American Heart AssociationAmerican Heart AssociationQuality of Care and Outcomes ResearchQuality of Care and Outcomes Research

Comparative Effectiveness ResearchComparative Effectiveness Research

Patrick Conway, MD, MScPatrick Conway, MD, MScChief Medical Officer, Dept of Health and Human Services, OS/ASPE Chief Medical Officer, Dept of Health and Human Services, OS/ASPE

(transitioned out to being HHS expert consultant in April)(transitioned out to being HHS expert consultant in April)Executive Director, Federal Coordinating Council on CER (former)Executive Director, Federal Coordinating Council on CER (former)

Cincinnati Children’s Hospital, Director of Hospital MedicineCincinnati Children’s Hospital, Director of Hospital Medicine

DisclosuresDisclosures

The opinions in this discussion represent my The opinions in this discussion represent my own and do not represent official policy or own and do not represent official policy or guidance from the Department of Health and guidance from the Department of Health and Human ServicesHuman 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 CriteriaThe 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 CERFCC Report: Strategic Framework for CER

Human & Scientific Capital

ResearchData 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

AHRQData 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 Enterprise1. National CER program must develop an overall

funding strategy - influenced by clinicians and patients and its stakeholder advisory board

2. Establish an initial list of priority topics and evaluate the current state of knowledge about each

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. Strive for a balanced portfolio of high impact research topics

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. 2010 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 2Increased Funding for the 2ndnd and 3 and 3rdrd T of the “3T’s” T of the “3T’s” Road Map to Transforming U.S. Health CareRoad Map to Transforming U.S. Health Care

Key T1 activity to testKey T1 activity to test what care workswhat care works

Clinical efficacy researchClinical efficacy research

Key T2 activities to testKey T2 activities to test who benefitswho benefits from from

promising carepromising care

Outcomes researchOutcomes researchComparative effectivenessComparative effectiveness

ResearchResearch

Health services researchHealth services research

Key T3 activities to testKey T3 activities to test how how to deliver high-qualityto deliver high-quality

care reliably and incare reliably and in all settingsall settings

Quality Measurement andQuality Measurement andImprovement Improvement

Implementation of Implementation of Interventions and healthInterventions and health care system redesigncare system redesign

Scaling and spread of Scaling and spread of effective interventionseffective interventions

Research in above domainsResearch in above domains

T1 T2 T3Basic biomedical

scienceClinical efficacy

knowledgeClinical effectiveness

knowledge

Improved health care quality and

value andpopulation health

Source: JAMA, May 21, 2008: D. Dougherty and Source: JAMA, May 21, 2008: D. Dougherty and P.H. ConwayP.H. Conway, pp. 2319-2321. The “3T’s Roadmap to Transform U.S. Health Care: The , pp. 2319-2321. The “3T’s Roadmap to Transform U.S. Health Care: The ‘How’ of High-Quality Care.” ‘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?Questions or Comments?