Hello. I am Dr Joe Selby, executive director of the Patient-Centered Outcomes Research Institute (PCORI). Welcome to this program titled "The Role of Patient-Centered Outcomes Research inthe Current Healthcare Landscape: Setting the Stage."

Joining me today are Dr Adrian Hernandez, director of Health Services and OutcomesResearch at Duke Clinical Research Institute at Duke University Medical Centerin Durham, North Carolina;

Ms Cheryl Jernigan, who is a lead patient advocatefor the Greater Plains Collaborative of PCOR net Clinical Research Data Network and a member of PCOR net Patient Council and Patient and Consumer Engagement Task Force;

and Dr Lisa Simpson, president and chief executive officer of Academy Health in Washington, DC. Welcome.

Let us start by just getting on common ground of what we mean by patient-centered outcomes research (PCOR).

The first thing that needs to be emphasizedis that it is comparative research. We compare the benefits and the potentialharms of alternative, preventive, diagnostic, therapeutic, palliative, orhealthcare delivery system interventions to inform decision making,highlighting comparisons and outcomes that matter to people.

It includes outcomes that are related to individual preferences and needs, outcomes thatpeople notice and care about, such as survival, but also function, symptoms,and the quality of one's life.

It incorporates a wide variety of settings and diversity of participants so that the findings will generalize to the same variety of settings where patients find themselves. It aims to optimize outcomes for individual patients.

The focus is in part on individual patientswhile addressing the burden to individuals, the availability of resources, andother stakeholder perspectives.

Let us start with a question at this point.How would you say PCOR differs from patient-reported outcomes?

One of thethings about PCOR that is different from patient-reported outcomes is thatpatient-reported outcomes is a segment of PCOR, so actually PCOR is a broad setof research that includes clinical outcomes that are meaningful to patients aswell as other patient-reported outcomes, such as health status or functionalstatus.

Patient-reported outcomes are usually found in PCOR studies, but PCOR studies are much more thanpatient-reported outcomes.

I think that another important dimension of PCOR that may not always be present in a studythat does include a patient-reported outcome is patient engagement throughoutthe research proposal. That concept of patient engagement is a central part ofwhat PCOR is as well.

Let us talk a little bit more about that. Obviously, if we want to get to patient-centered research, it's nota bad idea to ask patients. Is that right, Cheryl?

That is a good idea. Patient engagement means meaningfully involving patients, their families,their caregivers, and/or their advocacy organizations as partners, ascollaborators, and as advisers throughout the research process. This wouldstart from topic selection, move through design and conducting of the research,and the dissemination of the results.

When you engage patients, the researchhas the tendency to become more patient centered, more useful, and verytrustworthy, ultimately leading to greater use and uptake by both cliniciansand patients in the greater healthcare community of the research results.

Dr Selby: Who are these patients andpatient advocates that we are talking about?

Ms Jernigan: Besides being everyday people,they are survivors or co-survivors. That means they are the family members ofloved ones affected by the disease or by the medical condition. They aremotivated to make a broader impact by working to understand the science behind their disease or medical condition and also to understand its treatment.

They are committed to becoming involved in research to make a difference forpatients. They are connected to a broader community of patients. They are not just bringing their own perspective, but bringing a broader perspective.

We view our role as a profession. We needto do our homework. We need to help increase our knowledge about what theresearch is all about and about the science behind it.

A member of the research team.

Exactly.

Let us talk more about patient engagement and how we make sure that our research really is patient centered and the critical role that patients play in helping assure that the patient perspective is incorporated into the research we do. Adrian, you have an example.

Yes. Patients actually help with developing the concept of the question. What is actually the most meaningful question to patients or patient groups?

Patients work with clinicalinvestigators to develop that study concept, to go out and secure funding, toprepare the study protocol, to actually create the study procedures, implement the study, and to follow the track along the study progress. Ultimately, whenthe results come out, patients actually review the results, interpret theresults, and understand what they need and how they are meaningful to patientsin the communities that they represent.

Then, ultimately, they help withdisseminating results to clinical practices, patient communities, and thoserepresenting a variety of perspectives, the various stakeholders, whether it ishealth plans or health systems, to ensure that those results are implemented inpractice.

