Comparative Effectiveness
R
Research
h iin P
Pediatrics:
di i
HSR & P
Policy
li Impli
Implications
ti n
Wade M.
M Aubry
Aubry, M
M.D.
D
PRL--IHPS, UCSF
PRL
AcademyHealth ARM
June 27
27, 2010
High Hopes for CER
in Health Reform





Ri i h
Rising
health
l h care costs are unsustainable
i bl
Costs are driven by technologytechnology-related changes
i medical
in
di l practice
i
When evidence is unclear, geographic variations
in care are high
Some US communities achieve lower costs and
high quality
Therefore, p
production and use of better
evidence may improve quality and lower costs
Th Evidence
The
E id
P
Paradox
d

How to reconcile:
 18,000+
18 000+ RCTs
RCT are published
bli h d each
h year
 Tens of thousands of nonnon-experimental
p
studies
 Many systematic reviews and clinical
guidelines conclude that the “available
evidence
id
is
i limited
li i d or studies
di are poor
quality”
Common Gaps in Evidence





Research subjects are highly selected
Research settings are not typical of settings in
which care is usually delivered
Mi i or iincorrect comparators
Missing
Physiologic
y
g or surrogate
g outcomes,, not
functional status, long term impacts, QoL
R lt are nott available
Results
il bl when
h decisions
d ii
made
d
The Key Controversies in CER




What is it?
Who should pay for it?
How should it be directed?
How should it be used?
5
CER definition: FCC
FCC--CER
The conduct and synthesis
y
of research
comparing the benefits and harms of
different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions
di i
iin ‘‘reall world’
ld’ settings
i
Federal Coordinatingg Council on Comparative
p
Effectiveness Research
IOM Committee’s Definition of
CER
The generation and synthesis of evidence that
compares the benefits and harms of alternative
ds to prevent,
p v , diagnose,
d g s , treat,, andd monitor a
methods
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
b h the
both
h iindividual
di id l and
d population
l i llevels.
l
IOM’s Characteristics of CER






Objective of directly informing clinical or
health p
policyy decision
Compares at least 2 alternatives, each with
potential to be best practice
Results at population and subgroup level
Measures outcomes important to patients
M th d and
Methods
d ddata
t sources appropriate
i t for
f the
th
decision of interest
Conducted in real world settings
8
Categories of CER Methods





Systematic
S t
ti reviews
i
off existing
i ti research
h
Decision modeling,
g with or without cost
information
Retrospective analysis of existing clinical or
administrative data
Prospective nonnon-experimental studies,
includingg registries
g
Experimental studies, including
randomized
d i d clinical
li i l trials
i l (RCT
(RCTs))
Charge to IOM Committee:
Recommend national priorities for comparative
effectiveness research to be conducted or
supported by funds from the American
Recovery and Reinvestment Act of 2009.
•
Obtain national input from a wide variety of stakeholders,
to develop a list of no fewer than 50 priorities
•
Define how these recommended priorities should be
incorporated into a balanced portfolio
•
Recommend priority actions for ensuring the infrastructure
and workforce for a long-term, sustainable national CER
enterprise
Secondary Research Area
Primary Research Area
40
30
20
Number of Priiority Topics
N
Distribution of the recommended research priorities
by primary and secondary research areas
60
50
10
0
Reproduction
n
End-of-Life Care
C
Hepatobiliaryy
Gastrointestin
nal
Emergency/C
Critical Care
Respiratory
Oral Health
Genitourinaryy
Genetics
EENT
Complementtary/Alternative
Dermatologyy
Nutrition
Rheumatolog
gy
Developmental
Infectious Disseases
A
Substance Abuse
Women's Health
Oncology/He
ematology
Musculoskele
etal
Endocrinolog
gy
Pediatrics
Neurology
Psychiatry
Geriatrics
Cardiovascular
Disabilities
Disparities
Health Delive
ery
TABLE 5-3 Committee’s Recommended Research
Priorities by Types of Intervention
Types of Interventions
Systems
y
of Care
Pharmacological Treatment
Standard of Care
Behavioral Treatment
Prevention
Procedures
Provider Patient Relationships
Treatment Pathways
Testing, Monitoring, and Evaluation
Devices
Al
Alternative
i Treatment
T
Other
Total
Number of Topics
43
36
33
29
24
23
20
19
17
13
9
18
284
NOTE: The
NOTE
Th total
t t l exceeds
d the
th total
t t l number
b off priority
i it topics
t i as respondents
d t were allowed
ll
d to
t select
l t
multiple interventions to be compared for each topic.
TABLE 5-2 Committee’s Recommended Research Priorities
by Study Populations
Study Population*
Adults (including elderly)
Population at Large (general population)
Women
S i l Populations
Special
P
l i
(e.g.
(
pregnant women, low
l income,
i
disabled)
di bl d)
Men
Children/Adolescents Only
Eld l Only
Elderly
O l
Other
Long-Term Care
Ethnic Sub-Populations Only
Adults (excluding elderly)
Rare Diseases
Total
Number of
Topics
36
28
27
24
22
20
15
13
7
5
4
2
203
*Multiple populations could be assigned to a topic
IOM Recommendation 6

