CRISP Presentation on PCT Study Design: Case Study for Patient-Centered PCTs C. Daniel Mullins, PhD Professor and Chair Pharmaceutical Health Services Research Department University of Maryland School of Pharmacy

Acknowledgment: Parts of this presentation are based on work completed under contract PCORI-SOL-PCWG-002 funded by the Patient-Centered Outcomes Research Institute (PCORI) and grant 1R24HS funded by the Agency for Healthcare Quality and Research (AHRQ). Disclaimers: The views expressed in this presentation are solely those of the speaker and do not necessarily represent the views of the Patient- Centered Outcomes Research Institute (PCORI), its Board of Governors, or Methodology Committee. The findings and conclusions in this presentation are those of the author, who is responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this presentation should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services. 2

What does it mean to be patient centered? 3

4 right drug or treatment + right patient

Right Drug or Treatment How do we know what is the right treatment? Evidence-based medicine Reliable Meaningful Comparative effectiveness Relative value Implies choices 5

Right Patient Patients are unique: no one-size fits all medicine Heterogeneous response to treatment Genetic factors Patient behavior and culture Patient-centered outcomes research (PCOR) Subgroup analysis Patient preferences 6

7 Provide evidence to answer patients’ questions such as: Given my illness and the treatment outcomes that are most important to me, what should I expect to happen to me? What are my treatment options? What are the benefits and harms of those options? What can I do to help improve the treatment outcomes that are most important to me? How can my health care team help me make the best decisions about my care? What does it mean to be patient centered?

Patient-Centered PCTs 8 Design: How best to answer patients’ Qs Outcomes: Meaningful/actionable for patients Follow-up: Patient burden/fatigue; recall bias Analysis:Heterogeneity of patients

9 WHAT IS PATIENTS? PATIENTS is an AHRQ-funded Patient-Centered Outcomes Research (PCOR) Research Infrastructure Development Grant (R24HS )

PATIENTS:  Empowers patients to propose questions about their health care concerns and actively participate in studies to answer them  Combines the expertise of: Researchers from the University of Maryland Health care systems Patient communities Advocacy groups  Challenges the status quo by embracing patients and health care systems in every component of our research studies 10 WHAT IS PATIENTS?

PCOR is a Win for Everyone PATIENT RESEARCHER Informed decision making Meaningful research Not a “one- size-fits-all” result Interaction Continuous Topics and research questions Translate/disseminate results Comparators and outcomes 11

Why? Patient Engagement: Why? Makes PCOR meaningful – Framing the question so it is understood – Framing the answer so it is understood – Framing for cultural appropriateness – Does meaningful imply significant or impactful? Helps with patient activation Having the essential knowledge, skills and confidence for managing one’s own health and health care It shows an honest commitment to being patient centered 12

How? Patient Engagement: How? 13 IdentifyingPartneringExplainingDoing Updating Building/Maintaining Trust and Respect

10-Step Process for Conducting CER * 1.Topic Solicitation 2.Prioritization 3.Framing the Question Public Announce- ments Patient Forums Delphi Process * Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15):

* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): Selection of Comparators and Outcomes 5.Creation of Conceptual Framework 6.Analysis Plan 7.Data Collection In-person Meetings Focus Group Interviews Telecon- ferences Electronic Social Media Telephone Calls 10-Step Process for Conducting CER * * Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15):

* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): Reviewing & Interpreting Results 9.Translation 10.Dissemination Teach-Back Method Critique Documents (e.g. Patient Guides) Media 10-Step Process for Conducting CER * * Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15):

Pragmatic clinical trials (PCTs) should be – Pragmatic – Patient centered Patient engagement can enhance PCTs – Design – Execution – Translation and dissemination Summary 17