Transcript 02/06/04 NHPF - International Society For

Overview of Health Science
Policy Council Activities
May 16, 2005
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OBJECTIVES
• Describe Mission and Membership
• Discuss Structure and Activities
• Present Outcomes for 2005
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OBJECTIVES
• Describe Mission and
Membership
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MISSION
To advise the Society on important
science, research and policy issues
in pharmacoeconomics and
outcomes research
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MEMBERSHIP
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Jon Clouse
Peter Davey
Michael Drummond
Robert Epstein
Jean Paul Gagnon*
Bryan Luce
Eva Lydick
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Joel Hay
William McGhan
Donald Patrick
Jim Smeeding
Sean Sullivan
George Torrance
Milton Weinstein
Marilyn Dix-Smith
ISPOR Scribe - Daniel Klim
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OBJECTIVES
• Describe Mission and Membership
• Discuss Structure and Activities
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STRUCTURE
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• Council members identify and freely discuss wide
ranging issues at bi monthly meetings (Think Tank
approach)
• One month after meeting, lead constructs a draft
brief following approved format
• After Council discussion at next meeting second
draft constructed
• Final approval by Council and referral to Board for
discussion and action
• Lead turns brief into Board action plan
• Two weeks before meeting new issues submitted
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to Committee Scribe
COUNCIL FORMAT FOR TWO
PAGE BRIEF ON ISSUE
• TITLE
• DESCRIPTION
• ANALYSIS
• ALTERNATIVE RESPONSES
• RECOMMENDED ACTION
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BOARD FORMAT FOR
APPROVED ISSUE
• DEFINE ISSUE
• DESCRIBE SPECIFIC TASKS AND/OR PRODUCTS OF
ACTION PLAN
• PRIORITIZE THE INITIATIVES’ WORK ACTIVITIES
FOR CURRENT YEAR
• PROVIDE CLARITY FOR OPERATIONALIZING THE
WORK ACTIVITIES, E.G., ORGANIZATION,
TIMELINES, AND SUGGESTED WORK GROUP
MEMBERS
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ACTIVITIES
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MEET EVERY TWO MONTHS
HELD SIX MEETINGS IN 2004/2005
DISCUSS CURRENT ISSUES
SURFACE NEW ISSUES AND GAUGE REACTION
REVIEW BOARD COMMENTS AND DISCUSS
ACTION PLAN
• OPTIONAL – LEAD OR OTHER COUNCIL
MEMBERS PARTICIPATE IN IMPLEMENTATION
OF ISSUE ACTION PLAN
• PRODUCED SIX ISSUES FOR BOARD
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OBJECTIVES
• Describe Mission and Membership
• Discuss Structure and Activities
• Present Outcomes for 2005
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POLICY ISSUES
• BOARD APPROVED
 BRIDGING
THE HEALTH MEASUREMENT GAP:
MISSION IMPOSSIBLE:
Lead – George Torrance -
 ROLE
OF OUTCOMES RESEARCH IN EVIDENCEBASED HEALTH CARE DECISION-MAKING:
Lead – Bryan Luce, Founder & Senior Research Leader, the
MEDTAP Institute at UBC
 CONTINUOUS
QUALITY IMPROVEMENT FOR
COST EFFECTIVE HEALTH CARE RESEARCH AND
GLOBAL POLICY:
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Leads – Bill McGhan, Professor, University of the Sciences
POLICY ISSUES
• BOARD APPROVED

DEVELOPING STANDARDS FOR DRUG COSTS IN
PHARMACOECONOMIC STUDIES:
 Lead – Michael Drummond, Director, University of York, Centre for Health
Economics, Heslington, York

TRANSFERABILITY OF ECONOMIC DATA: WHEN DOES A
DIFFERENCE MAKE A DIFFERENCE
 Lead – Michael Drummond, Director, University of York, Centre for Health
Economics, Heslington, York, Jim Smeeding, President JestaRx Group
• COUNCIL DISCUSSION
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ACCELRATING USE OF COST EFFECTIVENESS AND OUTCOMES
DATA BY HEALTH CARE DECISION MAKERS
 Leads – Rob Epstein, Vice President of Medical Affairs, Medco, John Clouse,
Director, Pharmacoeconomic Evaluations, United Health Care, Jean Paul
Gagnon, Director Public Policy, Sanofi-Aventis Pharmaceuticals
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NEW ISSUES
• Anyone can submit issue to Science
Policy Council for development and
discussion
• Members are open to ideas
• Each issue will be discussed and
debated
• Issue author will be recognized and
informed of issue status
• Send issues to Jean Paul Gagnon at
[email protected]
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Bridging the Health
Measurement Gap: Mission
Impossible?
