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Welcome

Please be seated by 10:40.

The teleconference will go live at 10:45.

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Assessment of Prevention, Diagnosis, and Treatment Options Advisory Panel Meeting

January 13 – 14th, 2014

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Welcome: 10:45 am – 11:15 am

David Hickam, MD, MPH

Program Director Clinical Effectiveness Research PCORI

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Housekeeping

Today’s teleconference is open to the public and is being recorded

• Members of the public are invited to listen to this teleconference
• Meeting materials can be found on the PCORI website
• Comments may be submitted via email to

advisorypanels@pcori.org; no public comment period is scheduled

For those in the room, please remember to speak loudly and clearly into a microphone Where possible, we encourage you to avoid technical language in your discussion

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Meeting Objectives

Discuss important research gaps for 2 clinical topics and prioritize high priority research questions for both topics.

• Treatment of migraine headaches
• Treatment of osteoarthritis

Prioritize 14 new clinical topics and select a subset

• f topics for further consideration as research

priority areas.

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Background: Topic Prioritization

1,300+ Research Topics Received 841 Topics Accepted

• Program director screened and

consolidated topics

• Topics scored on 4 criteria

594 Assigned to Assessment of Options

• In April 2013, Advisory Panel reviewed

and prioritized 20 topics

20 High Scoring Topics Considered

• (1) Bipolar Disorder
• (2) Ductal Carcinoma in situ
• (3) Migraine Headache
• (4) Osteoarthritis

4 Priority Topics Selected

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Introductions: The Clinical Effectiveness Research Program Team

Diane Bild, MD, MPH David Hickam, MD, MPH Katie Rader Julie McCormack, MA Sandi Myers Stanley Ip, MD Jana-Lynn Louis, MPH Hal Sox, MD

Moderators

Alvin I. Mushlin, MD, ScM

Chair, Panel on the Assessment of Options Chairman, Department of Public Health, Weill Cornell Medical College; Public Health Physician-in-Chief, New York Presbyterian Hospital/Weill Cornell Medical Center

Margaret F. Clayton, RN, PhD

Co-chair, Panel on the Assessment of Options Associate Professor, College of Nursing and Co-Director of the PhD Program, University of Utah

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Welcome New Panel Member

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Angela Smith, MD

Assistant Professor, Surgery / Urology Lineberger Comprehensive Cancer Center UNC-Chapel Hill School of Medicine

Advisory Panel Members

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Thank you

Denise Kruzikas, PhD, MPH

Director, Health Economics & Reimbursement, Ultrasound; Director, Healthymagination, Global Ultrasound Strategy, GE Healthcare

Margo Halm, PhD, RN, ACNS-BC

Director of Nursing Research, Professional Practice and Magnet, Salem Hospital

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Agenda Overview

Time Agenda Item 10:45-11:15 a.m. Welcome and Overview of the Agenda 11:15-11:30 a.m. Procedures for Research Question Prioritization 11:30-1:00 p.m. Discussion of Key Research Questions for Migraine Headache 1:00-2:00 p.m. Lunch 2:00-2:15 p.m. Voting – Migraine Headache 2:15-3:30 p.m. Discussion of Key Research Questions for Osteoarthritis 3:30-3:45 p.m. Break 3:45-4:00 p.m. Voting – Osteoarthritis 4:00-4:15 p.m. Update on the Bipolar Topic 4:15-4:30 p.m. Update on the DCIS Topic 4:30-4:45 p.m. Review Voting Results 4:45-5:00 p.m. Next Steps/Announcements 5:00pm Adjourn

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Voting Process for Research Question Prioritization

Forced-ranking prioritization method

• For migraine, 5 votes per person, which could be

allocated to any of the 13 research priorities

• For osteoarthritis, 5 votes per person, which could be

allocated to any of the 12 research priorities

• Maximum of 3 votes per item

Rankings will be completed after discussion of topic

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Future Research Prioritization: Management Strategies for Migraine Headache: 11:30 am – 1:00 pm Douglas McCrory, MD, MHS

Duke Evidence Synthesis Group

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Future Research Prioritization: Preventive Treatments for Episodic and Chronic Migraine in Adults

Douglas McCrory MD, MHS Duke Evidence Synthesis Group January 13, 2014

Treatment Strategies for Episodic and Chronic Migraine

Migraine is a recurring disorder characterized by severe headache, generally associated with nausea, vomiting, and

• ther neurologic symptoms during attacks, with no symptoms

between attacks

• Can be episodic or chronic (occurs on 15 or more days per month for

more than 3 months)

Precipitating factors include stress, menstruation, weather changes, fasting, wine (aged or fermented food/drink) Focus of management is on prevention Management options include: drug treatments, behavioral therapies, spinal manipulation, other physical treatments Uncertainty in the effectiveness of preventive treatment for migraine

During the April 2013 meeting, this PCORI Advisory Panel ranked preventive treatments for episodic and chronic migraine in adults as HIGH PRIORITY for targeted future research funding

Overall Project Goal

To work with stakeholders to help PCORI identify, refine, and prioritize future research evidence gaps in the area of strategies for treating patients with episodic or chronic migraine

