Miscellaneous
Huntington's disease is a rare genetic condition that impacts over 30,000 people in the U S and 200,000 worldwide. It is a neurological disorder that impacts movement mood and the thinking process. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. On this episode of our podcast, we ask patient advocate BJ Viau, co-founder of the Huntington's Disease Youth Organization, to join us in a conversation about patient advocacy on People Always, Patients Sometimes. Janet Kennedy: (00:35) Hi, my name is Janet Kennedy, and I am your host for People Always, Patients Sometimes, a production of Spencer Health Solutions. I'm really looking forward to a conversation today with BJ Viau. BJ is passionate about positively changing the lives of people impacted by rare conditions. He has a history of working to support patient advocacy and rare disease non-profits by holding grassroots fundraisers. BJ also helped create an international nonprofit for Huntington's disease, the HD Youth Organization, or HDO. He has an MBA and has been working in the pharmaceutical industry for over a decade. Currently he's the Director of Patient Advocacy at Horizon Therapeutics. Welcome to People Always, Patients Sometimes BJ. BJ Viau: (01:20) Hey Janet, thanks for having me. Excited to be here, look forward to a conversation. Janet Kennedy: (01:24) Absolutely. Well, I think one of the things I'm most interested in learning about right now is what is Huntington's disease and how does it impact young people? BJ Viau: (01:35) Yeah, I mean, it could be a 20 hour answer, but essentially broken down, Huntington's is a rare genetic disorder impacting about 40,000 individuals across the United States. It's a neurological disorder that unfortunately takes over one's ability to do anything and everything in life. Most commonly described the combination between more common conditions like ALS, Alzheimer's, and Parkinson's all wrapped into one. How it affects young people is typically symptom onset starts for individuals later in life, probably in their thirties or forties, although it can vary. Although that's not technically a young person, where it starts to affect the young person is it's their parents. So you start to see decline in your parents, they lose their jobs, they lose their ability to function. Young people end up having to be caregivers for their parents. And what some might say the worst part about Huntington's is its autosomal dominant genetics, so every child of a parent who has Huntington's has a 50% chance of also getting Huntington's disease later in their own lives. So it's a little bit of looking in the mirror and wondering, am I going to have this future of my parents, or am I going to have a future of no Huntington's? And that's a really emotional decision that young people have to make. Janet Kennedy: (02:50) Now you mentioned it's genetic. So does that mean you can take a little test and find out, or do you have the sword of Damocles hanging your head your whole life? BJ Viau: (03:00) Yeah, take a little test. It sounds very simple and easy. Essentially it is. It's a blood test or saliva test that will provide you pretty much, if you will have Huntington's or if you will not have Huntington's. But even though it's simple, it's got huge social, emotional, psychological kind of bucket that comes with such a simple task. Janet Kennedy: (03:22) I'm curious about the fact that you're focusing on young people. Is this to help prepare them for a future of thinking through how does Huntington's going to affect them, or is this actually helping them deal with their parents? BJ Viau: (03:38) Yeah, a little bit of both. 15 years ago, when we were kind of dreaming up the Huntington's Disease Youth Organization A few of us young people were attending conferences and all the sessions and all the meetings were really focused for our parents, and there wasn't much support or education for us young people. In fact, a lot of people said, "Well, we don't even want to talk to young people about it because they shouldn't even be in the conversation." They were afraid to bring young people into this conversation, and that's where we thought that's not the case. We need to learn. We need to get educated. We need support. Not just to understand how to deal with our parents today that are going through Huntington's, but how we can start to shape and plan for our own future, which may or may not have Huntington's in it. So how do you prepare for school? How do you prepare for a job? How do you prepare for having kids, getting married, buying a house? Those are all really tough decisions that young people in Huntington families have to make very different from kind of the the normal person. Janet Kennedy: (04:35) Absolutely. And I think it begs the question - do young people consider that they have to make a lot of different decisions than other people because their issue is genetic in nature? BJ Viau: (04:48) I think they do. A lot of it relies on when the parents tell the child that Huntington's is in the family. Unfortunately in some families it's still a hidden secret and parents hold that secret from their children until