CER 2016 Trontell pcori cer presentation 2016 02 02 final

1. The Patient-Centered Outcomes Research
Institute: “New” Initiatives
Anne Trontell, MD, MPH
Senior Program Officer
Clinical Effectiveness Research Program
PCORI Office of Science
February 2, 2016

2. • Old new: PCORI’s distinctive approach to research
• Relatively new: Emphasis on pragmatic and targeted
clinical studies
• Truly new
– PCORI Study Advisory Committees
– Medical specialty societies and other stakeholder
outreach
– Dissemination of findings from PCORI-funded
studies
“New” is Relative

3. • Focus on patient-centered outcomes
• Stakeholder and patient engagement and participation
throughout research planning, conduct, and dissemination
• Focus on important and relevant real-world questions, patients,
and clinical care settings
The focus on real-world, stakeholder-shaped questions has led to
PCORI’s emphasis on large pragmatic and targeted clinical studies
PCORI’s Distinctive Research Approach – Old New

4. • Enroll broadly representative and heterogeneous patient populations
• Conducted in typical clinical and health care settings
• Large in size
– To discern hypothesized differences in effectiveness
– To evaluate differences in patient subgroups
• As simple as possible in design and conduct
– Maximal inclusion, minimal exclusion criteria
– Minimally intrusive on routine clinical practice in terms of procedures,
data collection
• Examples: ALLHAT1 , MI-FREE2
Pragmatic Study Characteristics
1ALLHAT Collaborative Research Group. Major outcomes in high-risk hypertensive patients randomized to angiotensin-
converting enzyme inhibitor or calcium channel blocker vs. diuretic. JAMA 2002; 288:2981–97;
http://www.ncbi.nlm.nih.gov/pubmed/12479763
2Choudhry, N.K., et al. Full coverage for preventive medications after myocardial infarction. N Engl J Med. 2011 Dec
1;365(22):2088–97;http://www.nejm.org/doi/full/10.1056/NEJMsa1107913

5. Pragmatic Clinical Studies – PCORI’s Approach
Overview
• Anticipated Awards per
Funding Cycle: Six to Nine
• Funds Available per Cycle:
Up to $90 Million
• Maximum Project
Duration: 5 Years
• Maximum Direct Costs per
Project: $10 Million
Seek to produce information that can be
directly adopted by providers:
• Compare two or more options for
prevention, diagnosis, treatment, or
management of a disease or symptom
• Address decisional dilemmas faced by
patients, caregivers, clinicians, and health
care systems
• Address a priority topic identified by
PCORI’s Advisory bodies, Institute of
Medicine, Agency for Healthcare Research
and Quality, and other stakeholders
Closely related are Targeted funding opportunities

6. • Similar goals & approaches to pragmatic clinical studies
• Targeted topics broach two or more related research questions
in a single funding opportunity (PFA)
• Funding caps for individual questions can vary
• Examples: 4 Targeted PFAs with applications due 2/16/2016
– Clinical strategies for managing/reducing long-term opioid use for
chronic pain ($40 million)
– Treatment of multiple sclerosis ($50 million)
– Management strategies for treatment-resistant depression ($30 million)
– New oral anticoagulants in extended treatment of venous
thromboembolic disease ($30 million)
Targeted Studies

7. • Emphasizes patient- and other stakeholder- initiated
research
• Allows focus and dedicated resources on high-priority
topics
• Approximately 2,000 topics submitted directly to
PCORI to date, from patients and other stakeholders
– Pathway and topic status available on our website
– Physician organization nominations have led to
~20% of PCS and targeted awards
PCS Priority and Targeted Topics Development

8. List 6
Approved for a
Targeted Funding
Announcement
List 2
Approved for Topic
Brief Development
List 3
Approved for Advisory
Panel Review
List 4
Reviewed by
Advisory Panels
List 5
Approved for
Refinement
List 1
Nominated Topics
List 7
Listed as a Priority in a Pragmatic
Clinical Studies Funding
Announcement
Lists 6
and 7
Topic Prioritization Pathway