Overview of Project

• 1. Identifying Known Evidence Gaps
• 2. Creation of Stakeholder Group
• 3. Expansion of Evidence Gaps
• 4. Analytic Framework
• 5. Stakeholder Prioritization
• 6. Horizon Scan
• 7. Study Design Considerations

Identifying Known Evidence Gaps

Review of published systematic reviews, clinical practice guidelines, and future research needs documents Initial 30 evidence gaps explored:

• Specific populations or subgroups of patients
• Comparative safety and effectiveness of available

interventions and comparators

• Impact of treatment of specific outcomes of interest
• Optimal timing or setting for treatment

Creation of Stakeholder Group

Alliance for Headache Disorders Advocacy American Academy of Family Physicians American Academy of Neurology American Academy of Pain Management American Academy of Pain Medicine American Council for Headache Education (ACHE) American Headache Society American Migraine Foundation American Neurological Association American Society for Clinical Pharmacology and Therapeutics International Headache Society Migraine Research Foundation National Headache Foundation Society for Pain Practice Management Society of Behavioral Medicine World Federation of Neurology Patient Advocates

Stakeholders

Andrew Blumenfeld, M.D. Kaiser Permanente Headache Center of Southern California Roger K. Cady, M.D. Associate Executive Chairman Headache Care Center Seymour Diamond, M.D. Director Emeritus and Founder National Headache Foundation Frederick Freitag, D.O., F.A.H.S. Associate Professor Medical College of Wisconsin Cathy Glaser President Migraine Research Foundation Richard B. Lipton, M.D. Director Montefiore Headache Center Bray Patrick-Lake, M.F.S. Patient Representative Elizabeth Loder, M.D. Chief, Division of Headache and Pain Brigham and Women’s Department of Neurology Donald Penzien, Ph.D. Director, Head Pain Center Professor, University of Mississippi Alan M. Rapoport, M.D. Clinical Professor of Neurology David Geffen School of Medicine UCLA Stephen D. Silberstien, M.D., F.A.C.P. Director Jefferson Headache Center Heather Zantisch Patient Representative

Stakeholder Discussions

Expanded initial list of 30 gaps to 34 evidence gaps

• 4 questions deleted
• 2 questions merged
• 9 new questions created (3 related to populations, 2 to

interventions, 4 to outcomes)

Recognition of central role that patients play in the prevention and management of their migraine Overlapping nature of the clinical conditions commonly referred to as chronic migraine, chronic daily headache, and medication overuse headache Consideration of comorbidity (i.e., anxiety, depression, and other pain syndromes)

Future Research Needs Broad Topic Areas Covering 34 Questions

• 1. Population differences (disease/patient

characteristics, treatment modifying effects, comorbidities)

• 2. Pharmacologic and nonpharmacologic

interventions

• 3. Episodic and chronic migraine (and

transformation)

• 4. Decision making interventions
• 5. Patient-centered outcomes
• 6. Timing and setting

Outcomes (both intermediate and long-term):

• 20. Effect of stigma on outcomes or care seeking
• 21. Emergency department utilization

22, 23. Quality of life with pharmacologic and nonpharmacologic treatment

• 26. Long-term effectiveness of combined pharmacologic +

nonpharmacologic treatment

• 27. Most important patient-centered outcomes
• 28. Effects of pharmacologic treatments on healthcare system

utilization/costs

• 29. Role of pharmacologic treatments in advancing obesity,

etc.

• 30. Adverse effects of pharmacologic treatment
• 31. Adverse effects of nonpharmacologic treatment

Population: Adults with episodic migraine Impact of population factors:

• 1. Biomarkers

2, 3. Disease and patient characteristics affecting efficacy

• 6. Family history, comorbidities,

duration of illness, treatment history

• 7. Factors linked to transformation from

episodic to chronic migraine 8, 9. Barriers, adherence to prevention

• 10. Comorbid psychological, metabolic,

gastrointestinal disorders

• 11. Refractory migraines
• 12. Disease model

Effectiveness of interventions: Pharmacologic and nonpharmacologic interventions for preventive treatment Impact of intervention factors, timing, and setting:

• 13. Pharmacologic combinations vs.

single agents

• 14. Nonpharmacologic vs. pharmacologic
• vs. combined
• 16. Decision-making tools
• 17. Prevention of medication overuse

headache

• 18. Insurance coverage

32, 33. Factors associated with discontinuation and success of preventive approaches

• 34. Management by headache specialists
• vs. interdisciplinary teams vs. neurologists
• vs. primary care providers

Analytic Framework: Episodic Migraine

Outcomes (both intermediate and long-term):

• 19. Defining treatment success
• 20. Effect of stigma on outcomes or care seeking
• 21. Emergency department utilization

24, 25. Long-term effectiveness of pharmacologic, nonpharmacologic treatment

• 26. Long-term effectiveness of combined pharmacologic +

nonpharmacologic treatment

• 27. Most important patient-centered outcomes
• 28. Effects of pharmacologic treatments on healthcare system

utilization/costs

• 29. Role of pharmacologic treatments in advancing obesity,

etc.

