more information could lead to confusion for patients on what choices are the right ones for them and also confusion for providers on how they should treat their patients"

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Gail Hunt, is the President and CEO at the National Alliance for Caregiving, a non-profit coalition dedicated to conducting research and developing national programs for family caregivers and the professionals who serve them.  Read her full bio here.

"I think CER is a wonderful opportunity for patients and caregivers to get invovled in the research.  Those families and caregivers shoudl be involved in helping to work on the reserach, involved in helping to think through what the results mean, how they can best be disseminated, and help in the dissemination.  Which is an opportunity they haven't had before."

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 CER

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Other Leading Articles and Resources

Analysis of Comparative Effectiveness
RAND Compare
Overview covers nine performance dimensions against which comparative effectiveness may be measured: spending, waste, patient experience, coverage, operational feasibility, consumer financial risk, reliability, health, and capacity. 

CER Inventory
The Partnership to Improve Patient Care provides this inventory of federally sponsored comparative effectiveness research projects.

Comparative Effectiveness Research: Health Policy Brief
Health Affairs | October 5, 2010
This helpful overview looks at the issues, the history, the law, the key concerns, and next steps.

Comparative Effectiveness Research and Medicare: Gail Wilensky's View
Health Affairs Blog | February 2, 1011
Gail Wilensky of Project Hopereacts to a proposal from Steven Pearson and Peter Bach proposing a new Medicare payment model incorporating CER.

Comparative Effectiveness Research and Medicare: Sean Tunis' View
Health Affairs Blog | February 2, 2011
Sean Tunis of the Center for Medical Technology Policy shares his thoughts on a proposal from Pearson and Bach proposing a new Medicare payment model incorporating CER.

Comparative Effectiveness: How Health Plans and Employers Will Translate Evidence Into Practice
Naomi Aronson, BCBS | September 14, 2010
In her presentation at The Second Annual Comparative Effectiveness Summit, Dr. Aronson looks at how CER improves the evidence for decision-making and how it's actually used in those decisions.

Comparative Effectiveness Resource Center
ECRI Institute
The ECRI Institute provides an educational site intended to increase understanding of this complicated topic. Resources include position statements by stakeholder groups, national policy conference recordings, and perspectives from leading experts.

Finding What Works in Health Care: Standards for Systematic Reviews
Institute of Medicine | March 23, 2011
The IOM consensus report recommends standards for systematic reviews of the comparative effectiveness of medical or surgical interventions.

HTA 101: Introduction to Health Technology Assessment
National Library of Medicine | January 2004
A detailed and comprehensive primer on HTA by Clifford S. Goodman of the Lewin Group.

Health Policy By Comparison
CQ Health Beat | August 16, 2010
Rebecca Adams offers an analysis of the integration of CER into the health care system. She provides case studies on the effects of CER and details about PCORI.

How CER Could Pay for Itself--Insights from Vertebral Fracture Treatments
NEJM | April 13, 2011
Elshaug and Garber explored the history of procedures to treat pain and disability associated with osteoporotic-vertebral fractures, finding that rigorous CER questioned the benefits and safety of the procedures. Reducing the procedures by 80% would save $725 million annually.

How Medicare Could Use Comparative Effectiveness Research in Deciding on New Coverage and Reimbursement
Health Affairs | February 2011
Steven Pearson of the Institute for Clinical and Economic Review and Peter Bach at Memorial Sloan-Kettering Cancer Centerpropose a payment model that incorporates CER to encourage Medicare to pay equally for services that provide comparable patient outcomes.

How Best to Engage Patients, Doctors, and Other Stakeholders in Designing Comparative Effectiveness Studies
Health Affairs | October 2010
Through a qualitative assessment of case studies, the authors identify five key principles for effectively engaging a broad coalition of stakeholders in CER research intended to improve health care and control costs--1)ensure balance among participating stakeholders, 2) get participants to buy into the process as well as understand their roles, 3) provide neutral experts to facilitate research discussions, 4) establish connections among participants, and 5) keep them engaged throughout the process.

Identifying and Eliminating Roadblocks to Comparative-Effectiveness Research
NEJM | July 8, 2010
Experts involved in the CATT trial discuss some of the roadblocks that the trial brought to light.

Lessons for Health Technology Assessment: It Is Not Only About the Evidence
Value in Health | September 2, 2009
Peter J. Neumann of Tufts Medical Center explores the role of non-evidentiary factors in the differences in HTA systems around the world. 

