1. Patient Engagement Celiac Disease Network Workshop
Only Session One is in PPT format, but introductory slides are provided as well. Workshop goals, objectives and agenda precede Session One.

2. Workshop Goals
Participants will increase their medical, scientific, and advocacy skills so they can improve the design, implementation, and dissemination of celiac disease research.
Purpose of baselines assessment

3. Medical and Scientific Learning Objectives
Participants will develop a working knowledge of:
Current practices in the screening of celiac disease
Current practices in the diagnosing of celiac disease
The underlying genetics that cause celiac disease
The pathway to drug development
Types and phases of clinical trials
Label reading to prevent gluten exposure
Purpose of baselines assessment

4. Advocacy Skills Learning Objectives
Participants will develop a working knowledge of:
The purpose and unique features of patient-centered outcomes research (PCOR)
The roles for patients and stakeholders as partners in research
The purpose of the Celiac Disease Foundation’s Patient Engagement Celiac Disease Network (PECDN)
Purpose of baselines assessment

5. Workshop Agenda Welcome
Session One: Patient Centered Outcomes Research
PPT Presentation
Understanding Check
Session Two: Celiac 101
Peter Green Lecture
Research Questions
Purpose of baselines assessment

6. Workshop Agenda Annette Taylor Lecture Understanding Check
Break
Session Three: Genetics
Annette Taylor Lecture
Understanding Check
Research Questions
Session Four: Clinical Trials
Dan Adelman Lecture
Purpose of baselines assessment

7. Workshop Agenda Janelle Smith Lecture Understanding Check
Break
Session Five: Nutrition Labels
Janelle Smith Lecture
Understanding Check
Research Questions
Kristen Yarema Lecture
Gallery Walk
Small Group Discussions
Tables Report Out
Session Six: Patient Centered Research Questions
Purpose of baselines assessment
Wrap up and Assessment

8. Session One Patient Centered Outcomes Research

9. Session One Objectives
You will learn:
The purpose, goals and unique aspects of Patient Centered Outcomes Research (PCOR)
The purpose and goals of the Patient Centered Outcomes Research Institute (PCORI)
The purpose and goals of the Patient Engagement Celiac Disease Network (PECDN)

10. Acronyms We’ll try to avoid them, but… CDF = Celiac Disease Foundation
PECDN = Patient Engagement Celiac Disease Network
PCOR = Patient Centered Outcomes Research
PCORI = Patient Centered Outcomes Research
CER = Institute Comparative Effectiveness Research
icure Celiac
MOSAIC

11. What is Patient Centered Outcomes Research?
Comparative Effectiveness Research (CER) The direct comparison of existing health care interventions to determine which work best for which patients and which pose the greatest benefits and harms.
What works best?
Patient Centered… What?
So let’s back up and put this in context. Comparative Effectiveness Research is a specific kind of health care research. Not all medical research compares two interventions directly. Comparative Effectiveness Research does just that. You can see the word “patient” in the definition. Comparative Effectiveness Research should be focused on what is best for the patient, but this has not always been the case in the past.
For which patients?
Under what circumstances?

12. Patient Centered Outcomes Research
Patient Centered Outcomes Research is a type of Comparative Effectiveness Research that answers patient-centered questions, such as:
Given my personal characteristics, conditions, and preferences, what should I expect will happen to me?
What are my options, and what are the potential benefits and harms of these options?
How can clinicians and the care delivery systems they work in help me make the best decisions about my health and health care?
What can I do to improve the outcomes that are most important to me?
Patient Centered Outcomes Research is a kind of comparative effectiveness research. It specifically and directly answers questions that are important to patients. Here are four questions for the Patient Centered Outcomes Institute to give you a sense of what questions PCOR is supposed to answer.

