 So, I'm Holly P.A. I'm trained as a genetic counselor and social scientist and I work predominantly at an advocacy group called Parent Project Muscular Dystrophy. I'm going to tell you a little bit about the community engagement approaches specifically on building relationships within this advocacy milieu. And I will also say that we have a patient report registry and a lot of what I may be talking about are efforts to engage the community through a patient self-report registry because this is a pediatric disease. I'll tell you more about it. And second, it's predominantly caregivers who are actually doing the reporting. And our network is part of PCORnet, which is probably something many of you have heard of and I know some people in the room are involved with as well. So I'm going to tell you a little bit about community engagement, predominantly relationship building predominantly to identifying compelling needs. So I represent a community that has a severe pediatric neuromuscular disease called Duchenne and Becker Muscular Dystrophy. It is a disorder that is progressive and fatal. Most children die in the young adult age. It's of 20 to 30, but we do have a greatly reduced lifespan with children dying in the early elementary years through the teen years as well. It causes progressive muscle weakness and there is no approved treatment. So this is a community where there are an enormous amount of unmet needs and a big part of our relationship building within the advocacy framework is to talk about how we prioritize among these compelling needs and in many cases competing needs because there are limited dollars to go around. Okay. So I'm going to talk a little bit first about what we have found is required for meaningful engagement and I'm going to give you a major caveat. So I talk about patient engagement and citizen science. I think they're related. If we have that Venn diagram, they're related but slightly different and I will also say that listening to these talks makes me realize that I think I'm stuck in like the 80s because the research I'm doing is not using the full range of technology in the ways that have been presented. I'm talking about sort of very traditional kinds of engagement activities. So when we talk about requirements for meaningful engagement from the advocacy perspective, this is a great community to do community engaged research and community engagement in general because advocacy really comes from this idea that every patient and every family member has an important voice to give to the research context and to priority setting. Now I think there are still some very important lessons that are both for the researchers and the clinicians engaging with the community as well as for people who are being called upon to be these experts in their own disease experience. One of them is a real strong focus on identifying research of importance and impact. I think it's very easy for someone who used to do research in the context outside of the advocacy setting to assume that you know what these families need and if there's nothing that these five years of community engagement with this community have taught me it is that really the understanding that clinicians and that researchers have of these families day-to-day lives is very narrow and truly it is very difficult to anticipate their most compelling needs. It's vital that we define the roles and responsibilities upfront. I think a lot of what happens in community engagement is we're sort of feeling our way along and I think we've gotten to the point as a set of you know there's a set of expertise now that really does no longer let's us do this. We can't feel our way through these engagement activities. The roles need to be defined very clearly. The responsibilities need to be to be defined and that really solves a lot of the problems that I've seen in community engagement where the expectations are known and clear upfront. But also you need to integrate support to allow stakeholders to develop what I call a research imagination and this is a phrase that I borrowed from Colleen McBride who used to be the social behavior research branch director at the genome institute. These and patients don't necessarily think like researchers and when you ask them what their research priorities are that's a very difficult ask. You need to allow people to vell up this imagination about how the research process can help them in ways that are meaningful to their day-to-day lives. So we've done a lot of work within our community to help create that research imagination and some of it is the way you frame the questions and ask to them but some of it really is helping people think in a slightly different way about how researchers can help them in a way that has impact. Another requirement is that when we think about engagement and developing these relationships that we focus on the impact of the relationships not the amount of engagement. I feel like people have gotten very caught up in the amount of engagement that happens and that really isn't the meaningful thing. The meaningful thing is the impact on the process. Professional researchers have to be willing to make changes. So this is the other thing I see time and time again is people will come to our community and say we have this protocol sign off on it, give us a rubber stamp and engagement is not a rubber stamp but we all know this intuitively and yet people do it over and over again and so you have to be willing to make changes in your research approach and your research protocol even your very your objective in aims even very high level things if you're going to do true engagement. And then one more requirement is the idea that one patient represents one patient and this is really kind of a tricky issue in the advocacy world. So patient leaders are extremely important and patient advocates can make a huge role but even the most knowledgeable patient advocate represents a small amount of the lived experience and so we have to be careful that we have we don't allow one patient to represent all patients. Another thing that's really important and this has come up already is the idea of motivations and incentives. So it's very important to explore people's motivations going in be transparent about them. People do not have to have the same motivations and they won't. There are different motivations for program initiation. There are different motivations for the collaboration. People have different motivations about what the outcomes are hoped to be and those will vary but they should be realistic. So it's important that everyone in the research team comes in with motivations and expectations that are realistic and it's important that everyone gets something out of the process and that something might be really different but everyone needs an incentive to participate that will be very likely realized. I'm going to just take one moment to talk about the culture of patient advocacy because I think one of the things that happens often is that researchers expect patient advocates and patient citizen scientists to engage in their culture and that really is not a realistic expectation. There are different perceptions about the value of research as a process. Right. So one of the things that is a real challenge is getting patients and patient advocates to appreciate the value of research and sometimes they have very real critiques. This process takes too long. This process takes too much money. This process is not fitting within the way that we're expecting things to happen and often it is related to timeline. So those timelines create a lot of pressure and researchers have to learn how to work within timelines of patient advocacy. For example, a lot of the motivations that we have within our advocacy organizations for our research is we want to influence policymakers and when you want to influence policymakers, you have a very tight timeline to work within. We've already talked about ownership and results dissemination and actually interpretation of those results. And that is very important. And for us, the Holy Grail is getting those things published and for patient advocates, often the outcomes are quite different. So waiting for publication to disseminate your results is not going to fly and you need to figure out middle ground approaches. So I'm going to end with just a few examples of benefits and challenges that we've run into. So one of the major benefits is asking the right questions. So, you know, you can ask, I'll give you an example. So we did a study on long-term well-being of moms and many of the predictors of their well-being were negative. So things like burden, stigma, negative impact. And the family said, no, that's not what we want. Look at things that are positive. Let's look at, they didn't use these terms, of course, but let's look at benefit findings, look at resilience. So we reframed our research and we came up with a really nice research study that really spoke to what they were looking for. Addressing ethically challenging and sensitive topics. This is a great time to use CBPR. This is when it's worth the time and investment. So if you have something very ethically challenging in our community, it's therapeutic misconception. How are we dealing with the therapeutic misconception around clinical trials? In those cases, that stakeholder engagement and using a full-blown CBPR approach was incredibly valuable. And advancing policy deliberations. And I'll just close with a few challenges. So one of the main challenges we have is that no matter how good you are at relationship building, it does not promise that what you do will be acceptable to all community leaders. And that's where that flexibility comes in. And I'll give you one example. We did a study on risk tolerance within treatments. And there was a big component of very vocal community leaders who felt like we were minimizing the impact of their testimony. And so we developed an alternative strategy where we also collected testimony in a systematic way. So you have to be willing to be flexible and responsive to the community. I'll end with one last one, which is the idea of crowdsourcing. So privacy and confidentiality in a rare disease. If you put data out there and you have any sort of identifiers, if you genetic mutation status, you have identified your community. So we have real issues and rare disease with the idea of crowdsourcing, but also the idea of protecting good science. This registry is a community resource and we want to be able to protect the good science that's done there. And so crowdsourcing, honestly, as a genetic counselor, we try to be very self-aware. I'm trying to be very self-aware. I mean, where is this anxiety coming from? But I think truly we have a responsibility to our stakeholders who have contributed data year after year that good science comes out of it. So I will end there and I'm looking forward to some more discussion about these issues.