The Patient-Centered Outcomes Research Institute--Promoting Better Information, Decisions, and Health
A. Eugene Washington, MD and Steven H. Lipstein, MHA for NEJM | September 2011
PCORI's board chair and vice chair describe the efforts of the board and methodology committee to build a world-class, patient-centered research institute from scratch.

The State of Comparative Effectiveness Research and the Environment for Decision Making
National Pharmaceutical Council | April 2011
A survey of stakeholders explored where they believe we are today with CER.

What is Comparative Effectiveness Research
AHRQ
The Agency for Healthcare Research and Quality (AHRQ) discusses the CER research process, the practical benefits of this research, and whether or not it can be trusted.

What is Comparative Effectiveness Research and What Will It Mean for Patients, Physicians, and Payors?
CMTP | July 30,2010
This whitepaper, distributed at the CMTP conference in July, focuses on various aspects of CER, addresses questions on the differences between CER and traditional clinical research, and methods by which CER should be carried out.

Cost Effectiveness

Comparative Effectiveness Research: From Clinical Information to Economic Incentives
Health Affairs | October 2010
James C. Robinson writes about "the mechanisms through which new evidence actually will influence patient practice, patient preference, and manufacturer investment."

Health Reform and Cost Cutting
The New York Times | October 19, 2010
David Leondhart discusses the potential role of CER cost cutting in the new health care law.

How Medicare Could Use Comparative Effectiveness Research in Deciding on New Coverage and Reimbursement
Health Affairs | October, 2010
Steven Pearson and Peter Bach propose a payment model for Medicare that would incorporate CER to encourage Medicare to pay equally for services that provide comparable patient outcome.

How Much is a Life Worth?
BBC News | August 24, 2010
Health economist Zack Cooper talks about the tough job of making coverage decisions based on cost and attempts to explain why NICE rejected cancer drug Avastin.

The Impact of CER on Health and Health Care Spending
Tomas J. Philipson | Septebmer 14, 2010
At The Second Annual Conference on Comparative Effectiveness, Philipson gave this presentation on the predicted effects of CER on cost-effectiveness of care, a case study for Medicare coverage for antipsychotics, and a wish list for PCORI.

Is the United States Ready for QALYs?
Health Affairs | October 2009
Peter Neumann and Dan Greenberg review the debate and use of QALYs in other countries, and the prospect of using them in the U.S.

The Role of Costs In Comparative Effectiveness Research
Health Affairs | October 2010
Alan Garber and Harold Sox argue that investigating the comparative value of interventions is necessary for decision makers who are attempt to raise quality of care while reining in health spending.

Shorter Lives, Less Prosperity: The Impact of Comparative Effectiveness Research on Health and Wealth
CMPI | May 2011
John Vernon, PhD, and Bob Goldberg, PhD explore the potential costs of CER.

Use of Comparative Effectiveness Research in Drug Coverage and Pricing Decisions: A Six Country Comparison
The Commonwealth Fund | July 2, 2010
Corinna Sorenson of the London School of Economics examines the use of CER in six countries—Denmark, England, France, Germany, the Netherlands, and Sweden.

CER Policy Organizations

Agency for Healthcare Research and Quality(AHRQ)
The Agency for Healthcare Research and Quality's (AHRQ) mission is to improve the quality, safety, efficiency, and effectiveness of health care for all Americans. As 1 of 12 agencies within the Department of Health and Human Services, AHRQ supports research that helps people make more informed decisions and improves the quality of health care services.

Center for Medical Technology Policy (CMTP)
CMTP is a private, non-profit organization that provides a neutral forum in which patients, clinicians, payers, manufacturers, and researchers can work together to design and implement prospective, real world studies to inform health care decisions. The primary goal of CMTP is to improve the process for generating reliable and credible information about the real world risks, benefits, and costs of promising new medical technologies.

ECRI Institute
Nonprofit organization dedicated to “bringing the discipline of applied scientific research to discover which medical procedures, devices, drugs, and processes are best, all to enable you to improve patient care.”

EUnetHTA
Supports effective HTA collaboration in Europe. Creation of collaboration was a response to a request from the EU Commission and EU Member States in the Work Plan 2009 of the Health Programme to continue fostering the development of HTA in Europe. Meant to inform policy decisions and connect public national HTA agencies, research institutions, and health ministires—enabling an exchange of information and support of policy decisions by member states. Over 60 partners—reflects government focused vision for the usefulness of HTA.

