Listening Session of the Federal Coordinating Council for Comparative Effectiveness Research

Introduction

The purpose of this meeting was to gather input from a broad range of diverse stakeholders on how the Council should approach its mission and to obtain public input on priorities, concerns, and ideas on how this comparative effectiveness research (CER) can empower patients and providers and improve care for all Americans. 

An analysis of comparative effectiveness is simply a rigorous evaluation of the impact of different options available for treating a given medical condition for a particular set of patients, according to the Research on the Comparative Effectiveness of Medical Treatments Report released in 2007 by Congress.

Such a study may compare treatments, drugs, technologies, or other approaches, and the analysis may focus only on medical risk and benefits, costs and benefits, effectiveness, appropriateness, or other measures.

CER provides information on the relative strengths and weakness of various medical interventions. This information is essential for clinicians and patients to decide on the best treatments, and it also enables our nation to improve the performance of the health system.

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 the agencies of the Federal Government, including HHS and the Departments of Veterans Affairs and Defense, in coordinating comparative effectiveness and related health services research.

Although the Council will not recommend clinical guidelines for payment, coverage, or treatment, it will consider the needs of populations served by Federal programs as well as opportunities to build and expand on current investments and priorities. The Council will submit a report to the President and Congress on these priorities and recommendations by June 20, 2009.

The Council will hold two additional listening sessions over the next 6 weeks and also will be taking online written comments at least until the end of May 2009.

Panel Recommendations

Public comment was obtained from 3 consecutive panels, each consisting of roughly 10 individuals. Each panel member was allowed 3 minutes to provide comments and recommendations. The Council then had the opportunity to ask questions. Following the last panel, a period of open public comment was extended to as many speakers as time allowed. Below are some of the most salient recommendations from the panelists.

Stakeholder involvement

• Patients need a defined road in the CER process. There needs to be transparent processes and venues for patient voices to be heard. The Council should consider:
  • Developing a national citizen’s advisory board, which could be modeled after the United Kingdom’s NICE Citizens Council model (www.nice.org.uk)
  • Establishing an explicit channel for patients to advise HHS on CER, which could be modeled after the FDA Patient Representative Program (http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/PatientInvolvement/ucm123858.htm)
  • Establishing an HHS Office on Patient Values and Inclusion to ensure patients assist in all aspects of developing the systems to deliver therapies for the major medical problems we face as a nation
  • Incorporating all health community stakeholders in the governance
• The Council should establish procedures for meaningful and ongoing public input and invite opportunity for public comment on any draft reports of recommendations

Transparency

• The Council should commit to full openness and transparency
• The Council also should describe criteria and rationale for any recommendations it makes and priorities for CER investments. Priority setting should be explicit so that people can trace backwards from results to inputs to satisfy themselves that the process was fair

Encourage collaboration

• Encourage collaboration at the Federal, State and local levels early and often. Also collaborate early and often with policymakers
• Federal Government must draw together data from various Agencies including those covering public health, behavioral health, labor, housing, criminal justice, defense, and veteran’s affairs. The same collaboration should be required from their State agency counterparts
• Encourage public/private collaborations on the design, conduct, analysis, and reporting of CER

Prioritize agenda

• The scope, selection, and prioritization of research are critical. The agenda should be prioritized focusing on areas that have major clinical significance and the greatest impact on health care delivery in the U.S. Preventive care and behavioral health interventions should also be included
• Priorities should focus on medical conditions with the greatest impact on morbidity and costs (these are most often chronic conditions). CER should encompass all health care services used to treat those conditions including prescription drugs, medical and surgical procedures, diagnostics, medical devices, and health care delivery methods. The research should also include insurance benefit designs

Define effectiveness broadly

• Effectiveness should be defined broadly and be inclusive in scope. In other words, quality, safety, and efficiency of care should be considered independently so that there are no tradeoffs between studies
• Consider using the Institute of Medicine’s definition of evidence-based practice which combines three factors: best research evidence, best clinical experience, and consistency with patient values

Use and develop current data sources

• Use as many currently available data as possible including
  • Phase IIIb and IV studies
  • Electronic health record systems
  • Data systems developed by regional health improvement collaboratives
  • Health care claims databases
  • Administrative databases
  • Databases of various Government health plans such as CMS and VA
• A coordinated effort to link currently isolated public and private databases has the potential to generate an unprecedented amount of information for a variety of research activities and could be a useful hypothesis-generating tool
• With these and other new data sources expanding the number of observations and the richness of clinical detail, new analytic methods to aid valid inference from observational research should be a high priority
• Consider using the Perioperative Nursing Data Set (PNDS), a standardized language for the documentation and evaluation of care in surgery in operating rooms of our nation’s hospitals and ambulatory surgery centers