the last possible day. And then, you know, the kids end up finding out some improper unique way. So what HDO's model is all about, not just helping the kids, but also helping the parents have that conversation with their kid to say, "Hey, you know, mom or dad has Huntington's. And that unfortunately means you may get Huntington's one day, but it's okay. You know there's people out there who can support you. We can get education, we can do this together." And it's creating that safe environment to have safe conversations that we truly believe at HDO is really best for everybody. Janet Kennedy: (05:36) On your website, you actually have a section for kids. Is there an age that is too young to learn about this, or does that actually take away a lot of the veiled mystery or concern by just making it part of a natural conversation at a very young age? BJ Viau: (05:54) It's a tough question. You're going to get a lot of different answers on who you talk to. I mean, I can speak from my personal experience. My parents told my sister and I, when we were about eight, nine, ten years old. And I would say them sharing that information with us. So young helped shape conversations that happened over many years to help my sister and I have the knowledge to create power for our own lives and feel motivated and empowered to understand what we're facing. I know a lot of other young people whose parents treated it similarly, and I see them these days dealing with HD in a very positive manner. I've seen a lot of families do the opposite. They hold it till the last possible minute until their kids are out of college, have their careers in front of them, are about to have a kid, and then they drop what I would call the HD bomb on them. And I've seen it go really poorly. So I would love for there to be a true research paper on when is the right time to talk to young people and tell them about Huntington's we don't have any statistical data to show when the right time is, but it would be great to provide that kind of statistical evidence to the parents who are unfortunately gonna face this decision with their children in the coming future. Janet Kennedy: (07:09) That sounds like something the NIH ought to fund, because it isn't just hard conversations about HD that parents need to have. There are innumerable number of conditions or diseases that are genetic, that have a serious connotation. Even things that aren't genetic - it might be considered a disease that isn't quite visible - and at what point do you share with your children a serious condition that they need? BJ Viau: (07:33) Totally. Yeah. It always seems like probably in the parent's perspective that tomorrow is a better day than today, but looking back, yesterday was always the best day. But that's just really difficult. It's tough. We're not trained in school, to have those conversations or trained as professionals. So it's a tough situation, but yeah, that'd be a great research study to get funded by somebody. Janet Kennedy: (07:55) Alright. Well, you've planted a seed there. We'll see where we go with this. Well, let me ask you a personal question, If you don't mind. I know that you are a new dad. How did that conversation go with your then fiance? BJ Viau: (08:08) Well, I'm pretty open and honest about my involvement with Huntington's. I am one of the few. There's actually a statistic that does show that about 90% of the people who are at risk for Huntington's choose not to get tested because there's, unfortunately, there's not much you can do about it as far as medication treatment wise today. So there's only about 10% who say, "Hey, I'm ready to know, even though I can't necessarily do something about it medically." But I, when I got tested, I learned that I would not get Huntington's in my future, which would eliminate the ability for my wife and I to pass Huntington's on to any of our children in the future. So it was a little bit of an easier conversation to have with my wife versus many decisions or many conversations that a lot other HD impacted family members have to have with a spouse. But anyway, her understanding of my involvement was very positive. So she got HD. She understood it. And I do think even if I had HD, we would have made a decision to have a kid, same way we did. Janet Kennedy: (09:10) Okay. I'm a little bit shocked here. And I, I want you to reiterate - you said that nine out of ten people do not take a test to confirm whether they're going to actually experience Huntington's or not? BJ Viau: (09:21) Yeah. Before they have symptoms. I mean, it's a tough question. I think if you ask the general public, would you want to know if you're going to get Huntington's disease or not? I think the general public you'd probably get the opposite. You'd probably get nine out of ten, who would say, "Heck yeah, I'd want to know." But when you live in a family with HD and you understand HD, you don't want to know. That that's a life changer. That's a game changer. That that is incredible weight on your shoulders, knowing that you will one get HD and not everybody's ready for that. So it is a true stat. I hope it will change in the future because