Health Technology Assessment international (HTAi)
HTAi is the global scientific and professional society for all those who produce, use, or encounter HTA. HTAi embraces all stakeholders, including researchers, agencies, policymakers, industry, academia, health service providers, and patients/consumers, and acts as a neutral forum for collaboration and the sharing of information and expertise. With members from 59 countries and six continents, HTAi is a thriving global network.

International Society for Pharmacoeconomics and Outcomes Research (ISPOR)
ISPOR promotes the science of pharmacoeconomics and outcomes research and facilitates the translation of this research into useful information for healthcare decision-makers to ensure that society allocates scarce health care resources wisely, fairly, and efficiently.

Institute for Clinical and Economic Review (ICER)
ICER's mission is to lead innovation in comparative effectiveness research through methods that integrate considerations of clinical benefit and economic value. Through a unique collaboration with patients, clinicians, manufacturers, insurers and other healthcare stakeholders, ICER develops tools to support patient decisions and medical policy that share the goal of achieving maximum value for every healthcare dollar.

Partnership to Improve Patient Care (PIPC)
PIPC exists to advance proposals for comparative effectiveness research (CER) that are focused on supporting providers and patients with the information they need, improving health care quality and supporting continued medical progress.

 Global CER

Use of Comparative Effectiveness Research in Drug Coverage and Pricing Decisions: A Six Country Comparison
The Commonwealth Fund | July 2, 2010
Corinna Sorenson of the London School of Economics examines the use of CER in six countries—Denmark, England, France, Germany, the Netherlands, and Sweden.

Comparative Effectiveness Research: International Experiences and Implications for the United States
Academy Health | July 2009
This report looks at how CER programs operate in other countries and how they address important policy issues.
 

Austria

Health Care Systems Road Map--Pharmaceuticals
ISPOR
An overview of the Austrian health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.
 

Denmark

Health Care Systems Road Map--Pharmaceuticals
ISPOR
An overview of Denmark’s health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.
 

France

Health Care Systems Road Map—Pharmaceuticals
Health Care Systems Road Map—Medical Devices and Diagnostics
ISPOR
An overview of France’s health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.

Haute Autorité de Santé
The French National Authority for Health’s Evaluation Committee for Medical Products is an independent public body that makes coverage and pricing recommendations to the Ministry of Health.
 

Germany

Health Care Systems Road Map
ISPOR
An overview of Germany’s health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.

Germany's IQWiG Calls for Wider Availability of Comparative Effectiveness Data
The Pink Sheet Daily | October 14, 2010
Proposals in Germany to prioritize more use of CER to cut drug costs prompted IQWiG (Germany's cost-effectiveness body) to call for the data to be made more available to doctors and patients.

Institute for Quality and Efficiency in Health Care (IQWiG)
Germany’s CER body which serves an advisory role, making coverage and pricing recommendations to the Federal Joint Commission and the Ministry of Health.
 

Italy

Health Care Systems Road Map—Pharmaceuticals
ISPOR
An overview of Italy’s health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.
 

The Netherlands

Health Care Systems Road Map—Pharmaceuticals
ISPOR
An overview of the Netherlands’ health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.
 

United Kingdom

Health Care Systems Road Map—Pharmaceuticals
Health Care Systems Road Map—Diagnostics
ISPOR
An overview of the UK’s health care delivery system with a focus on the reimbursement and pricing approval processes for pharmaceuticals, medical devices, and diagnostics.

National Institute for Health and Clinical Excellence (NICE)
NICE provides guidance, sets quality standards, and manages a national database to improve people’s health and prevent and treat ill health in the United Kingdom.

From Research to Health Care Practice: How the UK Uses Data on Comparative Effectiveness
JAMA | September 8, 2010
A look at how the National Institute for Health and Clinical Excellence (NICE) develops comparative effectiveness data, assesses it, and translates it into policy.
NICE: How Does it Work and What Are the Implications for the U.S?
National Pharmaceutical Council | May 2008
This studies authors--Sorendon, Drummond, Kanavos, and McGuire--examine key learnings from NICE, and overview of its operations, and implications for CER in the U.S.
 

