NIH and Comparative Effectiveness Research
Martin L. Brown, Ph.D.
National Cancer Institute
AcademyHealth
Boston
June 29, 2011
DHHS Definition of CER
Comparative effectiveness research is the conduct and synthesis
of research comparing the benefits and harms of different
interventions and strategies to prevent
prevent, diagnose
diagnose, treat and
monitor health conditions in “real world” settings. The purpose of
this research is to improve health outcomes by developing and
di
disseminating
i ti evidence-based
id
b
d iinformation
f
ti tto patients,
ti t clinicians,
li i i
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, CER must access a comprehensive
array of health
health-related
related outcomes for diverse patient populations and
sub-groups.
 Defined interventions compared may include medications,
procedures,
d
medical
di l and
d assistive
i ti d
devices
i
and
d ttechnologies,
h l i
di
diagnostic
ti
testing, behavioral change, and delivery system strategies.
 This research necessitates the development, expansion, and use of a
variety of data sources and methods to access comparative
effectiveness and actively disseminate the results.
All ti
Allocation of Funds
fF d
3
$400M
NIH
$300M
AHRQ
$400M
HHS
OS
$1 1B
$1.1B
IOM CER Report
p
4
• Required under ARRA
Legislation
• Released 6/30/09
• Lists 100 national priorities
for CER
• Informed by testimonials
given by advocacy,
industry, and other groups
• Guides HHS CER funding
decisions
IOM Recommendations for long-term
g
investment
5
• Ensuring
E
i meaningful
i f l consumer, patient,
ti t and
d
caregiver participation
• Building robust information systems and
research methods
• Development and support of a highly skilled
CER workforce
• Support efforts to translate CER knowledge
into everyday clinical practice
practice.
NIH and AHRQ have Complementary
Roles
NIH
Evidence
Generation
AHRQ
 Research Analysis
 Systematic
S t
ti Reviews
R i
 Evidence Synthesis
Informs
Payers and Providers
NIH and ARRA CER
 Active leadership role in Federal Coordinating
Council and the HHS Coordinating and
Implementation Team (CER CIT)
 NIH CER Coordinating Committee coordinates NIH
CER programs and develops funding
recommendations for the NIH Director
 CER CC Subcommittees to
coordinate and integrate
Inter-Agency activities:
–
–
–
–
NIH-AHRQ CER Workgroup
NIH VA CER Workgroup
NIH-VA
NIH-FDA CER Workgroup
Discussions underway with CDC
CER Activities Approved for ARRA
Funding from NIH’s $400
$
Million
(as of April 2010)
Funding
Mechanism
# Submissions
Total Costs
(millions)
Grand Opportunity Grants (RC2)
31
$144.9
Challenge Grants (RC1)
82
$76.5
Pay-line Expansions
12
$55.0
“Other”
5
$39.3
Competitive Revisions
7
$7.3
Administrative Supplements
29
$19.2
Administrative supplements for CER
Workforce Development
19
$7.8
TOTAL APPROVED
185
$350.0
CER Requests for Applications – to
allocate the remainder of NIH
NIH’s
s $400
Million
 RFA-OD-10-009, Methodology Development in CER
($10M)
 RFA-OD-10-008, CER on Upper Endoscopy in
GERD,, Eradication Methods for MRSA,, and
Dementia Detection and Management Strategies
($15M)
 RFA-OD-10-011, Institutional CER Mentored Career
Development Award ($25M)
 NOT-OD-10-037, Administrative supplements for
CER Workforce Development ($8M)
Examples of ARRA funded CER projects
10

Center for Comparative Effectiveness Research in Cancer Genomics

Comparative effectiveness of breast imaging strategies in community
practice

Contemporary Treatment and Outcomes for Atrial Fibrillation in Clinical
Practice

Comparative Effectiveness of Interventions for Chronic Pain
Management

Comparative effectiveness of FIT vs. colonoscopy for colon cancer
screening

Minimally Invasive Surgical Pulmonary Vein Isolation vs. Medical
Management in Patients with AF and Stroke

