Keynote: PCOR, PCORI, PCORnet: Oh My!- Maryan Zirkle MD, MS, MA

okay as the Silla said my name is Marian circle I see some familiar faces but I am a program officer at the quarry if you don’t know me or haven’t heard me on a call or something when you’ve been working with Lucilla um I manage p scanner as she said but also the other clinical data research networks that are involved in bacoor net and sort of the data aspects that are coordinating center works on and then also methods projects that have to do with big data research networks and distribute analytics so I’ll get started I want to first state that I’m not a wizard of oz fanatic or anything but when I talked with lassila about what we might discuss here and thought about all the questions that I get when I go speak at conferences or just go to conferences about the things that I assume people know I started thinking okay p corpa koriba cornet and i thought oh my that no wonder everyone gets confused because unless you come from a federal government agency and everything you do and everything you say has to do with acronyms this can be really confusing and to understand what we’re talking about so today I’m just going to talk a little bit about the peak or trust fund that basically is funding the quarry which is the patient-centered outcomes Research Institute I’ll talk to you a little bit about the Institute where I work and the type of funding and research that we have and some opportunities are out there that might align with networks such as P canopy scanner excuse me and then the cornet of course because that’s the work that I do so again I’ll try to keep it interesting because this is a bit of a long talk I thought again the yellow brick road seemed to fit right in because those that have been involved the cornet Orpah Corey I guess I’ve been there for two years it’s not a linear process in any way we do a lot of winding and moving around and trying to go towards the best things I think we’re doing a really good job we are all going towards something and we’re moving quite fast so it seemed to fit well so again I’ll start out so the patient-centered outcomes research trust fund funds the patient-centered out research institute it was established by Congress through Patient Protection and Affordable Care Act of 2010 and receives income from three funding streams and that’s the Treasury CMS and peak or fee now who and what is funded basically eighty percent of the funds from these three streams go to the quarry for research funding and operations but twenty percent go to the apartment of Health and Human Services majority HR q for dissemination research capacity building and this seems pretty simple but this has been something that’s really hard for I think people to get you know how are we doing this and where’s all going now / Cory again this is not a government Institute we are an independent research institute we’re governed by a 21-member board representing the entire health care community for those of you who have received rewards or attempted to receive awards for the quarry our Board of Governors meetings are public and that’s where you find out whether or not you were awarded and you can take a look on the website you can always go in and listen in anyone can listen mmm the quarry funds comparative clinical effectiveness research which we term CER you’ll hear that a lot and they engage patients and other stakeholders throughout the research process and they seek answers to real world questions and you can basically say that patients are true north of the quarry so basically in a nutshell pocari is research guided by patients caregivers in the broader health care community that’s sort of what you want to come away with we have three main goals increase quantity quality and timeliness of useful trustworthy research information available to support informed decisions we want to speed the implementation and use of patient-centered outcomes research evidence and we want to influence research funded by others to be more patient centered so what makes us different there’s a few things um but again the takeaways are we fund research on which care options work for whom and under which circumstances we focus on answering questions most important to patients and those who care for them and we aim to produce evidence that can be easily applied in real world settings and we engage patients caregivers clinicians ensures employers and other stakeholders throughout the entire process so who are stakeholders we have a bunch and as you might imagine it is a lot of effort to engage all of these different stakeholders so we have

a few ways that we do this we have merit review panels speaker’s bureau advisory panels ambassadors webinars workshops I’m going to talk to a little bit about the engagement Awards because I think these are things that sort of get kind of swept to the side people don’t really know they exist and for networks out there in particular CDR ends it’s a supplemental funding you know way to supplement i should say your funding for engagement so we have four awards that are currently available under the eugene washington but Corey engagement Awards these awards are up to 250 thousand dollars per project and up to two years in duration the first of the knowledge Awards which increased knowledge about how consumers and healthcare information excuse me view receive and make up use excuse me make use of the core and CER training and development awards that build capacity for participating in peak or and CER and create ways to connect patients caregivers clinicians and other health care stakeholders within the research community the dissemination Awards they develop and strengthen channels for disseminating and implementing p corn see our findings and the meeting and conference support awards which provide organizations with funding for meetings and conferences that align with the quarries mission again this is something that some of