Transcript Comparative Effectiveness Research

Congressional Budget Office definition for CER
“As applied in the health care sector, an analysis of Comparative
Effectiveness is simply a rigorous evaluation of the impact of
different options that are available for treating a given medical
condition for a particular set of patients. Such a study may compare
similar treatments, such as competing drugs, or it may analyze very
different approaches, such as surgery and drug therapy. The
analysis may focus only on the relative medical benefits and risks of
each option, or it may also weigh both the costs and the benefits of
those options. In some cases, a given treatment may prove to be
more effective clinically or more cost-effective for a broad range of
patients, but frequently a key issue is determining which specific
types of patients would benefit most from it.”
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Why are we here?
Projected Federal Spending on Medicare, as share of GDP
Can Comparative Effectiveness Research live up to it’s promise?
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Regional variations in spending
What have we learned?
Peter Orszag, N Engl J Med, 2007
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What do higher spending regions get?
Lower Quality?
Resources – and
Content of Care
Health Outcomes
Patient-Perceived
Quality
Physician’s
Perceptions
30% more beds and
MDs; 65% more
specialists
Slightly higher mortality
Worse access to
primary care
Worse communication
among physicians
Worse technical quality
No more elective
surgery
No better function
Lower overall rating
of medical care
Lower satisfaction with
hospital care
More hospital stays,
visits, tests
(1) Fisher et al. Ann Intern Med: 2003; 138: 273-298
(2) Baicker et al. Health Affairs web exclusives, October 7, 2004
(3) Fisher et al. Health Affairs, web exclusives, Nov 16, 2005
(4) Skinner et al. Health Affairs web exclusives, Feb 7, 2006
(5) Sirovich et al Ann Intern Med: 2006; 144: 641-649
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(6) Fowler et al. JAMA: 299: 2406-2412
Greater difficulty
ensuring continuity
Greater perception of
scarcity
Lower satisfaction with
career
Trends Over Time
Regions with greater
growth in resources
have no better gains in
survival after AMI
Health Expenditures Unsustainable
Cost crisis has been inexorably linked to
Comparative Effectiveness research (CER).
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Assumption is that we are paying for items that
are not as effective as less costly alternatives and
research will guide use of resources.
In fact the CER linked to cost is CostEffectiveness- Research-measuring the effect of
an intervention in relation to the resources it
consumes. Is it worth it?
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Cochrane A. BMJ (1999) 319:652-653
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The trillion dollar questions
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How to collect evidence on value and then
incorporate this evidence into decisions on coverage,
reimbursement, and payment for healthcare services?
How to develop value-based, cost effective
healthcare that is trusted and not perceived as only
cost cutting for profit/balancing federal budget.
Lack of evidence is a real impediment to value-based
healthcare. Collecting this data will take time.
Policy decisions based on incomplete data is subject
to serious negative consequences. Half truth
sometimes more dangerous than nothing.
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NIH Comparative Effectiveness Research
Approved for ARRA Funding
Grand
Opportunity
Grants (RC2)
31
$144,734,120
Challenge Grants
(RC1)
82
$76,510,300
Pay-line
Expansions
8
$35,838,658
“Other”
9
$58,473,346
Competitive
Revisions
7
$7,272,466
Administrative
Supplements
29
$19,081,118
TOTAL APPROVED
166
$341,910,008
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Remaining Funding Opportunities
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Remaining for FOAs
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FOAs
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Methodology Development in CER
02/26/2010
Research Gaps (MRSA, GERD, Dementia) 02/26/2010
Training
-Mentored Career Development Award 03/25/2010
- Administrative Supplements for CER Training
Total
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$ 58,089,992
Due date
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Total Costs
$10 M
$15 M
$25 M
$8 M
$58 M
Comparative effectiveness research
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Metric:
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Clinically significant outcome
Significant to whom
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Decision makers
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Patients
Doctors
Payors
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Insurers, HMOs,
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Hospital and Healthcare services CEOs
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Government Healthcare- VA, DOD,
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QALYs: The coin of the realm
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QALYs- Quality adjusted life years
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Each year assigned a health state and weighted by quality of life
associated with that health state.