13. Different Types of Trials
Efficacy Trials
Does it work (produce the intended effect) in ideal circumstances?
Effectiveness Trials or Pragmatic Trials
Does it work in the real world?
To understand Patient Centered Outcomes Research, it’s important to understand that there are different kinds of clinical trials. The ones that have a strict “protocol”– that is, a lot of very particular rules about who can be included and under what circumstances, those trials are often “efficacy” trials. In those trials, the researchers set the trial up to increase the chances of getting a positive result. The theory being that if the drug is not effective under ideal circumstances there is no point in taking the trials any further into development. These efficacy trials may exclude older patients or patients with other health problems; sometimes the exclusions are really extensive.
Pragmatic or Effectiveness trials have a different focus. They are intended to test whether an intervention actually works, with actual patients in actual settings. There still will be a protocol– certain people are eligible to participate and others are not– but usually these trials includes a broader group of people. Patient Centered Outcomes Research tends to focus on pragmatic trials, because those are the ones that actually answer the questions patients want answered.

14. A Few Terms Patient Engagement IS NOT THE SAME AS Patient Centered
Inclusion of patients in the research process, from topic selection through study design and conduct to dissemination of findings.
IS NOT THE SAME AS
Patient Centered
Addresses questions that patients and their families care about in clinical settings.
So here are two terms that are easy to confuse. One is about a process and the other is about substance. Research that has patient engagement is the process of including patients in all aspects of the trial. Research that is patient-centered is actually focused on a question of concerns to patients. It is possible for research to be one and not the other. Say a thoughtful researcher came up with a great idea for a study, which addressed a really important question about celiac disease. She quietly writes a proposal, gets funded and conducts excellent research. She answers an important question that improves the quality of life for celiac disease patients. That is an example of research that has no patient engagement but is patient-centered. The opposite can happen as well. The point of including patient in the process is to improve the quality of the research, not simply make patients feel included. So if a researcher collaborated with a patient group to develop a research question and continued to work side by side with patient advocates throughout the research, that could be considered research with patient engagement. But by itself that doesn’t guarantee it is patient centered research. It is patient centered research if the patient/researcher team has successfully designed research about an issue of serious concern to patients and their families. That’s not inevitable. Still, the expectation is that, through meaningful patient engagement, we will end up with more and better patient centered outcomes research.

15. A Few More Terms Patient Reported Outcomes Patient Centered Outcomes
Any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else.
IS NOT THE SAME AS
Patient Centered Outcomes
Research that is specifically designed to meet the most important needs of patients. Relevant data can come directly from patients, or from anywhere else.
A couple other terms that might be confusing. Patient reported outcomes is anything that comes directly from the patient. Temperature or blood pressure is not patient reported, but pain usually it, along with mood, energy level, etc. Although certainly patient reported outcomes can be part of patient-centered outcomes, there is nothing inherently patient-centered about patient-reported outcomes. Someone could be collecting data about a patients’ mood to see if it is possible to more efficiently gather that data– this may or may not be a worthy cause, but that would not be an example of a patient-centered research question. Of course if the research was focused on how to get more accurate information about emotional well-being because patients identified this as an unmet need, then it could be considered patient-centered.

16. Terms you also may hear…
Patient-Focused Research Person-Focused Research Community-Based Research Participatory Research Collaborative Research
You’ll hear other terms so I will mention them here. Some people prefer the term “focused” rather than centered– perhaps avoiding the self-centered insinuation? Many folks resent being called “patients,” especially those with chronic conditions that they will live with for decades, so “person-focused’ works for them. Community-Based, Participatory, Collaborative, each of these have their own nuanced meanings, all that is important here is to know that the notion of engaging the end-user – that’s us– into the health care research to end up with more research that actually matters to patients– is the goal.

17. Patient Centered Outcomes Research Institute (PCORI)
Research Done Differently
Comparative effectiveness research that focuses on questions that matter most to the community affected. And now there is an Institute devoted to this. They call the research they do “research done differently.”

18. Patient Centered Outcomes Research Institute (PCORI)
Created by Congress under the Affordable Care Act to figure out which medical treatments work best
Criteria for evaluation includes patient engagement and patient-centeredness
Applications are reviewed by scientists, patients, and other stakeholders
Began funding research in December 2012
Expects to spend $3.5 billion by the end of the decade
PCORI is not the first funder to include patients in the funding process, but they are the largest and the depth and breadth of engagement is really extraordinary. A few things to know about PCORI– not a government agency but fully funded by the federal government, the funds come from hospital and other fees that were part of the Affordable Care Act. They include both patients (which includes family members) and other stakeholders, which is a big bucket. That includes doctors, insurers, payers, nurses– pretty much everyone who has a stake in the health care system.
The funding for PCORI expires in 2019.

