Trials with Maya Z

Maya TrialHub (00:01.928)
Hello everyone. This is trials with Maya Z and Maya and I'm here with Ella Balasa a patient advocate that I recently met thanks to my awesome network and community of patient advocates. I didn't know that the patient advocates are so well connected and so so helping each other and praising each other, which is really awesome. Ella, welcome. Welcome to trials with Maya Z and I

would love to hear more about yourself.

Ella (00:34.033)
Yeah, thanks, Maya, for having me and thanks for reaching out and making this connection. It's been a pleasure to get to know you over the last couple of weeks. my name is Ella Balasa I am a patient advocate based in the US who has cystic fibrosis, which is a rare genetic lung disease.

Maya TrialHub (00:53.676)
Mm.

Ella (00:55.065)
It is a multi organ affecting disease, primarily affects the lungs and it causes a buildup of thick mucus that leads to ultimately lung deterioration.

And over the course of my life, I've had to receive a lot of countless intravenous antibiotic courses, many hospitalizations, and through these experiences have really learned the value of what it means to self advocate for myself in the healthcare setting. And now more recently, how to be a voice for not just myself, but also on behalf of the rare disease and more broadly patient communities globally. And so

I now advocate to bring patient voices throughout drug development as early in the process as possible so that patient perspectives and voices are heard and are considered when developing treatments and solutions to improve health in people's lives.

Maya TrialHub (01:57.592)
Thank you, Ella. When we met first, you mentioned that you are consulting different companies on how they are to strategize their clinical trials, how they're going to make them more aligned with patients. I wonder, what is your experience with advising these companies? Have you been doing only directly to sponsors, but also working with CROs? Is there any difference? And what's the overall experience? Can you share with us?

Ella (02:24.391)
So I have a wide array of experience. Initially, I started working with CF -specific nonprofit groups, but that quickly evolved. So initially, it was just helping to create patient -facing materials, sit on a number of advisory boards where the patient perspective is considered in...

Maya TrialHub (02:28.428)
you

Maya TrialHub (02:37.142)
Hmm.

Ella (02:50.371)
in what areas to fund research in and what are some considerations about clinical trial participation from a patient perspective. And this included a number of focus groups and such. But as that has evolved and as I've gained more experience about and interacting with the healthcare industry as a whole, I've now taken on roles as a consultant independently with

A number of startup companies as well who are delivering solutions for the management of a clinical trial from like the patient -facing softwares, things that help, you know, where patients can do patient -reported outcome measures on the apps and things. So,

Maya TrialHub (03:21.153)
Yeah.

Ella (03:42.501)
I've been involved in a number of projects on that front. I've also worked with some CROs and some smaller biotech companies and helping them to build relationships with certain patient populations. Specifically in the rare disease arena, that's where I have most experience, but helping to strategize ways that they can connect better with patient communities, how they can build that kind of that

Maya TrialHub (03:59.831)
Yeah.

Ella (04:11.301)
rapport or not trust and how important that is. And then, you know, I say that's been the extent I haven't worked too closely with larger pharmaceutical companies as you know, they have a lot of their own internal patient advocacy and patient engagement teams where I think individual patient voices are only brought in for small projects here and there, not really being

Maya TrialHub (04:37.398)
Interesting.

Ella (04:40.855)
fully integrated into the work of the company. In some of these smaller companies, I've had opportunities to help build out patient advisory boards where they're systematically

Maya TrialHub (04:55.426)
nice.

Ella (04:58.041)
on bringing in these perspectives to understand how they can create a better product, create better solutions for patients and helping to bring in those voices from diverse disease areas and diverse backgrounds so that there is really a breadth of understanding from how various different patients experience healthcare and how they may be able to utilize a certain platform or a certain treatment. So that's been the example.

Maya TrialHub (05:28.166)
I have a question. You mentioned that you're advocating and consulting these companies for how important it is to understand the patients, to engage them, like to build these advisory boards. Why is it important, Ella? Like, can you summarize? I know it's probably a long topic, but when you're advocating for that, what are these big things that make it so important? What will happen, let's say, if they don't do that?

Ella (05:55.035)
Sure. Well, I think that they're truly, and I frame this in this way quite a bit, is that it truly is a mutual benefit. It's a benefit for patients to have this type of involvement and be research partners, you could say. Because ultimately we are, as a whole, gaining more interface and involvement with how

treatments are created, what goes on kind of behind the scenes? We get more of that sitting at the table, right, you could say experience of being involved in drug development and being involved in how treatments come to us. So one, it's valuable in that it helps patients be more educated and be more involved and

help to be better advocates for themselves, but it also helps to bring about patient, it helps to create better patient experiences as a whole. Because when we share what works, what doesn't, what could be made better, and how that can be done, then companies can take that information

and hopefully utilize it to make the process better, to make a treatment that's better, to make a clinical trial that's easier to access and more inclusive of diverse populations and utilizes innovations and technologies that help us manage our disease better. Right. And so

Maya TrialHub (07:26.722)
Hmm.