Address subpopulations

• It is important that various treatments be compared for specific subpopulations including the working age population, women, minorities, diabetics, children, and people with comorbid conditions
• CER must take into consideration the need for personalized care. Findings should not overlook special subpopulations that may benefit from a particular treatment or neglect the needs of patients for whom the findings are not necessarily supportive
• Consider CER of multiple treatment options available for those with chronic, debilitating and life-threatening conditions. This could help educate individuals on their options and better equip them for the decisions they make with their physicians regarding the best course of action in treatment
• Clinical trials generally underrepresent older people as well as people with multiple chronic illnesses. The former is particularly true for people over 75. Therefore, results of clinical studies that focus on younger populations cannot be presumed to be applicable to older adults who have reduced kidney function, multiple chronic illnesses, and may already be taking five or more medications
• Consider drug safety. CER often focuses on drug effectiveness, but for older adults, an important issue is drug safety. In some cases, a medicine that is considered less effective in the general population will be preferred for older adults because it is less toxic and has a lower risk of producing adverse effects and drug interactions

Develop infrastructure and research

• Build a strong quality improvement infrastructure at the regional level building on the regional health improvement collaboratives and the charter value exchanges. Funding could be used to maintain and expand patient registries, pool claims databases and health information exchanges, and to expand the comparative performance reporting they do for providers, researchers, and the public
• Support research to inform clinical care delivery and the development of delivery system reforms
• Extend research training and the disciplines relevant to comparative effectiveness. Such investments are greatly needed to enhance the skills, supply, and diversity of the research work force

Consider real-world settings

• Research is needed not only to provide the evidence base for the best disease prevention, health promotion, or clinical interventions but also to know how to best implement findings in real-world complex organizational settings in communities
• Consider reliable, relevant, and usable research as the key criteria for CER
• Due to the current critical shortage in the health care work force, research should reflect the reality that an optimally trained clinician may not be available to provide preferred treatments
• CER results must not drive de facto coverage or reimbursement recommendations until they have been evaluated in real-world settings so we can determine their impact on individuals in various subpopulations
• CER information should be relevant to policymakers by providing information for local decisions to improve quality

Support innovation

• Encourage innovation while providing ongoing evidence of value
• Support the development and use of CER for means that are synergistic in the continued discovery of clinical innovations in biomedical science
• The Council should look at approaches that address how to perform CER when introducing innovative therapies, which by definition have less known about them in the real world than existing therapies

Include other areas, factors, and data sources

• Examine other areas of the health care system including health care delivery system reform, as well as prevention and wellness programs
• CER should include a wide range of factors that affect health, including nonclinical programs and interventions, organizational system characteristics, policies, and regulations
• CER should consider data from a wide range of studies and designs. For many programs and policies, it’s not feasible to conduct randomized trials. Therefore, in addition to randomized trials, CER should consider designs that are more common for evaluating comprehensive population-focused interventions, such as observational cross sectional studies, quasi experimental designs, and time series analyses

Address disabilities

• CER relating to persons with disabilities is required in four areas
• Research is needed about the most effective interventions to prevent or mitigate disability and the disabling effects of chronic diseases
• As persons with disabilities age, they risk the same diseases of aging as other individuals (e.g., breast cancer and cardiovascular disease). However, persons with disabilities are often excluded from clinical trials for these conditions. Because of this, often there is not a lot of information to guide their treatment decisions. Future clinical trials should exclude people with disability only when there are clear and compelling reasons
• Studies should investigate comparative effectiveness in person-focused interventions aimed at restoring or maintaining functioning or minimizing its loss. Much more research is needed to identify effective physical and occupational therapy interventions as well as various assistive technologies that address sensory deficits, communication impairments, and physical and motor limitations
• Research is needed to expand on the effectiveness of environmental modifications and to improve an individual’s quality of life as well as his/her participation in daily life. Examples include technologies and design changes within the built environment to make a home safer, more accessible, and to allow persons with disabilities to live independently in communities