Donald Patrick, University of Washington
Eva Lydick, New Mexico
George Torrance, McMaster University, Innovus
Research Inc., and Health Utilities Inc.
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The Issue
• There is no standard measurement of health
• Different agencies use different methods
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WHO, Statistical Agencies, NICE, etc.
• QALYs are not necessarily comparable
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SG, TTO, VAS
Public, Patients
EQ5D, HUI, QWB, etc.
• Should ISPOR propose a “reference case”
method?
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Analysis
• Is lack of comparability across studies a problem?
• Reference case does not preclude other methods
• How broadly to define the problem?
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QALY
QALY, WTP
QALY, WTP, HRQOL, PRO
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Tentative Recommendations
• ISPOR should take this on
• Organize a special workshop(s),
consensus, publish
• Maintain and update over time
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Where Does Outcomes Research
fit into Evidence-Based Health
Care Decision-Making?
Bryan Luce
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Selected Organizations
Using EBM
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BCBS’s Technology Evaluation Center
US Preventive Services Task Force
Clinical Practice Guidelines
CMS Medicare Coverage Advisory
Committee (MCAC)
• AHRQ’s Evidence-Based Practice
Centers
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Organizations Using EBM
(Cont.)
• UK’s National Institute for Clinical Excellence
(NICE)
• AMCP’s Format for Formulary Submission
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Multiple MCOs
• OHSU Drug Effectiveness Review Project
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13 Medicaid Agencies
Consumers’ Union BestBuyDrugs website
AARP’s ResearchRx website
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Organizations Using EBM
(Cont.)
• CMS’s MMA: Comparative Effectiveness
• CMS Interim Coverage: PCT/Registries
• Institute of Medicine:
EBM/Comparative Effectiveness PrivatePublic Initiative
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EBG in Practice: Three
Groups
• The “efficacy” group
• The “effectiveness” group
• The “cost-effectiveness” group
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The EBG “Efficacy”
Group
• Mantra is “minimize bias”
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Opt for maximizing internal validity of
studies at expense of generalizability
• Examples of organizations:
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Cochrane Collaboration
OHSU’s Drug Effectiveness Review
Project
Medicaid agencies (?), Consumers Union, AARP
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The EBG “Effectiveness”
Group
• Mantra: “Is it effective in the real world?”
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Opt for generalizability to populations and clinical
settings of interest (incl non-experimental evidence)
• Examples of organizations:
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CMS (MCAC, MMA, PCT/Registry Interim Coverage
Policy)
AHRQ’s EPCs
BCBS TEC
Clinical Practice Guidelines
IOM’s Effectiveness Initiative
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The EBG CostEffectiveness Group
• Mantra is “real world effectiveness and
real world value for money”
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Opt for generalizability often over long haul
and accuracy over precision
• Examples of organizations:
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Academy of Managed Care Pharmacy
U.K.’s National Institute for Clinical
Excellence (NICE)
AHRQ’s EPCs
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Specific Tasks:
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1. Recruit a senior advisory panel to
consist of both traditional clinical EBM
experts (e.g. Cochrane participants) and
HEOR researchers to recommend the
objective to be achieved, to help define
the problem and approach and to oversee
the work plan and work products
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2. Review, describe and report on
existing EBM applications by key
organizations in US and the rest of the
westernized world
3. Review and report on the EBM
methods literature including how
various parties define the of the words
“evidence-based medicine”, “best
evidence”, “systematic review” with
the intention of defining these words
for different contexts.
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4. Develop a consensus for the
definitions of words and phrases
above
5. Review and report on methods for
combining disparate sets of
evidence that include RCT evidence
and outcomes evidence
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6. Convene a 2 day workshop of key
opinion leaders who have different
concepts of the EBM application and
charge the group with engaging the
issues and developing a consensus
concerning the role that HEOR
(including observational data, modeling,
patient-reported outcomes, including
patient preference) should play in
different applications of EBM and the
methodological and reporting solutions.
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ISPOR Health Science Policy Council
Continuous Quality Improvement for
Cost-Effective
HealthCare Research
and Global Policy
Presenter:
William McGhan, PharmD, PhD
University of the Sciences
Philadelphia, Pennsylvania USA
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Issue Description
• While most ISPOR members and
practitioners are able to follow major
economic indicators and the release of
major economic papers, it has been
suggested that a process be developed
whereby ISPOR would systematically report
on trends in the overall quality of costeffectiveness studies and global policy.