Maya TrialHub (07:34.814)
They're meaningful. Yeah. They're meaningful. don't spend just like yet another bucket of money into something that doesn't make sense for the patients.

Ella (07:44.483)
Exactly. And then, you know, on the other side, it helps, you know, I said it's a mutual benefit. So it helps companies in that they're then not spending as, hopefully not as much money in a product or treatment that isn't going to be that effective for patients that isn't going to have that much uptake that is going to be hard to enroll if there are so many requirements of a patient in a trial.

Maya TrialHub (08:01.368)
Mmm.

Ella (08:09.741)
And that's just going to ultimately slow down innovation and creating new solutions for treatment for diseases. And so it's really a benefit on both sides to have patients involved at the table.

Maya TrialHub (08:24.91)
Well, I have a question because last time we spoke, you also mentioned that you have quite good connections with the European landscape of like patient advocates. I'm going to ask you about like the differences between the United States and the European landscape. But before that, I was more interested by communicating with these other patients outside of the United States. How different is the patient journey?

Ella (08:35.387)
Thank

Maya TrialHub (08:53.782)
in the different countries compared to in the United States? Is it very different or like the rare disease is more of the same thing?

Ella (09:03.197)
Well, you know, I think obviously not having lived outside the US, can't exactly speak to it, but having a number of friends in European countries who have cystic fibrosis as well, you know, one thing that I will say, which is we are very fortunate in the US is...

generally speaking, having that early access to novel therapeutics and treatments that unfortunately, you know, in other parts of the world, that doesn't come as quickly. And so that, you know, just putting that out there is a really great privilege and is amazing to have. I also think that in the US, there is a lot of focus on, there is an increasing focus on patient, the need for

evaluating and considering patient perspectives and asking patients what works and what doesn't and how it can be made better. So really this shift in patients, this focus on patient centricity really I think has initially stemmed out of the US. It's definitely moving into Europe and other parts of the world too. there is companies are considering what patients needs are and how they can best support that.

But when it comes to the patient advocacy organizations, think there's a lot, patient advocacy organizations in the US have a lot more, can have a lot more reach, can have a lot more involvement with the creation of new treatments and therapeutics. There's a lot of fundraising that goes into a lot of these organizations, whereas in Europe,

significantly much more lacking. They don't have those funding and those resources. They're not getting those kind of investments. They're not really being able to have any sort of pull or pull and push with what kind of

Ella (11:04.475)
innovations should be researched and having that voice of the patient and where there might be, know, there's no money to put forth. Here in the US, a lot of patient organizations can put that money forth to specific investors or to companies to research various areas.

Maya TrialHub (11:22.638)
Where does this difference come from, For example, why patient advocacy groups in, and sometimes they're not only advocacy, but they're a huge research foundations. Why do they have the money and in Europe, they don't have the money? Do you have any observations or not?

Ella (11:41.373)
You know, I'm not certain, but I just don't know if it's the culture that hasn't evolved like that. That's just not been the way that it's been set up or the way that organizations and businesses have been run or whether it's not really thought to be, previously, there hasn't been thought to be a value in that. I'm not entirely sure, but I think given that,

Maya TrialHub (11:45.976)
Mm -hmm.

Ella (12:08.665)
you know, here in the US, there's this opportunity for a lot of different organizations, a lot of different kinds of companies to kind of take hold and, you know, find and have those connections. I guess I'm not quite sure. I don't really have an answer. You know, it's actually something that I'm interested to understand more about too.

Maya TrialHub (12:27.009)
Yeah.

Maya TrialHub (12:32.578)
Yeah, well, I'll definitely make sure to interview more people on their European, like I actually already did from a different lens, but like you and I would discuss that we both know you EUPATI as the patients Academy in Europe actually is very interesting because I never found this like a similar institution in the United States where you have an institution that is like funded by let's say like

Ella (12:37.958)
Yeah.

Ella (12:44.401)
Who's who?

Maya TrialHub (13:01.312)
and so on and so forth that actually prepares patients to become patient advocates. It trains them, it certifies them, it's really great. That's what EUPATI does. But at the same time, like you said, in Europe, one of the hurdles that I've seen is that there is not an easy way to become a patient advocate professionally. I mean, that there are not that many opportunities like you mentioned. And I wonder, is it because companies think that when they ask the US

patients, that's just enough to understand the patient journey of these patients. And then whenever they go for global clinical trials in Europe, it should be the same. Do you think that's the case?

