American Heart Association Quality of Care and Outcomes Research Comparative Effectiveness Research Patrick Conway, MD, MSc Chief Medical Officer, Dept of Health and Human Services, OS/ASPE (transitioned out to being HHS expert consultant in April) Executive Director, Federal Coordinating Council on CER (former) Cincinnati Children’s Hospital, Director of Hospital Medicine

Disclosures  The opinions in this discussion represent my own and do not represent official policy or guidance from the Department of Health and Human Services

Today   CER definition, priority-setting, and framework   Brief overview of Recovery Act CER funding   Next Steps and Considerations

Federal Coordinating Council CER Definition Comparative effectiveness research is the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in “real world” settings. The purpose of this research is to improve health outcomes by developing and disseminating evidence-based information to patients, clinicians, and other decision-makers, responding to their expressed needs, about which interventions are most effective for which patients under specific circumstances.

Prioritization Criteria The criteria for scientifically meritorious research and investments are:   Potential impact (based on prevalence of condition, burden of disease, variability in outcomes, costs, potential for increased patient benefit or decreased harm)   Potential to evaluate comparative effectiveness in diverse populations and patient sub-groups and engage communities in research   Uncertainty within the clinical and public health communities regarding management decisions and variability in practice   Addresses need or gap unlikely to be addressed through other organizations   Potential for multiplicative effect (e.g. lays foundation for future CER such as data infrastructure and methods development and training, or generates additional investment outside government)

AHRQ Priority Conditions for CER   Arthritis and non-traumatic joint disorders   Cancer   Cardiovascular disease, including stroke and hypertension   Dementia, including Alzheimer's disease   Depression and other mental health disorders   Developmental delays, attention-deficit hyperactivity disorder and autism   Diabetes mellitus   Functional limitations and disability   Infectious diseases including HIV/AIDS   Obesity   Peptic ulcer disease and dyspepsia   Pregnancy including pre-term birth   Pulmonary disease/asthma   Substance abuse

FCC Report: Strategic Framework for CER Human & Scientific Capital Research Data Infrastructure Dissemination and Translation Priority Populations Priority Conditions Types of Interventions Cross- Cutting Priority Themes Investments can be made in a single category and/or be cross- cutting in one of the themes

Recovery Act (ARRA) CER Funding   $400M NIH   $300M AHRQ   $400M Office of the Secretary (OS)

Office of Secretary ARRA CER Data Infrastructure Related Projects Longitudinal Claims Data – –Medicare data CMS – –Medicaid data CMS – –Multi-payor Claims Design and Implementation ASPE/CMS/AHRQ – –Enhanced state data claims linked to clinical data AHRQ Data Networks – –Distributed Electronic Clinical Data Networks AHRQ – –Community Health Applied Research Network HRSA

Data Infrastructure and Research Patient Registries – –Patient Registries AHRQ – –Cancer Registries CDC – –Registry of Registries AHRQ Other – –Building FDA CER clinical data and standards infrastructure, tools, skills, and capacity FDA – –Persons with Multiple Chronic Conditions Data and Research – AHRQ/IHS – –Pediatric Care Networks and CER, HRSA

Office of Secretary ARRA CER Dissemination, Translation, and Adoption   Dissemination of CER to Physicians and other Providers, Patients and Consumers - AHRQ   Implementation strategies in AHRQ networks - AHRQ   Accelerating Dissemination and Adoption of CER in Delivery Systems - HHS/ASPE

Office of Secretary ARRA CER Research   Behavioral Economics and Change - NIH/AHRQ   Delivery System - AHRQ   Regionalized Emergency Care delivery - ASPR   Comparative effectiveness of chronic disease prevention - CDC   Centers of Excellence for Racial and Ethnic Minority-focused CER - OMH/NIH   Centers of Excellence for Persons with Disabilities - OD

Inventory and Evaluation   Inventory of CER   Evaluation and Impact Assessment

Five Next Steps for CER Enterprise 1. 1.National CER program must develop an overall funding strategy - influenced by clinicians and patients and its stakeholder advisory board 2. 2.Establish an initial list of priority topics and evaluate the current state of knowledge about each 3. 3.Select the research methods, with input from advisory board and decision-makers, appropriate to fill the gaps in knowledge on a particular topic/question 4. 4.Strive for a balanced portfolio of high impact research topics 5. 5.Evaluate progress and report to the public Source: Source: VanLare JM, Conway PH, Sox HC. Five Next Steps for a New National Program for Comparative- Effectiveness Research. N Engl J Med Mar 18;362(11):970-3.

Health Reform   Establishes Patient-Centered Outcomes Research Institute   Duties – –Identify Research priorities – –Establish research project agenda   Board of Governors - 17 members   Expert advisory panels (e.g clinical trials, rare diseases)   Establishes methodology committee (may consult with IOM)   Staff for the institute   Budget ramps up over time to likely over $500M per year

Methods Meet Decision-makers   Methods should attempt to address the level of evidence necessary to influence decision-makers   Will vary based on the question and decision involved   This should be explicitly considered in researcher’s proposals and by funders of research   Funding needed for methods development and building understanding of how methods and communication of findings will best meet decision- makers needs

Clinician and Patient Input to Guide CER Enterprise   Original FCC and IOM reports on CER strongly influenced by public input and ARRA funding (e.g. AHRQ) focused on horizon scanning and actively seeking input   New PCOR Institute should focus efforts on clinician, patient, and other stakeholder input   Need to consider utilizing technology and other methods to get more “real time” input from broad sample of patients, clinicians, and other stakeholders   Feedback loop from users and implementers of research back into the research enterprise

Tools and methods to increase adoption of comparative evidence   FCC report and subsequent ARRA funding (especially OS) focused on dissemination and adoption   Without this investment in adoption, we will fail to translate comparative evidence into improved health outcomes   Significant research opportunity for further understanding factors underlying successful adoption interventions   Health information technology can be a tool for both driving and measuring adoption

Increased Funding for the 2 nd and 3 rd T of the “3T’s” Road Map to Transforming U.S. Health Care Key T1 activity to test what care works what care works Clinical efficacy research Key T2 activities to test who benefits from who benefits from promising care Outcomes research Comparative effectiveness Research Health services research Key T3 activities to test how to deliver high-quality how to deliver high-quality care reliably and in care reliably and in all settings all settings Quality Measurement and Improvement Implementation of Interventions and health care system redesign care system redesign Scaling and spread of effective interventions Research in above domains T1T2T3 Basic biomedical science Clinical efficacy knowledge Clinical effectiveness knowledge Improved health care quality and value and population health Source: JAMA, May 21, 2008: D. Dougherty and P.H. Conway, pp The “3T’s Roadmap to Transform U.S. Health Care: The ‘How’ of High-Quality Care.”

Evaluating the CER investment   Research funders often do not systematically evaluate the impact of their research investments   Evaluation can be critical not only for measuring impact but also understanding how to improve future research investments   Office of Secretary ARRA CER investment included evaluation of ARRA CER funding   Ongoing CER research enterprise should be accountable to the public by evaluating investments and publicly reporting the results of the evaluation

Example Opportunities in Cardiovascular disease and stroke   NIH (NHLBI)   AHRQ   PCOR institute   Private sector, foundations, and other funding sources

New Research or Repackaging old Ideas?   Both   Not completely new – has been ongoing in evolving forms at VA, AHRQ, NIH, private sector, etc. for years   Concept of research and related funding decisions being driven by needs of patients, clinicians, and other decision-makers is a paradigm shift   Increase in funding and likely increase in CER studies in published literature   Patients, clinicians, and payors increasingly calling for comparative evidence

Questions or Comments?