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Issue Description (cont’d)
• Recommendations and reports from
ISPOR would be intended to advance
international health care efficiency and
quality and become a mainstay of our
global effort to improve the economic
and quality-of-life research and practice
in various health care sectors.
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Analyzing the Options
• Reports should be continually fostered inside and
outside ISPOR that quantify the adequacy in the quality
of publications and regional CEA analyses.
• ISPOR needs to monitor what qualitative, quantitative
and statistical methods require modification,
improvement or further development.
• It is important to monitor the quality of CEA guidelines
being used by various journals, organizations and
nations for analyzing new therapies and allocation of
resources.
• Educational materials and forums need to be provided
to improve CEA quality.
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Options for Action
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Commission a white paper on the state of CEA science & policy.
Generate report card system on the overall state of CEA science and
practice.
Issue periodic assessment reports on the overall quality of papers,
abstracts, journal guidelines, government guidelines.
Examine ISPOR awards program to assure that excellent reports and
research advances are recognized and encouraged (ranging from
researchers, policy makers, to clinical practitioners?).
Organize a special workshop on the topic bringing together representatives
of the various stakeholders (e.g., Statistical Agencies, technology
assessors, health economists, clinical researchers, health policy
researchers, payers and regulators). Publish the results.
Organize a special session on the topic at an upcoming ISPOR annual
meeting.
In relationship to improving economic modeling and transparency, it has
been suggested that ISPOR become an
online repository for published and “reference case” CEA models and
perhaps databases for which print journals have inadequate space.
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A List of ISPOR Initiatives:
How Well Are These Linked to Overall
Continuous Improvement?
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Research Practices
· ISPOR Research Initiatives
- ISPOR Health Science
Initiatives
- ISPOR Quality of Life
Initiatives
- Pharmacoeconomic Guidelines
· ISPOR Good Research
Practices
- ISPOR Code of Ethics
- Modeling Studies
- Retrospective Database
Studies
- CEA with Clinical Trials
- Real World Data Task Force
- Budget Impact Analysis Task
Force
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Research & Communication
Issues
· General
Pharmacoeconomics
Research and Use Issues
· Quality of Life Regulatory
Issues
· Health Science Research
Use Issues/General
· Use of Research in
Decision Making
· ISPOR Communications
Task Force
· ISPOR Abstract Quality
Assurance Task Force
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Changes in CEA Report Quality Over Time
1976-1997
N = 305
1998-2001
N = 228
* p<0.1 ** p<0.05
0%
20%
40%
60%
52%**
Presented study perspective
73%**
23%
30%
73%**
82%**
Discounted both costs and QALYs
Identified modeling software
43%
50%
83%
80%
Calculated net costs
68%**
82%**
Stated year of currency
Reported incremental ratios
46%**
69%**
89%
93%
Performed sensitivity analyses
Compared to results of related
CEAs
Discussed limitations
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100%
65%
65%
Disclosed funding source
Societal perspective
80%
57%
64%
84%**
74%**
Source: P. Neumann, N..V. Olchanski; A.B. Rosen; D. Greenberg; R. Chapman; P.W. Stone; J.
Nadai. ARE PUBLISHED COST-UTILITY ANALYSES IMPROVING? (Poster) ISPOR: Arlington,
VA, May 18-21, 2003.
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CONTENT ANALYSIS ISPOR ABSTRACTS
METHODS INDICATORS
Markov
Abstracts
Bootstrap
400
300
200
100
0
Sensitivity
Bayes
Confidence
Intervals
1998 1999 2000 2001 2002 2003 2004
Discounting
Source: 1. Smith MD and McGhan WF. ISPOR 10th International Meeting
(Poster) Washington, DC. May 15-18, 2005.
2. www.ispor.org/research_study_digest/index.asp (N=4605 abstracts)
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CONTINUOUS QUALITY IMPROVEMENT
Feedback
Analysis
Guideline
Guidelines
CEA / CUA
Economics
PRO
/ QOL
PRO/QOL
Outcomes
Outcomes
Measure
Measurement
Design
Intervention
Intervention
McGhan WF and Briesacher B. Implementing Pharmacoeconomic Outcomes
Management. PharmacoEconomics. Vol 6 (5): 412-416.
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REFERENCES
Reed SD et al. Conducting economic evaluations alongside multinational clinical
trials: Toward a research consensus. American Heart Journal. March
2005;149:434-43.