Dr Selby: From the beginning to the end ofthe research process, at all points. Lisa, we also see patients and otherstakeholders as well involved in the review of applications. What are theydoing there? What is their role?

Dr Simpson: This is a really important stepbecause Adrian has really outlined all the steps along this continuum of thelife cycle of the research project from beginning to end. One of the criticalsteps is going through review to get your funding to do the research.

One ofthe things that is really important in PCOR is that the review process involves patient representatives so that the voice of that patient is involved at that critical step as well.

Some of the questions that come up in thereview process, first and foremost, are as follows: Is this study important topatients and their families (from their perspective)? Is it important toimprove clinical practice so that patient care gets better? Who is eligible forthis study? Are they representative of the broader patient population?Importantly, does this study fill a gap in the research that we -- clinicians,patients -- currently do not have answers to, that it will really improve care?

The second-level question, once it has beendetermined this is a good study to fund, is: How are patients engaged in theprotocol, in the study itself? It is not just enough to ask those questions upfront, then write your study, and forget about the patients. They have to beinvolved throughout. That is taken into account during the review process.

One of the critical questions gets towhether the study will actually be able to accomplish what it lays out. Is theburden of participating in the study for patients too great? Then the wholestudy will not work. Is the study feasible? Once it is completed, the resultsare shared with patients and communities. It is not just shared with theclinicians and the professional side, but for the other side of the equation,importantly, so the patients understand the results as well.

Dr Selby: Are we talking about the reviewof research at PCORI? Are we talking about review of this type of researchacross the board?

Dr Simpson: Well, I think that one of thethings that PCORI has done is really put patient engagement on the map. This isa central feature of your institute's review process. There are some otherplaces that fund research that also incorporate this, but I think it is reallya central part of what PCORI, which we will talk about in a few minutes, doesfor the research enterprise.

Dr Selby: Thank you.

Ms Jernigan: PCORI really has bumped up theissue of involving patients.

Dr Selby: We have heard a lot frominvestigators about this notion. I just invite anybody on the panel to take alook at the slide that the audience is seeing now and make comments. We seethat in addition to a number of benefits that researchers are telling us, wealso have some concerns as well. Anybody want to speak to some of these?

Dr Hernandez: Yes. I can say before Ireally started doing PCOR where patients were really engaged, I had this ideathat what would they really bring? What would the patient engagement bring? Iwas worried that they would not have enough experience to actually make asuccessful research program or get answers that we needed. I was totally wrong.From beginning to end, the patients brought perspectives that we had notthought about. They were inspirational to our research team. We have taken thatmodel, actually translated it across our own research group and institute, andreally focused on the questions that matter and how they will ultimately matterto the patients we are caring for.

Ms Jernigan: Joe, I think people sometimesget confused that when patients are brought in to work with researchers or toreview research that they think we are trying to do that from a scientificperspective. We are not. We are viewing it as an expert having lived theexperience, of having been a patient or caregiver or family member of apatient. We are not bringing the research side of it. We are bringing thereal-life experience side of it.

Dr Selby: That is an important point. Whatwe hear and what we see in the data, in fact, is that the scientists on thereview panel drive the assessment of the methods and are an important factor inthe final scores. The scientists, the patients, and other stakeholders cometogether after the discussions as well.

Dr Simpson: I would just add that one ofthe concerns that we hear from the research community is that doing meaningful patientengagement, really incorporating their views and perspective, does sometimesadd to the cost or time of doing the research. That is important for theinvestigators to understand and be well versed in doing this so that they candesign their study appropriately to do that well. Too often, I think we maybethink about the patient perspective as an add-on or a last minute, and that isnot meaningful.

Dr Selby: That is right. Particularly, thatissue of the cost and time just points out that we really have to investigatewhat we are doing absolutely as quickly and as well as we can because we needto do it efficiently. This is like every other aspect of research. It needs tobe done efficiently. PCORI is very busy at trying to evaluate all aspects of this.

Adrian, here is a question for you. It maybe a trick question in a way, but I will ask you anyway. Once an investigatorhas a good idea about the research question, what does he or she do to go outand find patients or patient organizations to work with?