• 30. Adverse effects of pharmacologic treatment
• 31. Adverse effects of nonpharmacologic treatment

Population: Adults with chronic migraine Impact of population factors:

• 1. Biomarkers

4, 5. Disease and patient characteristics affecting efficacy

• 6. Family history, comorbidities, duration of

illness, treatment history

• 7. Factors linked to development of chronic

migraine/transformation from episodic to chronic

• 8. Barriers to prevention
• 10. Comorbid psychological, metabolic,

gastrointestinal disorders

• 11. Refractory migraines
• 12. Disease model

Impact of intervention factors, timing, and setting:

• 13. Pharmacologic combinations
• vs. single agents
• 15. Nonpharmacologic vs.

pharmacologic vs. combined

• 16. Decision-making tools
• 17. Prevention of medication
• veruse headache
• 18. Insurance coverage

32, 33. Factors associated with discontinuation and success of preventive approaches Effectiveness of interventions: Pharmacologic and nonpharmacologic interventions for preventive treatment

Analytic Framework: Chronic Migraine

Stakeholder Prioritization

Online ranking of evidence gaps Forced-ranking prioritization method

• 12 votes per stakeholder, which could be allocated to

any of the 34 research priorities

• Maximum of 3 votes per item

Asked to rank based on “most important unanswered research question in strategies to treat episodic or chronic migraine” Questions divided into a top, middle, and lower tier Only top tier moved on to final stage of horizon scan and study design considerations

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Stakeholder Prioritization – TOP TIER

Question

Score N

What strategies for treatment of individual headache episodes are most effective and least likely to promote medication overuse headache in patients with high frequency episodic or chronic migraine? 9 6 How should research on preventive pharmacologic or nonpharmacologicb treatments for chronic migraine define treatment success? How should patients participate in defining treatment success? 8 6 Are combinations of pharmacologic treatments effective in preventing episodic migraine in patients for whom treatment with a single preventive drug has been either ineffective or intolerable? Are there fewer or more adverse events when using low dose polypharmacy versus single agents at higher doses? Do treatment efforts that align with patient-defined values improve the efficacy of preventive treatments for episodic and chronic migraine? 8 5 What biomarkers help predict response to preventive treatment in patients with episodic or chronic migraine? 8 4 What is a valid disease model to accurately describe disease characteristics and treatment mechanisms for patients with episodic or chronic migraine? 8 4 Which disease and patient characteristics (duration, severity, frequency of attacks, comorbidities) affect the efficacy of preventive pharmacologic treatments for chronic migraine? 7 6 How does the comorbidity of depression and related psychological disorders, metabolic disorders, and gastrointestinal disorders affect the efficacy of preventive pharmacologic or nonpharmacologic treatments? 6 6 What patient, treatment, and other factors (e.g., age of onset, diet, psychosocial stress, early life trauma) are associated with the development of chronic migraine, or the transformation from episodic to chronic migraine? 6 4 What are the effects of decision-making tools compared to usual care to assist patients and providers with shared decision-making regarding migraine, with a focus on individuals with low health literacy and limited health access compared to usual care? 6 4 Which disease and patient characteristics (duration, severity, frequency of attacks, comorbidities) affect the efficacy of preventive nonpharmacologic treatments for chronic migraine? 5 4 What are the patient factors that determine whether patients with episodic migraine are prescribed, take, and adhere to preventive approaches? 5 4 What are the most important patient-centered outcomes for patients receiving pharmacologic or nonpharmacologic treatments for the prevention of episodic or chronic migraine? How do these differ depending on patient or provider perspective? 5 4 What are the comparative safety, tolerability, and effectiveness of nonpharmacologic versus pharmacologic versus combined (nonpharmacologic + pharmacologic) treatments in the prevention of chronic migraine? 5 3

Horizon Scan

PubMed

• 2323 articles identified
• 99included as potentially relevant to top tier questions
• 9 systematic reviews, 30 RCTs, 37 cohort studies, 1

case-controlled study, and 22 other studies

ClinicalTrials.gov

• 93 open protocols identified
• 24 included as potentially relevant to top tier questions
• Sample sizes ranged from 28 to 1,400 patients
• Estimated completion ranged from overdue to

November 2017

Horizon Scan Summary

Question SR s RCTs Cohort Case Control Other Ongoing

Least likely to promote MOH 1 2 3 4 Define treatment success 1 1 3 1 Combination pharmacologics 1 2 2 6 Biomarkers 2 6 1 4 Disease model 1 1 1 1 Pt/disease characteristics  pharmacologics for chronic migraine 4 1 4 Comorbidities 1 3 Transformation episodic chronic 1 12 7 3 Decision making tools 3 1 Pt/disease characteristics  nonpharmacologics for chr. migraine 3 1 1 Patient adherence 2 1 2 2 Most important PC outcomes 1 2 2 1 CER prevention chronic migraine 7 22 7 3 15

Study Design Suggestions

Question RCT Meta Analysis Observational Model

RCTs Observ . New Data Existing Data

Least likely to promote MOH Yes No No ? No ? Define treatment success No ? ? Yes ? No Combination pharmacologics Yes ? ? ? ? ? Biomarkers ? ? ? Yes Yes No Disease model No No No ? No ? Pt/disease characteristics  pharmacologics for chronic migraine ? ? Yes Yes Yes ? Comorbidities ? ? Yes Yes Yes ? Transformation episodic chronic No No ? ? Yes No Decision making tools Yes ? No Yes Yes Yes Pt/disease characteristics  nonpharmacologics for chr. migraine ? ? ? Yes Yes ? Patient adherence No No ? Yes Yes No Most important PC outcomes No No No Yes ? No CER prevention chronic migraine Yes ? ? Yes Yes ?