there'll be something that people can do about it. We're still a long ways from truly a disease modifying treatment. But I do believe that when we have some more progress in the medical field that everyone's going to want to know if they will one day get HD, because there's something they can do about it. Janet Kennedy: (10:14) Absolutely. Well let's talk a little bit about a big disappointment that came to the HD community over the past year, and that was that not just one, but two studies were canceled regarding treatments for Huntington's. How did that go over in the community, and is there still hope out there for other types of interventions? BJ Viau: (10:36) Janet, as you know, as you've talked with a lot of other people on this podcast about trials, it's an experiment. There's a hypothesis that people have that if you do X at Y time at this amount, it will do this, and we think that if it will do this, it will slow a disease down or decrease symptoms. One of them was a phase three trial. It was the largest trial ever run in the Huntington's community with over 800 people around the world. So we were hopeful. We put a lot of our marbles into this bucket, hoping it would lead the way. It was supposed to go until next year. And kind of out of the blue, it was a little bit of a gut punch to the HD community. Not just because it wasn't the results we wanted, but also because we didn't really, as a community, understand that this was even an option to get stopped halfway through for the reason it got stopped. BJ Viau: (11:28) So I think if we would've known a little bit more as a community, it may not have been as much of a gut punch. It still would have hurt, it still would have stung, but it just came out of nowhere. That was kind of part one, and then part two was just the trial with another company for totally separate reasons - no correlation between the two - their trial showed that there was no significant efficacy in their medicine doing what it was trying to do. So two in a week - that hurts. Is there still hope? Yeah, there's a ton of hope, and we still have a lot of companies invested in the community whose programs are built fully enrolling and moving forward. But it's sets us back. For those who needed that treatment today, some of them may be out of luck and that hurts. That hurts a lot of us. It was tough. It'll be tough for awhile. Janet Kennedy: (12:18) Where does the HD community go to find support? BJ Viau: (12:23) Yeah, I think everybody has their own ways of finding support. This is Huntington's, or this is just any rare condition. There are patient advocacy groups, a lot of people reach out and have connections with each other through those advocacy groups. A lot of people go to their physician, their healthcare professionals to find support, and they want the most up-to-date relevant medical information and they want to hear it from a licensed practitioner. And I think others turn to social media. They turn to Facebook, private Facebook groups to find support and education from those that are going through the condition or going through the battle themselves. And I'm sure everybody else has other ways too. But I would say those are probably the three most common ways of finding support for the HD community, but in general, probably anywhere disease. Janet Kennedy: (13:08) I'd like to talk a little bit about patient advocacy. You have been involved in patient advocate programs, whether it was fundraising or helping to found HDO for a long, long time, and you also interestingly work in the pharmaceutical industry. So you have a unique view on the inside of how complicated it is to set up and run clinical trials, to test out drugs and things of that sort. A lot of patients don't have that vision, and a lot of patients probably have never even heard of patient advocacy. So if you were talking to someone who is maybe new to a disease, new to a condition, newly diagnosed, how would you explain to them what a patient advocate is and how they can be their own patient advocate? BJ Viau: (14:03) I don't think that's a one-answer-fits-all. Everyone's situation is so different. So there's different forms of patient advocacy. I would say there is being your own patient advocate, which is advocating for yourself, for your child, or your loved one, your mom, dad, whomever. And that can be done in many ways. That can be done in the doctor's office, advocating and communicating what's going on and what may be needed with the physician. It could be I think of advocating regulatory-wise, going to your governmental folks and advocating for change based on rules and regulations that aren't what's best for your personal condition. And then I think you could also then quantify or qualify that there's professional patient advocacy, which is also a large bucket, but that can be working as a professional. Your paid job, either for a patient advocacy organization, typically nonprofit organizations in the US that have 501c3 designation, or you start to see it and not just pharmaceutical companies, but healthcare companies in general have folks who are titled as patient advocates internally, which can have