Dashboard ReviewEnd of FY 2015

Joe Selby, MD, MPHExecutive Director

Michele Orza, ScDSenior Advisor to the Executive Director

Discussion Questions• What is your view of our performance in FY 2015 and our

status as of the end of FY 2015?

• What are your thoughts about our funding commitments over the next four years?

• Is our approach to targeted topics optimal?

Our Goals: Increase Information, Speed Implementation, and Influence Research

0 100 200 300 400 500 600

Actual

Budget

$ Millions

Q1 Q2 Q3 Q4

0

10

20

30

Q1 Q2 Q3 Q4

Num

ber o

f Pro

ject

s

Targeted

Pragmatic

Broad

Engagement

PCORNet

NANeeds Attention

On TargetOff Target

Legend

Funds Committed to Research – up to $640M Percent of Projects on Track

Board of Governors FY2015 Dashboard – Q4 (As of 9/30/2015)

Journal Articles Published

Projects AwardedNA=Not Applicable

0

5

10

15

20

25

30

35

By Awardees About or By PCORI

Num

ber o

f Art

icle

s

Q1

Q2

Q3

Q4

0

1,000

2,000

3,000

4,000

5,000

Web Views0

5

10

15

Citations

Q1

Q2

Q3

Q4

0

5

10

15

20

25

30

Q1 Q2 Q3 Q4

Num

ber o

f Pro

ject

s

Expected Actual

Uptake of Methodology StandardsProjects Completed as Expected

Expenditures – Total Budget, up to $362M

Phase II PFA

Released

Version 2.0 of CDM

Complete

Governance policies

approved

Aspirin Trial

Awarded

Obesity Cohort Project

Awarded

Health Systems

Convening Event

Phase II awardedQ1

2015

Q22015

Q32015

Q42015

Progress of PCORnet – Completion of Phase I

Q1=Q1 2015Q2=Q2 2015

Q3=Q3 2015Q4=Q4 2015

0102030405060708090

100

Research Engagement PPRNs CDRNs

Perc

ent

Q1 Q2 Q3 Q4

NANA

ResearchAwardsBudget

ResearchAwardsActual

All Other Budget

All OtherActual

0 50 100 150 200 250$ Millions

Q1

Q2

Q3

Q4

*Influencing Research*A new graduate level Health Psychology course at Tulane University

is modeled on PCORI’s National Priorities for Research

Goal 3 Results: PCORI Influence on Curriculum

*Influencing Research*A new graduate level Health Psychology course at Tulane University

is modeled on PCORI’s National Priorities for Research

Michael Hoerger, PhDAssistant Professor of Psychology and PsychiatryTulane University

Designed a new Health Psychology course for the Masters Program in Behavioral Health based on PCORI’s National Priorities for Research• Course objective: prepare students for doctoral training in psychology so that they

can ultimately pursue careers in clinical practice, research, and policy• 10 modules, with 6 focused on PCORI• Hoerger, M. (2015). Educating the Psychology Workforce in the Age of the Affordable Care Act: A Graduate

Course Modeled After the Priorities of the Patient-Centered Outcomes Research Institute (PCORI). Training and Education in Professional Psychology. E-pub ahead of print.

“PCORI’s five national priorities are noteworthy for psychologists and psychology trainees in terms of suggesting potential funding areas and clarifying trends in US healthcare for psychologists.”

