organization engagement in PCORIs pragmatic clinical studies program - - PowerPoint PPT Presentation

Jaye Bea Smalley, MPA Melissa Delbello, MD MS

Engagement Officer, PCORI Professor of Psychiatry, Pediatrics & Psychology, University of Cincinnati

Anne Trontell, MD, MPH Angie Day

Associate Director, Science, PCORI Chapter and Volunteer Services Manger, Depression Bipolar Support Alliance

Jean R. Slutsky, PA, MSPH

Chief Engagement and Dissemination Officer, PCORI

Innovative practices in patient and stakeholder

• rganization engagement in PCORI’s pragmatic

clinical studies program

Academy Health Research Meeting, June 27, 2016

• Introduction to PCORI and the Pragmatic Clinical Trial Program
• Introduction to PCORI engagement and early engagement

learnings from Pragmatic Clinical Trials Program

• MOBILITY* Trial and Impact of Engagement
• Patient organization perspective

* METFORMIN FOR OVERWEIGHT &OBESE CHILDREN AND ADOLESCENTS WITH BIPOLAR

SPECTRUM DISORDERS TREATED WITH SECOND-GENERATION ANTIPSYCHOTICS

Agenda

PCORI

• An independent research institute authorized by Congress in 2010

and governed by a 21-member Board representing the entire healthcare community

• Funds comparative clinical effectiveness research (CER) that engages

patients and other stakeholders throughout the research process in

• rder to compare alternative options in
• Prevention, diagnosis, treatment, or management of a disease or

symptom

• Healthcare system–level approaches to managing care
• Elimination of disparities in health or healthcare
• Seeks answers to real-world questions about what works best for

patients based on their circumstances and concerns

3

• Addresses compelling choices or “decisional dilemmas” facing

patients and clinicians today

• Compares the effectiveness of 2 or more options as they are

used in “real-world” (non-research) settings

• Maximizes the diversity of patients, care providers, and

health care settings where the study is done

• Limits the research burden on participating patients and

clinicians

• The goal: To be relevant, applicable, and readily implemented

in care

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What is a Pragmatic Clinical Study?

• The potential to fill a defined, critical knowledge gap that is

important to patients, clinicians, and others in health care

• Based on clinical burden, a gap identified from a synthesis of

available data or guideline efforts, or from well-established research priorities

• Suggested by variations in practice that suggest clinical

uncertainty

• The potential for the study’s findings to be adopted into clinical

practice to improve health and health care

• The ability to assess if outcomes vary amongst different types or

subpopulations of patients

What Does PCORI Seek in Funding Pragmatic Clinical Studies?

• Launched in 2014 to support large scale but simplified studies

that meet requirements previously described

• Current awards: 20 studies with total funding of $242.2 million
• Individually and collectively a significant investment
• Targeted studies similar but with a specified research focus
• Engagement is critical

– To define the problem, design the study that can address it, assure the study is realistic and feasible, and help its findings be adopted into practice

PCORI’s Pragmatic Clinical Trials Initiative

We Fund Research That…

• Patients are partners in research, not just “subjects”
• Active and meaningful engagement between scientists,

patients, and other stakeholders

• Community, patient, and caregiver involvement already

in existence or a well-thought-out plan

“Patient and stakeholder engagement” What we mean by…

• The project aims to answer questions or examine outcomes that

matter to patients within the context of patient preferences

• Research questions and outcomes should reflect what is

important to patients and caregivers

“Patient-centeredness”

The PCORI Approach to Engagement-Our Engagement Rubric

Who Are Our Stakeholders?

Purchasers Caregivers/Family Members Payers Patients/Consumers Clinicians Training Institutions Policy Makers Hospitals/Health Systems Industry Patient/Caregiver Advocacy Organizations

Elliot Israel, MD Brigham and Women’s Hospital

Engagement

• Patients, patient advocacy groups,

providers (including pharmacists), professional societies, healthcare policy experts, and content experts have been engaged in formulating the research question, selecting the intervention, and

• utcomes, and will continue to guide the

conduct of the study. Potential Impact

• Intervention could address knowledge gap

and reduce asthma exacerbations in a manner concordant with patient treatment preferences.

• Findings could be included in the National

Asthma Education Prevention Program (NAEPP) guidelines and influence clinical practice.

Examines whether symptom- based use of inhaled corticosteroids (ICS) reduces asthma exacerbations compared to daily use of ICS in African-American and Hispanic/Latino patients with asthma.

Pragmatic Clinical Studies, Awarded January 2016

Patient-Empowered Strategy to Reduce Asthma Morbidity in Highly Impacted Populations (PESRAMHIP)

Shelley Hwang, MS, MPH Duke University

Engagement

• Study team includes a Patient Leadership

Team comprised of cancer survivors who will coordinate with the ALLIANCE Cooperative Group Patient Advocate Committee Potential Impact

• Inform treatment decision making and

potentially help to mitigate overtreatment and treatment-related morbidity of DCIS therapies Methods

• PCT

Examines whether active surveillance is non-inferior to guideline-based care for women diagnosed with low-risk ductal carcinoma in situ (DCIS)

Pragmatic Clinical Studies, Awarded January 2016

Comparison of Operative versus Medical Endocrine Therapy for Low-Risk DCIS: The COMET Trial

Thank you!

www.pcori.org info@pcori.org