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Live from SCOPE 2024: Talk of the Towne featuring Rare Patient Voice

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We’re here with Antidote’s tenth installment of Talk of the Towne, and it’s a very special one! We recently attended SCOPE 2024, where we met up with our partners at Rare Patient Voice and recorded our first Talk of the Towne episode live and in person. 

Our host, Rich Towne, had a great conversation with Pam Cusick, the Senior Vice President at Rare Patient Voice. They had the opportunity to chat about the mission of Rare Patient Voice, how the organization helps patients and their loved ones, and the advancements their work has made in research and representation. We’re also thrilled that this episode is coming out just in time for Rare Disease Day, which takes place on February 29th. Be sure to give this special episode a listen and read the transcript below!

Rich Towne:
Welcome to Talk of the Towne, the podcast from Antidote that bridges a gap between clinical research and conversation. I’m Rich Towne, a trained pharmacist and the senior clinical informatics manager at Antidote. Today I’m thrilled to welcome Pam Cusick, the senior Vice President of Rare Patient Voice, one of the members of Antidote’s Partner Network. Pam Cusick is an experienced research professional with more than 30 years of expertise in study design, implementation and analysis. Her background in public health communications and research, coupled with her passion for patient advocacy, dovetail with Rare Patient’s voice mission and visit. As senior vice president, Pam’s focus is on the continued growth and success of RPV with an eye on client services, business development and oversight of patient outreach, panel management and marketing. Pam, welcome to Talk of the Towne.

Pam Cusick:
Thanks so much for having me.

Rich Towne:
So in the United States, a rare disease is defined as any disease, disorder, illness or condition affecting fewer than 200,000 people. There are currently over 7,000 rare diseases identified and it is estimated that 30 million people are living with one of these conditions in the United States. Because they’re unfamiliar to many people, rare diseases can evade detection for years. However, through research, awareness and organizations such as Rare Patient Voice, more individuals and medical professionals are becoming aware of these conditions. One key component of this is Rare Disease Day, which falls on February 29th this year and serves to raise awareness and garner support for individuals and their loved ones who are impacted by rare condition.

Rare Patient Voice, which recently celebrated its 10th anniversary, currently covers more than 1,500 diseases and conditions across nine countries. They have paid patients and family caregivers over $13 million for participating in research and work with thousands of organizations, patient advocacy groups and individuals to spread the word. Today I’m so excited to chat with Pam about her work at Rare Patient Voice with a special focus on recent research breakthroughs, clinical trials, and the importance of awareness when discussing these conditions.

So in the past 10 years, Rare Patient Voice has touched the lives of countless individuals living with a rare condition as well as their caregivers. Are there any specific highlights that stand out for you in the past decade?

Pam Cusick:
There’s so many interesting things that we have connected patients to. So we started out as a market research company really connecting patients with interviews and focus groups and surveys so that they can give their input about products being developed and input on things coming on the market. And so over time we’ve expanded to clinical research, medical affairs, usability studies where patients get to test devices. A cool thing that patients participated in and a couple of years ago was a company that was looking to create accessible clothing. And so they wanted to have patients test the clothing with people with mobility issues. So we connected them with them and they sent clothing to the people and they got to test them out and then give their input about what worked and what didn’t. And then the company ended up developing that line.

So that’s sort of where we started and over time there’s been more and more interest in clinical trials and obviously everyone knows here at Scope, the funnel and that it’s hard to find patients. The interesting thing with the way that we connect with patients is we go to events. So we’ll go to the National Hemophilia Foundation Conference or Sickle Cell Conference and meet patients and connect with them and explain what research is and then they decide if they want to join us and we connect them with studies. So we actually have met a lot of these patients in person, connected with them at events, and then they spread the word, they share with their communities and their families and friends.

And so over time we’ve built up to 145,000 patients and caregivers, which is pretty amazing given we’ve only been around for 10 years. And I know that some of the companies here have millions of patients that they’re connected with through EHRs and so on, but not necessarily people who signed up because they want to share their voice and share their experience. So that’s kind of a neat thing about us because we’re a little bit different in the way we connect with patients.

Rich Towne:
So do you feel like the interest was always there for rare patients to connect with clinical trials, they just didn’t feel like they had a voice to do so?