$196

$58

FY 2014Total=$254M

$244

$50

FY 2013Total=$294M

$115

$179

$78

FY 2015Total=$372M

Broad

Pragmatic

Targeted

We are making progress on our Strategic Priority to “Increase the proportion of research funding going to focused and targeted topics”

Funds Committed in Each Fiscal Year by Type of PFA

And the result is a substantial shift in the distribution of our portfolio

$440

$108

End of FY 2014Total=$548M

$244

$50

End of FY 2013Total=$294M

$555$179

$186

End of FY 2015Total=$920M

Broad

Pragmatic

Targeted

Funds Committed by Type of PFA, Cumulative Total by Fiscal Year

LIST 5

LIST 1

LIST 2

LIST 3

LIST 4

LIST 6

Targeted PFA

Pragmatic PFA

LIST 7

Topic Prioritization PathwayTo make list 1, Staff determine topic eligibility

To make list 2, SOC reviews and endorses topics for topic briefs

To make list 4, Advisory Panel reviews & prioritizes topics

To make list 5, SOC endorses topics for further refinement

To make list 3, SOC reviews topic briefs and approves them for Advisory Panel review

To make lists 6&7, SOC assigns questions to Targeted or Pragmatic PFA

SOC reviews and approves questions for

Pragmatic PFA

Board reviews and approves forTargeted PFA

Topic Prioritization Pathway: FY15 Q4 Snapshot

Under Consideration

Approved Topics

Funded Topics

For Targeted PFAs (List 6)• Chronic Pain/Long Term Opioid Therapy• Multiple Sclerosis• NOACs for blood clots• Treatment-Resistant Depression

Funded via Targeted PFAs• CAD-Aspirin Dose• Care Transitions• Falls in Elderly• Hepatitis C- New Therapies• Hypertension• Obesity in Diverse Populations• Severe Asthma in African

Americans & Hispanics• Uterine Fibroids

SOC Endorsed for Refinement (List 5)• Asthma• Chronic Low Back Pain• Diabetes• Mental Health Integration• Palliative Care• Sickle Cell DiseaseReviewed by Advisory Panels (List 4)• Antimicrobial Resistance• Autism Spectrum Disorders- risk assessment• NOACs for stroke prevention• Care coordination• Cognitive Impairment• Communication• Coronary Artery Disease- Statins• Dementia• Genetic Testing for Rare Disease• Glaucoma• Health IT & Evidence-Based Treatment• High Cholesterol• Implantable Cardiac Defibrillators• Links btw Providers & Community• Neck Pain• Orthopedic Surgery• Pneumonia• Post-Acute Care Transitions• Role of Spacers in Asthma• Substance Abuse- Adolescents/Alcohol• Weight Maintenance & Reduction

For Pragmatic Studies PFAs (List 7)• Autism Spectrum Disorders-

behavioral analysis• Cardiovascular Disease• Caregiver Engagement & Support• Carotid Artery Disease• Dental Caries• Diabetes• End Stage Renal Disease• Insurance Design• Medication Management• Migraine• Musculoskeletal Pain• Opioid Abuse Treatment• Osteoarthritis • Pelvic Floor Dysfunction• Pre-term & Low Birth Weight• Suicide Prevention• Tobacco Use Prevention & Cessation• Traumatic Brain Injury

Funded via Pragmatic PFAs• Bipolar Disorder• Breast Ductal Carcinoma in Situ• Crohn’s Disease- Biologics• Chronic Back Pain• Hip Fractures• Mental Health & Primary Care• Particle Beam Therapy• Pulmonary Nodules & CT

Surveillance

Funded Projects by Primary Disease/Condition

(N=283) - Cycle I through Spring 2015

*Categories are mutually exclusive*Does not include Methods projects

12

38

1011

1212

1617

1920

2122

2640

43

0 10 20 30 40 50

Skin DiseasesAllergies and Immune Disorders

Digestive System DiseasesKidney Disease

Infectious DiseasesMuscular and Skeletal Disorders

Trauma/InjuryReproductive and Perinatal Health

Multiple/co-morbid chronic conditionsRespiratory Diseases

Neurological DisordersNutritional and Metabolic Disorders

Rare DiseasesCross-cutting

Cardiovascular HealthCancer

Mental/Behavioral Health

We have projects focusing on a wide range of conditions, with the largest concentrations in mental/behavior health and cancer