Dr Hernandez: Ideally, questions areactually coming from patient communities that you already engage with. Workingwith a patient community, we find that there is an unmet need that needs to beaddressed, and so we develop the questions with that patient organization. Forexample, the American Heart Association is a patient community that we do a lotof work with. We want to understand what is the unmet need or questions thatare related to that patient community? Then, how can we work together to findwhat is the right approach? What is the right outcome to address?

Dr Selby: Excellent. Adrian, can you giveus an example?

Dr Hernandez: Sure. We have been doing astudy called PROSPER, which is actually evaluating the comparativeeffectiveness of different therapies for stroke. As we were developing thestudy with our patient partners, we recognized that we were working together indifferent areas. While we thought that a certain area, such as thrombolytics,was really important, it turns out that was not as important for our strokesurvivors. What were more important were areas like depression.

We also realized that we were thinkingabout different outcomes and that the outcomes that mattered to the patientswere actually the time at home, or so-called home time. That caused us tochange our study approach, addressing the questions actually patients werecoming up with through their organization and then developing methods toaddress their questions. That has really made a big impact on PROSPER and whatwe have done with that study.

Dr Selby: Excellent. Not all PCOR involvesprimary data collection and recruitment of patients. You can do PCOR analyzingsecondary data, and we want to show an example of that as well. This PCORproject is called the Diabetes Prevention Program (DPP). It was partiallyfunded by PCORI.

Basically, the DPP showed that you canreduce the risk of developing diabetes in patients who are at high risk by 2ways. One is by lifestyle modification (physical activity and weightmanagement). Or you can reduce the risk of developing diabetes by givingpatients at risk a drug called metformin.

This secondary analysis of DPP found, veryimportantly, that only those patients who were at the very highest risk, thequartile at the highest risk of developing diabetes, usually because theirblood sugar was already trending toward looking diabetic, they were the onlyones who benefited from metformin, but all patients at any level of increasedrisk benefited from lifestyle. In fact, the patients at lower risk looked likethey did a bit worse on metformin.

The news to patients is do not overusemetformin. Only use it if you are in that highest risk quartile. The news toclinicians, as well, was very useful. Lisa, one more example?

Dr Simpson: Yes, I have another example,but I love the one you just described because it really gets to the point aboutunderstanding how results play out for individual patients, their subgroups,and that is a big part of PCOR as well.

Another example is a pediatric examplewhere PCORI funded a very important study comparing the effectiveness ofintravenous (IV) vs oral antibiotics for children with osteomyelitispostdischarge from the initial IV course in the hospital.

This is a really relevant question forpatients, for children and their parents, because having the child go home onIV antibiotics means a peripherally inserted central catheter (PICC) line, riskof infection, and all kinds of other complexity, whereas an oral antibiotic ismuch easier.

What this study found, again, throughsecondary data analysis, was that the risk of treatment failure, defined byvarious outcomes, was the same in the 2 patient groups. In fact, there weremore adverse events in terms of infections from the PICC line and some othercomplications. In the practice community, the question really was that thevariation in practices is quite wide.

This study helps clinician talk in a verydifferent way with the parents, patients, about should we do an antibioticorally. Should we continue IV therapy? Tailor care and treatment choices moreto that family. I think it is another great example of digging down deeper intoreally understanding how to tailor care to individual patient needs.

Dr Selby: In that study, they found noincrease in benefits but a definite increase in harms with the IV lines. Wetake the point that we still need to think carefully about tailoring.

Dr Simpson: Exactly.

Ms Jernigan: Joe, you said no benefits, butthe benefits would be for the patients not to have to worry about the PICCline.

Dr Selby: Correct. I meant there were noextra benefits from using the PICC line or the IV line.

Dr Selby: Let us talk a bit about the roleof PCOR in the dissemination of research. A very important priority at PCORI isthat we do not want our research to sit on the shelf. It cannot sit around for17 years. That is why we do engagement. All the panelists have shown verynicely how patients involve themselves in helping to disseminate the findings.

It is important to note that PCORI partnerswith the Agency for Healthcare Research and Quality in the dissemination ofresearch findings. We also fund research on dissemination because disseminationis hard. At its best, with the most compelling findings, it can be difficult toget the message out. One of the types of comparative research that PCORI fundsis research on how to communicate and disseminate.