Ella (13:42.279)
mean, that could very well be. Yeah, I think that maybe these companies, because they're primarily stemming, a lot of these pharmaceuticals are stemming from the US, are based in the US. so I think that, yeah, maybe they think that they can just gain the perspectives of the community and the population here, and then that would be representative of the...

Maya TrialHub (13:50.071)
Yes.

Ella (14:02.637)
the population globally, which is very much not the case. As we know that patients have varying and vastly different experiences, obviously even to just based on the differences in the healthcare system as a whole and how that's set up and the way that they access treatments and ensure access to care and all of those sorts. So yeah, think that yes, patient advocates have much more opportunities to have these

Maya TrialHub (14:05.887)
and

Ella (14:31.799)
these direct connections to pharmaceutical companies and organizations and are able to share their voices and perspectives and, importantly, be compensated for it, which is, I think, really not something that is understood or really considered at all for Europe and the rest of the world.

Maya TrialHub (14:51.512)
Hmm.

Ella (14:55.771)
And I know that is is changing, right? There is more consideration to it, but that's still not really it's I think patients are just expected to want to be altruistic and kind of give back or, you know, help the broader community, which I truly that's honestly where my how my involvement stem from my passion. And it still does. I want to help other people who experience this to have

less hurdles and less barriers when it comes to getting care and to living the best quality of life that they can. But at the same time, when you become, I think, to a level that I have where it's a level of professionalism that you're not going to find in the general population, think that's where it requires, you know, there needs to be a fair measure of compensation for that.

Maya TrialHub (15:50.734)
compensation. Yeah, and at the end of the day, Ella, in order to become so professional and be helpful to the companies, you have to spend a pretty good amount of time on trying, perfecting, reading. It's like a, it's a mini university education. Maybe it's not even a mini, if you ask me, because for years I do have a friend with cystic fibrosis as well. She went through a lung transplantation and like...

Ella (16:01.405)
time.

Maya TrialHub (16:19.34)
Yeah, that helped her and like she's now healthy, but I know for how many years she and her family struggled. So it's like not mini education, but it's like several lives of education because you literally every single minute of your life you're going through that. And then once you also get connected to other people from your community, then you learn about exactly the fact that the access to standard of care access is very different. People are going through different

experiences and so on and so forth. So at the end of the day, also you have to pay your bills. Like everyone should pay their bills. So if you want to continue doing this work professionally, then you need to have like some sort of a compensation so that at the end of the day, you can continue doing that. Otherwise you can't sustain that.

Ella (16:56.198)
Right.

Ella (17:10.803)
That's right. And hefty bills at that, especially for patients in the US with the cost of a and such.

Maya TrialHub (17:17.654)
Yes, yes, that's actually that's very to the point Ella. I recently found out that a huge part of Americans are excluded from clinical trials exactly because of the financial constraints and the way like the trial reimbursement is fixated. So actually turned out that for you to join a clinical trial if you're in the States, you actually have to be financially sustainable.

and definitely not be on welfare. Otherwise it's almost impossible. But even if you have some income, even then it's quite challenging for families to ensure that the patient can actually go and participate in the clinical trial. I have a question, Ella You mentioned that you're advising different companies and you're telling them about the patient journey, but also concerning the patient facing materials.

I wonder how open they are to listen to your feedback, how open they are to adapt and change.

Ella (18:21.041)
Mm -hmm. So it definitely varies. You know, I think that for the most part, I have not been involved in any, I guess, clinical trial development earlier than once a protocol is already established. And then I'm reviewing a protocol and giving feedback.

And sometimes some of that is implemented. Sometimes I've never received feedback on whether it's implemented or not. And so I would say that's in a large part in the clinical research space, that's as early as I've been involved. When it comes to, and my belief is that this should obviously happen a lot sooner, but just going back to the whole conversation that we've just had of why that's important and really

You know, I think that, but that being said, it does, it does vary based on what type of company I'm working with and how interested they're in. So it's interested in gaining this patient input when it comes to, you know, smaller, more like, like vendors and other kinds of solution providers that kind of are either.

companies that are used by sponsors to support the clinical trial experience or, you know, be involved in clinical research in some capacity, they're increasingly seeing the need for bringing in these patient voices to be on advisory boards or, you know, consult independently and give feedback on certain aspects of their products and their solutions. But

As a whole, will say it is shifting. Patients are increasingly being involved and consulted with. But I think larger pharma companies, as I said before, they have their internal teams. They maybe bring in a patient or a handful of patients to advise on certain materials, patient -facing materials, even once a drug is in the market. Or maybe they bring in, it's kind of a new area, I think, of pharmaceuticals.