the other CDR ends have taken advantage of so basically as i said we find comparative clinical effectiveness research what does that mean so this is research that generates and synthesizes evidence comparing benefits and harms of at least two different methods to prevent diagnose treat and monitor a clinical condition or improve care delivery research that measures benefits in real world as I said and research that informs a specific clinical or policy decision it is also research that describes results in subgroups of people research that applies appropriate methods and data sources and research that helps consumers clinicians purchasers and policymakers make informed decisions that will improve care for individuals and populations of a note we do not fund cost effectiveness research as per our mandate so this isn’t our air work it’s kind of tricky and I get a lot of questions we have national priorities for research each one of these that I’m going to talk about is actually a department within the quarry they each have their own program officers just like I work for infrastructure and methods with an epic or net Network and so when I go through these things if you have ideas or even thoughts about how you might request or apply for research in this area you’re more than happy to reach out to program officers listed on our website that work in this department or me and I will get you to someone in this department so the first is assessment of prevention diagnosis and treatment options this is research that compares the effectiveness and safety of alternative prevention diagnosis and treatment options it determines which one’s work best for different people with particular health problem the next is improving health care systems this is research that compares health system level approaches to improving access again another one that could be really well aligned with the cornet network affiliates supports patient self-care innovative use of health information technology care coordination for complex conditions and effective workforce deployment our communication and dissemination research priority is research on providing information produced by CER empowering people to ask for and use the information and supporting shared decision-making between patients and their providers we have a national priority for research on addressing disparities this is research on prevention diagnosis or treatment effectiveness preferred clinical outcomes across patient populations and healthcare required to achieve best outcomes in each population we actually have two of our CDR ends from phase one that are working on an award in this area right now lastly we have our national priority for research on accelerating patient-centered outcomes research and methodological research this is where picure net falls in this is research on building data infrastructure improving analytic methods and training researchers patients and other stakeholders to participate in this research so okay this is what i consider pakoras magic slippers ruby red slippers however you want to call it special sauce so our patient centeredness and patient and stakeholder engagement is like no other this is what we do and i often get what do you mean how do we do that how do we write that on proposal so patient

centeredness is surrounds project aims answering questions or examining outcomes that matter to patients within the context of patient preferences the research questions and outcomes should reflect what is important to patients and caregivers a patient and stakeholder engagement is the patient’s being and partners and research being engaged not as subjects but again as partners so it’s active and meaningful engagement between scientists patients and other stakeholders and community patient and caregiver involvement already an existent or well-thought-out plan within your proposal and again when proposals come in if this is a misunderstanding we often work with them to clarify what we mean and what the differences are so to go a little bit further if you think about all of those national priorities that I mentioned within that we fund research that focuses on high priority conditions these are affecting large numbers of people across a range of populations placing a heavy burden on individuals families specific populations in society and including rare diseases which are difficult to study that this is why we had rare disease cohorts built into our picure net network affiliates to neurons and we fund research that pays particular attention to specific populations and you’ll see a lot of this come out and several of our networks and for instance we have older adults women veterans which as Lucilla mentioned obviously p scanners bringing in the largest amount of veteran population of any network with the Vinci database we fund research that target specific high priority topics this is a long list you can see that let’s see about two of them are projects administered by the NIH so we work closely with them and then we also have projects or a project that is administered by the HR q and we’re building and building and building always our collaborations with the other funding institutions in addition to this list that’s ongoing just i believe it was yesterday pakora posted i think it’s what is it for new funding opportunities that are targeted they are around long-term opioid use for chronic pain treatments of MS studies in the area of treatment resistant depression and then also studies in the area of new oral anticoagulants so again this just got posted on our website so I wanted to let everyone know I’m quite sure you don’t go there daily to find out what new and innovative ways we have for funding but it is something to think about it if you’re considering applying for research studies so this is something that I will talk a little bit more about later sir for some funding opportunities but as you know we also work to improve research methodology