Neumann PJ, Stone PW, Chapman RH, Sandberg EA, Bell CM. The Quality of
Reporting in Published Cost-Utility Analyses, 1976-1997. Annals of Internal
Medicine. 2000; 132(12):964-72.
Neumann PJ, Greenberg D, Olchanski NV, Stone PW, Rosen AB. Growth and
quality of the cost-utility literature, 1976-2001. Value in Health. 2005;8(1):39.
Rosen AB, Greenberg D, Stone PW, Olchanski NV, Neumann PJ. Quality of
Abstracts of Papers Reporting Original Cost-Effectiveness Analyses. Medical
Decision Making. In press.
Neumann PJ, Greenberg D, Olchanski NV, Stone PW, Rosen AB. Growth and
quality of the cost-utility literature, 1976-2001. Value in Health. 2005;8(1):39.
Neumann PJ, Stone PW, Chapman RH, Sandberg EA, Bell CM. The Quality of
Reporting in Published Cost-Utility Analyses, 1976-1997. Annals of Internal
Medicine. 2000; 132(12):964-72.
Rosen AB, Greenberg D, Olchanski NV, Chapman RH, Neumann PJ. Reporting of
Key Data in Abstracts of Cost-Utility Analyses. Abstract SMDM, Chicago, IL,
October 19-22, 2003.
McGhan WF and Briesacher B. Implementing Pharmacoeconomic Outcomes
Management. PharmacoEconomics. Vol 6 (5): 412-416, 1994.
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TRANSFERABILITY OF
ECONOMIC DATA:
WHEN DOES A DIFFERENCE
MAKE A DIFFERENCE?
Michael Drummond
Centre for Health Economics
University of York
United Kingdom
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OUTLINE OF THE ISSUES
• Several factors, varying from location to location,
are thought to limit the transferability
(generalisability) of economic data.
• These factors include differences in relative
prices, practice patterns, availability of healthcare
resources, community values for health states.
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OUTLINE OF THE ISSUES
(Continued)
• Existing guidelines for economic evaluation
(formal and voluntary) take differing
positions on the relevance and admissibility
of data from outside the country of interest.
• Too much flexibility could lead to misleading
cost-effectiveness estimates; too much
restriction could lead to unnecessary
duplication of research.
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OUTLINE OF THE ISSUES
(Continued)
• A recent review of economic evaluations of
drugs conducted in Western Europe has
shown that:
 there
are variations in cost-effectiveness
from country to country;
 the differences are not systematic;
 they depend on the methods employed by
the analyst;
 the implications of these variations for
decision-making are not clear.
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Barbieri et al. Value in Health 2005; 8(1): 10-23.
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ISPOR’s PLANS
• To tackle the following issues:
 Which elements of economic data vary most
from setting to setting?
 Given the known variability, what would be
reasonable guidelines for accepting (or not
accepting) data from outside the country of
interest?
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ISPOR’s PLANS (Continued)
• Review existing national guidelines to extract
more detail on transferability recommendations.
• Analyse the studies in the Barbieri et al review
in more detail, to identify more precisely the
variation (from place to place) in the key
parameters.
• Organise an issues panel at a future meeting,
involving researchers and decision-makers, to
discuss these findings.
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DEVELOPING STANDARDS
FOR DRUG COSTS IN
PHARMACOECONOMIC
STUDIES
Michael Drummond
University of York
Jim Smeeding
University of Texas & JestaRx
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OUTLINE OF THE ISSUES
• Drug costs (of the study drug and comparator)
are major cost drivers in pharmacoeconomic
studies.
• The cost of the drug regimen involves not only
the price but also the impact of wholesale
discounts, pharmacy on-costs and assumptions
about wastage.
• In the USA, the Center for Medicare and
Medicaid Services (CMS) is proposing to base
reimbursement on Average Sales Price (ASP).
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OUTLINE OF THE ISSUES (Continued)
• Until recently most cost studies in the USA have
quoted Average Wholesale Price. ASP factors in
discounts and rebates.
• In addition, there is a growing theoretical
literature, often linked to discussions about
patent protection, that suggests that the market
prices for drugs are not good approximations to
the social opportunity costs.
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JANUARY 1, 2005 – ASP PLUS 6%
• Policy:
- Average Sales Price plus 6% (ASP plus 6%)
shall apply to payment(s) for drugs and
biologicals (under Medicare Part B – HCPCS)
that are furnished on or after January 1, 2005.