Pam Cusick:
I think actually people were not very familiar with research in general. So when we go to these events and explain what people can be involved in, one of the common things that I hear is, “Why would anyone want to talk to me? Why would they want to hear what I have to say about a product or a service?” Which blows my mind because they’re the experts in what they do. They live with their condition every day. They know more than most people about what they go through. So their input on a project or service is really important.

So I think the lack of familiarity with research in general is one thing. I think COVID, with all its bad things, introduced people a little more to the fact that there are clinical trials and people started to realize that was something that they could be involved in. But it’s still not something everybody knows about or knows how to be involved. We did a study a couple years ago internally and it was about how people want to learn about clinical trials and they want to hear about it from their doctor. Well, if they’re in a small rural town in, pick a state, and their doctor isn’t on the research academic side of things, they might not ever find out about a trial that’s available to them. And it being up to them, they’re already worried about dealing with their disease, finding that is kind of hard to do on their own.

So I think it is definitely an awareness thing. And for rare patients, first of all, it takes them on average like 10 years to get diagnosed with their condition, so they wouldn’t even know what trial to look for because they don’t know what they have for many, many years. And then of course with there being so few people, there’s not always a trial available either.

Rich Towne:
So you actually just took one of my questions with the difficulty to get a diagnosis, maybe even difficulty to get a physician. How does that also play into the access to trials that are for newly diagnosed patients, how do you get someone who might suspect they have a condition living out in the rural country? Is there any way that Rare Patient Voice helps these patients get a diagnosis or reach out to the appropriate medical care?

Pam Cusick:
So we don’t do that. We connect them with opportunities to participate in research, but none of us have any kind of a medical background, so we wouldn’t want to necessarily do that. We do have a lot of resources through advocacy groups that we are partnered with. So we have an internal patient advocacy team and they’re connected with 4,800 referral partners around the world that help us to identify and connect with more patients who want to participate in research. And some of those organizations also are either disease specific awareness organizations, some of them may be focused on clinical trials, and those organizations also help those patient groups to find research that they can participate in. For us, it’s more of being like a matchmaker and connecting them with those opportunities. And then when they might have questions, they can go to our trusted partners page or they can go to, they might find something on our Facebook page that a partner shares, but we don’t want to mistakenly tell somebody something that we’re not really qualified to do.

Rich Towne:
So what kind of challenges do rare patients face with clinical trial recruitment? Let’s say there is a match, are there any things that consistently cause an obstacle? And are some study types more challenging?

Pam Cusick:
For the patients that I’ve talked to, the logistics are mostly a barrier. So finding a trial, because they’re going to be rare, that’s in your neighborhood is not going to be very likely. So you’re going to have to travel there. If you live very far away, you’re going to have to fly, drive. You may have kids, you have to figure out what to do with those kids. Or those kids are in the trial. And so there are a lot of logistical challenges and I think there are some companies out there that help with that. But not all sponsors do that. They don’t necessarily always take advantage of those services. But there are companies that help with travel, that help with, they’ll do payments, they’ll coordinate your babysitter. I mean they actually help. So that helps with take that one burden off of the parent or off of the individual who wants to participate because it’s really hard for them maybe to actually find that one trial and may only have one site and that’s difficult.

Rich Towne:
When you say that, what I’m thinking is study design as well, like the number of visits that these patients have to commit to, are there ever trials where patients might have to stay in an area for an extended period of time, especially with some of these more rare diseases?

Pam Cusick:
Sometimes. And that’s again where those types of organizations can come in and help. They can identify housing for them. I know that there are people who have set up townhouses for a whole family to stay in a place for a month or so so that their child could be in a trial and it makes life a little bit easier for everyone in the family. But again, it’s the logistics that make it difficult.

Rich Towne:
Do you feel like there’s a rise in rare disease trials recently? Do you feel like there’s enough incentivization to sponsors to help these patients that maybe only 5,000 in the United States or even the world have a condition?

Pam Cusick:
I think that that rare patients are becoming a force to be reckoned with and they’ve kind of banded together in larger groups. We are all rare, so rare disease day, everyone’s rare. And in that way they’ve brought attention to the n-of-1 trial and that just because their child has something that only five other children in the world might have doesn’t make them less valuable. And I think that people are hearing that and sponsors are hearing that and making an effort. Sometimes products that are created can be used at another time for some other disease or condition. So there are good reasons for sponsors to create those or to do those trials so that will be available, not only for that one child or that one family, but for everyone else who may be diagnosed.