Summary: Stakeholder Priorities

Future research priorities should focus on:

• better information about which patient and disease factors

are associated with key outcomes

• better understanding of which outcomes patients with

chronic migraine value most and how treatment success should be defined in research on chronic migraine

• efficacy of preventive treatment in specific understudied

areas

Questions related to chronic migraine higher priority than questions pertaining to episodic migraine

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Discussion: Research Gaps in Migraine Headache

How well do the research questions meet the 5 PCORI criteria?

• Patient-Centeredness
• Impact of the Condition on the Health of Individuals and

Populations

• Options for Addressing the Issue
• Likelihood of Implementation in Practice
• Durability of Information

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Lunch 1:00 pm – 2:00 pm

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Voting – Migraine Headache 2:00 pm – 2:15 pm

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Future Research Prioritization: Osteoarthritis: 2:15 pm – 3:30 pm

Jennifer Gierisch, PhD

Duke Evidence Synthesis Group

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Future Research Prioritization: Effectiveness of Alternative Strategies for Stabilizing Symptoms in People with Osteoarthritis

Jennifer Gierisch PhD Duke Evidence Synthesis Group January 13, 2014

Strategies for Stabilizing Symptoms in People with Osteoarthritis

Osteoarthritis (OA) is characterized by damage to cartilage and bones of joints, causing symptoms of pain and stiffness in the affected joints OA is a very common condition, particularly in people

• ver age 45 and is a major cause of physical disability,

decreased quality of life, and increased health care costs Most commonly affected joints are knees, hips, hands, spine, and feet Various treatment options: pain relievers, anti inflammatory drugs, weight loss, exercise, physical therapy, combination management, joint surgery

Strategies for Stabilizing Symptoms in People with Osteoarthritis – Uncertainties

Optimal strategies for implementing existing recommendations for care and patient interventions Comparative effectiveness of specific nonsurgical therapies or combination of nonsurgical therapies Comparative effectiveness of strategies to increase patient adherence to nonsurgical therapies and self-management strategies (e.g., patient engagement tool or prompts) Identification of optimal strategies for different patient subgroups Key patient-centered outcomes

During the April 2013 meeting, this PCORI Advisory Panel ranked evaluating strategies for stabilizing symptoms for people with osteoarthritis as HIGH PRIORITY for targeted future research funding

Overall Project Goal

To work with stakeholders to help PCORI identify, refine, and prioritize future research evidence gaps in the area of strategies for stabilizing symptoms for people with osteoarthritis

Overview of Project

• 1. Identifying Known Evidence Gaps
• 2. Creation of Stakeholder Group
• 3. Expansion of Evidence Gaps
• 4. Analytic Framework
• 5. Stakeholder Prioritization
• 6. Horizon Scan
• 7. Study Design Considerations

Identifying Known Evidence Gaps

Review of published systematic reviews, clinical practice guidelines, and future research needs documents Initial 31 evidence gaps explored:

• Specific populations or subgroups of patients
• Comparative safety and effectiveness of available

interventions and comparators

• Impact of treatment of specific outcomes of interest
• Optimal timing or setting for treatment

Creation of Stakeholder Group

American Academy of Orthopaedic Surgeons American Physical Therapy Association Association of Rheumatology Health Professionals Arthritis Foundation Centers for Disease Control and Prevention (Arthritis Program) Centers for Medicare and Medicaid Services (Division of Medical and Surgical Services) Clinical Orthopedic Society National Institute on Aging National Institute of Arthritis and Musculoskeletal and Skin Diseases Orthopaedic Research and Education Foundation Osteoarthritis Research Society International Patient Advocates

Stakeholders

Kelli Allen, Ph.D. Associate Research Professor, Medicine Duke University Medical Center Teresa Brady, Ph.D. Senior Behavioral Scientist Arthritis Program Centers for Disease Control and Prevention

• G. Kelley Fitzgerald, Ph.D., P.T.

Professor, Department of Physical Therapy University of Pittsburgh Yvonne M. Golightly, Ph.D., P.T. Research Assistant Professor, Epidemiology University of North Carolina Lyndon Joseph, Ph.D. Health Scientist Administrator Division of Geriatrics and Clinical Gerontology National Institute on Aging Jeffrey Katz, M.D. Professor of Medicine and Orthopedic Surgery Harvard Medical School Gayle Lester, Ph.D. Program Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases Dave Mekemson Patient Representative Paul Rockar, D.P.T. Adjunct Assistant Professor, Physical Therapy University of Pittsburgh Jyme Schafer, M.D. Director, Division of Medical and Surgical Services Centers for Medicare and Medicaid Services Nancy Simington Patient Representative Kim Templeton, M.D. Director, Musculoskeletal Oncology Service University of Kansas Medical Center Patience White, M.D. Professor of Medicine and Pediatrics George Washington University

