Unlocking Success in Rare Disease Trials

Rare disease trials come with unique challenges, and independent research sites are well-equipped to tackle them. Hamish Baird and Jennifer Botte of Remington-Davis discuss the role of independent sites in rare disease trials and their logistical and operational challenges when executing complex studies. Tune in to hear about effective strategies for patient recruitment, site-sponsor collaboration, and the benefits of flexible, patient-focused approaches in rare disease research.

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Jimmy Bechtel 0:02

Welcome to SCRS Talks presented by the Society for Clinical Research Sites. Thank you for joining us as we explore the latest insights, trends, and innovations shaping clinical research today. I'm Jimmy Bechtel, the Vice President of Site Engagement with the society. Today, I'm joined by Hamish Baird, the President and CEO of Remington-Davis, as well as Jennifer Botte, the Director of Clinical Operations with Remington-Davis. We're going to be talking today about rare disease trials and how independent sites make their way into these studies and tackle them and bring the best they can to executing these clinical studies. I'd love to start off, Hamish, with you, a little bit of an introduction of yourself, and then we'll go to you as well, Jennifer, and learn a little bit more about you.

Hamish Baird 0:48

Thank you Jimmy. I'm excited to be here. My name is Hamish Baird. As you mentioned, I'm the President and CEO of Remington-Davis. I've been here for almost 28 years. It'll be 28 years in October. I started as a study coordinator, did that for about six years, moved into project management and operations. And then in 2018, I had an opportunity to purchase the company. And since that time, I have been the CEO here. And just a little bit of background about our company. We were founded 32 years ago, in 1992, we're actually sort of founded by accident within hospital. There was an infectious disease physician that was doing HIV trials out of his sort of private practice that was located in the hospital. But he was also doing antibiotic research like nosocomial pneumonia, skin and soft tissue infection studies through the emergency room and even some sepsis studies in the ICU. And his office manager was both the regulatory person and his study coordinator as well as the office manager for his practice and they did a good job. They started getting more and more studies. They were eventually offered acute respiratory distress syndrome study and the infectious disease physician physician was not interested. So they went across the hall to a pulmonology practice. And then they had 2 investigators, hired a regulatory personnel. And that's when they realized that they'd been doing this for a couple of years, and that they needed to to incorporate now that they actually had dedicated employees to the research unit. I joined in 1996, and I did actually the very first outpatient study for our company. It was a Zantac versus Pepsid study and we enrolled 180 patients in a couple of months and that sort of gave us the impetus to sort of launch an outpatient division which I headed up over the years.

Jimmy Bechtel 2:45

Thanks, Hamish. Jennifer, a little bit more about you.

Jennifer Botte 2:48

Hi, my name is Jennifer Botte. I am the Director of Clinical Operations at Remington-Davis in Columbus, Ohio. I started here at Remington-Davis 23 years ago as an inpatient study coordinator managing infectious disease and critical care trials at one of the local hospitals. And then over the years as our company grew and our shape changed, I took on the role of Clinical Director and I'm also now a co-owner of the company.

Jimmy Bechtel 3:18

Awesome. Well, thank you both. It's great again to have you here. It's really cool to hear, you know, 20 years in the industry. Not every organization out there can say that, but maybe Hamish start us off with sharing some of the most, maybe challenging or logistically complex trials that you guys have been involved in. And again, based on our focus today, particularly some of those in the rare disease space.

Hamish Baird 3:42

Absolutely. So, you know, being an inpatient company to begin within our career, we had to work with a lot of different departments within the hospital. And so that becomes logistically challenging as far as getting everybody on board and making sure that you're following the protocol and within windows and that type of thing. So, just from our very founding, we were always sort of problem solvers and the great collaborators to try to get everybody to work together for this common mission. And some of the studies that we did that were exceptionally complicated and challenging. We did the vent study. That was actually a device study. It was one way valves that were placed in the lungs of people who had COPD and emphysema so badly that they were a candidate for lung volume reduction surgery. And this was actually to take the place of that and for example, we had to get the operating department on board. We had to get the pulmonologist on board. We had to get you know, the, the entire, basically the entire hospital, but we also had to send our investigators to I believe it was Boston. And they had to do trainings where they actually were placing these valves in the lungs of sheep and pigs before they could, you know, be be cleared to actually do the procedure. So that's an example of a very, you know, very logistically and medically complex trial. You know, since then, we've also done quite a few rare disease trials that have been very, very complicated. The most recent one is in EPP, erythropoietic polyporphorism. And the trick with that one is, is that there are a couple of clinics that specialize in that, but they can be difficult to get to. And so we were actually approached for this study We've actually done three of these studies now with the same sponsor. We were approached primarily because of our geographic location. It was easy to get to us in Columbus, Ohio, from a large swath of the nation. And so this was an example where you know, the sponsor could help us by advertising. We engaged with the porphyria registry service and advocacy group. And what they did was help us to spread the awareness. And then we would field the calls and reach out to the patients. We worked with a travel agent to bring the patients here. And some of the advantages of that is it gave the sponsor, rather than selecting 20 or 30 sites that all maybe gave one or two patients, we became the Midwestern hub. And then that way, we had the experience where we were actually randomized 22 patients in the trial. And I think that there's, you know, obviously there's a cost advantage to only opening one site versus, you know, perhaps, 11 sites to deliver the same number of patients, but it also offers the ability for our physicians and our staff to sort of become the experts of both the disease, but also the standard operating procedures of the protocol in the, in the way that the study was run. And I think I'll probably kick it over to Jen to discuss that more, but also other other examples of some rare diseases that we've been involved in.