9. Topic Prioritization Pathway: January 2016
Under
Consideration
Approved
Topics
Funded Topics
Approved for Targeted PFA (List 6)
• Chronic pain/Long term opioid therapy
• Multiple sclerosis
• NOACs for blood clots
• Treatment-resistant depression
Funded via Targeted PFA
• 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
Endorsed for Refinement (List 5)
• Asthma
• Chronic low back pain
• Community-acquired pneumonia
• Diabetes
• Mental health integration
• Palliative care
• Decreasing opioid initiation for chronic pain
• Sickle cell disease
Reviewed by Advisory Panels (List 4)
• Antimicrobial resistance
• Adolescents and alcohol abuse
• Autism Spectrum Disorders- risk assessment
• Care coordination
• Cognitive Impairment
• Communication
• Coronary artery disease- statins
• Dementia
• Genetic testing for rare disease
• Glaucoma
• Health IT & evidence-based treatment
• High cholesterol/PCSK9 inhibitors
• Implantable cardiac defibrillators
• Links btw providers & community
• Neck pain
• NOACs for stroke prevention
• Orthopedic surgery
• Post acute care transitions
• Role of spacers in asthma
• Weight maintenance & reduction
Priority Topics for Pragmatic
Clinical Studies PFA (List 7)
• Autism Spectrum Disorders-
behavioral analysis
• Care transitions
• Chronic pain management
• Dental caries
• Diabetes
• End-stage renal disease
• Medication management
• Migraine
• Osteoarthritis
• Pelvic floor dysfunction
• Pre-term birth & low birth weight
• Substance abuse – Tobacco cessation
• Suicide prevention
• Traumatic brain injury
Funded via Pragmatic Clinical
Studies PFA
• Bipolar disorder
• Breast ductal carcinoma in situ
• Crohn’s disease- Biologics
• Chronic back pain
• Hip fracture
• Mental health & primary care
• Particle beam therapy
• Pulmonary nodules & CT
surveillance
PFA= PCORI Funding Announcement

10. • Study Advisory Committees for pragmatic and targeted studies
– Established by awardees in collaboration with PCORI
– To include national patient organizations, professional
organizations, and/or payer or purchaser organizations as
partners and participants in research
• PCORI engagement with general and specialty medical societies
– important role of medical societies in access and application
of research findings by their member physicians
– opportunity to identify new ways to make research evidence
more accessible to practitioners
Truly New – Engagement & Dissemination

11. With PCORI-funded studies now nearing completion and results
• PCORI will start to exercise its obligation to
• conduct peer review of study reports for scientific
integrity and conformance to methodology guidelines
• assure timely release of findings to end-users
• PCORI is initiating funding support mechanism to funded PIs for
dissemination of study findings
Truly New – Engagement & Dissemination

12. Limited Competition Dissemination Funding for Current
Awardees - Truly New
• Offers additional funding for current awardees to disseminate their
research findings
• Seeks approaches that are informed and guided by established
dissemination and implementation models and frameworks
• Strategies expected to vary based on:
– the results and/or products being disseminated
– the populations being targeted
– the goals of the dissemination and implementation effort

13. What does ‘new’ mean in a
fast-changing world? Recent,
innovative, or unfamiliar?

14. Additional Information
• Questions about applying for PCORI funding
https://help.pcori.org/hc/en-us
• Questions about whether your research is a fit for PCORI
funding: sciencequestions@pcori.org or
https://pcori.zendesk.com/agent/dashboard
• Characteristics of pragmatic and explanatory trials
Thorpe, K.E., et al. A pragmatic-explanatory continuum indicator summary
(PRECIS): A tool to help trial designers. CMAJ. 2009:180:E47–E57;
http://cmaj.ca/content/180/10/E47.full.pdf
Loudon, K. et al. The PRECIS-2 tool: designing trials that are fit for purpose. BMJ.
2015: 350:h2147 doi: 10.1136/bmj.h2147

