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GPC-USA
FIRST ANNUAL MEETING
November 12th, 2013
Sean R. Tunis, MD, MSc. President and CEO
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MEETING GOALS
Report and discuss progress since May 1 launch of
GPC-USA
Panel discussions on several interesting topics and
initiatives relevant to the work of GPC-USA
Discuss uptake and impact of EGDs: what does
success look like; how do we promote and measure
Explore potential future areas of work
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GPC-USA UPDATE
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GREEN PARK COLLABORATIVE - USA
• A multi-stakeholder forum to support dialogue and
consensus on methodological standards for clinical
research
‒ Focus on comparative effectiveness and value
‒ Informed by evidence expectations of patients,
clinicians and payers
• Primary outputs are “effectiveness guidance documents”
‒ Condition and technology-specific study design
recommendations for researchers
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WHAT IS THE PROBLEM?
• “Because of the paucity of high quality
evidence, the data available – though
voluminous – may have little meaning or
value for informing clinical practice”
• Abernethy AP, Coeytaux RR, Carson K, et al. Technology assessment
report: report on the evidence regarding off-label indications for targeted
therapies used in cancer treatment. Agency for Healthcare Research and
Quality 2010
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GPC – USA ORGANIZATIONAL STRUCTURE
GREEN PARK COLLABORATIVE – USA
GPC Advisory Committee
Chair: Sean Tunis, MD, MSc
Technical Working Group Place in Therapy
Chair: Anne Schott, MD
Technical Working Group Patient-Centered Outcomes
in Diabetes
Chair: Nisa Maruthur, MD, MHS
Oncology Consortium
Lead: Donna Messner, PhD
Endocrine – Metabolic Diseases Consortium
Lead: C. Daniel Mullins, PhD
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GPC – USA ADVISORY COMMITTEE
Advisory Committee Structure
Gary Palmer Alexandra
Clyde Alan
Rosenberg James Murray
Sean Tunis
Chair
Mark
Skinner Amy
Abernethy Ethan Basch
Janet Corrigan
Ellen Sigal
Other
Payers
Life Sciences
Board Composition Key
Murray Ross
Lisa
Simpson/
Erin Holve
Lew Sandy
Ex Officio
FDA CMS PCORI NIH AHRQ VA Avalere
Bob
Temple
Patrick
Conway
Rachael
Fleurence
Michael
Lauer
Jean
Slutsky
David
Atkins
Tanisha
Carino
Murray
Sheldon
Louis
Jacques
Jason Gerson
Peter
Marks
Donna Cryer
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ADVISORY COMMITTEE PRIMARY ROLES
Provides input on technical work of GPC-USA, including:
o Feedback on EGD topics and scope of EGDs
o Process for developing methodological standards
Advice on dissemination and mechanisms to promote uptake of
EGD recommendations
Communications
o Between AC members and broader GPC membership
o Inform/advise CMTP about related work
Helps identify new consortia, EGD topics, other GPC work
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PANEL DISCUSSION
What are the Evidence Gaps Affecting Clinical Use of
Next Generation Sequencing Tests in Oncology?
Moderator: Patricia Deverka, MD, MS, MBE
Center for Medical Technology Policy
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ANNUAL MEETING PANEL DISCUSSION
What are the Evidence Gaps Affecting Clinical Use
of Next Generation Sequencing Tests in Oncology?
Panelists: Robert Lechleider, Chief Scientific Officer
United States Diagnostics Standards, Inc.
Alberto Gutierrez, Director, Office of In Vitro Diagnostics
Center for Devices and Radiological Health, FDA
Sarah Zimmerman, Medical Science Liaison
Illumina
Gary Lyman, Professor of Medicine
Duke University School of Medicine and the Duke Cancer Institute
Elaine Jeter, Medical Director
Palmetto, GBA
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11
DNA SEQUENCING COSTS
Wetterstrand KA. DNA Sequencing Costs: Data from the NHGRI Genome Sequencing Program (GSP) Available at: www.genome.gov/sequencingcosts. Accessed 9/16/13.
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12
WHAT ARE THE DIFFERENCES?