a lot of different hats to themselves. But I guess long-winded answer: the word patient advocacy Isn't a very clear definition, but it's definitely either advocating for yourself or for others based on the health condition that they're battling. Janet Kennedy: (15:26) What about your work as a professional patient advocate and the other ones that you have met from other companies? Is there a particular focus that you're in, for instance, if there are meetings, are you sitting in there saying yes, but from the patient's perspective, or is a little bit broader than that? BJ Viau: (15:47) Yeah, I think that's part of it. I mean, companies are learning the more we can partner with the patient communities - the earlier the possible - I just think it's best for everybody. It's best for the company in my mind, because they're going to hopefully create a product that's most meaningful for the patient community, and it's beneficial for the patient community because they're getting a product that they really want and they really need, and hopefully it makes a big difference. So I think there's just so much overlap and benefit of working together. And I would say as a professional in the pharmaceutical patient advocacy space, the more that that role can work with patients and caregivers and advocates and loved ones and patient advocacy groups, and make sure that their voices heard and make sure that their needs are heard, that their problems are heard, that their abilities are heard, the better off in the long run for everybody. Janet Kennedy: (16:47) I sense that there's a little bit of a sea change with the impact of the Corona virus on the clinical trial community that a year and a few months ago, pretty much pharma companies, sponsors, CROs sort of set the procedure and the protocol, and this is how it works. And the inability and the now lack of desire of patients to actually come to an office has really spurred decentralized clinical trials, has spurned virtual trials and hybrid trials. And I think has really put the patient front and center. You cannot have clinical trials without patients, and as desperate as they may have been in the past, they have also stepped up and said, I have to consider my own health. From your perspective, as both a patient advocate, a professional patient advocate, and someone who has experienced the pharmaceutical industry - what are your thoughts on what the past year has done from the patient's perspective? BJ Viau: (17:54) I think we, as just a general community, trying to bring new medicines to market that fit an unmet need -we were going in that direction. COVID maybe sped it up a little bit and maybe made folks change the way they think a little quicker than what they were hoping for. This is all my personal opinion. I really, as a patient advocate, just transparently, I've never participated in an actual clinical trial. I've participated in observational trials. And in my professional space, I have worked more on the commercial side with products that have already been approved. So don't have a ton of clinical experience, but I just think in general, we should have been always, and we should always, and we should always in the future always be asking the question "What's best for the people that this medicine is meant for, and how do we get it approved and find out if it's truly providing value with that person in mind?" So if that's the centralizing clinical trials, is that what's best for the patient and the advocate and the loved one that are participating? Is that best for the clinicians that are collecting the data? Is it going to provide as much data is it needs to be provided remotely? Can it still be the same positive influence? I don't know. that's not my expertise, but those are the questions I think we should be having. Janet Kennedy: (19:18) Well, let's talk about going beyond the pill. It's really becoming much more a conversation about the full patient experience. And I have to wonder if somebody just writing a script is enough. Do you think that the pharmaceutical industry should actually be taking a bigger role in a patient's full experience with their disease state, whether it's providing educational videos or support tools or digital health tools to help them manage and monitor? Is there a need to do more than just provide a medication? And really what I'm asking is if you could wave the magic wand and tell pharmaceutical companies, "Hey, you really need to be thinking outside this pill box." What would you actually recommend and suggest that companies think about as they want to work with patient communities? BJ Viau: (20:16) I mean, in general, especially in the rare disease community, which I know best, I think pharmaceutical companies actually do a really good job trying to provide information, education, resources, and programs beyond the pill. There are a lot of legal and regulatory firewalls that unfortunately doesn't make it as easy as we would love it to have. But I would say in general, the rare disease companies know that if they're going to get into this space, that's part of it. Part of it is education beyond the medicine - disease