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Weights generated by psychometric techniques asking patients to rate
their health relative to perfect.
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Factor probability of staying, or moving into other health states over
lifetime and expected lifetime.
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Sum the QALYs of future years lived in various health states
weighted by their quality of life, probability and time into the future.
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Cost effectiveness then examined as expenditure/QALY
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Judged against monetary incentive needed to get people to take a
job with increased risk of death!.
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Generally $50-200k/QALY
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Could develop a rationing based on fixed total health budget.
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Library of data on QALYs (CUAs) *https://research.tufts-nemc.org/cear/Default.aspx
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National Institute for Health and
Clinical Excellence (NICE)
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“An independent organization responsible for
providing national guidance on promoting
good health and preventing and treating ill
health”.
Secretary of State directs that NHS provide
funds for medicines recommended by NICE
appraisals usually within 3 months.
http://www.nice.org.uk/
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Cost effectiveness analysis of
Acetylcholinesterase Inhibitors in AD.
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Use AHEAD model to arrive at cost/QALY (Caro et. al. Neurology 2001; 57: 964-971.
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Probabilistic model gives difference due to drug(s) in time spent in full-time care over 5 years of 1.4-1.5
months, QALYs gained from 0.032-0.035, and 70k-97k pounds CQG.
Add in carer utility and CQG improves to 36-50k pounds.
Model on only those with moderate AD and CQG as low as 20kpounds for rivastigmine.
DSU examines the model and results and makes further adjustments/recommendations.
Interest groups have ability to analyze and submit their assumptions.
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Based on metanalysis of trials reporting ADAS-cog
Assumed only 70% of full time care would be met by NHS
Used US x-sectional heatlh-state utility data
Applied utility value of 0.6 for pre-full time care and 0.34 for full time care health state - based on literature and
EuroQol.
Alzheimer society reanalyzed with their own assumptions, (cost of behavioural improvement) and get to 14.5k
pounds CQG.
Companies redid the model and one identified technical errors assoc. with less than 2k/CQG difference.
The committee
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Use of AchE inhibitors lied outside the range of cost effectiveness that might usually be considered appropriate for
the NHS.
Found no support for use in mild AD, but acceptable for AD with moderate cognitive impairment.
Concluded that a number of technical inaccuracies in the model.
Concluded that evidence supporting memantine’s ability to prevent institutionalization in moderate to severe AD is
insufficient.
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Guidance on use of acetylcholinesterase
inhibitors in AD (last reviewed 2009)
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Recommended only in management of AD of
moderate severity (MMSE 10-20).
Only specialists in dementia should initiate Rx.
If initiated, evaluate every 6 months.
Memantine is not recommended except in a study.
Quite different than what occurs in US.
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15% of persons in nursing homes receive ChEIs
50% of persons in ADNI with MCI receive ChEIs
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Coverage by private insurers
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Usually defined as “medically necessary”
Some do not cover a service that is more
costly than an “equivalent” .
Multiple cost containment practices
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Tiering, Physician edits,
Most contracts with providers are linked to
Medicare rates: The Elephant in the room.
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Conditional Coverage
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Examples: Herceptin use only in those with
Her2/neu receptor after discovery that 20% of
treated UnitedHealthcare patients had the
marker.
HealthPartners in Mn restrict adalimumab in
RA pts to prescription by rheumatologist,
doseage limit, and only in pts who failed a 3
month trial of another agent.
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Cost generated processes that require
evidence.
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Ordering choices in correct context, “tiering”
 First choice default is least expensive and more
expensive choices when fail first option, “step edits”.
(Meds, referral to specialists, nurse vs. physician).
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Physician edits- some drugs only prescribed by
specialists.
Health services models vs. doctor/patient.
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Medical home, chronic disease programs, communications
technology vs. doctor visits.
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Center for Medicare and Medicaid
Services
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Operates under regulations on coverage.
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Require payment not be made for interventions that are not
“reasonable and necessary”
Default has been to provide coverage if there is no evidence of harm.