15. Additional Information
• Information on all PCORI Funding Opportunities, including pragmatic, targeted,
and dissemination research
http://www.pcori.org/funding-opportunities
• Physician Roundtable on how PCORI can work with physician specialty
organizations to disseminate research results
http://www.pcori.org/events/2016/physician-roundtable
• PCORI in Practice: Highlighting Opportunities for Medical Specialty Societies
http://www.pcori.org/events/2014/pcori-practice-highlighting-opportunities-
medical-specialty-societies
• For information about PCORI’s pragmatic clinical studies
atrontell@pcori.org

16. Contact Us
www.pcori.org
info@pcori.org

17. Additional Information

18. Pragmatic Clinical Studies – Priority CER Topics as of
Cycle 3 2015 Funding Cycle
• Surgical options for hip fracture in the elderly
• Pelvic floor mesh implants – use or not, surgical techniques
• Treatment of autism spectrum disorder with long-term follow-
up
• ESRD dialysis options overall and in subgroups
• Reduction of tobacco use in high risk, vulnerable populations
with disparities**
• Integration of mental and behavioral health into primary care
• Management of DCIS**
• Treatment of adults with migraine headache**
• Treatment of OA, including surgical and nonsurgical therapies

19. Pragmatic Clinical Studies – Priority CER Topics as of
Cycle 3 2015 Funding Cycle
• Perinatal care models to improve mothers’ & babies outcomes
• Systems interventions (eg. team-based care) to reduce lower
extremity amputations in high-risk populations with diabetes
• Support services for families & infants after NICU discharge
• Rehabilitation programs for TBI
• Primary care management of chronic, noncancer pain
• Dental care delivery models to prevent caries in medically
underserved children
• Strategies to integrate pharmacists or pharmacy services into
patient care to improve patient centered outcomes
• Screening and prevention to minimize adolescent suicidality

20. 20
Cycle Title
Fall 2014 Integrating Patient-Centered Exercise Coaching into Primary Care to Reduce Fragility Fracture
Fall 2014
Mobility: Improving Patient-Centered Outcomes Among Overweight and Obese Youth with Bipolar Spectrum
Disorders Treated with Second-Generation Antipsychotics
Fall 2014 Pragmatic Randomized Trial of Proton vs. Photon Therapy for Patients with Stage II or III Breast Cancer
Fall 2014
A Practical Intervention to Improve Patient-Centered Outcomes after Hip Fractures Among Older Adults (Regain
Trial)
Fall 2014 Anti-TNF Monotherapy versus Combination Therapy with Low Dose Methotrexate in Pediatric Crohn’s Disease
Spring 2014 PCS Early Supported Discharge for Improving Functional Outcomes After Stroke
Spring 2014 PCS Enabling a Paradigm Shift: A Preference-Tolerant RCT of Personalized vs. Annual Screening for Breast Cancer
Spring 2014 PCS
Pragmatic Trial of More versus Less Intensive Strategies for Active Surveillance of Patients with Small Pulmonary
Nodules
Spring 2014 PCS Targeted interventions to Prevent Chronic Low Back Pain in High Risk Patients: A Multi-Site Pragmatic RCT
Spring 2014 PCS A Pragmatic Trial to Improve Colony Stimulating Factor Use in Cancer
PCORI’s 19 Funded Pragmatic Clinical
Studies