• There is meager science regarding the long-term functional benefit of many rehabilitation therapies and equipment for children. The lack of evidence base is reflected in the wide regional variation of services and programs
• Effectiveness comparison should determine the indication for rehabilitation therapies in major disabling conditions, their components, timing, duration, outcomes, and quality of life expectations
• There is a need for effectiveness comparison of conventional treatments to newer interventions for which evidence exists but is more time consuming or costly (e.g., constraint induced therapy to improve hand use or body weight support to facilitate walking)
• Consider comparing health care that provides active health promotion to conventional services. Although no evidence supports continuous lifetime therapy, there is evidence that health-promoting behaviors including exercise can improve health, function, and quality of life in those with disabilities
• Families often seek alternative therapies and improvements when conventional treatment is lacking. It is important to establish rigorous comparison of alternative therapies to conventional care in order to clarify their role in patient care
• Children with disabilities and complex health care needs eventually become adults, and as adults, they often have difficulty transitioning to health care services. They need exceptional services and yet their providers may be penalized by becoming outliers in quality audits. Successful transition programs that incorporate many elements already exist. Such programs improve not only satisfaction but also health outcomes. CER of transition programs could help identify the elements that are required for successful programs

Address disparities and cultural differences

CER must be mobilized to improve the health outcomes of various racial and ethnic populations in order to end the gap that exists between the health status of some minority populations and other Americans. Clinical trial design and infrastructures of CER research must accommodate this goal

CER can add to the body of knowledge for reducing disparities with the following priority areas

• Knowledge about health disparity interventions between hospital systems and clinics that have long-standing experience with minority physicians, their patients, and other individuals
• Cultural competence in health literacy research in order to ultimately change behaviors and improve lifestyles
• Effective ways of communicating with minority patients and their families
• Integrative care that is outcome-based and includes mental health, oral health, and physical health
• Developing minority health professionals

Given the growth of the Hispanic population, there is a need to create regional areas for Hispanic health research and to follow the health care decision making within the health systems in that region

Personalized medicine

• Findings should not result in one-size-fits-all coverage recommendations
• To support the growth of personalized medicine, the analysis of data from integrated networks can help identify those factors that contribute to disease susceptibilities and characteristics that result in different outcomes to treatment
• CER should sponsor research that is designed to improve clinical decision making by both clinicians and patients. This would include research that considers individuality and health conditions as well as factors such as family history, patients’ values and preferences, and individual experience and treatment
• The Council should continue the Personalized Health Care Initiative at the Department of Health and Human Services and incorporate the initiative into CER
• CER should outline a process for decision reconsideration. Comparative approaches such as metanalysis and systemic reviews of the literature reward old medicine and one-size-fits-all methods that are inefficient and expensive. Therefore, the Council should outline a plan by which comparative effectiveness findings can be reconsidered such as when the FDA changes the label of a drug or when a professional organization changes the suggested standard of care

Focus on patients

• CER must address the needs and the life circumstances of individual patients. It must integrate clinical expertise with the best evidence and individual patient’s predicaments, rights, and preferences
• Research should include complex behavioral, environmental, and genetic components of comorbid conditions
• CER research should not only be about one product against another or treatment process or combination thereof. It should also include health care delivery issues such as the organization, design, and management of patient care
• CER should never be employed by payers or regulators to diminish the importance of clinical experience or patient/family values and experiences

Address potential conflicts of Interest

• CER should develop a strong and clear policy for conflict of interest in biomedical research and publishing
• It should consider steps that would make the CER program, over time, essentially free of conflict of interests
• It should strongly favor researchers and institutions that are devoted to doing research in the public interest and have no current conflicts
• It should require from the outset 100 percent disclosure and transparency of all conflicts from all researchers and institutions

Consider the potential legal implications and legal barriers

• Studies should scrupulously account for pre-treatment severity of illness in all of the conclusions that are made, as this is always a major issue in liability actions
• There is a need to have very statistically significant conclusions. Physicians are very concerned about being able to obtain clinically specific guidance from these studies
• The Council should ensure that there aren’t any clinical or non-clinical factors that can impact the outcome (e.g., preventive measures, lifestyle changes, or environmental influence). These need to be teased out from any conclusions made concerning recommendations
• The Council should also address any possible legal impediments to comparative effectiveness tools such as practical clinical trials and CED

Cost

• Opinions on cost were conflictive and included the following
  • Do not focus on cost, rather keep the focus on clinical effectiveness. A focus on cost could lead to limiting access and benefits
  • Reconsider the framework that providing informative cost information would limit access
  • Study all factors that are contributing to increased costs in the health care system including the examination of some of the inherent root causes (e.g., obesity epidemic). Tackling such issues early on can have a far greater impact on cost savings in the future than our current thinking on CER indicates