Rich Towne:
Rare Patient Voice supports the rare disease community year round, but things tend to ramp up around this time of year as Rare Disease Day approaches. What is the impact you’ve seen from Rare Disease Day as a whole and what are you most excited about for Rare Disease Day this year?

Pam Cusick:
We love going to Rare Disease Day at NIH. It’s really fun because we get to see everyone in one place that we don’t always get to see. A lot of advocates. A lot of the patients who participate with us will come into town too because there are a lot of events around Rare Disease Day too. So it’s not just that one event, but there are things on Capitol Hill, there are all sorts of things going on. So that’s a really fun thing for us, seeing more patients, more advocates and meeting new people. A lot of times it’s new families who are coming into the area. They may have just received a diagnosis fairly recently and are joining this community and it’s nice for what we do, we’re providing them an opportunity to participate in something that they might not even know about and get paid for their time. They earn $120 an hour to participate in things like focus groups and surveys and so on. And that’s kind of a nice thing for them.

The other thing we hear about that is that it’s almost therapeutic in some ways because they get to share with a moderator or researcher all about what they’ve gone through. And those people really want to hear every detail. They want to know what your journey was like. And sometimes people don’t want to share that with their family. They’ve already told their family one or 10 times and don’t want to burden them with some of the details. And being able to participate and share their experience, they’re not doing it for the money, they’re doing it to help people coming after them. So maybe that shortens someone else’s diagnosis, maybe that helps someone else to avoid a pitfall. And so we hear that a lot from patients that they’re really doing it because they really want to help other people.

Rich Towne:
And that altruism is just incredible when there’s no treatment, when there’s no symptomology, it probably is incredible for these people to come. And is there anything specific during this year’s Rare Disease Day that is maybe different than previous years or a specific forum or moderator that you’re specifically excited for?

Pam Cusick:
We like to go and listen to all the different [inaudible 00:13:54]. It’s hard to pick one or something particularly exciting. One of the things that they often have are they’ll have some art exhibits that some kids are presenting their artwork or other paintings and things, and that’s always neat to see. And then certainly any legislative actions and changes that are being reported, because they often share them at these events, that would make things easier for patients to get treatment diagnosis to help them to be identified more quickly. Those are things that are really important.

One of the biggest things recently, it was for Krabbe disease being added to the newborn screening testing. For some of these conditions, if you can find and identify them early, there might be something you can do. I’m not sure that’s true with every condition, but if you can find that there might be an intervention. And if it’s not on the newborn screening, you may not know for years that your child has a particular condition. And then the impacts might already be felt. So that’s super important. And I think those are the kinds of things that I like to hear about.

One of the years there was talk about the four people who are tube feeding to get their supplements covered by their insurance. And there are all kinds of things, and I don’t know if they are or not right now, but I think it’s interesting to hear about those things and learn about advances because when you’re just doing your day-to-day, you don’t hear about those things and it’s kind of neat to see it all at one time.

Rich Towne:
And that’s an angle I’ve actually never thought about before is the legislative angle, especially for patients, whereas more prevalent conditions, those might be established, there might be a whole structure  and hierarchy. I mean something like this step system in Medicare where you have to try one drug before the other and it’s very laid out. How do these legislative actions usually start, especially for patient populations that are very small? Is there anything that, let’s say you even mentioned it before, people that are trying to help the journey for people that come after them, is there any advice or ideas for patients that might want to see something like a disease being added to the newborn screening list?

Pam Cusick:
So a lot of times working with their disease specific advocacy group is one way. There’s also the rare disease legislative advocates, that’s what they focus on all the time, getting things passed through and improving the lives of people with rare diseases. There are other organizations like NORD and Global Genes which focus on different things, but they also focus on trying to move things forward in rare disease areas. But I think it’s banding together with a group makes you stronger. So finding your people, whether it’s your disease specific area, whether it’s the rare disease community in general, and finding out how did this one organization get their condition added to the newborn screening registry, what did they do and what are the steps they followed so that maybe you could use that with your organization or your disease.