21. 21
Cycle Title
Spring 2015
Active surveillance versus traditional treatment options, such as surgery and radiation, for patients
diagnosed with DCIS.
Spring 2015
The effectiveness of two medications – roflumilast and azithromycin – commonly used to treat patients
with chronic obstructive pulmonary disease (COPD).
Spring 2015
The effectiveness of different approaches involving community health workers, tailored education, and
other means to help adults with serious mental illness quit smoking.
Spring 2015
The effectiveness of daily use of an inhaled corticosteroid versus symptom-based use for reducing
asthma exacerbations in African American and Hispanic adults with asthma.
Spring 2015
The effectiveness of two approaches to help people manage their chronic migraines and reduce the risk
medication overuse.
PCORI’s 19 Funded Pragmatic Clinical
Studies (continued)
Winter 2015 Integrating Behavioral Health and Primary Care
Winter 2015
Comparative Effectiveness of Pulmonary Embolism Prevention after Hip and Knee Replacement (PEPPER):
Balancing Safety and Effectiveness
Winter 2015 Comparing Outcomes of Drugs and Appendectomy (CODA)
Winter 2015 Integrated Versus Referral Care for Complex Psychiatric Disorders in Rural FQHCs

22. 22
Award Date Topic PFA Title
Number of
Projects
12/17/2013 Asthma
Treatment Options for African Americans and Hispanics/Latinos with
Uncontrolled Asthma
8
6/4/2014 Fall Prevention Clinical Trial of a Multifactorial Fall Injury Prevention Strategy in Older Persons
1
MOU with NIH
9/30/2014 Fibroids
PCOR for Treatment Options in Uterine Fibroids: Developing a Prospective
Multi-Center Practice-based Clinical Registry (P50)
1
MOU with AHRQ
9/30/2014 Care Transitions The Effectiveness of Transitional Care 1
9/30/2014 Obesity Treatment Obesity Treatment Options Set in Primary Care for Underserved Populations 2
5/4/2015 Aspirin (PCORnet)
Optimal Maintenance Aspirin Dose for Patients with Coronary Artery Disease
(PCORnet Demo)
1
8/18/2015 Obesity (PCORnet) Obesity Observational Research Initiative (PCORnet Demo) 2
9/28/2015 Hypertension
Testing Multi-Level Interventions to Improve Blood Pressure Control in Minority
Racial/Ethnic, Low Socioeconomic Status, and/or Rural Populations
2
MOU with NIH
9/28/2015 Hepatitis C Clinical Management of Hepatitis C Infection 2
PCORI’s Funded Targeted Studies

23.  American Academy of Asthma, Allergy and Immunology
 American Academy of Child and Adolescent Psychiatry
 American Academy of Family Physicians
 American Academy of Orthopaedic Surgeons
 American Academy of Pediatrics
 American Association for the Study of Liver Diseases
 American Association of Hip and Knee Surgeons
 American College of Cardiology
 American College of Chest Physicians
 American College of Emergency Physicians
 American College of Physicians
 American College of Preventive Medicine
 American College of Sports Medicine
 American College of Surgeons
 American Orthopaedic Association
 American Society for Radiation Oncology
 American Society of Anesthesiologists
 American Society of Clinical Oncology
 American Thoracic Society
 Canadian Orthopedic Trauma Society
 Infectious Diseases Society of America
 North American Society for Pediatric Gastroenterology, Nutrition and Hepatology
 Society of Hospital Medicine
Physician Organizations Engaged in Large
Studies
Includes PCS and Targeted studies awarded as of January 2016

24. The examples provided below are intended to be illustrative, not exhaustive or
prescriptive. Areas of interest include, but are not limited to the following:
• Translation/adaptation of the content/delivery mechanism of effective CER
results/products to improve their penetration and use at the policy, health
systems, clinical practice, caregiver, and patient levels
• Development, demonstration, and evaluation of processes or products to
incorporate PCORI research results into decision making settings for patients,
clinicians, policy makers and other stakeholders
• Demonstration of the capacity and ability to take research results and products
found effective through PCORI research studies “to scale” in diverse settings and
populations
• De-implementing or reducing the use of strategies and procedures that are not
evidence-based, have been prematurely widely adopted, or are harmful or
wasteful, in place of evidence-based approaches
Specific Areas of Interest for Dissemination Funding