Data Infrastructure for Post-Marketing for Comparative Effectiveness
Studies

Conservative Versus Dialytic Management in Stage V Chronic Kidney
Disease

Developing a Community-Based Autism Spectrum Disorders Research
Registry
Key NIH CER Activities
 Research to generate evidence that enables
physicians and patients to make optimal
health care decisions
 Research Training to develop the CER workforce of
tomorrow
 Personalized Medicine highlights uniqueness of
individuals and special populations
 CER Centers to support research
research, training and
dissemination of evidentiary knowledge
 Behavioral Economics to increase “uptake”
uptake of CER
findings by providers and payers
Health Care Legislation and CER:
Patient Centered Outcomes
Patient-Centered
O tcomes Research
Institute
 Establishes non-profit corporation (PCORI) to
organize, fund CER, paid for by new trust fund
– Overseen by Board of Directors (including
Directors/designees of NIH, AHRQ)
(incl. Directors/designees
– Standing methodology committee (incl
of NIH, AHRQ) to establish research priorities; set research
agenda
 Charged to identify
– National Research Priorities
– Evidentiary Gaps
– New Clinical Evidence
– R
Relevance;
l
standards;
t d d
economic correlates
Health Care Legislation and CER:
Patient Centered Outcomes
Patient-Centered
O tcomes Research
Institute
 Will ensure
– Rigorous peer review process to select best research
– Consideration of health disparities; individual genetic
differences; rare diseases
 Will build
b ild capacity
it b
by establishing
t bli hi grantt program ffor ttraining
i i
researchers to conduct CER (this will managed through AHRQ)
 Funding
– Direct appropriations:
• FY 2010: $10M
• FY 2011: $50M
$
• FY 2012: $150M
– FY 2013 - 2019: PCORI trust fund,
fund $150M plus per capita
charge per enrollee (per year)
Health Care Legislation and CER:
Patient Centered Outcomes
Patient-Centered
O tcomes Research
Institute (PCORI)
 AHRQ, through PCORI, is directed to consult with
NIH on:
–
–
CER Manpower Development Strategies
Communication and Dissemination
 NIH will continue its legacy of independently
supporting and conducting cutting edge CER
Summary
 The NIH is committed to CER as a research priority
 CER can be an effective tool to:
– Generate evidence that demonstrate “what
what works
works”
– Inform medical decision-making
– Support decisions based upon quality and value
– Possibly “bend the curve” on health care costs
 A key challenge is getting the
results of NIH supported CER
studies implemented by
providers, payers, and the public
15
Upcoming NIH CER Activities
 Conference on CER and Personalized Medicine
– October 19 -20, Bethesda, Maryland
 Conference on CER Methodolgy
– December 2-3, Bethesda, Maryland
Extra Slides
AHRQ CER Spending Plan
 AHRQ plans to use the $300 million they received
from ARRA to expand and broaden pre-existing
pre existing CER
activities initiated in response to Section 1013 of the
MMA (2003).
 AHRQ views CER as a process that includes the
following steps, and for which they will fund various
projects/initiatives:
– Horizon Scanning: identification of current or emerging
medical interventions
• Contracts
C
($9
($9.5 M))
– Evidence Synthesis: review and synthesis of current
medical research
• Contracts ($25 M)
– Identification of Evidence Needs and Gaps
• Contracts ($25 M)
AHRQ CER Spend Plan (cont.)
– Evidence Generation ($173 M)
• CHOICE Studies
• Requests for Registries
• DEcIDE
DE IDE C
Consortium
ti
S
Supportt
• Unfunded Meritorious Applications
– Dissemination and Translation ($
($34.5 M))
• CE Dissemination and Translation Innovation Grants
• Eisenberg Center Modification
– Research
R
hT
Training
i i and
dC
Career D
Development
l
t ($20 M)
• Institutional Training Awards and CE Fellowship
 In addition,
addition AHRQ plans to convene a Citizen Forum
on Effective Health Care in order to formally engage
stakeholders in the CER enterprise
p
(($10 M))
Office of the Secretary CER Spend
Plan
 The Secretary’s $400 Million has been
allocated to specific projects across the
following categories:
– Data Infrastructure
– Dissemination & Translation
– Research
– Inventory and Evaluation
Office of the Secretary CER Spend
Plan
 NIH will take the lead on the following
projects being funded by the Office of the
Secretary
– NCMHD Competitive Revision Application to Support CER
for Eliminating Disparities (NIH/OMH) ($7M)
$
– Behavioral Economics for Nudging the Implementation of
CER: Clinical Trials(NIH/AHRQ) ($24.9M)
– Behavioral Economics for Nudging the Implement of CER:
Pilot Research (NIH/AHRQ) ($5M)
– Impact of Decision-Support Systems on the Dissemination
and Adoption of Imaging-Related Comparative Effectiveness
Findings (NIBIB/NIH) ($3M)
AHRQ Funding Decisions
 $9.5 Million (3.2%) to Horizon Scanning
 $25
$ Million ((8.3%)
%) to Evidence S
Synthesis
 $25 Million (8.3%) to Identification of Evidentiary
N d and
Needs
dG
Gaps
 $173 Million (57.6%) to Evidence Generation
 $34.5 Million (11.5%) to Dissemination and
Translation
 $20 Million (6.7%) to Training and Career
Development
DHHS Funding Decisions
 $210.5 Million (52.6%) to Data Infrastructure
 $89.5 Million (22.4%) to Dissemination and
Translation
 $71 Million (17.75%) to Research
 $7.6 Million (2%) to Inventory and Evaluation