Rich Towne:
So with Rare Patient Voice do you often see a lot of cross disease kind of assistance where different patients with rare disease band together?

Pam Cusick:
Well, so at Rare Disease Day, there’ll be people from the National Organization of Rare Disorders and Global Genes, as I said, RDLA, all these groups, those are kind of collective, larger umbrella organizations that help rare disease patients. So in that way, I think they become a larger community. As opposed to the groups that make up those larger organizations, they may have very small numbers, their child may be one of three in the world that’s diagnosed with this one thing. So coming together and working with a larger advocacy group could be very helpful.

Rich Towne:
And that brings me to kind of shift the topic here because it just brought this to my attention. So one of the biggest trends right now in clinical trials and extremely important is DEI. How does that affect the rare disease community where there’s already so few patients with the disease and a minority within a minority might be even more difficult to engage and get to trust research and participate in it. Is there anything that Rare Patient Voice has observed or done with the DEI considerations?

Pam Cusick:
For us, it is the way that we meet patients and connect with them. So prior to COVID, we would go out to events, we went to 300 and some odd events in a year. And we still go to events now, but not 300 and some odd. And some of those were in very rural areas, some were in urban areas. And we were able to connect with people of different races, ethnicities, genders. Gender is the hardest one for us because we have way more women who have signed up with us than men. They like to do research more than men. Different age groups that we would meet at these different events.

So I think that we’ve done a good job by being kind of boots on the ground and meeting people in person and being good faith actors in the research community, people then trust us and then share with their own communities. So where we might meet a group in Houston that’s a Hispanic community, those people then, once they participate with us and they realize, wait, they’re legitimate. They’re connecting you with good opportunity, then they share with their own community. So we are able to find more people who are diverse through those methods.

I think too, and this is my own part of DEI that I’m most interested in is disabilities. So people with disabilities make up 25% of the world population, which is amazing. They’re apparently one of the most diverse groups. And within our community, most people have, not everybody, but have some sort of either visible or invisible disability. So outside of their ethnicity and gender, they are quite diverse in their abilities. And so what I believe is that not enough people with disabilities are included in research. And in clinical research, that is true too. Talking to one colleague, she was telling me of her experience bringing her, I believe 17, 18-year-old child to the doctor’s office. He was in a wheelchair, couldn’t pick him up to put him on the table and there was no table that went up and down. So I mean, why would you want to bring your child to participate in a trial where it wouldn’t accommodate them?

Rich Towne:
And do you feel like that’s something that sponsors or sites have done anything with or do you think there’s a more systemic change needed in protocol development, in site selection?

Pam Cusick:
So at another conference I was at last year, there were a couple sessions including people with disabilities and making sure that the sites were… It’s not enough just to have a ramp. You can’t just have a ramp. There’s a lot more to it. And so just checking the box is not enough. You have to make sure that is it appropriate for people with hearing loss? Is it appropriate for people with low vision or blind people? There’s so many more things to consider. And so if you think of somebody with Crohn’s disease or an autoimmune condition that could flare up, they might not want to travel across the country three times a year for whatever the protocol requires. Could you do something in their local area? Could you bring it to their house?

There was another woman who I talked to with, she had, Fabry, was very involved in this community and she wanted to make sure that she was in a trial because of course it’s genetic and impacts her family. She did it for 12 years and she traveled back and forth either several times a year, at least once a year back and forth to this one site where they drew blood and took a health history. She had to fly. They could have done that anywhere, but no one even thought about it. And having Fabry is exhausting. So those sorts of things where you’re looking at, yes, this is something that you want to do to improve, treat, cure this one condition, but you have to think of the whole person and how are they going to get there and how can you make it easier for them to want to participate? Because everything that they had her do in that short day and a half, it was exhausting. So she had to ask to kind of take a break. But her care team probably should have known about that or thought about that. If they’re working on the disease, it’s really important that they think of those things because they’re not just subjects. They’re people and they have real challenges and it’s important to consider that.

So to your question, I think it’s becoming something that people think more about. And I think if you do and you think about that and you really, really think about the human being who is part of that, I think more people will then stay in a trial. They’ll enroll and they’ll stay in a trial because it suits their lifestyle, it makes it easier for them to do. And I think that will lead to more success.