Stakeholder Discussions

Expanded initial list of 31 gaps to 48 evidence gaps (3 new questions related to population, 14 new questions related to interventions) Stressed importance of future research focusing on:

• engaging patients early in the disease process
• helping patients navigate treatment options that optimize

patient-centered outcomes

• developing strategies that promote successful long-term

engagement in nonsurgical treatment options

Consideration of OA as a chronic disease needing early identification, patient engagement, strategies for adherence/maintenance, shared decision making regarding nonsurgical and surgical treatments

Future Research Needs Broad Topic Areas Covering 48 Questions

• 1. Population differences (sociodemographic,

comorbidities, disease aspects)

• 2. Identification and screening
• 3. Therapeutic interventions
• 4. Promotion of adherence and maintenance of

effects

• 5. Decision making interventions
• 6. Healthcare, policy, and ecological approaches
• 7. Patient-centered outcomes

Defining Patient-Centered Outcomes (FRN #44)

• Sleep
• Negative

affect/depression/worry

• Delay to surgery
• Reduction in medications
• Pain/independence in activities
• f daily life/instrumental

activities of daily life

• Patient satisfaction
• Time to return to work/activities
• Quality of life
• Reliability of pain treatment

Impact of Population Factors (FRN #1-6)

• Sociodemographic differences
• Comorbidity differences
• Previous surgical intervention
• Prevalence/severity of comorbidities
• Comedications
• Disease aspects of OA (e.g., severity,

functional limitations, location, multi- joint) Identification & Screening (FRN #7-18)

• Patient management of OA
• Standardized screening tools
• Sensitivity/specificity of

evaluations

• Strategies for engaging

patients

• Optimal ways to track and

identify OA risk factors

• OA-specific barriers to

physical activity

• Functional ability self-

assessment tool

• Walkability audit/checklist
• Different models of health

care

• Optimal timing for

interventions

• Threshold level of symptoms
• Need for transition from

nonsurgical to surgical interventions Adverse Effects of Therapeutic Interventions Promotion of Adherence & Maintenance of Effects (FRN #30-38)

• Patient engagement tools/prompts
• Followup treatments
• Financial incentives
• Self-management strategies
• Strategies for long-term behavior

change

• Nonsurgical management strategies
• Cultural group differences
• Socioeconomic differences
• Supervised exercise program

Decision Making Interventions (FRN #39-40)

• Decisionmaking tools
• Patients raising issue

Population Adults with OA Therapeutic Interventions (FRN #19-29)

• Nonsurgical therapies
• Physical therapy
• Orthotics
• Self-administered transcutaneous

electrostimulation

• Biomechanical strategies
• Complementary and alternative

approaches

• Individual treatment modalities
• Method of delivery
• Duration/intensity/frequency
• Sequenced weight loss program
• Setting up patients to succeed

Impact of Outcome Measurement Factors (FRN #45-48)

• Validity/reliability of patient-centered
• utcome measures
• Validity/reliability of intermediate
• utcome measures
• Defining intermediate outcomes
• Confounding variables

Health Care, Policy and Ecological Approaches (FRN #41-43)

• Promoting care available in today’s new

health care delivery system

• Policy and environmental strategies to

promote physical activity

• Insurance coverage

Stakeholder Prioritization

Online ranking of evidence gaps Forced-ranking prioritization method

• 16 votes per stakeholder, which could be allocated to

any of the 48 research priorities

• Maximum of 3 votes per item

Asked to rank based on “most important unanswered research question in strategies to stabilize symptoms of osteoarthritis” Questions divided into a top, middle, and lower tier Only top tier moved on to final stage of horizon scan and study design considerations

49

Stakeholder Prioritization – TOP TIER

Question

Score N

What are the most important patient-centered outcomes for patients with foot, ankle, knee, or hip OA?

13 8

What are the optimal duration, intensity, and frequency of examined nonsurgical interventions for OA to create sustained changes in patient-centered outcomes?

12 9

Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by sociodemographic differences? How do these strategies differ in specific underrepresented patient populations?

9 7

What are effective ways for patients or providers to determine the need for the transition from nonsurgical to surgical interventions for OA?

8 7

What are the comparative safety and effectiveness of strategies promoting long-term behavior change in the context of chronic pain and functional limitations associated with OA?

8 6

Are there potential standardized screening tools and indicators of OA that can improve early diagnosis

• f OA?

7 6

What are the comparative safety and effectiveness of biomechanical strategies to improve OA symptoms and slow progression of disease?

7 6

What are the comparative safety and effectiveness of strategies to help patients engage in key self- management behaviors for managing OA in real-world settingss?

7 6

What opportunities for promoting coordinated, proactive, longitudinal, chronic care for OA are now available in today’s new health care delivery system?

7 6

What are the comparative safety and effectiveness of strategies for identifying and engaging patients early in the OA disease process, particularly fostering healthy behaviors to prevent progression and disability from OA?

7 5

What are the comparative safety and effectiveness of different usual care nonsurgical therapies or combination of different usual care nonsurgical therapies to prevent progression and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time?