The majority of studies in our portfolio are clinical trials

67%7%

26%

Study Design byNumber of Awards

(N=283)

Randomized Controlled Trials

Secondary Data Analysis

Observational (Prospective)

$634 M76%

$37 M4%

$168 M20%

Study Design byAward Amount ($)

Total: $838M

*Does not include Methods projects

Funded Portfolio by Study DesignCycle I through Spring 2015

The majority of our studies focus on treatment and 18% of our funding is in screening and prevention studies

82%

10%

4% 2% 2%

Care Continuum by Proportion of Projects

N=283

Treatment Prevention Screening Diagnosis Other

77%

14%

4% 3%3%

Care Continuum by Proportion of Funding

Total: $838M

*Does not include Methods Projects

Funded Portfolio by Care ContinuumCycle 1 through Spring 2015

AD18%

Methods8%

CDR7%

IHS26%

APDTO41%

Total= 355 Projects, $920M

The majority of our research funding has gone to our APTDO and IHS portfolios

We actively monitor our projects, support them to be successful,and classify their progress as shown below

The “Percent of Projects on Track” shown on the Dashboard is the percent of projects in the green zone

-AND- -AND- -AND-

-OR- -OR- -OR-

-OR- -OR- -OR-

The majority of our projects are on track and we are giving additional attention to those that are not

130

180

183

47

54

67

16

9

11

11

14

13

1

1

0 25 50 75 100 125 150 175 200 225 250 275 300

Q2 2015

Q3 2015

Q4 2015

Number of Projects

Distribution of Project Status by Quarter

Green Zone

Yellow Zone

Orange Zone

Red Zone

Award Terminated*

*Notice of Termination Issued

Some projects require contract modifications in order to be successfully completed

67 8

0

12

45

0

8

32 2

5

2 20

0

5

10

15

20

25

30

35

40

Milestones Scope of Work Period of Performance Budget (Increase)

Q1 (N=195) Q2 (N=222) Q3 (N=259) Q4 (N=278)

• Includes all research projects with at least one progress report due• Does not include infrastructure or pilot projects

%

Reasons for Contract Modifications Percent of total projects by Q of FY15Number (N) of total projects differs by Q

Almost half of our first completed cohort of studies (the Pilot Projects, average length 26 months) required an extension (5 months on average) and 88% were then completed within the contract period

54%46%

PILOTS Contract Extensions

(N=50)

No ExtensionRequired Contract Extension

88%

12%

PILOTS Completed Project within

Contract Period (N=50)

Completed in Contract Period(includes modified contracts)

Not Completed within ContractPeriod (Late)

Our First Completed Cohort of Projects

3

47

1

40

127

46

21

2

113

36

19

8

0

20

40

60

80

100

120

140

2014 2015 2016 2017 2018 2019 2020 2021Fiscal Year

Expected Number of Contracts Ending by Fiscal Year

Pilots

CER

Methods

We currently expect the contracts for our first cohort of CER studies to end late in FY 2016

Includes completed contract modifications; Does not include pending modifications to incorporate peer review of draft final research report

The number of journal articlesconcerning PCORI increased in FY2015

0 3 006

10

1

18 17

511 9

27 3033

110

46

84

0

20

40

60

80

100

120

Articles Resulting from PCORI-funded Projects

Articles by or about PCORI Articles that Cite or MentionPCORI Work

Number of Articles

By Fiscal Year

2010 2011 2012 2013 2014 2015

Cumulative total=143 Cumulative total=114 Cumulative total=153

Three Published Protocols for CER Studies

Field CA, et al. Cultural adaptation of a brief motivational intervention for heavy drinking among Hispanics in a medical setting. BMC Public Health. 2015 Jul 30;15:724.

Culturally-adapted therapy vs non-adaptedtherapy for heavy drinking among Latino men

Taveras EM, et al. Connect for health: Design of a clinical-community childhood obesity intervention testing best practices of positive outliers. Contemp Clin Trials. 2015 Sep 28.