Rich Towne:
So with that, one of the other trends that we’re seeing a lot at Scope and in the clinical trial industry at large is decentralized trials. How has decentralized trials and the approaches used within it, how has that specifically impacted the rare patient community? And are there any particular advances that you’re excited in?

Pam Cusick:
I think for one thing, it will enable more people to participate because there are so few. I mean, rare is rare. So when you have a small number of people who could participate anyway who might have trouble getting to that site, going to them makes it much more likely that you’ll be able to fill your trial. And then to that woman that I mentioned before, if someone went to her house to gather her blood and her health history, it would’ve made her life so much better.

So I mean I think it really just comes back to what I said before, which is thinking about the human element, think about that person and those are the, when you’re looking at a decentralized trial, what are those things that can make somebody’s life a little easier would make them want to join a trial and participate? I tried to join as a healthy subject one time and I was going to have to spend the night at NIH. Why? I am not sure that it was needed and I didn’t end up enrolling in that. And I didn’t live that far away and I wouldn’t do it.

Rich Towne:
I wouldn’t either.

Pam Cusick:
Yeah. So I mean you have to really think about that and I think that decentralized means that it’s available to more people. So people in rural areas, if you have somebody who speaks another language, find the care team that speaks that language and bring it to them. It just makes so much sense. We can do it, we all found a way to work remotely when we had to. It’s a good thing.

Rich Towne:
So are there any other ways that you feel like the human element… I really like the way of putting that, just remembering the whole person. Are there any other ways, we’ve talked about travel, we’ve talked about lack of accessibility for disabled patients, is there anything else that specifically stands out? you even brought up your experience as a healthy volunteer, are there any things that you feel like are a trend that just consistently stay in place and maybe don’t consider the whole person or maybe are just, for lack of a better word, archaic in a way?

Pam Cusick:
Well, I think trial design without involving patients early on in a process, that would be the archaic part. Because patients getting input about, I mean, for lack of a better words, what a patient can bear, what will they tolerate? can they come to my house? Or do I have to go to the site? How many days do I have to stay there and how can we make that easy? I think involving patients early on in the process is becoming more common, but not everyone does it. But it is a really important element because getting that input, it will make it easier for other patients in that disease community to want to participate.

Rich Towne:
I think that’s something that we’ve definitely heard. And I remember that there was a panel that I listened to with the FDA saying that they would more heavily consider patient input, and potentially even trying to, not favor in some way, but support sponsors that were including this sort of step into their protocol design. But on the topic of protocol design, one of the challenges we see with a lot of other therapeutic areas is that the protocol is looking for an unrealistic population. The stat in one webinar I heard was that the average patient fails at least three criteria in a trial and that 30% of all patients are eligible for a trial, while rare is 70% of patients will receive the drug. Are there any specific challenges that you’ve seen with protocol development in terms of strictness or maybe unnecessary criteria or even study design?

Pam Cusick:
I think, I mean I’m not a doctor or a lab scientist, so some of the things that might seem like they’re a little extra, they may be required and I don’t pretend to know those things necessarily. But I do think that sometimes, and we find this in other research too, that they want a pure patient. So you don’t have anything else. You haven’t been treated by anything else. There’s so many of those things that if you haven’t been diagnosed for 10 years with something, you will inevitably have had some other medication, you’ll have had some other treatment. And now that you have that thing, you know what the indication is, now you’re not eligible for a trial, you’ve been treated by all of these other things.

And again, there’s a scientific reason for that. So I’m not saying it’s not important, but I think it makes it so much harder for the rare patient because they’ve been diagnosed with multiple other things before they finally get their real diagnosis. And if there is a trial, they’re probably not, but they may not be eligible just because they’ve been treated with other things already or they have multiple other diagnoses because now other organs have failed and other things have happened over time when they haven’t gotten a treatment. So now they have too many other indications and they don’t qualify. So it’s a hard thing. I think it’s part of the fact that the journey to diagnosis is so long that there are all these complicating factors that evolve over time.

Rich Towne:
One of the things that we’ve talked about and we’ve heard is expanding the cohorts of a study to just enable more people to at least participate, even if that data is not used for that population finally, allowing people to be a treatment experience cohort or a comorbidities cohort. I don’t know if you’re familiar with any of that talk, but is there anything that you would suggest sponsors or sites that have a rare patient come in, they’re ineligible for a trial, is there anything that these sponsors or sites can do?