at the quarry as far as we’re concerned in any study obviously methods matter and so we’ve developed methodology standards that patient-centered CER should follow at a minimum so if you apply to pick quarry there are these 11 broad categories you can see that if if you are a network applying obviously there’s several that pertain to you and if applicable you are expected to respond that you are following the standards as set forth by the quarry so as of the 28th we actually have 468 projects funded in all fakhouri right now we have over a billion dollars awarded at this time and we are in 41 states so we’re doing pretty well and again this is what I was sort of alluding to earlier this is our pack or a funding opportunity page and I would urge you to go to this page and where you see the upcoming tab if you click on that as of yesterday they listed a bunch of new opportunities that will be coming out those four I just listed in addition to a the new improving methods PFA will be released as well and they’ll all be released in october 12 are open for you to look at the PFA and then submit your loi and/or application whatever is applicable so i said i was going to talk a little bit more about the methods program again this is a portion of my department and I’m lucky enough to be able to manage about I think six studies right now dealing with a variety of different things overall the our entire portfolio on methods has 73 funded projects right now at about 70 1 million dollars awarded and we’re in 24 States DC and Quebec our current portfolio is research on methods to excuse me research related to a patient and stakeholder engagement in the research process patient centered in patient reported outcomes human subjects

protection against uh D design analytic methods and then data research networks and I don’t know if all of our networks within the cornet are aware of the opportunities here I mean you’ll see when I jump into the cornet talk that there have been a lot of struggles there’s been a lot of lessons learned and things that you know could possibly take us on another one of those yellow brick roads that that would be interesting for some of our networks to study and these are opportunities to do so so again this is a little bit more detail on what you’ll find if you go look on the website like i said october 12 there will be a new PFA released and to my knowledge it will look very much similar to the one that was in cycle 3 or excuse me cycle to this will be cycle 3 you can see the previous announcements and take a look these are all the areas of interests that we will be funding so again i always encourage people to take a look at our website because it helps you get ahead a little bit because our turnaround times are quite short now so after talking about pecora obviously you can see that we do research differently for a lot of different reasons obviously patient senators patient engagement our focus on CER mpcore in general so if that’s the case that that’s research done differently obviously pic or net is research infrastructure done differently and you know this is one of the tag lines that we say all the time but it actually means a lot there’s a lot in there but the Coronet brings together the expertise the populations resources and data of its participating organizations to create a national infrastructure that enables more efficient patient-centered research and it is just that so you can see that we had in phase 1 11 c drn’s more than 70 participating health systems within that we had portal la sierra and pedes net path some of these you may know about if you’re a member of p scanner you I’m quite sure you’ve worked with the other groups because I know as Lucilla said not only with the PPR ends are they collaborated here at P scanner but with every other network in the cornet and PPR ends we had 18 and phase 1 and that was a combination of 80 different organizations again this information is all on the cornet org so you know if you aren’t speed readers and obviously this is kind of loaded slide so I don’t want you to have to waste your time looking through that but you can learn more about each one of the organizations and the networks at peak or net org so again when I was speaking with Lucilla what to talk about I we thought that past present and future sounded like a really good thing we’re always looking to solidify our vision at Cory ferpa cornet and for me I felt like here we go again it’s a little bit more of The Wizard of Oz so we have you know leading experts in picure net across the entire nation we have so much heart it’s unbelievable the amount of passion that goes into the work that is done it must be unreasonable hours of the night because I don’t know how its I don’t know how they do it and just the determination and the boldness of the participants at all levels and this network is amazing and it continues it’s been 18 months and i’m quite sure it’s going to go into the next three years so when i thought about how do you look at phase one and sort of summarize it to look back or evaluate whether we did what we were supposed to do or what we wanted to do in the end and in my mind there were sort of research readiness not for each individual Network per se but for their network as a whole is what was important to me and if you can break that down based on technical infrastructure clinical trial infrastructure administrative simplicity and people power so I’m going to go through all four of those things and sort of just let you know what’s stuck out in my mind that brought us more towards that what opportunities that gave us and how we’re evaluating it as we move forward so technical data infrastructure and as Lucilla said you know there’s more than a million patients involved in each network as she said they have 30 million or more but at this point every one of our CD errands have transformed greater than 1 million patients into the cornet common data model now that took a lot of time and a lot of effort but it also then gave us the opportunity to work toward optimizing processes for obtaining complete data and this is means administrative claims data but also patient reported outcomes any other supplements or supplementary data that you can use to complete the