 No Grace Period
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ASP METHODOLOGY:
PHARMACEUTICAL SALES DATA
 Calculated
Quarterly (calendar):
3rd Quarter 2004 ASP for January 1, 2005 implementation;
4th Quarter 2004 ASP for April 1, 2005 Implementation.
 Average
Sales Price for each NDC, along
with the number of units sold (Weighted
Average).
 Average Sales Price shall include: volume
discounts, prompt pay discounts, cash
discounts, charge backs, and rebates to
the first point of sale.
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PROS & CONS
Pros:
• Market driven (not set by manufacturer or other source).
• Factors in discounts, rebates.
Cons:
• Very difficult to establish an ASP which is fair to all
providers.
• Very difficult to maintain – contracts are continually
changing with status of product.
• Variation from quarter to quarter.
• Difficult to capture all discounts (early payment, etc).
• ASP data – One Quarter Lag at beginning of New
Quarter.
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ISPOR’s PLANS
• To establish a Task Force or Working Group to
develop standards for drug costs in
pharmacoeconomic studies.
• To review current practice for estimating drug
costs in pharmacoeconomic studies undertaken in
major markets.
• To review the conceptual and methodologic
literature on drug prices, patent protection and
social opportunity costs.
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ACCELRATING USE OF COST
EFFECTIVENESS AND OUTCOMES
DATA BY HEALTH CARE DECISION
MAKERS
Rob Epstein, Vice President of Medical
Affairs,Medco
John Clouse, Director, Pharmacoeconomic b,
United Health Care
Jean Paul Gagnon, Director Public Policy,
Sanofi-Aventis Pharmaceuticals
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ISSUE DESCRIPTION
• Health care decision makers not incorporating
CEA in decision-making
• Inferior translation process, few decision makers
understand how to use CEA information
• Decision makers not getting information, e.g.,
don’t read “Value in Health”
• ISPOR not demonstrating value to decision
makers
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REDEFINING COMPETITION IN
US HEALTH CARE*
• Healthcare competition today works on the
wrong level, players engage in zero-sum
competition (dividing value rather than creating it)
• Players transfer costs onto one another, limit
access to care, hoard information and stifle
innovation
• Competition should occur at the level of
preventing, identifying and treating patients’
conditions and disease
*Porter M, Olmsted Weisberg, E., June 2004
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FIXING HEALTH CARE COMPETITION*
• Increase transparency in pricing to reduce
cost shifting, discrimination and other
inefficiencies
• Improving quality often substantially
reduces costs
• Use information to support value-based
competition
• Focus on value for patients, not just cost
*Porter M, Olmsted Weisberg, E., June 2004
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PORTER SUGGESTS THREE WAYS TO
DEMONSTRATE VALUE
• “Compete on it”, e.g., plug into, become part of
decision making systems used by decision makers,
i.e., work with PDPs, MAs, physicians, pharmacists,
patients and others
• ISPOR should proactively design decision making
system that includes all stakeholders and
demonstrates value
• ISPOR should direct its promotional activities at
insuring patients correctly use products and
services
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ANALYSIS
• Movement to accelerate availability of CEA,
e.g., FMCP FORMAT program for collecting PE
data
• However, no movement to translate data or
show decision makers how to contrast and
compare drugs or devices
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ALTERNATIVE RESPONSES
• Develop training courses or Internet modules that
show decision makers using cases how to
translate CEA data and together with other
variables make decisions
• Develop model procedure with steps decision
makers can follow to synthesize, understand and
use CEA and outcomes data to select drugs and
devices
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ALTERNATIVE RESPONSES
• Start process by using focus groups of
stakeholders to:
 Describe
and discuss how they arrive at formulary
or drug product decisions
 Suggest programs and tools needed to effectively
use CEA and other variables to make decisions
 Use later as advisory panel to provide feedback
on prototype translating tools and procedures
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RECOMMENDATIONS
• Build a solid set of procedures and tools that equip
decision makers with the skills to use CEA and other
variables to make decisions
• Use focus group to build and then evaluate the prototype
procedures, tools and educational program for using CEA
with other variables to make decisions
• Offer tested program of procedures and tools to decision
makers
• Once launched develop newsletter for users of ISPOR’s
decision making procedure and tools program that shares
tips for better decision making
• Work with AARP and CU to implement patient based
procedures and tools for using CEA and other data to
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Summary
• Described Mission and Membership
• Discussed Structure and Activities
• Presented Outcomes for 2005
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REACTION &
DISCUSSION
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Contact Information
Jean Paul Gagnon
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[email protected] 800-648-9499 Ext 6379
sanofi-aventis
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