The CER Program should fully involve
consumers patients
consumers,
patients, clinicians,
clinicians payers,
payers
policymakers, and other key stakeholders
i key
in
k aspects off CER,
C R including
i l di strategic
i
planning,
p
g, priority
p
y setting,
g, research proposal
p p
development, peer review, and
di
dissemination.
i ti
AHRQ
Q EHC Program
g
2003-Present
2003Goal: To support
s pport informed health care
decisions by consumers, clinicians, and
policymakers and improve the quality,
effectiveness, and efficiency of health care
Authorized in 2003 by Section 1013 of the Medicare Prescription Drug,
Improvement, and Modernization Act
The program was established and received an initial appropriation of $15
million in 2005
Conducts scientific comparisons of the effectiveness of different health
care interventions
American Recovery and
R i
Reinvestment
t
t Act
A t off 2009 (ARRA)

$1.1 billion for comparative
effectiveness research




AHRQ: $300 million
NIH: $400 million
Office of the Secretary: $400 million
Federal Coordinating Council appointed to
coordinate CER across federal agencies
AHRQ CER & ARRA
AHRQ,
$9.5
$9 5 million for horizon scanning
$50 million for evidence synthesis
$172 million for evidence generation
$34.5 million for evidence translation and dissemination
$20 million for training and career development
$10 million for citizens’ forum
AHRQ
Q Conceptual
p
Framework
Stakeholder input
& involvement
Horizon
Scanning
Research training
Evidence
Synthesis
Evidence
Need
Identification
Evidence
Generation
Career development
Translation &
Dissemination
EHC Program
g
Components
p
Stakeholder Group
Scientific Resource Center
Evidence--based Practice Centers (EPCs)
Evidence
Developing Evidence to Inform Decisions about
Effectiveness Centers (DEcIDEs
(DEcIDEs))
Centers for Education & Research on Therapeutics
(CERTs)
John M. Eisenberg Clinical Decisions and Communications
Science Center
R
Researchers
h
Stakeholder Group
Volunteer committee representing clinicians,
consumers,
n m r government,
rnm nt p
payers,
r and
nd ind
industry
tr




Provides perspective on research gaps and methodology
Provides user perspective on EHC Program accountability and
transparency
Id ifi products
Identifies
d
most useful
f l to decisiondecision
d i i -makers
k
Identifies opportunities for increased stakeholder involvement
NIH Challenge Grants:
69 CER Topics

Focus on:




Developing research trial
protocols that may be
initiated later
Completing analyses using
existing data
Building data registries and
other tools to allow for
future analyses
Funding capped at $1M for
two yyears; $500K per
p year
y
Analysis by Avalere, Inc
CER in Health Reform

Creates PatientPatient-centered Outcomes Research
Institute (PCORI)
Independent nonnon-profit
 Contracts with research entitites (g
gov & non
non--ggov
gov))


Focus on Comparative Clinical Effectiveness
Priority
P
i i setting
i
 Methodology
 Research Implementation
 Dissemination and use of findings

22
Patient-Centered Outcomes
PatientResearch Institute
Financed through PatientPatient-Centered Outcomes
Research Trust Fund
Appropriations of $10$10-150 million for years
2010--2012
2010
2013 and later: fees on different forms of
insurance
Potentially $500 million of annual revenue
Patient-Centered Outcomes
PatientResearch Institute
Expert advisory panels (eg clinical trials) with
broad representation
p
Methodology Committee to develop standards
within 18 months
Board of Governors – NIH and AHRQ
directors and 17 other members
Medical treatments,, services
and items
“…health care interventions, protocols for
treatment, care management, and delivery,
procedures,
p
d s, medical
d
devices,
d v s, diagnostic
d g s tools,
s,
pharmaceuticals (including drugs and
biologicals) integrative health practices,
biologicals),
practices and any
other strategies or items being used in the
treatment, management, and
d diagnosis
di
i of,
f or
prevention of illness or injury in, individuals.”
HR 3590, Subtitle D, Section 6301
Limitations on uses of CER
“The
The Secretary shall not use evidence or findings
from comparative clinical effectiveness
research in a manner that treats extending the
research…
life of an elderly, disabled, or terminally ill
individual
d d l as off lower
l
value
l than
h extending
d the
h
life of an individual who is younger,
nondisabled, or not terminally ill.”
R l off costRole
cost-effectiveness
ff i
“The PatientPatient-Centered Outcomes Research Institute established
under
d section
i 1181(b)(1) shall
h ll not develop
d l or employ
l a dollarsdollars
d ll -per
per-quality adjusted life year (or similar measure that discounts the
value of a life because of an individual
individual’ss disability) as a threshold to
establish what type of health care is cost effective or
recommended. The Secretary shall not utilize such an adjusted life
year (or such a similar measure) as a threshold to determine
coverage, reimbursement, or incentive programs under title
XVIII ”
XVIII.”
CER Policy Option: Coverage
with Evidence Development

Links payment to requirement for
prospective
p
p
data collection



Has been adopted selectively in both public
programs and commercial health plans
Intent is to guide clinical research to
address
dd
hi
high
h priority
i i questions
i
Goal to support
pp evidence and innovation

Lower evidence threshold for coverage with
commitment to generate better information
28