Listening Session of the Federal Coordinating Council for Comparative Effectiveness Research
The purpose of the third listening session on comparative effectiveness research (CER) was to continue to gather public input from a broad range of diverse stakeholders on priorities, concerns, and ideas about how CER can empower patients and providers and improve care for all Americans.
The 2009 American Recovery and Reinvestment Act authorized $1.1 billion for comparative effectiveness research, including $300 million for the Agency for Healthcare Research and Quality, $400 million for the National Institutes of Health, and $400 million for the Secretary of Health and Human Services to support CER. The same law also created a 15-member Federal Coordinating Council for Comparative Effectiveness Research, which will assist federal agencies in coordinating comparative effectiveness and related health services research. The Council will submit a report to Congress on these priorities and recommendations by June 30, 2009.
The Council held its first listening session in Washington, DC, on April 14, and its second listening session on May 13 in Chicago, IL. This was the third and final listening session prior to the completion and delivery of the Report to Congress.
The Council obtained public comment from a wide range of speakers. A summary of key comments and recommendations follows.
Why CER Matters
The CER Definition
The CER Prioritization Criteria
Concerns about CER
Create a Global Network
Consider The Whole Health Care System
Dissemination is Critical
Prioritize Around Vulnerable Populations & Co-Morbidities
Focus on Care Delivery
Other Specific Research Priorities
Council’s Questions to Panelists
Dr. Graham asked Drs. Grover and Fasules about the importance of clinical databases (as opposed to administrative databases), and about the diversity in their organizations’ databases. Dr. Fasules replied that with registries, as opposed to with clinical trials, you are able to capture everyone who has a particular procedure or treatment. He added that the strength is the breadth of the registry, adding that when you include a large claims database then you get a double impact. Dr. Grover concurred, adding that the Society of Thoracic Surgeons specifically collects data on race and ethnicity in order to have reliable clinical data to make treatment decisions at the individual patient level. Pressed further about whether these databases can adequately capture some of the impact of various interventions on priority populations, Dr. Fasules noted that his organization was trying to move the databases longitudinally.
Dr. Valuck also Dr. Fasules to elaborate on his two-step process for incorporating cost into the comparative effectiveness discussion. In response, Dr. Fasules noted that cost is important—but that the cost value should not be done while comparing the science or the treatment; otherwise, he said, you may not be evaluating the effectiveness of the difference in treatment.
Dr. Hunt asked Mr. Kanter how long it took him to make a treatment decision, and what tools he used to do so, once he discovered he had prostate cancer. Mr. Kanter replied that it had taken him almost two years. He said he had started with WebMD, then talked to his urologist, and then spoke with the medical director of a nonprofit cancer institute. Mr. Kanter said that the medical director recommended a new treatment protocol at another institution—and he decided to go with that despite the lack of evidence to support the decision.
Dr. Kupersmith asked Dr. Fasules to what extent studies from the cardiology databases had informed the ACC’s guidelines (versus randomized clinical trials). Dr. Fasules replied that he could not give a percentage because randomized clinical trials are the first step. He added that the ACC was starting to mine several of the databases to look at how the data can be used to affect care, and then changing the guidelines as that happens.
Ms. Tanden asked Mr. Kanter for his recommendation about how to access international CER information. Mr. Kanter replied that much of the international data that his foundation has looked as thus far is not very good. He added that some major groups in the U.S., including Kaiser, have a lot of data that they have indicated they are willing to share with his global health sharing network.
Mr. Millman asked Dr. Cuddeback about the federal investment that would be needed to help operations such as his to use data warehouses. Mr. Cuddeback said that the Council’s strategic framework, as laid out, hits on a number of areas where there are needs, including methods development and looking at methods for enhanced adoption.
Dr. Delany asked Mr. Fox what has to change in order to put in place a viable “expert patient” model. In response, Mr. Fox said that technologies and methodologies need to be developed within the health information technology systems that allow a patient to enter clinically significant information into the system. Mr. Fox added that the starting point needs to be the clinical suite, and that the doctor and patient need to work together at a very primary level with the findings coming out of comparative effectiveness research.
Dr. Kilpatrick asked Mr. Fox if he could elaborate on the concept of involving the patient in the design, selection, and process of developing CER studies. Mr. Fox responded that patients are looking to get into the game. He noted that, when given the opportunity to participate, patients want to do so. They understand their symptoms, he said, so this is really a health literacy problem.
Ms. Tanden asked Dr. Vojta whether UnitedHealth’s efforts to design insurance plans to interact with comparative effectiveness research was something unique to that company or a new arena. Dr. Vojta replied that UnitedHealth did a great deal of qualitative and quantitative research before it rolled out its personalized benefit design plan, which reduces out-of-pocket costs for people with diabetes in exchange for compliance with American Diabetes Association standards.