7 5

How do we best set up patients to succeed with nonsurgical management for OA incorporating available

• ptions and their specific potential barriers for an individual patient?

7 5

Horizon Scan

PubMed

• 4692 articles identified
• 427 included as potentially relevant to top tier

questions

• 92 systematic reviews, 254 RCTs, 41 cohort studies, 3

case-controlled studies, and 37 other studies

ClinicalTrials.gov

• 153 open protocols identified
• 55 included as potentially relevant to top tier questions
• Sample sizes ranged from 8 to 20,000 patients
• Estimated completion ranged from overdue to June

2017

Horizon Scan Summary

Question SRs RCTs Cohort Case Control Other Ongoing

Most important PC outcomes 1 4 5 Duration, intensity, frequency 5 15 3 1 9 Sociodemographic differences 1 2 3 4 Transition to surgical 1 2 Long-term behavior change 5 21 5 5 Screening tools 1 7 2 3 Biomechanical strategies 6 16 1 6 2 Patient engagement strategies 7 18 1 4 2 Healthcare delivery system 1 6 1 4 Early identification/engagement 2 3 2 2 CER nonsurgical therapies 79 228 19 1 12 47 Set up patients to succeed 4 3 4 5 2

CER of Nonsurgical Therapies (N=339)

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Intervention SR s RCTs Cohort Case Control Other Tota l

Oral medications 29 84 3 3 119 Injections 19 54 8 5 86 Physical therapy/exercise 23 55 2 5 85 Weight management 3 7 1 11 Combination strategies 1 1 Biomechanical strategies 3 1 4 Other 15 53 5 1 2 76

• Of the 119 oral medication studies, all but 20 of these studies evaluated oral

medications compared with either another oral medication or placebo.

• Relatively few studies evaluated treatments across categories or combined

categories of treatment

Study Design Suggestions

Question RCT Meta Analysis Observational Model

RCTs Observational New Data Existing Data

Most important PC outcomes No No No Yes ? No Duration, intensity, frequency Yes ? No ? ? ? Sociodemographic differences Yes Yes ? ? ? ? Transition to surgical Yes ? No ? ? Yes Long-term behavior change Yes ? ? ? ? ? Screening tools ? ? ? Yes Yes ? Biomechanical strategies Yes ? ? ? No ? Patient engagement strategies Yes ? ? ? ? ? Healthcare delivery system ? ? ? Yes ? ? Early identification/engagement Yes ? ? ? ? ? CER nonsurgical therapies Yes Yes ? ? ? ? Set up patients to succeed Yes Yes No ? ? ?

Summary: Stakeholder Priorities

Future research priorities should focus on attending to patients’ needs early and across the spectrum of the disease

• Disease identification  treatment decisions  transition

to surgery

Engage patients early in the disease process Help patients navigate treatment options that

• ptimize patient-centered outcomes

Develop strategies that promote successful long- term engagement in nonsurgical treatment options

55

Discussion: Research Gaps in Osteoarthritis

How well does the research question meet the 5 PCORI criteria?

• Patient-Centeredness
• Impact of the Condition on the Health of Individuals and

Populations

• Options for Addressing the Issue
• Likelihood of Implementation in Practice
• Durability of Information

56

Break

3:30 pm – 3:45 pm

57

Voting – Osteoarthritis 3:45 pm – 4:00 pm

58

Bipolar Disorder and Antipsychotic Use in Children, Adolescents and Young Adults: 4:00 pm – 4:15 pm Diane Bild, MD, MPH

Senior Program Officer Clinical Effectiveness Research PCORI

59

Work Group Members, December 20, 2013

Participant Organization

Ronald Means, MD (Chair) Outpatient, Child and Adolescent Psychiatrist Catholic Charities of Maryland Emilie Becker, MD Mental Health Medical Director, Texas Medicaid James Becker, MD Medical Director in the Bureau for Medical Services, West Virginia Boris Birmaher, MD American Psychiatric Association Robert K. Heinssen, PhD National Institute of Mental Health Patrick Hendry Mental Health America Teresa King National Federation of Families for Children’s Mental Health Marcia Leiken, MD Psychiatrist Adelaide Robb, MD American Academy of Child and Adolescent Psychiatry Benedetto Vitiello, MD National Institute of Mental Health

60

Meeting Objectives

Provide feedback on a set of research questions that PCORI may consider pursuing Identify other high-priority research topics (in the same area) that PCORI should consider Assure that multiple perspectives are accounted for in the development of a possible funding announcement

61

Highest priority questions for CER

Diagnostic uncertainty for bipolar disorder in children, adolescents and young adults

• Consider study of treatment based on full DSM criteria vs.
• ther symptom-based criteria

Comparative long-term benefits and adverse effects of different treatment strategies

• Functional, social, and symptomatic outcomes
• Adverse effects, particularly weight gain
• Consider pharmacologic and nonpharmacologic therapies,

including case management

Full report will be posted on PCORI website. Topic planned for Pragmatic Studies announcement

62

Update on the DCIS Targeted Funding Announcement: 4:15 pm – 4:30 pm

Stanley Ip, MD

Senior Program Officer Clinical Effectiveness Research PCORI

63

DCIS follow up

Met with stakeholders interested in breast cancer

• n 10/30/2013.

