Comparative Effectiveness Research: Now and in the Future

1. Comparative Effectiveness Research: Now and in the Future Children’s Hospital of Philadelphia July 13, 2009 Patrick Conway, MD, MSCE Chief Medical Officer, OS/ASPE Department of Health and Human Services

2. Disclosure • I am an employee of federal government • Views expressed are my own and do not represent official policy or guidance from the Department of Health and Human Services

3. Agenda • Short Background • Comparative Effectiveness Research – What is it and where we are currently • Potential implications for academic community of comparative effectiveness research and broader health reform

4. Background • RWJ Clinical Scholar at Penn (and attending at CHOP) – thesis was comparative effectiveness study with Ron Keren as mentor • Faculty (now on leave) Cincinnati Children’s Hospital • White House Fellow 2007-08 – split between Secretary of HHS and Director of AHRQ • Chief Medical officer in policy division for Office of Secretary • Executive Director of Federal Coordinating Council on Comparative Effectiveness Research

5. Portfolio at HHS • Quality measurement and link to payment • National Quality Forum • CHIPRA – quality measures • Health Information Technology (e.g. meaningful use) • Health reform • Comparative Effectiveness Research • Whatever is asked

6. Background on American Recovery and Reinvestment Act Legislation • Allocated $1.1 billion for CER: $400 million to Office of the Secretary, $400 million to NIH and $300 million to AHRQ • All money obligated (not spent) by end of FY10 • Established Federal Coordinating Council for CER composed of senior federal officials and over half physicians • Required FCC report to the President and Congress by June 30 on recommended priorities for OS CER funds • Required IOM report by June 30 on CER priorities • Mandated operational plan from Secretary for combined OS, AHRQ, and NIH CER funds by July 30

7. Federal Coordinating Council for CER Objectives • Develop a definition, establish prioritization criteria, create a strategic framework, and identify priorities that lay the foundation for CER. • Foster optimum coordination of comparative effectiveness research conducted or supported by relevant Federal departments. • Formulate recommendations for investing the $400 million appropriated to the HHS Office of Secretary as part of this Report to Congress.

8. 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. • To provide this information, comparative effectiveness research must assess a comprehensive array of health-related outcomes for diverse patient populations and sub-groups. • Defined interventions compared may include medications, procedures, medical and assistive devices and technologies, diagnostic testing, behavioral change, and delivery system strategies. • This research necessitates the development, expansion, and use of a variety of data sources and methods to assess comparative effectiveness and actively disseminate the results.

9. Minimum Threshold Criteria • Included within statutory limits of Recovery Act and FCC definition of CER • Potential to inform decision-making by patients, clinicians, and/or other stakeholders • Responsiveness to expressed needs of patients, clinicians, and/or other stakeholders • Feasibility of research topic (including time necessary for research)

10. 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)

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

12. CER Investment and Activities Four major categories: • Research (e.g., comparing patient outcomes between different treatments or care delivery models for a specific condition) • Human and Scientific Capital (e.g., training new researchers to conduct CER, developing CER methodology) • CER Data Infrastructure (e.g., developing longitudinal, linked administrative or Electronic Health Record (EHR) databases, or patient registries) • Dissemination and Translation of CER (e.g., improving methods to disseminate CER findings to clinicians and patients and translate CER into practice)

13. Examples of Potential Focus Areas for Cross-Cutting Theme Investments Priority Populations • Children • Patients with multiple chronic conditions • Racial and ethnic minorities • Elderly • Persons with disabilities Priority Conditions • Cancer • Heart disease • Diabetes Types of Interventions • Medications, devices • procedures • Behavioral change • Delivery system changes • Prevention

14. Dissemination and Translation Priority Populations Priority Conditions Types of Interventions Council’s Recommended OS Investment Strategy Human & Scientific Capital Research Data Infrastructure Crosscutting Priority Themes Legend Secondary investments Supporting investment Primary investment

15. Coordinated complimentary funding • AHRQ and NIH will likely devote significant amounts to research category (as well as training and cross-cutting themes) • OS could make short term investments in infrastructure (both data and translational) to lay foundation for CER and complement AHRQ and NIH funding

16. Purpose: - to prepare a list of priorities for research funding and recommendations to implement a sustained CER effort Similar definition: “CER is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels.” IOM Report

17. Recommended100 priority CER topics and research questions divided into four quartiles Examples: Compare the effectiveness of treatment strategies for atrial fibrillation Compare the effectiveness of comprehensive care coordination programs, such as the medical home model Compare the effectiveness of various screening, prophylaxis and treatment interventions in eradicating methicillin resistant staph aureus IOM Report

18. IOM General Recommendations • Establish coordinating advisory body • Develop and promote robust data and information systems • Strengthen the CER workforce • Promote rapid adoption of CER findings • Obtain public input and maintain transparent processes

19. Interested to Read More • Full FCC report and IOM report available online • Short summaries in NEJM online June 30th1,2 1 Conway PH, Clancy C. Comparative Effectiveness Research – Implications of the Federal Coordinating Council’s Report. N Engl J Med 2009 June 30. 2 Iglehart R. Prioritizing Comparative Effectiveness Research – IOM Recommendations. N Engl J Med 2009 June 30.

20. Where might CER fit in broader health care reform and research funding in the future • If we want to improve quality and value in health care, we need to know:1,2 • What works for which specific patient subgroups • The quality outcomes for a given cost • How to implement across systems of care and measure results 1 Dougherty D, Conway PH. The T3 Roadmap to Transform U.S. Health Care: The How of High Quality Care. JAMA. 2008 May 21; 299(19): 2319-21. 2 Conway, PH, Clancy C.  Transformation of Health Care at the Frontline. JAMA. 2009 Feb 18;301(7):763-5.

21. Health Reform • All of the major current reform bills include comparative effectiveness research or similar research with different name (e.g. patient-centered outcomes research) and typically with significant funding • Different proposals for where CER might primarily reside (e.g. within a current government agency, new entity, public-private governance structure)

22. Thoughts on future • In terms of growth of research funding, this may be a major area for future growth • Data and link to HIT is critical for these questions • Will require further methods development and training of researchers • Need to strengthen link between questions that patients and clinicians need answered and the research enterprise

23. Immediate timeline • Spend plans under review and ARRA calls for release of combined plan July 30th • Once plans approved (NIH, AHRQ, OS), funds can be obligated • All funds obligated by September 2010 • Spend may be able to extend 2-3 years from date obligated at least for significant portion of funds

24. Acknowledgement • Mentorship of faculty at CHOP such as Dr. Ron Keren and faculty through RWJ Clinical Scholars program and CCEB

25. Questions?