Jennifer Botte 6:48

Yeah, so to Hamish's point, the EPP study there are big medical centers that in academia that everybody knows MassGen and UTMB that are sort of experts on managing this condition, but because of their size and the fact that they are academic centers, and there's unfortunately a lot of layers of bureaucracy that exist in those environments sometimes they're not able to do things as quickly as sponsors would like. And so by utilizing a combination of smaller, more nimble sites alongside these larger academic centers or perhaps even networks. I think a sponsor can get the best of both worlds. And also serve a more diverse population of patients because the patients that perhaps live in Boston and are going to Mass Gen are not the same patients that are coming to Columbus, Ohio to be enrolled into a trial. So they can kind of achieve two goals simultaneously.

Jimmy Bechtel 7:49

That's great. You guys, it's that really unique way of thinking. And some of those unique approaches that you guys had mentioned and talked about brought to the table for that clinical trial that I'm sure has been the impetus for much of the success of your organization, which is really, really great to hear. And I know we're going to talk a little bit more about some of the specifics of those. So, hamish, what do you believe sponsors then should prioritize when selecting sites for a trial, having been in the game for a while and participating in what sounds like simple, you know, sort of phase four studies all the way through to these very complicated to clinical trials for rare diseases. And then how do those, I guess, getting against specific here, those priorities change when we talk about those rare and complex trials.

Hamish Baird 8:32

Absolutely. So obviously all sponsors want to find sites and research centers that have experience in the indication, have a track record of success, have quality data, are very responsive to their startup timelines and recruitment timelines. And those are sort of things that you see across the entire industry. But there's also some things, especially as they relate to rare diseases. one thing that's very important, I think, especially when you're bringing people in from far away you have to be respectful of how difficult it is to actually participate. If you're asking somebody to travel multiple hours to a site the customer service becomes becomes a huge issue. And so if you're open on the weekends, if you're open in the evenings you have to be responsive. You have to get them their travel plans quickly so that they can take time off work. Some of these things are areas where having a small independent site, we can be very responsive to that and we can be flexible and change even our own operating hours to make sure that we are successful. And we don't have patients conforming to us, we can conform to the patient's needs. And I think that that's gone a long way to our success in recruiting for, and retention for a lot of these EPP studies. Do you have anything to add, Jen?

Jennifer Botte 9:51

Yeah, I was gonna say, because we're not practicing medicine, we are conducting research, we can treat each patient that comes through the door as someone who's come here by choice and work to make their experience as easy and simple as possible. But when you're practicing medicine there's a different mentality, I think, that happens in a lot of clinical practices. The patient needs the doctor more than the doctor needs the patient. And when you're focused solely on research, the researcher needs the patient more than the patient needs the researcher. And I think that shift in that dynamic really leads us to be as patient centric as we possibly can which also speaks to our high retention rates and our patient satisfaction rates. We have had lots of patients that have come back to us, you know, maybe they participated in a trial and years could have passed until there was another study that met their needs or that they're eligible for. But we do end up with a lot of repeat business and a lot of patients who refer their friends and their family because of the experiences they've had with us.

Jimmy Bechtel 10:58

That's incredible, right? And it's like you said, it's that mentality shift and it's that culture that you build and you instill in the organization to have that mentality and that focus. And I can imagine, especially in rare disease trials, that patient centric approach and really making them feel that they're the most important thing to what you're doing. And this process is Even more important because we're talking about, oftentimes we're talking about conditions here that don't necessarily even have treatments or the treatment that they are on is the only treatment available. And maybe it's not as effective for them. We see this all the time with rare diseases. So it's great to hear that that priority on patient centricity and patient care is something that's at the forefront of what you guys do at Remington-Davis. Other considerations then when sponsors are deciding between maybe an independent trial site versus maybe a larger network, or are there a particular, I guess, scenarios or trials that might be more effective for independent research sites versus larger organizations, the IRO, the SMO type of models.

Jennifer Botte 12:09

Part of it depends upon the tests and procedures that need to be done within that protocol and whether or not they're very specialized in nature. As an example, we did a purple artery disease trial, which is not by any means a rare disease. However, the sponsor had a very rare requirement for a very specific type of MRI. And so in order to overcome that logistical hurdle, they partnered with a local medical center that could provide that service. And then they made that the vendor for any site within 200 miles. So they eliminated one of those logistical hurdles on our behalf. Other sponsors for rare disease trials, obviously, geography is the most blaring obstacle because for example, with EPP, there's approximately 4,000 to 6,000 people within the United States that have this disease. So obviously, geography is a big issue. And so the sponsors worked with us to eliminate that hurdle by having basically travel concierge service that will book their hotel, book their car service, book their flights, all of those things to make it easier for them to get to us. And then once they reach us, they're not going to a giant megaplex of buildings and parking garages and tunnels you know, our office is, right off the main road and is fairly simple to find, you know, parking is free and plentiful. And those kinds of things also speak to the patient experience.