patient picture now in order to test this technical data infrastructure we have our obesity demonstration projects and again I just very high level going into this we have to obesity projects the bariatric study and then effects of antibiotics on childhood growth these are the CD our hands and PP errands that are involved in each for the bariatric study we’re looking to compare three bariatric surgical procedures our outcomes measured will be changes in BMI at years 13 and five obesity related outcomes for resolution of type 2 diabetes and incidence or recurrence of type 2 diabetes and then adverse outcomes will be hospitalization reoperation and death and then for the short long short and long term effects of antibiotics on childhood growth will be comparing effectiveness of different antibiotics used during the first two years of life and also evaluating the comparative effectiveness of different types and amount of antibiotics used in the first years of life on the rates and patterns of childhood growth during the first five years of life so the outcomes measured there will be body mass index at ages five and ten and growth trajectory for height and weight through preschool ages so as I said that’s the work that will do to test our data infrastructure so what about our clinical trial infrastructure all phase one CDR ends are building the capacity to embed clinical trials into the clinical care workflow without disruption now this provided for the opportunity for innovative techniques to leverage electronic health data streamlined consent and contracting and reduce burden on providers and we will be testing this with a clinical trial demonstration project this is our adaptable trial again I don’t know if you all have heard about it and it’s all over our web page if you’d like to learn more but the gist of it is basically um it is comparative effectiveness of 81 versus 325 milligrams of aspirin for secondary prevention of cardiac events and serious bleeding and it’s led by dr. bow and dr Han des Duke now the third thing I mentioned was administrative simplicity so all phase one Syrians are streamlining their RB processes as Lucilla mentioned and most are using the vehicle of reliance agreements and this is an opportunity for us to make ourselves more attractive to external funders as a network and become a major success for the cornet at this current time we are developing the cornet wide policies and abba cornet master contract and these will also be tested on these these demonstration projects i just mentioned the to obesity in the clinical trial now last but certainly not least is the people power and transforming clinical research through PPR ends so our PPR ends as i said 18 in the first phase represent 18 different models of partnerships and levels of infrastructure and they also represent over a hundred diseases overall now the PPR ends provided an enormous wealth of opportunities but i will say in my opinion one of the greatest things has been the collaborations across the cornet and I’ll also mention that in talking to Lucilla one time we at the very beginning we were thinking about how can we work these collaborations between the PPR ends in the CD iran’s and it was a struggle for a lot of the p is for the CDR ends not because they couldn’t figure out what they wanted to do but how and when would we have the time because we were keeping them pretty busy but i remember a comment lee Silla made about the fact that you know of course we want to work with the PPR ends there are gateway to all these patient reported outcomes and it’s exactly the truth we have PPI on demonstration projects where we’re going to try and demonstrate just how much information we can get from these people the PPR ends their patients their communities and really harvest that that data and that knowledge so our pprn demonstration projects they’re pretty new I’d be surprised if people knew other than Mitch who I can’t find in the audience put so picure is investing in supporting communities or networks of patients motivated to participate in clinical research through the cornet and to develop their capacity to govern the research activities of their network we are going to fund up to eight individual projects the application deadline is today and we will award up to 2.5 million dollars for up to three years and all the phase two PPR ends were invited to submit a low eyes for this one the other demonstrations project started prior to phase two quite a bit prior so that isn’t necessarily an

option as yet but maybe in the future and awards will be announced and the project start date for these PPR and demonstration projects will likely be January 2016 and already right now we had ten PPR ends that were invited to submit applications so we’re really excited about that so to summarize this and what are the hallmarks of success for phase one mm I think we have highly engaged patients clinicians health systems researchers and other partners we have a collaborative community supported by robust governance we have analysis ready standardized data with strong privacy protections we have oversight the protects patients supports the timely conduct of research and builds trust and the research enterprise and we have research that has sustainably integrated into care settings and with communities of patients but what were our challenges so again you can choose a lot of different paths and I think that these four things for me basically sum up and you know it was a challenge but in in hindsight now it just gave us more direction I think and so establishing a network that is sustainable that is a sustainable now national resource while incentivizing