United States

AHRQ's Role in Advancing CER: State-of-the-Art Methods and Data Sources
Carolyn Clancy, AHRQ | June 15, 2010
In her presentation a Lewin Group symposium on CER, AHRQ Director outlines AHRQ's role in CER.

CER Inventory from the Partnership to Improve Patient Care (PIPC)
PIPC
Comprehensive source for federally sponsored CER.

GAO Announces Appointments to New Patient-Centered Outcomes Research Institute (PCORI) Board of Governors
GAO | September 23, 2010
The U.S. Government Accountability Office announced the appointment of 19 members to the Board of Governors for the new Patient-Centered Outcomes Research Institute.

The Implications of Comparative Effectiveness for Medicare
Center for Studying Health System Change | September 14, 2010
In her presentation at The Second National Comparative Effectiveness Summit, Elizabeth Docteaur discusses CER and Medicare and how the latest CER initiatives could impact Medicare policies.

 AMD Trials

The AMD Model

The wAMD Trials

CATT Trial
United States
The Comparisons of Age-Related Macular Degeneration Treatments Trials. CATT is funded by the NEI.

GEFAL
France

IVAN Trial
United Kingdom
A randomized controlled trial of alternative treatments to inhibit VEGF in age-related choroidal neovascularization. IVAN is funded by the Health Technology Assessment (HTA) Clinical Trials programme of the UK National Institute for Health Research (NIHR).

LUCAS
Norway

MANTA Trial
Austria

VIBERA Trial
Germany
Prevention of Vision Loss in Patients with Age-Related Macular Degeneration by Intravitreal Injection of Bevacizumab and Ranibizumab.
 

The Results

Bevacizumab verus Ranibizumab--The Verdict
NEJM | April 28, 2011
The official CATT results are discussed.

CATT in the Eye of the Beholder: Top government officials react to comparative study
The RPM Report | June 10, 2011
NIH’s Collins, AHRQ’s Clancy, and FDA’s Woodcock offer different reactions to the government-run comparative study pitting Lucentis against off-label Avastin for macular degeneration.

CATT Study Raises More Questions Than It Answers
Macular Degeneration Foundation | May 2011
The Macular Degeneration Foundation comments on the recent CATT results.

Cheaper Roche Drug As Good as Lucentis
Reuters | April 27, 2011
The results of the CATT trial could have a big financial impact on Roche.

Genentech Statement on CAT Trial Data Published in the New England Journal of Medicine
Genentech Newsroom |April 28, 2011
Genentech releases a statement on the CATT results.

New Answers On Macular Degeneration: Important Questions for Comparative Effectiveness Research
Health Affairs Blog | May 6, 2011
Steve Pearson, Director of the Institute for Clinical and Economic Review (ICER) shares thoughts on CATT.

NIH Study finds Avastin and Lucentis are Equally Effective in Treating Age-Related Macular Degeneration
NIH | April 28, 2011
The NIH reports on CATT trial results which will be released in NEJM on May 1, 2011.

Preparing for the CATT Study: Will Makena controversy have an impact?"
IN VIVO Blog | April 27, 2011
As the CATT results loom, will the situation surrounding pre-term labor drug Makena have an impact?

Royal College of Ophtalmologists' Statement on the CATT Study
April 5, 2011

Statement from the American Academy of Ophthalmology Regarding the Results of the Comparison of AMD Treatment Trials
American Academy of Ophthalmology | April 28, 2011
AAO comments on the results.

Test of Lucentis for Macular Degeneration Said to Show Success
The New York Times | April 27, 2011
Speaking on condition of anonymity, two people familiar with the results of the CATT trial said that regular injections of Avastin and Lucentis resulted in vision changes at the end of a year that were essentially the same.

Ranibizumab and Bevacizumab for Neovascular Age-Related Macular Degeneration
NEJM | April 28, 2010
The much anticipated CATT results.

UPDATE: NIH Study: Lucentis, Avastin Equally Effective for Eyes
The Wall Street Journal | April 28, 2011
An updated reporting on the CATT results.
 

Educational Resources on AMD

AMD Health Corner
Alliance for Aging Research
Patient and physician resources include brochures, quizzes, and a resource guide.

The National Eye Institute AMD Resources
Health information on AMD from the NEI includes patient brochures, AMD animations, statements from the NEI, and other helpful links.