19. Patient Centered Outcomes Research Institute (PCORI)
Patient-centered is not is enough— Good science is not enough— It must be BOTH!
Let me pause here to emphasize a very important point. Patient-centered research is useless is if isn’t good research. Unless rigorous methods have been used, any results will be completely unhelpful– to patients or anyone else. But note it is possible for a very clever, fascinating, well-designed and meticulously implemented study to focus on a question or an issue that simply doesn’t matter to patients. And a research question that doesn’t matter, even when designed and implemented using the best science there is, at the end of the day it doesn’t matter. So they are equally important.

20. Patient Centered Outcomes Research Institute (PCORI)
PCORI brings patients, other healthcare stakeholders, and scientists together in every step of the research process, including:
--Selecting research topics to study
--Identifying research projects to fund
--Collaborating on the research team
--Communicating research findings
--Building an inclusive merit review process

21. Patients Can Participate in Planning Research by ...
Developing the research question and relevant outcomes to be studied.
To ensure that the project and its results will be useful and important to patient and stakeholder communities.
Why?
Defining the characteristics of study participants.
To minimize the risk that certain patients will be included or excluded due to criteria that are not relevant.
Why?
This material is from PCORI, they have some amazing resources including a rubric that literally walks you through the process of designing patient-centered research.
Drafting or revising study materials and protocols.
Why?
To promote/increase retention of study participants.

22. Patients Can Participate in Conducting Research by ...
Drafting or revising study materials and protocols.
To ensure feasibility for clinicians and patient participants.
Why?
Participating in recruitment of study participants.
To increase and sustain recruitment, and to ensure viability of the study.
Why?
Participating in data collection and data analysis.
To lend unique and varied perspectives on interpretation of the data.
Why?

23. Patients Can Participate in Conducting Research by ...
Serving as a patient representative on a data safety monitoring board.
To make the board composition more robust and patient-centered.
Why?
Participating in the evaluation of patient and stakeholder engagement.
Why?
To ensure authenticity and value of engagement.

24. Patients Can Participate in Disseminating Research by ...
Identifying partner organizations for dissemination.
Why?
To ensure meaningful and direct connections with end-users.
Planning dissemination in the context of shaping study design and protocol.
Why?
To ensure dissemination is incorporated into the research from the very beginning. .

25. Patients Can Participate in Disseminating Research by ...
Authoring manuscripts and presenting study findings.
To offer the patient perspective and to reach new and different audiences.
Why?
Identifying opportunities to share information about the study, even as it is in progress.
To move away from traditional models of dissemination and think more creatively about how to get information into the hands of those who need it.
Why?

26. Translating Our Concerns and Questions into Research
Who are the people that should be studied? This is the population of interest. How can people make informed choices between options? These are the factors that people will consider when making a decision between/among options. What options should be compared? These are the decisions the research is intended to inform.
The People
The Options
The Outcome

28. Patient Engagement Celiac Disease Network (PECDN)
Purpose
To develop a nationwide network of patient advocates who can help to design, implement, and disseminate results of patient-centered outcomes research that are important to adults and children with celiac disease and caregivers.

29. Patient Engagement Celiac Disease Network (PECDN)
Goal
Establish a national network of 200 celiac disease patient advocates who are ready, willing, and able to collaborate with the research community by the end of 2017.

30. Review: Session One Objectives
You will learn:
The purpose, goals and unique aspects of Patient Centered Outcomes Research (PCOR)
The purpose and goals of the Patient Centered Outcomes Research Institute (PCORI)
The purpose and goals of the Patient Engagement Celiac Disease Network (PECDN)

31. Worksheet and Discussion
Session One
Worksheet and Discussion