participation from member networks is a challenge and you’ll see am I discussion about phase two and where we’re going and how we tried to kind of bridge the gap there and I think we’re moving in a much better direction the pp on CD on relationships again I alluded to that a little bit um it wasn’t for lack of trying or a lack of willingness it was certainly a time commitment and just trying to figure out how best to interact and I think as we move forward as you can see p scanner as one of our most collaborative networks in all of the cornet and it’s basically trailblazing for a lot of a lot of other networks and they’re learning from what Lucilla’s done and her network has done to work with PP errands and I think the pyrrans vice verse are also learning from what the CD errands can do for them and I think a third thing was determining the definition and the timing of research readiness I use that we’re before and again I’m speaking to the corner as a whole but we are still trying to figure out what does that mean and when will we be there and then lastly planning for future independence of the quarry and leads back to the first comments about sustainability and making sure that were you know able to have these networks live a long time with without funding from the quarry necessarily so well I said we are in transition we are building a groundbreaking national resource and I’m very proud to work with the with the cornet and the networks involved in it so what does that mean for Phase two so right now just to kind of you know wrap your head around this this is a 250 million dollar infrastructure investment that we have here and we now have as of tomorrow will will be officially in Phase two and we will be added to see dr ends and we added for PPR ends to the mix and we also have our coordinating center which provides technical assistance and logistical assistance they also existed in phase one as well so this is what i was talking about when i said we’d have sort of an new organizational structure as we move forward that’s more about bringing the expertise and the knowledge of the network participants together to make decisions for the network and we have a back or neck Council which essentially will be the new name for our steering committee although there will be a nominating committee within that and that has just been finalized we are currently working on the cornet advisory group Rachel Florence is hard at work determining the members for that group and we’ve always had an executive committee but we will now have engagement data and research committees and under those committees will be subcommittees and work ropes and things that basically it’s our our lead and have participants from the network so excuse me and phase 2 also where do the patients come in because a lot of people look at that organizational structure and say well why don’t you have something specifically for them well it’s because they’re embedded in it everywhere there in the PPR ends and CDR in governance they are part of the pic or net Council and they will have new committees and engagement work groups that fall under that engagement committee so our resources are shifting

a bit and sometimes it’s just transitioning names really but adding a few more bells and whistles so we’re going to have our governance policies they’re brand new there’s one out now but they’ll be more to come our phase one workgroups we termed them work groups but now they’ll be transitioning into subcommittees as I said our phase one resources were basically on central desktop and for anyone who’s used that I’m I would expect that you would cheer for the fact that we are moving in another direction and we’re looking at what’s called a Commons which will hold phase one documents such as the cornet minimum standard data standards previous presentations and webinars and special pprn workspace and contact lists and hopefully will be quite a bit more user friendly so again and phase 2 how are we going to harness this patient power now we’ve been talking about a national data infrastructure or data resource for a while but I think where we’re going is a national patient voice and so I think phase two will accelerate movement from single disease focus to community focus and sharing of knowledge expertise and infrastructure and then participation in key national discussions around research to that point we are leveraging a lot of national efforts pakoras made significant efforts to collaborate as I said with federal agencies working the space ask the FDA NIH ctsas and in particular CDC that’s of collaboration I’m leading and we meet weekly actually as if people are interested again you’ll all have my email at the end you can ask me it’s pretty exciting natural experiments network that we’re putting together with the CDC and the cornet networks again for Phase two obviously we have these natural partners we had them in phase one CTS a sentinel and we’re working faster and harder to align as we move forward into Phase two so again for for me research readiness for Phase two and what I’ll be looking to see that that we’re able to accomplish is implementing are the building blocks that we spent the last 18 months plus this will merge into our three years in Phase two that we’re harnessing the power of patients and participants to transform and support better research and that we are able to open pakorn it up for business so to speak and that we are scaling the capabilities of the network to support large volumes of studies not just these demonstration studies and lastly I usually always like to end with some quotes from our network participants because what you hear is usually my impression of how things are going but I’m a different perspective from those that are in the weeds and it helps me level set when I hear that they to feel the way I do about this network you can see see dr n basically said that it created a sense of collaboration we have a pprn that