education. I would say they can always do better. Not everybody is perfect. It's definitely creating those with the patient or the patient advocacy group at its forefront; making sure that the materials they are creating are relevant and actually needed. But I would also say it's a job as a patient advocacy organizations, the nonprofit businesses, and the physician to step up their game as well, because pharmaceutical companies don't always have a great way of getting the materials out to the patients and families that need it. BJ Viau: (21:17) And the advocacy groups or the physicians are the ones that typically have the better reach with families. So instead of just prescribing a medicine, can a physician prescribe that a patient goes to XYZ website, or prescribes that this patient participates in a camp or an education day put on by an advocacy group. I always had this idea that I wanted to create the Huntington's Disease Youth Organization to create a script pad, and we would send it to doctors and the doctor would actually give out a script and the script would be, "Hey, go to HDO's website and look at all their resources." And it's not just, I'm here to write a medicine, it's I'm here as a physician or healthcare provider to provide you with the best resources to help you navigate your condition. So we can all do better for sure. But hopefully we're headed in the right way. Janet Kennedy: (22:09) I agree. I think one of the things that we do face is that there are so many pieces of information. There are so many conditions and disease states. There's so much information out there. How do we help the physician? Now, if you're a specialist, that's fine. This is the area you're focusing on. But when I think about some of the folks that are maybe the first to diagnose, which is our internists, our family physicians, to try to find out everything there is to know about everything is really tough. And I think that's why organizations like HDO are so essential. That you can get them started on their journey, but patients really need to learn how to do smart research online and get to the authoritative critical sites that can really provide them, that kind of interaction. BJ Viau: (23:00) I'm with you. And again, I'm just brainstorming right here, but do hospital systems or physician's offices, like, are they thinking maybe we need a patient advocate? Who can work in our office to help find and then disseminate the information out there, especially for rare diseases? I, as a doctor, as a nurse - I may not have the time to spend an extra 15 minutes on Google looking this up for one patient or two patients who I see in my practice. But is it worthy of an investment to find somebody else who can do that? Who can truly help that physician's office be the best resource for each family who walks in the door, regardless if it's a condition they see regularly or conditions, and they might see, you know, once in a lifetime? Janet Kennedy: (23:44) Oh I love that. And I know it's come up in other conversations I've had on podcasts about the idea that, in essence, you need like a librarian. You need someone who is a health strategist or a health care manager who can then take that conversation further and really help get the patient off on the right foot, as opposed to hearing any kind of devastating news. I know for a fact as a college educated professional, once we get past the second data point, I'm not hearing a thing you're saying. It'd be really awesome if I felt like I could call someone at the office to say, "Okay, help me out here. How do I get started?" BJ Viau: (24:26) Exactly. Yeah, how can you connect me with somebody else I can talk to in the community that, beyond my physician's visit, once every six months, where do I turn? How do I find that group of people who understand me and understand my needs, who can help me along that journey? So anyway, there's a lot of technology that's improving and a lot of online communities that are forming, which I think are great, truly just helping all people involved, become engaged if they want to be engaged. I truly believe an engaged person is someone who's going to be more successful when battling their condition. Janet Kennedy: (25:00) Absolutely. Excellent words to the wise there, BJ. It was a great pleasure having you on the podcast. And I really look forward to learning more about HD and supporting the community and getting the word out about HDO. And I hope you have an absolutely wonderful time as a new dad. BJ Viau: (25:22) Thanks, Janet. Yeah, a lot of fun, but appreciate the opportunity to come on and speak, and yeah, I look forward to staying in touch and continuing to listen to the rest of your podcast, guests who have a great knowledge around the continuous hurdles we come up with, but it's great to see the conversation try to come up with a solution. So thank you. Janet Kennedy: (25:40) You're welcome. Hey, thank you for downloading this episode of People Always, Patients Sometimes podcast. If you've enjoyed our conversation, a review and rating on iTunes will help us find more listeners. This podcast is a production of Spencer Health Solutions.