Jimmy Bechtel 13:48

That's great. Hamish, anything to add there? It sounds pretty comprehensive, but...

Hamish Baird 13:53

It does. Good job, Jen. I think it just depends on, you know, not all rare diseases are created equal. So, you know, there are some centers of excellence for particular rare diseases. And I think that, obviously, they make sense to make them one of the sites but sometimes you don't want to put all your eggs in one basket. And you need to have a couple of other sites where, they can maybe pick up the slack or if it's too difficult to get to, as Jen pointed out with the geography you can sort of spread out and make little hubs throughout the United States, or people can sort of congregate to those those different sites. And I think that when you go with a large network, they can certainly accomplish that. But you also get a lot of consistency from smaller sites, including the flexibility and the sort of standardization that we provide.

Jimmy Bechtel 14:42

It's excellent. Thank you, both. I want to start to conclude our conversation today with a more lofty strategic vision type of question. So actually, Jennifer, I'd go to you on this one, kind of running the operations for the organization. What strategies have you found most effective in fostering that collaboration that we talk about all the time between sites and sponsors or other industry partners, particularly again, in those logistically complex rare disease studies that might require a little bit extra, a little bit of extra love and attention?

Jennifer Botte 15:15

So I think that really communication is key and you have to provide your feedback and then sort of evaluate how the person that you're giving it to is receiving it and decide whether maybe you need to take it to the next level or not. I think we've all had the experience of working with somebody who, is hearing us but not listening to us. And I think that that you need to make sure that if you have feedback to give, especially if you feel strongly that it is making or breaking a study that you're making sure that the person that you're giving that feedback to is actually listening. And then I think we also have to do something that, when Christine would be at summit, she would always tell us we weren't allowed to which we had to come with solutions. Well, everybody. Everybody's solution is always amend the protocol, but maybe really we need to look a little deeper than that. Maybe we don't actually need to always amend the protocol. Maybe it's something simpler. Maybe it's, you know, can we do, can some of these things be telehealth? Can some of these things be done in a different way? Maybe our advertising campaign is using verbiage or pictures or words that are too confusing or intimidating. Maybe we need, maybe the sponsor needs to develop a tool to help simplify the consent form or the protocol to put it in a more simplistic, humanistic sort of light. So sometimes asking for those extra tools, making adjustments to tools that already exist, things like that can help to, you know, simplify the process and and can work out better for all of us.

Hamish Baird 16:58

Yes, I agree. I would say that when you collaborate with sponsors on rare diseases, it really truly does need to be a collaboration. There can't be any type of adversarial relationship with the sponsor because they need to hear us. We need to hear them. And as Jen's point, you know, Certainly, they need to take the inclusion, exclusion criteria into consideration when there's such a small pool of patients that they don't want to unnecessarily exclude patients from a study, but even more important is making sure that logistically, patients can get here and it's not too much of a burden on them. You know, you can't have very small visit windows. You have to have contingency plans like Jen had mentioned, maybe have a home health agency go and get blood draws, especially if you do a blood draw for a visit and it comes back and there's an abnormality and all of a sudden you're going to ask them to get back on an airplane or drive several hours to come here just for an additional blood draw. So, those types of things if you can plan it out at the beginning of the study and anticipate where where these bottlenecks and choke points are going to be and and provide a mechanism to relieve that there's a long way to eliminating protocol deviations you know, improving retention so patients just don't get fed up and drop out. And then, of course, you have to have an infrastructure at the site that, as I mentioned, has a high level of customer service. And the patients want frequent communication, especially if they need to take time off of work and travel here and they have kids and dogs and that type of thing. So those are some of the things that I think can really make or break a trial probably even more so than the actual entry criteria.

Jimmy Bechtel 18:35

Well, that's, that's incredible guys. Really, really great to hear so much focus on collaboration and different ways in which we can more effectively collaborate together. And again, it sounds like these things are just part of what you guys do, right? They're just ingrained into the culture and the operations of the business, which is great to hear. And I think our site listeners hopefully can take a few areas of focus away, but that's definitely one that I take away is we talk about these things all the time, but, you know, building them into the culture, into the way that your business operates as part of how we see long term success. So thank you both for your time here today. Thank you for the interview and the discussions. It was great to learn more about you guys and how you're approaching executing these trials in a very unique way. So Hamish, thank you. Jennifer, thank you.

Jennifer Botte 19:21

Thank you, Jimmy.

Hamish Baird 19:22

Yes, thank you very much. This was very fun and I enjoyed speaking with you and thank you for giving us the opportunity.

Jimmy Bechtel 19:28

Absolutely. Thanks again, guys. As we conclude for those that are listening, don't forget to explore other site focused resources made available to you like the various publications and webinars on our website, myscrs. org. You'll also find a wealth of content and details about upcoming engagement opportunities, like our Site Solutions Summits being held throughout the year. Thanks again for listening and until next time.