The Silver Book: Vision Loss
Alliance for Aging Research
This unique almanac of references spotlights the impact of chronic disease on our aging population and health care system, and the significant impact medical breakthroughs will have on future health care use and expenditures. The Vision Loss Volume looks at vision loss generally, and highlights data on age-related macular degeneration (AMD), diabetic retinopathy, and glaucoma--which along with cataract are the eye diseases that disproportionately affect older Americans.

AMD and Vision Loss Organizations

Alliance for Aging Research
The Alliance is the nation’s leading non-profit organization dedicated to supporting and accelerating the pace of medical discoveries to vastly improve the universal experience of aging and health. Our website includes a number of resources on AMD at the AMD Health Corner.

AMD Alliance International
AMDAI strives to bring knowledge, help, and hope to individuals and families around the world affected by AMD.

American Macular Degeneration Foundation
AMDF is committed to the prevention and cure of macular degeneration and offers hope and support to those afflicted and their families.

Association for Macular Diseases
The Association offers support to individuals with macular disease, their caregivers, and the professional community.

Foundation Fighting Blindness
The FFB’s mission is to drive the research that will provide preventions, treatments, and cures for people affected by retinitis pigmentosa (RP), macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases.

Macular Vision Research Foundation
The Foundation’s mission is to find the cause, prevention, treatment, and cure for macular vision diseases with the goal of saving sight and providing public education, advocacy, and support to those with macular degeneration.

Macular Degeneration Foundation
MDF is dedicated to those who have and will develop macular degeneration. They offer this growing community the latest information, news, hope and encouragement.

Macular Degeneration Partnership
The mission of the partnership is to provide comprehensive, easily understood, and up-to-the-minute information about macular degeneration for everyone who needs it.

National Alliance for Eye and Vision Research
NAEVR is a non-profit advocacy organization comprised of a coalition of 55 professional, consumer, and industry organizations involved in eye and vision research. It’s goal is to achieve the best eye and vision care for all Americans through advocacy and public education for eye and vision research sponsored by the NIH, NEI, and other federal research entities.

National Eye Institute
As part of the federal government's NIH, the NEI’s mission is to "conduct and support research, training, health information dissemination, and other programs with respect to blinding eye diseases, visual disorders, mechanisms of visual function, preservation of sight, and the special health problems and requirements of the blind."

Prevent Blindness America
Founded in 1908, Prevent Blindness America is the nation's leading volunteer eye health and safety organization dedicated to fighting blindness and saving sight. Focused on promoting a continuum of vision care, Prevent Blindness America touches the lives of millions of people each year.
 

AMD Treatments and Research Developments

Additional Genes Associated with Age-Related Macular Degeneration Identified
National Eye Institute | April 12, 2010
Results of a large-scale collaborative study supported by the NEI were published in the Proceedings of the National Academy of Sciences. This press release highlights the findings, including the identification of three new genes associated with AMD.

AMD Treatments – Overview
A quick introduction to the available treatments for both wet and dry AMD—from AMD Alliance International.

Use of Retinal Procedures in Medicare Beneficiaries From 1997 to 2007
Archives of Ophthalmology | October 2010
Analysis of Medicare claims data reveals marked changes in the use of several retinal procedures between 1997 and 2007, particularly in treating macular degeneration and retinal detachment.

 Innovation

Valuing Innovation

Balancing Act: Comparative Effectiveness Research and Innovation in U.S. Healthcare
NEHI | April 20, 2009
The National Network for Health Innovation provides a framework for designing and implementing a federal CER program that will sustain innovation in healthcare.

Comparative Effectiveness Research: Effect on Pharmaceutical Innovation, Value of Health, and Longevity
CMPI | May 2011
John Vernon, PhD, and Bob Goldberg, PhD, explore the potential impact of CER on innovation, health, and even the length of people's lives.

Comparative Effectiveness Research (CER) and Innovation: Policy Options to Foster Medical Advances
National Institute for Health Care Reform | October 5, 2010
By promoting effective therapies, CER could increase rewards and incentives for beneficial innovations in medicine. However, it could also dampen the development of new, potentially effective therapies by creating additional hurdles for innovators. This paper explores the steps that could help ensure that CER encourages beneficial innovation.

Framing the Debate-Untangling the Potential Impact of Comparative Effectiveness Research on Innovation
Avalere Health | November 2009
This paper explores potential impacts of CER on pharmaceutical investments and innovation.

The Kennedy Study
NICE
Commissioned by Professor Sir Ian Kennedy, this study aims to respond to the concerns surrounding innovation.