First Generation
Sequencing (Sanger)
• Widely available
• Accurate, well-defined method
• Identifies linear sequences of
nucleotides (DNA fragments)
• Throughput is main limitation
• Would cost ~$5-30 million to
sequence human genome and
take 60 years (if using one machine)
Next Generation
Sequencing (Massively parallel)
• High throughput sequencing of large
numbers of different DNA sequences
in a single reaction
• Advantages are speed, breadth and
depth of coverage • Unclear whether claims of lower (total)
costs of testing are realistic
• Large volume of information
produced has shifted emphasis to
bioinformatics and data analysis
components, as opposed to
technical assay component to
generate data
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13
GENOMIC SEQUENCING* AS A CONTINUUM
Whole genome sequencing
Whole exome sequencing
Gene panels
Single gene testing
*Inherited/germline vs. acquired/somatic
Clin
ical Utility
Dat
a vo
lum
e
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14
NGS: WHAT ARE THE ISSUES?
• Need to distinguish discussions of
technology itself from discussions of
various clinical applications • NIPT
• Oncology
• Rare childhood disorders
• DTC
• Need standards for AV and CV in
addition to CU
• For many applications the results are
dynamic, not static • Reimbursement as well as ethical implications
• Complexity of issues argues for focusing
on near-term priorities • e.g., NGS testing in oncology
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15
NGS TESTING IN ONCOLOGY
Redefining cancer
• Use NGS test to measure large number of molecular markers at
a single time
• Goal is to identify a treatment which targets the biological
pathway involving the marker
• Results in a different treatment that usually selected for patient
based on the type of cancer and its stage
• Choosing a treatment based on biological pathways rather than
tumor site or histology is hypothesized to be a better method of
selecting treatment
• Is this test a ‘bundle of markers’ or a new capability facilitating
comprehensive classification & management of tumors?
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16
PAYER ASSESSMENT OF NEW TESTS
Analytic validity
Clinical validity
Clinical utility
Does the test accurately/reliablydetect analyte(s) of interest?
Is there a significant association between the test results and clinical phenotype?
Does use of the test lead to improved patient outcomes compared with the alternative? (test must impact clinical decision-making)
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17
PANEL DISCUSSION
• Does this current framework apply to
NGS testing in oncology?
• How will payers, clinicians and patients be
confident in the analytic and clinical validity of
oncology tests?
• How will test results differ across different
platforms?
• What is the clinical significance of these
differences? Of known issues re error rates?
• What role should FDA play?
• Will the current definition of clinical utility
need revisiting given small numbers of
patients with particular genomic profile?
• What role can group like GPC-USA play
to facilitate evidence for informed
decision-making?
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GPC-USA ONCOLOGY
CONSORTIUM
UPDATE
Donna A. Messner, PhD | November 12, 2013
Consortium team: Ellen Tambor, Srijana Rajbhandary
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POLICYMAKERS & REGULATORS
RESEARCH FUNDERS
LIFE SCIENCES INDUSTRY
PATIENTS AND CONSUMERS
PAYERS AND PURCHASERS
HEALTHCARE PROVIDERS
RESEARCHERS
Eli Lilly and Company Foundation Medicine, Inc. Genentech, Inc. GlaxoSmithKline Merck Millennium Novartis US Diagnostic Stds
National Coalition for Cancer Survivorship The Avon Foundation Breast Center (JHU) Research Advocacy Network Patient Advocates in Research
Aetna BCBSA CMS United Health Group WellPoint
Veterans Affairs Kaiser Permanente
Duke University U of Michigan/SWOG Angiogenesis Foundation
ASCO FDA NCCN
NCI PCORI
ONCOLOGY CONSORTIUM AT A GLANCE
CLINICIANS
Community oncologists (pending)
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PROCESS TO NARROW TOPIC LIST FOR DISCUSSION
Literature review,
analysis of evidence gaps
Creation of prospective
topic list
Narrowing of topic list for
in-depth consideration
20 phone calls with 23
people plus 2 written
comments
In-depth discussion of two topics in
inaugural meeting and group voting
DEC
ISION
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SELECTED TOPIC: “PLACE IN THERAPY”
• For situations where multiple therapeutic options exist
and the optimal priority order (individual or in
combination) of administration has not been established
• Methodologic recommendations and data collection
strategies for efficient evaluation of the most beneficial
ordering of therapies for cancer patient management
• The recommendations will include consideration of
patient-centered preferences in tradeoffs between
effectiveness or other benefits vs. toxicity, cost,
convenience of administration, life style alterations
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IMPORTANCE OF TREATMENT SEQUENCING
EXAMPLE: RENAL CELL CARCINOMA (RCC)
• Treatment Options RCC:
– Mid-2000s - Cytokine therapy was the treatment of choice for
mRCC
– December 2005 – February 2012 – 7 new agents approved for
the treatment of mRCC (sorafenib, sunitinib, temsirolimus,
everolimus, interferon/bevacizumab, pazopanib, axitinib)
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NCCN GUIDELINES – KIDNEY CANCER
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WHAT IS THE ‘EVIDENCE GAP’ IN TREATMENT SEQUENCING IN RCC?