Held teleconference with NIH/NCI experts on 12/20/2013.

Stakeholders in breast cancer research

The Center for National Breast Cancer Coalition Advocacy Training Susan G Komen Foundation American Cancer Society Avon Foundation for Women

• Dr. Susan Love Research Foundation

Friends of Cancer Research Patient stakeholder

Points raised by stakeholders and Federal partners

Difficult to study natural history (without any initial treatment) in the US. Difficult to randomize (treatment vs. active surveillance). Even if an RCT is feasible, it would require 10-15 years to detect survival differences. 5 year trial can look at differences in disease progression. Observational study is subject to self-selection bias.

Recommendations by stakeholders or Federal partners

Put effort into basic biology and imaging studies to better characterize which patients with DCIS progress to invasive breast cancer. Observational studies may be useful in summarizing characteristics of patients whose diseases do not progress despite not receiving standard treatments. Compare different approaches to inform patients of the DCIS diagnosis in terms of decisional conflicts, treatment options,…etc.

Voting Results 4:30 pm – 4:45 pm

68

Migraine Headache Prioritization (N=20)

Question Score # Stakeholders

• 17. What strategies for treatment of individual headache episodes are most

effective and least likely to promote medication overuse headache in patients with high frequency episodic or chronic migraine? 16 9

• 10. How does the comorbidity of depression and related psychological

disorders, metabolic disorders, and gastrointestinal disorders affect the efficacy of preventive pharmacologic or nonpharmacologic treatments for episodic

• r chronic migraine?

13 9

• 15. What are the comparative safety, tolerability, and effectiveness of

nonpharmacologic versus pharmacologic versus combined (nonpharmacologic + pharmacologic) treatments in the prevention of chronic migraine? 12 9

• 27. What are the most important patient-centered outcomes for patients

receiving pharmacologic or nonpharmacologic treatments for the prevention of episodic or chronic migraine? How do these differ depending on patient or provider perspective? 11 8

Migraine Headache Prioritization

Question Score # Stakeholders

• 7. What patient, treatment, and other factors (e.g., age of onset, diet, psychosocial

stress, early life trauma) are associated with the development of chronic migraine, or the transformation from episodic to chronic migraine? 9 8

• 13. Are combinations of pharmacologic treatments effective in preventing

episodic migraine in patients for whom treatment with a single preventive drug has been either ineffective or intolerable? Are there fewer or more adverse events when using low dose polypharmacy versus single agents at higher doses? Do treatment efforts that align with patient-defined values improve the efficacy of preventive treatments for episodic and chronic migraine? 9 7

• 19. How should research on preventive pharmacologic or nonpharmacologic

treatments for chronic migraine define treatment success? How should patients participate in defining treatment success? 8 8

• 1. What biomarkers help predict response to preventive treatment in patients with

episodic or chronic migraine? 5 4

• 9. What are the patient factors that determine whether patients with episodic

migraine are prescribed, take, and adhere to preventive approaches (as opposed to acute treatment for episodic migraines without preventive treatment)? 5 4

Migraine Headache Prioritization

Question Score # Stakeholders

• 16. What are the effects of decision-making tools

c compared to usual care to

assist patients and providers with shared decision-making regarding migraine, with a focus on individuals with low health literacy and limited health access compared to usual care? What are the optimal format, content, and timing for these? What is the value in terms of treatment outcome of providing patients multiple tools for treatment? 5 5

• 5. Which disease and patient characteristics (duration, severity, frequency of

attacks, comorbidities) affect the efficacy of preventive pharmacologic treatments for chronic migraine? 3 3

• 4. Which disease and patient characteristics (duration, severity, frequency of

attacks, comorbidities) affect the efficacy of preventive nonpharmacologic treatments for chronic migraine? 2 2

• 12. What is a valid disease model to accurately describe disease characteristics

and treatment mechanisms for patients with episodic or chronic migraine? 2 2

Osteoarthritis Prioritization (N=19)

Question Score # Stakeholders

• 18. What are effective ways (e.g., checklist of functionality/symptoms) for

patients or providers to determine the need for the transition from nonsurgical to surgical interventions for OA? 17 10

• 10. What are the comparative safety and effectiveness of strategies for

identifying and engaging patients early in the OA disease process, particularly fostering healthy behaviors (physical activity, weight management), to prevent progression and disability from OA? 16 11

• 19. What are the comparative safety and effectiveness of different usual

care nonsurgical therapies (pharmacotherapy, injections, physical therapy/exercise, weight loss) or combination of different usual care nonsurgical therapies to prevent progression and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? 9 7

• 29. How do we best set up patients to succeed with nonsurgical

management for OA incorporating available options and their specific potential barriers for an individual patient? 9 7

Osteoarthritis Prioritization

Question Score # Stakeholders

• 1. Do the comparative safety and effectiveness of nonsurgical management

strategies to prevent progression and disability from OA differ by sociodemographic differences (e.g., age, sex, race/ethnicity, socioeconomic status, insurance status, access to health care, those with physically demanding

• ccupations)? How do these strategies differ in specific underrepresented

patient populations (i.e., those with low literacy level, low SES, or less health care access)? 8 5

• 27. What are the optimal duration, intensity, and frequency of examined

nonsurgical interventions for OA to create sustained changes in patient-centered

• utcomes?