Has regulation to allow it in limited circumstances to cover only the
“least costly alternative” for durable medical equipment and IV drugs.
Reimbursement based on RVU, not clinical benefit.
Would need new legal authority to apply CER principles to
coverage decisions.
Fee for service model incentivizes “capacity” expansion.
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Center for Medicare and Medicaid
Services
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Coverage with evidence development.
Blended payment rates
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2 HCPCS codes judged to be identical are paid at
a single blended rate for both which incentives
payors to use the least expensive option.
Can deny coverage for adverse events
considered fully preventable.
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Coverage with Evidence
Development
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Links payment to requirement for
prospective data collection
Intent is to guide clinical research to
address questions of interest to Medicare
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Medicare must approve study design
Goal to support evidence and innovation
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Lower evidence threshold with commitment to
generate better information later
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Improving Incentives in the Medicare Program:
Medpac.
http://www.medpac.gov/documents/Jun09_EntireReport.pdf
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Peter Orszag and cost containment in
Health Care Package
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Bundled payments
Accountable-care organizations
Rapid path for bio-generic drugs to come to market
Independent Medicare commission of experts to
improve care and lower costs
Medicare commission to facilitate movement from
pilot programs to full practice “as we learn”.
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Bundled Payments
http://www.randcompare.org/policy-options/bundled-payment
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Middle ground between capitation (assume all risk)
and fee-for- service (pay multiple charges).
Case rates or episode-based payment
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Single payment for all services related to Rx or condition
spanning multiple providers in multiple settings.
Precedents:
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Medicare CABG demonstration
Geisinger CABG
Prometheus payment initiative- MI, hip, CHF, DM,
asthma
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Prometheus payment model
http://www.prometheuspayment.org/publications/pdf/Whats%20The%20Score.FINAL.pdf
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Explicitly designed to pay providers to “deliver what science
says patients should receive” for their specific constellation
of clinical problems associated with a condition.
Maximum payment based on Evidence informed Case Rate
Whether provider gets full payment depends on quality
performance (now 6 chronic disease scores). Akin to pay for
performance but:
Final scores depend 70% on what the provider does and 30%
on what every other provider treating that patient for that
condition has done- whether under the ECR or not.
Claim savings by pushing system toward less overuse due to
complications of care, or misuse to pay for less underuse.
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Accountable care organizations
http://www.medpac.gov/chapters/jun09_CH02.pdf
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Organization between a number of primary care and
specialty physicians, hospital, other health
professionals, >5000 pts.
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Fee for service billing but some withheld (spending
target)
Link bonus payments to quality and utilization of health
services
Coordinate care on shared Medicare patients to meet
quality benchmarks and “share in cost savings that stem
from quality gains.”
Brookings-Dartmouth ACO pilot- Vt. Carilion Clinic Va.
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Quality measures in ACO
http://www.medpac.gov/chapters/jun09_CH02.pdf
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Mortality, hospital admissions, readmissions, patietn
satisfaction, improvements in functionality.
Quality targets aggregated into a weighted average
score.
Both Quality and spending targets be met to achieve
bonuses.
Shared savings stem more from joint than individual
decisions.
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ACO: Shared savings stem more from joint than
from individual decisions.
http://www.medpac.gov/chapters/jun09_CH02.pdf
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Process to approve follow-on biologics.
http://www.medpac.gov/chapters/jun09_CH05.pdf
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The top 6 biologics account for 43% of
spending on separately billed drugs in
Medicare Part B ($13billion)
An FDA approval pathway for follow-on
versions would reduce prices. Save Medicare
9-12 billion over 10 years.
EU has approved 10 biosimilars and these
have entered the market at prices 15-25%
lower than the reference products.
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Real Targets
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Overuse, Misuse and Underuse
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Danger is decision making in absence of outcome
evidence. Cost will drive decision making.
Performance measure substitute for outcomes.
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Appropriate use requires evidence
Difficult to design a system without complete body of
evidence and consensus on all of medicine.
Difficult to accommodate a more expensive, more
effective, new products in a system that is costdriven. True health outcome data is key (QALYs).
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