• Recently approved products (except temsirolimus) were approved based on improvements in PFS.
• Oncologists can now sequence therapies from the available treatment options, however, there is a lack of H2H comparative evidence to enable their decision about which treatment option to select and the sequencing of different therapies.
• Limited comparative effectiveness evidence from clinical trials across multiple therapies has resulted in Oncologists relying on what is observed in clinical practice (“real world”) or retrospective studies.
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PROCESS FOLLOWING TOPIC SELECTION
Assemble appropriate TWG
and designate chair
TWG helps to develop scope,
frames recommendations
in collaboration with consortium
Consortium provides input,
reviews recommendations
Recommendations finalized by CMTP
with TWG and consortium
DEC
ISION
DISSEM
INA
TION
September 2013
April 2014
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TECHNICAL WORKING GROUP TARGET COMPOSITION
TWG Chair: Dr. Anne Schott,
U of Michigan, SWOG
Methodologist (PROs)
Methodologist (decision
modeling)
Methodologist (Registries ,
EHR, and data mining)
Patient (2)
Med center P&T (clinician,
pharmacologist)
Community practice
buying group member
Oncologists (2)
(community)
Oncology clinical trialist
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GPC-USA ENDOCRINE-
METABOLIC CONSORTIUM
PROGRESS REPORT
Nisa M. Maruthur, MD, MHS | November 12, 2013
Consortium team: C. Daniel Mullins, PhD, Srijana Rajbhandary
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POLICYMAKERS & REGULATORS
RESEARCH FUNDERS
LIFE SCIENCES INDUSTRY
PATIENTS AND CONSUMERS
PAYERS AND PURCHASERS
HEALTHCARE PROVIDERS
RESEARCHERS
Covidien Eli Lilly and Company GlaxoSmithKline Medtronic, Inc. Merck Novartis Sanofi
ADA National Alliance for Caregiving Individual patients
Aetna BCBSA Highmark Johns Hopkins Healthcare New Hampshire Medicaid United Health Group Wellpoint
The Johns Hopkins Hospital and Johns Hopkins Health System
Duke University HealthPartners Institute Johns Hopkins University MedStar Health Research Institute Universities of : Illinois, Maryland, Michigan
CDC Maryland Department of Health and Mental Hygiene
NIA PCORI
ENDOCRINE-METABOLIC CONSORTIUM AT A GLANCE
CLINICIANS
The Frist Clinic VA Maryland Health Care System
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ENDOCRINE-METABOLIC CONSORTIUM
TECHNICAL WORKING GROUP MEMBERS (TO DATE)
Member Affiliation
Carolyn Alexander The Community College of Baltimore County
John E. Anderson, MD American Diabetes Association, The First Clinic
Vanita R. Aroda, MD MedStar Health Research Institute
Ranee Chatterjee, MD,MPH Duke University
Jon Eaton, MCP, MCSA Leach Wallace Associates, Inc.