8 7

• 44. What are the most important patient-centered outcomes (e.g., sleep,

negative affect/depression/worry, delay to surgery, reduction in medications, pain/independence in activities of daily life/instrumental activities of daily life, patient satisfaction, time to return to work/activities or other employment

• utcomes, quality of life, reliability of pain treatment) for patients with foot, ankle,

knee, or hip OA? 7 6

• 41. What opportunities for promoting coordinated, proactive, longitudinal,

chronic care for OA are now available in today’s new health care delivery system? 6 4

Osteoarthritis Prioritization

Question Score # Stakeholders

• 33. What are the comparative safety and effectiveness of strategies to help

patients engage in key self-management behaviors for managing OA (physical activity, weight management) in real-world settings (community, primary care)? 5 5

• 8. Are there potential standardized screening tools and indicators of OA

that can improve early diagnosis of OA? 4 2

• 23. What are the comparative safety and effectiveness of biomechanical

strategies to improve OA symptoms and slow progression of disease? 4 4

• 34. What are the comparative safety and effectiveness of strategies promoting

long-term behavior change (e.g., weight management, physical activity) in the context of chronic pain and functional limitations associated with OA? 2 2

Announcements & Adjourn

4:45 pm – 5:00 pm

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Welcome

Please be seated by 8:40.

The teleconference will go live at 8:45.

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Agenda Overview

Time Agenda Item 8:45-10:00 a.m. Path to Funding Announcement Discussion 10:00-10:15 a.m. Procedures for Reviewing Topics and Voting 10:15-10:30 a.m. Review Topics 10:30-10:45 a.m. Break 10:45-12:15 p.m. Review Topics 12:15-1:00 p.m. Lunch 1:00-2:00 p.m. Review Topics 2:00-2:15 p.m. Voting 2:15-2:30 p.m. Voting Results/Next Steps 2:30 p.m. Adjourn

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Two Pathways to PCORI Funding

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Diverse portfolio addressing high-priority questions

Investigator-Initiated or “Broad” Pathway

Patient/Stakeholder-Initiated Pathway – “TARGETED”

PCORI and stakeholders generate and prioritize questions based on review criteria PCORI issues specific funding announcements for highest-priority topics Researchers and stakeholders develop responsive proposals Peer review prioritizes applications by level

• f alignment

with criteria

Focused portfolio addressing high-priority questions

Two Pathways to PCORI Funding

Procedures for Reviewing Topics and Voting

Review 14 new topics against PCORI’s criteria

• 12 minutes per topic
• Prior to discussion, one panelist will give a brief
• verview of topic (denoted with *)

Ranking will be completed after discussion of all topics

• SurveyGizmo drag and drop ranking method will

be used

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Topic 1: Treatment options for hypercholesterolemia

Bettye Green Margo Halm* Priti Jhingran Mark Johnson

81

Topic 2: Treatment options for psoriasis

Regina Dehen Priti Jhingran Mark Johnson* Daniel Wall

82

Topic 3: Management of arrhythmogenic right ventricular dysplasia (ARVD)

Margo Halm Bruce Monte* Alan Rosenberg Daniel Wall

83

Topic 4: Treatment options for pemphigus vulgaris

Margo Halm Sara Hohly Cynthia Mulrow* Daniel Wall

84

Topic 5: Treatment options involving mesh for the management of inguinal and abdominal hernia

Bettye Green Ronald Means Bruce Monte Seema Sonnad*

85

Topic 6: Assessment of benefits and harms

• f pelvic floor mesh implants

Karen Chesbrough Priti Jhingran* Seema Sonnad Angela Smith

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Topic 7: Robotic surgery for urologic and gynecologic cancers

Debra Madden Bruce Monte Angela Smith* Seema Sonnad

87

Break

10:30 am – 10:45 am

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Topic 8: Treatments for hearing loss

Sara Hohly Kathie Insel Mark Johnson Debra Madden* Marcia Rupnow

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Topic 9: Identifying lung cancer in people with lung nodules

Denise Kruzikas Debra Madden James Pantelas* Alan Rosenberg

90

Topic 10: Treatment options for opioid substance abuse

Karen Chesbrough Regina Dehen* Denise Kruzikas Ronald Means

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Topic 11: Biomarker testing for patients with malignancy

Karen Chesbrough Kathie Insel* Cynthia Mulrow James Pantelas

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Topic 12: Treatment options for multiple sclerosis

Bettye Green Kathie Insel Cynthia Mulrow* Marcia Rupnow

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Topic 13: Proton beam therapy for breast, lung, and prostate cancer

Denise Kruzikas James Pantelas Alan Rosenberg* Angela Smith

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Topic 14: Treatment options for autism

Regina Dehen Sara Hohly Ronald Means* Marcia Rupnow

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Voting

2:00 pm – 2:15 pm

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Topic Ranking Results (N=19)

Next Steps: 2:15 pm – 2:30 pm

Next in-person meeting tentatively scheduled for the Spring in Washington, DC (location TBD)

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Thank you for your participation.

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