said it basically truly made it truly possible to study millions of patients another pprn said it helped them learn what it means to be a learning health system and then lastly the creation of our CD RN and participation in procore net has been fundamentally transformative it has sparked conversations surrounding collaborative research at an unprecedented level and engage Health System CEOs senior leadership trustees and ongoing senior in conversations so again there’s my email as I said this is a lot of information to put in 30 minutes or however long that just took me if you ever have any questions you have ideas about research and you don’t know why where it might fit in the quarry you want to work with another network and you know they were funded by / Cory just shoot me an email and if I don’t know the answer all I’ll get you one so that’s a pleasure to be here and thank you everybody oh sure yeah okay so Daniella asked that I add some texture to the natural experiments Network which is the collaboration with the chorion CDC so um basically they are natural experiments so observational

studies about the impact of policy on diabetes and our impact of research or yes I’ll just stop there it gets kind of convoluted because it’s not something that the core is typically used to doing so what we have right now is there are CDC has made their awards there are five from CDC that are funded two of those happens to be the cornet networks which are advanced and a combination of Greater plains collaborative and cap corn which is our Chicago network and pakora had a funding announcement and limited funding announcement open for only those networks that were not funded by CDC could apply to us we will award up to three so we are in that process now so I can’t really actually talk in detail about that but when it’s all said and done and we’re working through what this is going to look like right now I have as I said weekly meetings with CDC to determine how we will collaborate because it’s an initiative with we actually are not co-funded we’re separately funded and so we’ll have to work out the management process and things like that what the advisory groups are going to look like and I’m not sure if anyone or danielle if you’re familiar with next D which was the previous five years that CDC did this sort of same collaboration and they took their lessons learned and basically are shooting into a next five years but we haven’t really landed quite yet on what that’s going to look like so i can’t after that much detail and but again it’s basically centered around diabetes and natural experiments of health policy impact in that area Thank You Marianne for that overview far from someone who comes from the more traditional clinical trials space how would you explain your concept of what a comparative effectiveness trial is which we understand this randomized you know maybe placebo-controlled trial of a versus B or it’s actually two drugs that are being used if its comparative effectiveness and then what transforms that from an NIH grant okay um so I would say oh okay so basically for clinical trials and comparative effectiveness how does how does an nih clinical trial basically look different from say a pic or e version of that same clinical trial and again i’m i don’t i’m not the program officer for our adaptable trial but i can speak a little bit to what our goal was there and basically as i said it’s the patients being involved every step of the way so the actual question that they even asked was vetted by patients and clinicians and stakeholders from the start it wasn’t something that just clinicians wanted to do didn’t come from the top came from the bottom up and then as you work through it and the way that you’re going to intervene in the study they work with patients to figure out what was this what you want to know is this the outcome you’re most concerned about how will this affect you is this are these the types of questions that you feel will be worth responding to will this get through the answers that you need so I think there’s a bit of a it’s hard to converge in those areas when you have as I said sort of top down versus bottom up because the perspectives are so different you don’t necessarily the answer is not going to be the answer on you everybody wants to get but I think it’s their complementary in a sense and I think you could find a way for the types of NIH studies that are out there versus the type of Corey study and find a way to gain insight from both in a way that’s impactful in my mind I I know we just have a couple minutes left and so I’m wondering if you can comment on how you’re seeing we’re hoping there will be synergy between picure net and the CTSA program okay again this is not something I particularly work on but I do know that for contracting purposes and I arbys and things of that nature we are trying to not reinvent the wheel so I think we’re trying to leverage a great deal of the work that’s going on or that’s been going on with the ctsas for the cornet because I think every one of our networks actually has includes a CTSA so we have groups of people within our networks that are already used to doing work in that area and will be will

help to again bridge that gap so that we can leverage that work do you have additional funding beyond the what was allocated to peak or like is their ongoing funding or are you only spending the interest or will you get get more research money in the future um again this is probably one where they they’d pull me off the stage you’re talking about something like this I’ll say that as I said those three feeds you know Philip trust fund that that that help fund our research but to the extent of you know sort of the finance issues and all of that I’m probably not the one to ask but there is a lot of information on our website I would be surprised if it was that detailed but yeah and again I could find someone who could answer that question for you