Hugh Fatodu, RPh, MBA Johns Hopkins HealthCare
Felicia Hill-Briggs, PhD, ABPP Johns Hopkins Medical Institutions
Mary Muth Kats and Associates/Keller Williams
Robert E. Ratner, MD, FACP American Diabetes Association
Deneen Vojta, MD UnitedHealth Group
Consortium Lead C. Daniel Mullins, PhD University of Maryland School of Pharmacy
TWG Chair Nisa M. Maruthur, MD, MHS The Johns Hopkins University
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EGD PROCESS: EMC-YEAR 1
Literature review,
analysis of evidence
gaps
CER Workshop
on Diabetes - October
2012
Preliminary draft recommendations;
TWG recruiting; early TWG
response to recommendations
Key Informant
Interviews -
Preliminary diabetes
topic
Discussion of draft recommendations
EMC Meeting
10/23/13
Refin
em
en
t
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EGD PROCESS: EMC-YEAR 1
In-person TWG
meeting –
12/16/13
TWG refines EGD scope and
recommendations based on EMC input
EMC input/review of
recommendations
Recommendations finalized by CMTP
with TWG and Consortium; EGD
released
Refin
em
ent
DISSEM
INA
TION
December 2013
April 2014
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EMC MEETING 10/23/13
SCOPE OF DIABETES EGD
• Focus on type 2 diabetes
• Focus on medication interventions
• Use PICOT framework rather than
focusing on PROs alone
• Devices deserve separate guidance
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EMC MEETING 10/23/13
DRAFT RECOMMENDATIONS
Population
• Selection criteria should purposefully oversample targeted
populations
– including patients with advanced age, diverse race/ethnicity,
level of diabetes control, and co-morbid conditions
• Subgroup analyses for age, race/ethnicity, and HbA1c subgroups
should be conducted uniformly across studies using pre-defined
categories
– Age: <65 and ≥65 years
– Race: white, African American, Asian, Native American
– Ethnicity: Hispanic and non-Hispanic
– Baseline HbA1c: <8.0% and ≥8.0%
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EMC MEETING 10/23/13
DRAFT RECOMMENDATIONS
Outcomes
• Evaluate patient-reported outcomes (PROs) in a
standardized fashion across studies using validated
instruments
– Domains: global health; physical health; mental health; and
social health
• Establish definitions for and measure clinically-relevant,
treatment- related weight change
• Provide credible evidence of the link between
intermediate or surrogate outcomes and long-term
cardiovascular and cerebrovascular outcomes
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EMC MEETING 10/23/13
DRAFT RECOMMENDATIONS
Methods
• Distinguishing subpopulation characteristics (e.g., cultural beliefs
about treatment) should be incorporated in study design and
analysis
– Consideration of appropriate covariates.
• System level studies should collect and analyze relevant patient-
level, provider level, and system-level variables
• Define measures of adherence and barriers to adherence and
assess these uniformly.
• Studies should use rational comparators based on the established
highest standard of care
– including optimal dosing, intensity, and follow up.
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EMC MEETING 10/23/13
DRAFT RECOMMENDATIONS
Additional Patient-centered Considerations
• Measure factors related to cost and patient burden which are likely
to impact outcomes of the diabetes intervention once applied in a
real-world setting.
• Evaluate decision support tools (incorporating benefits, harms, and
burden) which allow patients to participate in diabetes treatment
decision-making.
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LUNCHEON PRESENTATION
THE COMET (CORE OUTCOME MEASURES IN
EFFECTIVENESS TRIALS) INITIATIVE
PAULA WILLIAMSON
MRC NORTH WEST HUB FOR TRIALS METHODOLOGY
RESEARCH
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Core Outcome Measures in Effectiveness Trials
www.comet-initiative.org
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Acknowledgements
• COMET Management Group:
Doug Altman, Jane Blazeby, Mike Clarke, Paula Williamson
• COMET project coordinator: Elizabeth Gargon
• Collaborators: Peter Tugwell, Maarten Boers, Caroline Terwee, Holger Schunemann, Michael Rose, Sunita Vohra, Roberto D’Amico, Lorenzo Moja
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International Advisory Group
• Professor Jacques Demotes-Mainard (ECRIN)
• Dr Irmgard Eichler (EMA)
• Professor David Flum (PCORI)
• Dr Piero Olliaro (WHO)
• Dr Sean Tunis (CMTP)
• Ms Liz Whamond (Cochrane)
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DMARD trials for rheumatoid arthritis
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Health care research is untidy
• It needs to be tidied up if it is to achieve its aim of helping practitioners and patients to improve health care and health
• This needs initiatives such as the Cochrane Collaboration and Green Park Collaborative
• And COMET …..
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Core outcome set
• An agreed standardised set of outcomes that should be measured and reported, as a minimum, in all clinical trials in specific areas of health or health care
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Scope of a COS
• “The specific area of health or healthcare that the COS is to apply to, in terms of health condition, population and types of interventions needs to be determined.”