Ella (20:35.461)
pharmaceutical marketing is through work with patient influencers. These are people that are on patients that are very active on social media. They have a large following. so companies are starting to partner with these individuals. But that's still not, that is not patient involved in research and really ideating on how to improve the process and experience.

Maya TrialHub (20:39.543)
Mm.

Ella (21:02.565)
Another area where I think that there is, we've talked a lot about the clinical research and the clinical trial space and how I would say companies are starting to really consider patients in this realm. But another area that I've really come to realize where there isn't a lot of patient involvement in is more so on the side of patient access to care and access to payments. So when it comes to...

Maya TrialHub (21:21.485)
Hmm.

Ella (21:25.567)
a patient who is looking to be on a product once it's going through clinical trials and it's come to approval. And they're having to navigate, and this is maybe a unique situation to the United States a little bit, but having to...

get the prescription filled by their doctor, then it goes to the pharmacy. But the pharmacy can't fill it because they need a prior authorization from the insurance company, but the doctor has to fill that out. And so there is this convoluted

mix of back and forth of various stakeholders and individuals that have to be a part of providing a patient access to a treatment that then ends up being the patient has seen having or they get a denial from the insurance company and then the patient is having to call the doctor and call the pharmacy and call the insurance and understand why this is happening and figure out a solution.

you know, are these pharmaceutical companies have what are called patient support programs. But these programs often, to be honest, don't do much. They are not really able to facilitate any sort of hurdles or make anything happen because each of the other parties has to do their job. The physician has to sign these paperwork.

Maya TrialHub (22:26.774)
Hmm.

Ella (22:44.083)
They have to send it over to the pharmacy. it's like, this is an area where we want to really talk about reducing patient burdens too. These are significant burdens on the US patient population in trying to navigate access to drugs or access to care in the hospital setting too and dealing with insurances. so, yeah.

Maya TrialHub (23:03.33)
Yeah. Yeah.

Ella (23:05.819)
I'd say that as a part of this too, forget, and I certainly forget about this too, that insurance companies are part of this in the US. They're part of this conversation that they should be, but they're not really involved at all. And they're sort of this outlier that doesn't think they need to be patient -centric or support the patients in any way.

Maya TrialHub (23:25.674)
Yeah, actually that's a great point, Ella. Thank you so much for sharing. To be honest, it's not very different outside of the United States to my knowledge. I can speak definitely about Europe. The only caveat is that the insurance, the ultimate insurer is the government, which makes it even harder, but at the same time more centralized. The government wants to improve the access.

Ella (23:48.028)
Mm -hmm.

Maya TrialHub (23:50.892)
Like for example, in Europe, you have these situations where the doctor wants to prescribe you this drug because they believe it will have much bigger, much better effect on your recovery, for example. But they're only allowed to do that after another drug that is being, that's let's say, reimbursed as a first line treatment. And only after you fail on this first line treatment, then you can be prescribed the second line of treatment. And then a lot of things are going again, back and forth. And it's like really crazy. I do agree with you.

the patient journey and understanding this patient journey, how the standard of care defines that how the healthcare system define this patient journey, which are these bottlenecks are not only for clinical trials is for the entire healthcare, payers, insurance companies, like all sorts of vendors and so on and so forth. And I do agree, we need more people like you who like are doing for the sake of like doing that for the sake of helping others. But I think that there should be a more dedicated

Ella (24:33.543)
Right.

Maya TrialHub (24:49.94)
institutions, organizations to actually elevate this voice, make it as part of like not something nice to have, but actually a must have. And once again, what you said, bringing, bringing a patient after you finalize the protocol is probably the worst decision you can make because on one side you did so much work on finalizing this protocol. And at the same time, you're getting someone to give you feedback.

Ella (25:13.138)
Right.

Maya TrialHub (25:16.11)
hoping that they won't give you actually feedback so that you don't have to fix it. So you either don't do anything and then you go through amendments, which happens with 45 % of clinical trials, or you just have to go back and redo, not all, but a huge part of the work that you're doing.

Ella (25:20.679)
you

Ella (25:33.191)
Right, and that's a significant cost and resource burden on the part of the companies as well.

Maya TrialHub (25:40.566)
Yeah, Ella, thank you so much for spending your time with me, sharing your insights. Hopefully, sometime, we will have another conversation and then you give us even more positive news, also with more impact on the patient support programs. So cross fingers for you and thank you for the things you're doing.

Ella (26:00.979)
Thank you and thanks for this opportunity. It was a pleasure to talk to you.