Pam Cusick:
It is a really hard question. I mean there’s so much disappointment when they don’t qualify. I was talking to somebody else here, and when they finally find the trial and they are found ineligible, it’s so disappointing. So giving them something else that they can do. Again, they could participate in research with us as an example. Other types of things that a focus group, interview, survey where they’re still providing their input, they’re maybe not getting a treatment, but it makes them feel like they’re able to contribute. Because it’s a really hard thing being told you’re not good enough but you don’t fit. And then thanks so much, see you later. So giving them some other sort of thing to do is a good thing. I don’t know about the comorbidity trials and whether if I’m going to go multiple times a year to do something and you’re not going to use my data, I don’t know if I want to do that.

Pam Cusick:
But I think giving people a choice is always a good thing and those are important things. But again, back to the human, you have to ask them. What do the patients want? Do they want to be in a trial where they’re just there in the background? Or maybe they do and I’m not to judge that.

Rich Towne:
Are there any common misconceptions that rare disease patients have about clinical trials that maybe a sponsor or a site might not be aware of? And maybe it’s just patients at large, misconceptions about what participation or taking a study drug might be?

Pam Cusick:
To me, I think because people are not as aware of clinical trials, they don’t necessarily know. They come back to, well, I don’t want to be a lab rat. That’s one common one.

Pam Cusick:
And that they are just wasting their time because are they going to get the placebo? that’s not a misconception, but helping people understand what the whole process is and making it less scary because it’s not something that everybody really understands. And I think that not just rare disease patients, but patients in general don’t really know what a trial involves. And so they make assumptions like everybody does about everything. If you don’t know about it, you make assumptions. So maybe there’s more general education about what a trial is and what it means to participate and what if you do get the placebo and all of those things to sort of help people to realize that they’re not a lab rat, but it is that they’re participating in something bigger and that can be very helpful.

Rich Towne:
And one of the pieces of advice that we often hear is that often as part of these trials, there is a top quality medical team that will be monitoring the patient and will be doing assessments and making sure that their current care is up to code, for lack of a better word. But on the opposite side, we briefly talked about it already, how about misconceptions that you see with sponsors and sites that you might’ve talked to about the rare disease population and how patients, maybe there’s a misconception about the misconception. I was going to say is there anything you’ve heard specifically, maybe questions that seem to indicate a lack of understanding about how best to reach these people?

Pam Cusick:
Well, I mean that kind of goes back to what I had mentioned about that study we did internally a couple of years ago, patients trust their doctor. And so how do you reach them? Well through their doctor because they’re not necessarily going on ClinicalTrials.gov. Some people do. Or some people do go to an academic medical center and somebody will tell them about a trial. But for most of us who are not maybe in a high density area, you go to your family physician and that’s who you trust. And so finding a way to get that information to physicians who are not necessarily the study team or on the study team is a good thing. They’ll lose their patient to the study team for some period of time too, which can sometimes be a concern for people. But in general, I think most people would want to, if they knew that there was a way to treat or help their patient, they would do that.

So I think maybe it’s getting that information out to physicians at large. I don’t know if everyone goes to conferences where they can hear about all this information and share it with their patients. But if you have a patient with a rare disease and you’re able to find a trial for them, but you’re not, again, at an academic medical center or a site, how do you know?

Rich Towne:
And that’s something that I’ve never thought of before in the context of this population is losing the patient with rare diseases since they have specialists that maybe the only physician, maybe there’s only two physicians and it’s going from physician A to physician B. Is that a common cause of concern for these rare disease physicians where maybe they don’t want their patient to participate and potentially lose track of their care?

Pam Cusick:
It’s a unique question. I do think it’s a hard thing to answer because we’re all human, right? And you want to know that if I’ve diagnosed patient A and I’ve been working with them for the past five or 10 years and they’re stable and steady and then somebody else comes in and says, “I can do something for them,” do I know or trust that person? I don’t know. I mean, I don’t know from that perspective.

Rich Towne:
This is just me throwing out an idea, so full disclaimer, do you think there’s more opportunity for sponsors to reach out to physicians to participate as investigators in trials? So if there are a rare group of physicians or specialists, allowing that physician, maybe allowing them to dispense the study drug or procedure and observe their own patient in their own care?