• All stages or severity of a specific health condition or focussed on a particular disease category
- e.g. in colorectal cancer, a COS might be developed for all patients or it may focus on patients with metastatic disease
• All treatment types or for a particular intervention
- e.g. in morbid obesity, a COS may be created to use in trials of all interventions or just bariatric surgery alone
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Improvements over time (Kirkham et al, Trials 2013)
Studies reporting
full RA COS (%)
WHO/ILAR
RA COS
EMA
guideline
FDA
guideline
100
80
60
40
20
1985 1990 1995 2000 2005 2010
0
Mean number of
clinical outcomes
6.0
6.5
7.0
0.0
drug studies
non-drug studies
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The COMET Initiative • To raise awareness of current problems with
outcomes in clinical trials
• To encourage COS development and uptake
• To provide resources to allow practitioners to develop COS, e.g. COMET database, guidance
• An international network of trialists, systematic reviewers, health service users, practitioners, editors, funders, policy makers, regulators
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COMET Initiative
• ‘What’ to measure
• ‘How’ to measure (validity, reliability, feasibility)
- COSMIN
- PROMIS
- PROQOLID
- TREAT-NMD ROM
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Website
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Background
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COMET Database
• Systematic review - 28,000+ abstracts
• 198 published COS
- 62 explicitly for a particular intervention type (e.g. drugs, surgery, etc)
- 35 explicitly involved patients or carers
- 147 involved US/Canada
• Plus a further 50+ ongoing COS projects
• Other relevant articles
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Scope
Identifying existing knowledge
Stakeholder involvement
Consensus methods
Achieving global consensus
Regular review, feedback, updating
Implementation
Clear presentation
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Stakeholder involvement
• Health care practitioners
• Patients, carers, representatives
• Regulators
• Industry representatives
• Researchers
• Stage of involvement may vary by group
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Patient and public involvement
• Impact of involvement
– Rheumatology (OMERACT): fatigue
– Chronic pain (IMMPACT): expansion of previously proposed core outcome domains
– Multiple sclerosis: fatigue, continence
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Professor Hywel Williams, Chair of the NIHR HTA
Commissioning Board: ‘Patients and professionals
making decisions about health care need access to
reliable evidence. The new COMET database will help
researchers across the NIHR family and beyond when
choosing the outcomes to include in the studies that will
establish this evidence base'.
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Response to EMA consultation SINHA ET AL
REDDELL ET AL BUSSE ET AL
5-11 years 12+ years
TOP 6 PARENTS +/- CLINICIANS ESSENTIAL OPTIONAL ESSENTIAL OPTIONAL ESSENTIAL OPTIONAL
Symptoms
√ √ √
Exacerbations
√ √ √ √
QoL
√ √ √ √
Death
√ √
Normal activities
√ √
Exercise ability
√
Reliever use
√ √
Lung function
√ √ √
Tx side effects
√
Healthcare utilisation
√ √ √
Biomarkers
√
Hyper-responsiveness
√
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NICE clinical guidelines
• Selection of outcomes always been important
• GRADE since 2009 - assesses the quality of the evidence by outcome rather than study
• PICO framework for each clinical question
• Addition to methods within NICE to check the COMET database
• Project underway comparing published COS with outcomes in related NICE clinical guidelines
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Journal endorsement
• Core Outcomes in Women’s Health (CROWN)
- a consortium of 29 O&G journals to implement core outcome sets across the board, including AJOG, BJOG, Cochrane, etc
• Co-ordinating rapid peer-review of manuscripts describing core outcome sets and publishing these simultaneously across participating journals
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Future work
• Maintain and keep the database up to date
• Quality assessment tool
• Methods for COS development
• Methods for engaging consumers - initial meeting of UK PPI organisations
• Methods to promote implementation
• Monitoring uptake
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COMET and GPC
• GPC identifies priority clinical areas
• COMET searches database and non-database list (50+ ongoing projects)
• Existing COS: GPC appraisal
• Gap identified: GPC and COMET facilitate COS development
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ANNUAL MEETING PANEL DISCUSSION
Improving the Science & Methods of Patient-Centered Outcomes Research:
The PCORI Methodology Report
Moderator: Sean Tunis, CMTP
Panelists: Naomi Aronson
Blue Cross and Blue Shield Association
Ethan Basch
University of North Carolina at Chapel Hill
Deborah Collyar
Patient Advocates in Research
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ANNUAL MEETING PANEL DISCUSSION
Medical Devices: Prospects & Priorities for Developing Methodological
Standards
Moderator: Tanisha Carino, PhD, Avalere
Panelists: Robert Giffin, PhD
Vice President, Health Economics & Outcomes Research, Covidien
Louis Jacques, MD
Director, Coverage & Analysis Group, CMS
Danica Marinac-Dabic, MD PhD
Director of the Division of Epidemiology, FDA
Murray Sheldon, MD
Associate Director for Technology and Innovation, FDA