Contextually-tailored health coaching vs non-tailoredcoaching for weight loss in obese children ages 2-12 years

Williams QI, et al. Physical therapy vs. internet-based exercise training (PATH-IN) for patients with knee osteoarthritis: study protocol of a randomized controlled trial. BMC Musculoskelet Disord. 2015 Sep 28;16(1):264.

Standard physical therapy vs internet-based training for largelyuninsured patients with knee osteoarthritis

10

21

31

26

0

5

10

15

20

25

30

35

2012 2013 2014 2015

Num

ber

Citations of Methodology StandardsBy Calendar Year

Measures of Uptake of Our Methodology Standards:We are tracking citations and other early indicatorsExample – Citations, Page-views, and Downloads of: Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI). Methodological standards and patient-centeredness in comparative effectiveness research: the PCORI perspective. JAMA. 2012 Apr 18;307(15):1636-40.

JAMA Statistics

Measures of Uptake of Our Methodology Standards:In FY2016, we will be tracking PCORI-Funded CME/CEOur CME/CE activity is designed to introduce PCORI’s Methodology Standards, review the development and types of standards, and provide context and guidance for adherence relevant to the use and application of these standards in research and in responding to PCORI funding announcements

PCORI Methodology Standards Series with 6 Modules Released Sept 30, 2015

Module 1Basic Context, Role, and Development of Standards in CER

Module 2Role of Standards in PCORI Funding Announcements & the Application Process

Module 3Formulating Research Questions and Patient Centeredness

Module 4Data Integrity, Preventing and Handling Missing Data, and Heterogeneity of Treatment Effects

Module 5Data Registries, Data Networks, and Causal Inference

Module 6Standards for Specific Study Designs- Adaptive and Bayesian Trial Designs and Diagnostic Tests

Joel M. Gelfand, MD, MSCECenter for Clinical Epidemiology & BiostatisticsPerelman School of MedicineUniversity of Pennsylvania

MSCE with Concentration in Patient-Centered Outcomes Research (PCOR)• Students explore methods spanning qualitative research, clinical epidemiology, molecular epidemiology, and clinical

trials• Unifying theme is generation of data that helps patients, their care givers, providers, and health care delivery

systems make informed health care decisions that improve outcomes that matter most to patients• Students expected to pursue a thesis relevant to patient-centered outcomes research

Measures of Uptake of Our Methodology Standards:Incorporation into academic curriculum – Example

Class Topic

1 Overview of PCOR: PCORI Methodology Report

2 Formulating Research Questions, Identifying Evidence Gaps

3 Patient Centeredness: Stakeholder Engagement

4 Patient Centeredness: Methods to Increase Patient Enrollment

5 Patient Centeredness: Selection of Outcomes, PROs

6 Sensitivity Analyses

7 Missing Data

8 Data Registries & Automated Databases for CER

9 Pragmatic Trials & Bayesian Designs

EPID 624: Methods inPatient-Centered Outcomesand Effectiveness Research

Discussion Questions

• What is your view of our performance in FY 2015 and our status as of the end of FY 2015?

• What are your thoughts about our funding commitments over the next four years?

• Is our approach to targeted topics optimal?

Slide Number 1Slide Number 2Slide Number 3Slide Number 4Slide Number 5And the result is a substantial shift in the distribution of our portfolioSlide Number 7Slide Number 8Funded Projects �by Primary Disease/Condition�(N=283) - Cycle I through Spring 2015The majority of studies in our portfolio are clinical trialsThe majority of our studies focus on treatment and 18% of our funding is in screening and prevention studiesSlide Number 12Slide Number 13Slide Number 14Slide Number 15Slide Number 16Slide Number 17The number of journal articles concerning PCORI increased in FY2015Three Published Protocols for CER StudiesSlide Number 20Slide Number 21Slide Number 22Slide Number 23