Medical Innovation: Promises and Pitfalls
The Brookings Institution | Winter 2003
Alan M. Garber and Victor Fuchs discuss explain the dynamic relationship between spending and innovation.

Progress at Work: The Value of Innovation PIPC A resource from the Partnership to Improve Patient Care (PIPC) that explores innovation and CER.

Promoting Biomedical Innovation and Economic Value: New Models for Reimbursement and Evidence Development
Brookings | April 22, 2010
The Engelberg Center for Health Care Reform and the Leonard D. Schaeffer Center for Health Policy and Economics at USC hosted an event where key stakeholders discussed how to promote a system that fosters biomedical innovation and promotes economic value in the delivery of health care. Speakers included Dana Goldman, Mark McClellan, Newt Gingrich, Peter Orszag, and more.

Should U.S. Import U.K. Model for Medicare and Medicaid?
Forbes.com | October 5, 2010
Tomas J. Philipson discusses why controlling costs is important, but shouldn’t come at the expense of future innovations.

The Silver Book: Chronic Disease and Medical Innovation in an Aging Nation
Alliance for Aging Research
This almanac of statistics on the burden of chronic disease and the value of medical research in reducing burden, shares highlights from studies and reports that help make the case for innovation.

Use of Comparative Effectiveness Research in Drug Coverage and Pricing Decisions: A six-country comparison.
The Commonwealth Fund | July 2, 2010
Corinna Sorenson provides information on the role and use of cost effectiveness research in coverage and pricing decisions in Denmark, England, France, Germany, the Netherlands, and Sweden.

Balancing Act: Comparative Effectiveness Research and Innovation in U.S. Healthcare
NEHI | April 20, 2009
The National Network for Health Innovation provides a framework for designing and implementing a federal CER program that will sustain innovation in healthcare.

Comparative Effectiveness Research: Effect on Pharmaceutical Innovation, Value of Health, and Longevity
CMPI | May 2011
John Vernon, PhD, and Bob Goldberg, PhD, explore the potential impact of CER on innovation, health, and even the length of people's lives.

Comparative Effectiveness Research (CER) and Innovation: Policy Options to Foster Medical Advances
National Institute for Health Care Reform | October 5, 2010
By promoting effective therapies, CER could increase rewards and incentives for beneficial innovations in medicine. However, it could also dampen the development of new, potentially effective therapies by creating additional hurdles for innovators. This paper explores the steps that could help ensure that CER encourages beneficial innovation.

Framing the Debate-Untangling the Potential Impact of Comparative Effectiveness Research on Innovation
Avalere Health | November 2009
This paper explores potential impacts of CER on pharmaceutical investments and innovation.

The Kennedy Study
NICE
Commissioned by Professor Sir Ian Kennedy, this study aims to respond to the concerns surrounding innovation.

Medical Innovation: Promises and Pitfalls
The Brookings Institution | Winter 2003
Alan M. Garber and Victor Fuchs discuss explain the dynamic relationship between spending and innovation.

Progress at Work: The Value of Innovation PIPC A resource from the Partnership to Improve Patient Care (PIPC) that explores innovation and CER.

Promoting Biomedical Innovation and Economic Value: New Models for Reimbursement and Evidence Development
Brookings | April 22, 2010
The Engelberg Center for Health Care Reform and the Leonard D. Schaeffer Center for Health Policy and Economics at USC hosted an event where key stakeholders discussed how to promote a system that fosters biomedical innovation and promotes economic value in the delivery of health care. Speakers included Dana Goldman, Mark McClellan, Newt Gingrich, Peter Orszag, and more.

Should U.S. Import U.K. Model for Medicare and Medicaid?
Forbes.com | October 5, 2010
Tomas J. Philipson discusses why controlling costs is important, but shouldn’t come at the expense of future innovations.

The Silver Book: Chronic Disease and Medical Innovation in an Aging Nation
Alliance for Aging Research
This almanac of statistics on the burden of chronic disease and the value of medical research in reducing burden, shares highlights from studies and reports that help make the case for innovation.

Use of Comparative Effectiveness Research in Drug Coverage and Pricing Decisions: A six-country comparison.
The Commonwealth Fund | July 2, 2010
Corinna Sorenson provides information on the role and use of cost effectiveness research in coverage and pricing decisions in Denmark, England, France, Germany, the Netherlands, and Sweden.