Disseminate successfully

• All stakeholders, including patients, should be involved in disseminating CER findings
• The most effective dissemination approaches should be locally organized and implemented. They should be carried out through a collaborative effort of providers, payers, consumers, and employers and should also be implemented in conjunction with quality reporting and quality improvement initiatives
• CER must disseminate findings to the end user and the patient population. It should ensure that people are aware of the information, understand how to use it, and have few or no barriers to be able to use it
• All aspects of CER must be disseminated by an essential independent body to the provider, patient, and payer

Other

• The Council should take into account the significant disease burden and economic burden of oral health when establishing priorities areas
• The Council should take a holistic approach that includes factors such as addiction, mental illness, HIV, prevention, treatment, and early identification

Open Public Comment

HIV/AIDS

• CER should protect the existing Public Health Service's treatment guidelines for HIV/AIDS and opportunistic infections. These guidelines are well-established and periodically reviewed by experts in the field. CER should not limit treatment regimens that would interfere with these guidelines
• People with HIV/AIDS must be ensured access to all drugs for the treatment of HIV/AIDS, which today includes a combination of antiretroviral therapy

• • CER should examine the following areas

  • The role of support services, such as case management offered to Ryan White, in the health outcomes of people with HIV/AIDS
  • A comparison on how health care is provided, either by HIV or infectious disease specialists, for primary care providers
  • A comparison to health care outcomes for HIV/AIDS patients between stages with differing AIDS drug assistance programs or Medicaid programs
  • The impact of other comorbid conditions in certain drug regimens
  • Models to improve an adherence to taking medications and the impact of certain medications on those who are aging

Telemedicine/Teleradiology

• Existing teleradiology and telemedicine resources should be developed and utilized as part of the infrastructure to establish registries, networks, and other e-health data to analyze comparative effectiveness on diagnostic and treatment pathways for targeted health conditions
• Reimbursement policies and claims processing systems aimed at controlling cost are currently impeding rather than facilitating the development of teleradiology. Conflicting quality credentialing and State policies under Medicare/Medicaid also impede access to quality teleradiology. Comparative effectiveness studies involving diagnostic services must be cognizant of the impact of these forces and provide solutions to improve access
• One of the priorities should be coordinating, leveraging, and providing synergies with the Recovery Act and ongoing projects in developing and implementing the infrastructure for health IT, digital image transmission, e-health records, telemedicine, and teleradiology

Environmental control units

• The Council should recommend funding for an environmental control unit, which is a set of patient rooms in either the wing of a medical facility or a freestanding building. These rooms are constructed with special building materials that do not emit chemicals. The air in the unit is filtered, and the entire environment of these patients is strictly controlled. Using environmental control units can help us determine the influences on chronic disease, discern psychological illness from primary physical illness, and interpret symptom clusters previously thought to be unrelated

EHR-mediated interventions

• The Council should look at comparative effectiveness research to include evaluation of EHR-mediated interventions. Studies have resulted in conflicting results in this area. Some interventions and some sites improved quality, while seemingly similar interventions at other sites resulted in no effect, and, in a few cases, quality was negatively impacted. Our understanding of which specific EHR features impact which quality attributes needs to become as nuanced as our selection of chemotherapy agents for specific cancers. This will only happen if we conduct appropriately designed, sufficiently powered, and meticulously analyzed multi-centered studies of these interventions

Complementary and alternative medicine

• The Council should consider complementary and alternative medicine (CAM). Some CAM interventions can have a positive effect that is approximately equal to conventional medicine. However, more research is needed to know what kind of modalities work best for what kinds of conditions

CME

• The Council should consider using Continuing Medical Education (CME) in disseminating results to a widespread professional audience. CME reaches over a million physicians each month and almost as many nurses. Some studies have shown that CME participants were more likely than non-participants to make diagnostic and therapeutic choices based on clinical evidence. It is particularly critical to include educational components and outcome studies in order to determine the effectiveness of CER on changes in clinician behavior and patient health

Minority and rural groups

• Comparative effectiveness research should be designed to address the medical problems for minority groups and people living in rural areas. A disproportionate number of these people suffer from multiple chronic illnesses, such as diabetes, renal, and cardiovascular disease
• CER also should be designed to provide data for policy development and an organized system of care for those with multiple chronic conditions instead of comparisons for discrete treatments