An AHRQ Perspective on CER and Methodology Considerations Jean Slutsky Director, Center for Outcomes and Evidence New York City August 12, 2010
How Can We Make Better Healthcare Decisions? Improve research base – Relevance – Quality
Improve use of data in decision making
The Role of Clinical Decision Makers in CER CER +
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Researcher looking through lens of decision maker to do research
EBM =
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Decision maker looking through lens of research to make decisions
Comparing Evidence: Medical vs. Semiconductor Research “When I was doing semiconductor device research, it was expected that I would compare my results with other people's previously published results and that I would comment on any differences. But it seemed to be different in medicine. “Medical practitioners primarily tended to publish their own data; they often didn't compare their data with the data of other practitioners, even in their own field, let alone with the results of other types of treatments for the same condition.” Intel co-founder and prostate cancer patient Andy Grove Forbes 5/13/96
Questions for Setting Clinical Policy: A Systematic Process 1. What is the outcome I care most about? 2. How good is the evidence that the 3. 4. 5. 6.
interventions can improve those outcomes? How sure am I that it will work in “real world”? How do the potential benefits compare to possible harms and costs? What constitutes “good enough” evidence? What other considerations are relevant?
Some History on Comparative Effectiveness Research
Comparative Effectiveness: AHRQ Effective Health Care Program Created in 2005, authorized by
Section 1013 of the Medicare Prescription Drug, Improvement, and Modernization Act (MMA) of 2003 To improve the quality, effectiveness, and efficiency of health care delivered through Medicare, Medicaid, and S-CHIP programs by focusing on – What is known now – What research gaps are critical to fill – Clinical effectiveness
Definition: HHS 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. Recovery Act Spend Plan, Office of the Secretary, 11/09
A Framework for CER
Evidence Generation
Horizon Scanning Evidence Need Identification Evidence Synthesis
Strategies Interventions Conditions Populations
Dissemination Translation
Research Platform Infrastructure – Methods Development – Training
Improvements in Health Care
An Unprecedented Investment Allocations for the $1.1 billion in comparative effectiveness research funding in the American Recovery and Reinvestment Act of 2009:
Research Data Infrastructure Dissemination and Adoption Administrative support, inventory, evaluation
$681M (62%) $268M (24%) $132M (12%)
$19M (2%)
AHRQ FY 2008 – 2010 (including ARRA) Investments
3%
3% 6%
13%
57%
12%
6%
Public Engagement Topic nominations
for research Comment period for key research questions Comment period for draft reviews Focus testing translation products www.effectivehealthcare.ahrq.gov
Categorical Investments in Evidence Generation Distributed data networks and related
infrastructure development Methods development Non-experimental/observational studies, both retrospective and prospective Sponsorship of pragmatic clinical trials addressing key foci of comparative effectiveness
Evidence Generation Activities Under ARRA Request for Registries: Up to five awards for the creation or
enhancement of national patient registries, with a primary focus on 14 priority conditions defined under MMA ($48M) DEcIDE Consortium Support: Expansion of multi-center research system and funding for distributed data network models that use clinically rich data from electronic health records ($24M) Clinical and Health Outcomes Initiative in Comparative Effectiveness (CHOICE): Coordinated national effort to establish a series of ~10 pragmatic clinical comparative effectiveness studies ($100M) iADAPT: Innovative dissemination and implementation grants for CER ($29.5M)
Underlying Principles Attentive to subgroups Under-represented in clinical trials (elderly, children, racial and ethnic minorities) Inclusive of comorbidities Explore heterogeneity of treatment effects
Citizen’s Forum for Comparative Effectiveness Research ARRA funding will support the Citizens’ Forum
Initiative to develop new mechanisms and refine existing approaches to eliciting public input The Forum will increase our use of public input to inform health care policy, especially involving comparative effectiveness research for AHRQ’s Effective Health Care Program It will expand AHRQ’s efforts to obtain professional and consumer input, build methods and capacity for obtaining public input and allow the program to obtain guidance and insight from a broader public
Horizon Scanning for CER ď Ž Horizon Scanning
methodology and reporting for CER
Registry of Patient Registries The Recovery Act calls for AHRQ to fund
development of a registry that would serve broader research purposes. This type of registry would: – Benefit multiple stakeholders – Reduce unnecessary duplication of effort – Encourage communication between registry
developers
“For example, researchers undertaking a patient registry of cardiovascular disease among women under the age of 55 in a certain region may find several similar registries underway with which they might collaborate or choose to differentiate to better answer certain questions.” ARRA Spend Plan, Office of the Secretary
Educating Clinicians Concise Actionable Paired with
consumer guides Convey level of uncertainty/certainty of findings
Effective Health Care Web 2.0 and Social Media Tools Product Buttons Product Collections
RSS Feeds
Widgets Social Media
CME/CE Activities CME/CE activities are available for the following health conditions and methods areas: Diabetes Heart and Blood Vessel Conditions Muscle Bone and Joint Conditions AHRQ Training Modules for the Systematic Reviews Methods Guide Other Topics
Emerging Methods in Comparative Effectiveness, EBM & Safety
Variation in methods among systematic reviews undercuts transparency
Poorly done new research can be misleading Methods must continue to evolve and not remain stagnant AHRQ has and will continue to make investments in improving methods, esp. in understanding clinical heterogeneity.
New Resource on Comparative Effectiveness Research Methods June 2010 supplement to Medical Care on
CER Methods
22 original articles, special focus on: –
Ways to enhance the inclusion of clinically heterogeneous populations in clinical and comparative effectiveness studies
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Methods for implementing longitudinal investigations that capture longer term health outcomes, including patientreported outcomes
Printed copies available free of charge
through the AHRQ Publications Clearinghouse
www.effectivehealthcare.ahrq.gov AHRQ publication number: OM10-0067
Questions Related to Surrogate Endpoints Easy to measure but.. – How well do they link to true meaningful
health outcomes – What options exist to capture distal outcomes of interest – How well do we understand the natural course of some conditions
http://effectivehealthcare.ahrq.gov