Pam Cusick:
I would think that that would probably be a good thing, especially for the patient who then trusts that physician. So it makes it nice from both sides that the physician who’s been following them is then also the treating physician. In the eighties or nineties, people wanted to learn about that from their doctor. And that’s a big deal because again, if you’re in a rural area or you live in a community where there aren’t a lot of clinical trials going on, you might never know.

Rich Towne:
So one of the things that I always ask guests, and we might go down a tangent here, is I do the three pieces of advice. So there’s three big parties that we talk about often with clinical trials, patient is always number one, but there’s also the sponsor and the site. So we should start with the most important of all. If there is a rare disease patient that connects with Rare Patient Voice and they’re interested in clinical trials and listening to this podcast thinking this is all great, but what piece of advice would you give this person that may be on the fence, not quite sure what to do, but is interested in clinical research?

Pam Cusick:
I would say find out more information. Talk to your doctor, talk to your advocacy group. Go to, in the case of rare disease, look for those larger umbrella organizations and try to understand what’s available to you and what the perspective is on clinical research as a care option? There’s so many ways that it can be beneficial to people. There’s a lot of information online, but you don’t always know what’s online. So I would say go to a trusted organization and your physician and find out what’s available and your physician should be able to hopefully direct you to a resource of some sort. But then again, there are those larger rare disease organizations that might be able to help as well.

Rich Towne:
So the next one is the sponsor. We’ve talked briefly about ways that maybe they can do a little bit better, but if let’s say a sponsor, maybe even one here at Scope is listening to this podcast and they’re thinking, I would love to help the rare disease population. I want to work to make this experience better. Beyond just developing drugs, is there any piece of advice or guidance if a sponsor asks you, how can I best help this population?

Pam Cusick:
I would say, get more information out about clinical trials from patient friendly resources. So things that can get into the hands of patients, get them out to their physicians, get them out to the advocacy groups, because there’s an element of trust that people really need. They’re putting their lives in your hands. And so information is power. And so the more you can share with patients from the sponsors so that they don’t think that you’re a big evil company trying to do bad things, that you really are trying to make a difference in the lives of patients and show that, I think it’ll make it easier for patients to want to enroll.

Rich Towne:
And finally, one of the big focuses here at Scope with all these sponsors, patient advocacy groups and organizations, is the site that actually does the screening. And I think they’re often an under considered population. I think often it’s the patient and the sponsor. But what piece of advice, let’s say there’s an investigator out there who maybe owns a small clinical research site, or even maybe they’re a physician at a large research university and they’re listening to this podcast thinking, I didn’t think of a lot of these things. What piece of advice would you give them for interacting and working with these rare disease patients at their site or place of work?

Pam Cusick:
Make sure that you make it a welcoming, accessible site for those patients. A lot of the people in the rare disease community have, as I said, visible, invisible disabilities that are not always accommodated for. And I think a site that accommodates for that, have a table that goes up and down where somebody does not have to try to lift their a hundred and some odd pound child onto it. It’s an important thing and you need to make it easy for them. It might cost a little more to make your site accessible, but in the end, those people want to come back.

Rich Towne:
This has been just a really insightful interview. I thought about a lot of things that I haven’t considered, especially, I have to be honest, that aren’t really concerns with the larger patient groups that I think just get brushed over because people don’t consider them. And I really hope for anyone listening to this that they take all the pieces of advice you just gave them and implement them.

So one of the final things that I like to do as well is where would you point anyone that listens to this podcast? Is there any resources specifically for patient sponsored sites that you’d really want them to go to after this call? I know obviously the Rare Patient’s Voice’s website. Is there anything else outside of that?

Pam Cusick:
Gosh, I mean there’s so many. Support Rare Disease Day. Go to the NCATS NIH website and see what’s going on in the rare disease community, what legislation is changing and where there might be an opportunity for you to engage with rare patients because they oftentimes have had a really hard journey getting to where they are, so they need a little extra love.

Rich Towne:
All right. Well this has been awesome, Pam. Thank you so much for joining Talk of The Towne.

Pam Cusick:
Absolutely.

Rich Towne:
And it was a pleasure having you.

Pam Cusick:
I’m happy to be here. Thank you so much for the opportunity.