The Patient-Centered Outcomes Research Institute: “New” Initiatives

Anne Trontell, MD, MPH

Senior Program OfficerClinical Effectiveness Research ProgramPCORI Office of ScienceFebruary 2, 2016

• 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

• 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

• 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

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

• 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

• 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

List 6Approved for a

Targeted Funding Announcement

List 2Approved for Topic Brief Development

List 3Approved for Advisory

Panel Review

List 4Reviewed by

Advisory Panels

List 5Approved for Refinement

List 1Nominated Topics

List 7Listed as a Priority in a Pragmatic

Clinical Studies Funding Announcement

Lists 6 and 7

Topic Prioritization Pathway

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 diseaseReviewed 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

• 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

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

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

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

Additional Information

• Questions about applying for PCORI fundinghttps://help.pcori.org/hc/en-us

• Questions about whether your research is a fit for PCORI funding: sciencequestions@pcori.org orhttps://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

Additional Information

• Information on all PCORI Funding Opportunities, including pragmatic, targeted, and dissemination researchhttp://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 studiesatrontell@pcori.org

Contact Us

www.pcori.orginfo@pcori.org

Additional Information

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

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

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

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

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 1MOU with NIH

9/30/2014 Fibroids PCOR for Treatment Options in Uterine Fibroids: Developing a Prospective Multi-Center Practice-based Clinical Registry (P50)

1MOU 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

2MOU with NIH

9/28/2015 Hepatitis C Clinical Management of Hepatitis C Infection 2

PCORI’s Funded Targeted Studies

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

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