Bending the Cost Curve Through
Comparative Effectiveness Research
ASCO 2012
Sean Tunis MD, MSc
June 2, 2012
Evidence Summary: Radiation Therapy for
Clinically Localized Prostate Cancer
Comparisons
Disease specific
survival
Freedom from
biochemical failure
GU/GI toxicity
RT vs NT
insufficient
insufficient
insufficient
SBRT vs EBRT
insufficient
insufficient
insufficient
SBRT vs HDBRT
insufficient
insufficient
insufficient
SBRT vs LDBRT
insufficient
insufficient
insufficient
EBRT vs HDBRT
insufficient
insufficient
insufficient
EBRT vs LDBRT
insufficient
insufficient
insufficient
HDBRT vs LDBRT
insufficient
insufficient
insufficient
Combined mod.
insufficient
insufficient
insufficient
Intra SBRT
insufficient
insufficient
insufficient
Intra EBRT
insufficient
moderate
moderate
Intra LDBRT
insufficient
insufficient
insufficient
Source: Tufts Evidence-based Practice Center: Draft AHRQ Technical Assessment, March 25, 2010
Off-label Uses of Oncology Drugs
• Draft systematic review of off-label use of
oncology drugs
– Duke EPC, Amy Abernathy
– 428 pages, several thousand trials
• “Because of the paucity of high quality
evidence, the data available – though
voluminous – may have little meaning or
value for informing clinical practice”
• PROs missing or not comparable
4
http://www.cbo.gov/ftpdocs/89xx/doc8972/02-15-GeogHealth.pdf
High Hopes for CER
• “Better information about the costs and
benefits of different treatment options,
combined with new incentive structures
reflecting the information….is essential to
putting the country on a sounder long-term
fiscal path.”
Peter Orszag, CBO (later OMB)
Congressional Testimony, June 2007
6
Overview for Center for Medical Technology Policy
PATIENT-CENTERED OUTCOMES
RESEARCH INSTITUTE
Baltimore, MD
August 24, 2011
Joe Selby, MD MPH
Executive Director
The Evidence Paradox
• 18,000+ RCTs published each year
• Tens of thousands of other clinical studies
• Systematic reviews intended to inform
clinical and health policy decisions routinely
conclude that evidence is inadequate
The CER Hypothesis
• Gaps in evidence will be reduced with greater
engagement of end users (decision makers =
patients, clinicians, payers) in
– Identifying most important uncertainties
– Designing study protocols
• Useful CER studies must incorporate views of
patients, clinicians, payers in study design
9
10
P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTE
The Patient-Centered Outcomes Research
Institute helps people make informed health
care decisions – and improves health care
1 delivery and outcomes – by producing and
promoting high integrity, evidence-based
information – that comes from research guided
by patients, caregivers and the broader health
care community
Patient Perspectives: Psoriasis
• Regulatory studies report extent and severity of
total body surface area affected by plaques
– FDA does not allow any QoL measures on label
• Patients state that face and joint involvement
had biggest impact on QoL
• More pragmatic design would include measure
of face/joint severity as secondary endpoint
– and high priority to develop and validate a measure
for severity of face/joint involvement
12
Clinician Perspective – Dementia
• ACP guideline reviewed 5 drugs approved by
FDA based on ↑ cognitive function (ADAS-cog)
• “Weak” evidence for clinical recommendations
• No convincing comparative studies
• Outcomes used in trials not used in routine
clinical practice….and “not clinically important”
• Follow-up too short: generally less than 1 year.
• More pragmatic design would include active
comparator, include cognitive measures used in
practice, and longer follow up
13
Payer Perspective - Artificial Disc
• Medicare denied coverage of cervical artificial
discs based on FDA-approved PMA trial
• Trial limited to patients age 18 to 60
• Non-inferiority comparison to spinal fusion was
not most clinically relevant alternative
• SF-36 and radiographic changes “have no
clinical meaning”
• More pragmatic design would enroll older
patients, compare to conservative care, and
measure patients activity level
14
High Hopes for CER
• “Better information about the costs and
benefits of different treatment options,
combined with new incentive structures
reflecting the information….is essential to
putting the country on a sounder long-term
fiscal path.”
Peter Orszag, CBO (later OMB)
Congressional Testimony, June 2007
15
Implications for Clinical Practice
• The Learning Healthcare System
– Every clinical encounter generates knowledge
– Depends on accurate and complete data entry
• Bundled payments and quality-adjusted pay
– Clinical income linked to outcomes and efficiency
– Risks, benefits and costs of alternative diagnostic
and therapeutic options are essential resource
• Increased patient-cost sharing
– Clinical decisions must be cost sensitive
High Hopes for CER
• “Better information about the costs and
benefits of different treatment options,
combined with new incentive structures
reflecting the information….is essential to
putting the country on a sounder long-term
fiscal path.”
Peter Orszag, CBO (later OMB)
Congressional Testimony, June 2007
17
Who is to Blame?
•
•
•
•
•
•
Health insurance profits and salaries?
Overpriced drugs and devices?
Greedy malpractice lawyers?
Incompetent federal bureaucrats?
Demanding patients?
(Physicians?)
– My theory, a “the tragedy of the commons”
Contact Info
•
•
•
•
[email protected]
www.cmtpnet.org
410 547 2687 x120 (W)
410 963 8876 (M)
Extra Slides
CANCERGEN Structure
21
RxPonder Trial
Node-positive (1-3 nodes), HR-positive, and HER2-negative breast cancer
(N= 8,800)
Patients consent to study-sponsored RS testing, discussion of potential
trials, tumor tissue submission and linkage to cancer registry data
(N= 600)
RS already Available
RS < 25 ?
RS > 25
RS < 25
(N= 3,800
Discuss alternative
trials for high risk
patients
N= 5,600
Physician and
patients discuss
randomization
knowing the RS
Refuse
22
N= 1,600
Record chosen therapy
and followed for vital
status using cancer
registry
Accept
N= 4,000
Randomization
stratified by
1. RS 0-13 vs. 14-25
2. Menopausal status
3. Sentinel LNB only
vs. complete LND
N= 2,000
Chemotherapy;
appropriate
endocrine therapy
N= 2,000
No Chemotherapy;
appropriate endocrine
therapy
CER Defining Characteristics (IOM)
• Objective of directly informing clinical or
health policy decision
• Compares at least 2 alternative, each with
potential to be best practice
• Results at population and subgroup level
• Measures outcomes important to patients
• Methods and data sources appropriate for
the decision of interest
• Conducted in “real world” settings
23
Methods Emphasized in CER
• Heavy focus on extracting more knowledge
from existing studies and data
– Meta-analysis / systematic reviews
– Modeling
– Analysis of EHR and administrative data
• More efficient, informative prospective studies
– Registries
– Adaptive designs
– Attention to heterogeneity / subgroup effects
– Pragmatic clinical trials