Dr. Conway asked Drs. Cuddeback and Roberts about how the U.S. government can fund both R01 work as well as the types of studies that are broader than a single question (perhaps addressing registries, translation, adoption, and infrastructure). In response, Dr. Cuddeback talked about the value of the data themselves, including the ability to take observational data that are the byproduct of providing care and essentially raising the standards for those data. He added that there was value in funding the process of using existing real-world data and, with the understanding that the process itself may raise the quality of the data, improving the usability of the data and the inferences that can be drawn from that data. Dr. Roberts added that there were two types of research that need to be funded in this arena: the application of methods to particular problems that are not traditional basic science and the development of strong, rigorous methodologies that are advancing the field.
Dr. Emanuel asked Dr. Buckley what the structure might be should his organization create a CER program. Dr. Buckley replied by talking about the downsides of randomized clinical trials, and said that he would like to see a large, upfront investment in methodological issues. He also talked about the importance of incorporating all stakeholders into the process, including patients. Finally, Dr. Buckley talked about the need to focus on how the totality of the health care system (including care delivery and insurance benefit design) impacts patient care and outcomes.
Dr. Graham asked Dr. Fox what he would include in a true patient-provider partnership. Dr. Fox replied that what was needed was some kind of systematic measurement of the effectiveness of the communication between the clinician and the patient as CER is rolled out.
Dr. Clancy asked Dr. Roberts about the tension between standardization (i.e., RCTs) and continued methodological innovation. In response, Dr. Roberts noted that many involved in science have learned a lot not from randomized controlled trials but from thoughtful observations with rigorous underlying theory. He noted that there was some concern with the concept of certification in this area, in that certification itself assumes an understanding of the methods well enough to determine what an appropriate certification would be. Dr. Roberts added that the true wisdom isn’t the trial that says 10 percent died on therapy A, but rather to understand why it happened and whether the subjects would have fared better on therapy B.
Dr. Clancy asked Dr. Lerner to elaborate on the concept of a national patient library and how it might be distinguished from MedlinePlus and other existing online resources. Dr. Lerner noted that a lot of the existing information is repurposed information (with lots of science and statistics) that is not really usable by the general public. He added that the information should be purpose-built for use by consumers in conjunction with their physicians.
Dr. Delany continued on the same theme, noting that he thought that a lot of CER would be needed just to design the system. He suggested that it would likely need to be an iterative process, because a foundational set of research studies would be needed first. Dr. Lerner noted that the first step could be a significant planning process that looks at what exists, where the gaps are, and what would be needed to fill them.
Dr. Kilpatrick asked Drs. Carr and Stewart how they would approach the issue of medical literacy, and involving patients in the process, when there isn’t yet a lot of evidence-based data [on trauma and emergency care processes]. Dr. Carr replied that the first step in looking at planning for emergency care is to recognize that it must be done from a population perspective (and not from a hospital-based individual perspective). He added that the second issue is that this is a discussion of undifferentiated complaints—people who don’t know what’s wrong with them—and that this is not the emphasis of most of the medical literature.
Dr. Millman asked Drs. Carr and Stewart for their recommendations for priorities for CER in the areas of trauma and emergency care. In response, Dr. Stewart said that hemorrhages, resuscitation, infection, disaster preparedness, burns, and traumatic brain injury were all high on his list. Dr. Carr added that, from a systems standpoint, shared infrastructure is critical.
American College of Cardiology
American College of Emergency Physicians & Society for Academic Emergency Medicine
American Urological Association
Anceta – AMGA’s Collaborative Data Warehouse
Biotechnology Industry Organization
Eunice Kennedy Shriver NICHD Maternal Fetal Units Network
Fundamental Clinical Counseling, LLC
Institute for Health Technology Studies
Joseph H. Kanter Family Foundation
National Association for Rare Disorders
National Center for Patient Interactive Research
National Trauma Center
Society for Medical Decision Making
Society of General Internal Medicine
Society of Thoracic Surgeons
Universal American Corporation
Presenters During the Open-Comment Period
American Society of Anesthesiologists
Care Management Technologies
Executive Intelligence Review
Foundation for Environmentally Triggered Illnesses
National Research Center for Women and Families
Agency for Healthcare Research & Quality
Centers for Medicare and Medicaid Services
Department of Defense
Health Resources & Services Administration
National Institutes of Health
Office of the Assistant Secretary for Planning and Evaluation
Office of Management and Budget
Office of Minority Health, HHS
Office of the National Coordinator, HHS
Office of the Secretary, HHS
Substance Abuse and Mental Health Services Administration