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Clinical data from the patient's perspective with Ella Balasa
27 Plays1 month ago
In this episode of Clinical Data Talks, Sylvain Berthelot welcomes Ella Balasa, a patient advocate and leading voice in shaping collaborative approaches to clinical research. Together, they explore the critical importance of embedding the patient voice into every stage of the clinical trial lifecycle to ensure research is truly inclusive and effective.
With a deep personal history of navigating the healthcare system as a cystic fibrosis patient, Ella shares the stark realities of trial participation: from the struggle for awareness in rural communities to the heavy physical and logistical burdens of complex procedures. She provides a poignant account of how rigid exclusion criteria can prevent the very patients who need new therapies most from contributing to essential data.
Sylvain and Ella discuss why the industry must move beyond traditional endpoints to capture what truly impacts a patient’s quality of life. They also dive into the necessity of optionality, explaining how flexible trial designs can improve enrolment, reduce attrition, and accelerate timelines.
Tune in to discover how true partnership between sponsors, regulators, and patient communities can transform trial design from a "one-size-fits-all" model into a meaningful, data-rich collaboration.
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Transcript
Introduction and Guest Welcome
00:00:13
Speaker
Welcome to Clinical Data Talks, a podcast brought to you by CRS-Cube. I'm your host Sylvain Berthelot. Join me and industry experts as we discuss the latest trends impacting the world of clinical data.
00:00:28
Speaker
Today we're going to focus on clinical data from the patient's point
Patient Advocacy and Personal Experience
00:00:33
Speaker
of view. And I couldn't think of a better guest for this than Ella Balassa.
00:00:40
Speaker
A passionate advocate for patient-centric innovation, Ella has become a leading voice in shaping more inclusive and collaborative approaches to clinical trials.
00:00:54
Speaker
With deep personal experience navigating the healthcare system, she works closely with industry stakeholders to ensure the patient voice is truly embedded in clinical research.
Challenges in Trial Enrollment
00:01:09
Speaker
Hi Ella, thank you for joining me on the podcast. How are you doing? Wonderful. Syvonne, thank you for having me. Well, we've already recorded a podcast together in the past, so um um I know we're in for a treat and I'm very passionate as well about today's topic. So I'm sure it's going to be amazing.
00:01:29
Speaker
um And I'm going to start with ah maybe a tough question. You tell me if it is. But from your experience, what you see in the clinical trial industry, what do you think are the biggest challenges patients face when enrolling in clinical trials?
00:01:56
Speaker
You know, I think first and foremost, the greatest challenge and barrier is really awareness and knowing about trial opportunities that might exist for a particular patient for their disease area.
00:02:11
Speaker
I think that You know, it's widely known that a lot of clinical trial enrollment happens through the provider or through the physician at their particular care centers.
00:02:23
Speaker
But, you know, with First of all, you know heavy patient load, patient doctors are not necessarily aware of a lot of of trials that exist around the country. there is um you know if if it's If the patient is not seen at a medical institution that conducts a lot of their own clinical trials or is ah is a hub of of research, then a lot of patients you know outside of that don't have opportunities to learn about or to participate in trials. So um I know there are increasingly platforms and technologies that are trying to bridge these gaps and bring about you know through digital means and other ways by which to um reach patient populations. about clinical trial opportunities, but I think it's still like is a very big gap that exists. And unfortunately, those in more rural community hospitals or those that, as I said, are not seen at these large academic institutions are you know not getting the same opportunities to participate and learn about clinical trials and clinical research opportunities.
Burden of Clinical Trials on Patients
00:03:38
Speaker
um I think secondly, more so when it comes to the actual trial itself, the burdens that you know the burdens from having to travel to the sites or the complexities of the procedures and tests that are required and the the involvement for or the burden on the patient to um be a trial participant, it can be overwhelming, i think, especially for populations whom you know may not be used to a lot of medical involvement or intervening, right? If they are new to healthcare systems and you know relationships or connections with physicians and and other hospital staff, there may be a lot that is required of these patients and there may there are gaps in
00:04:34
Speaker
um the ease of how this patient is being walked through or supported throughout the trials. So I think there can be wide, you know, just broadly speaking, improvements in how that's managed and how the patient is being, you know, facilitated through the trial experience.
Navigating Trials and Advocacy Role
00:04:55
Speaker
yeah both very good points a a very short anecdote about knowing about trials that are available i so i've got my patient advocacy podcast and i talk to patients regularly and someone in one of the episodes shared that they go on ct.gov clinicaltrials.gov for those who don't know it and to look for trials that are upcoming or enrolling.
00:05:27
Speaker
ah But as they were explaining that, I was thinking, but how do they navigate it? Because it's not user friendly, not even patient friendly. It's just not user friendly at all. So yeah, we glad to hear that there are tools that hopefully will help with that in the future.
00:05:46
Speaker
Yeah, I was just going to add that you know there is a number of companies that are trying to solve this, but obviously you know it's it's fragmented. And and i think some approaches that are help that are bringing in the advocacy organizations to support this in their respective disease areas potentially could have the the most traction or you know benefit where by which there is you know sort of a vetting process or just sort of this uh trust kind of building layer where the patient can you know be aware of these trials through these organizations that are supporting you know the well-being of the patients and supporting
00:06:33
Speaker
holistically the lives of the patient, um not just, you know, they're not just the clinical trial kind of a physician perspective or experience. So I think that that's a great way to Yeah.
00:06:49
Speaker
I was half expecting you to also mention exclusion inclusion criteria that could be a ah barrier to patients participation because they can be restrictive, essentially.
Exclusion Criteria and Personal Advocacy
00:07:03
Speaker
ah ah What's your experience with that? And do you think it can restrict access for patients? Well, I'd love to sort of bring in my own personal experience into this topic because yes, absolutely.
00:07:19
Speaker
Exclusion criteria is a very significant barrier to participation. And I personally have experienced this myself. and Having cystic fibrosis, I have reduced lung functions. So my lung function less than 40%
00:07:37
Speaker
which is FEV1 is the measure of lung function. And in all new investigational drug trials for cystic fibrosis, um it it's only patients with above 40% have been included into trials. And um that's for you know over a variety of reasons, but I've been very strongly advocating for the broader inclusion of patients in trials because you know there is a ah whole subset of the population such as myself who could readily and you know meaningfully contribute to clinical trial data and the understanding of you know how there's a whole population of patients that um you know a lot of these these trials are not um capturing data from and so if we can really be innovative in the way that we're designing trials and making sure that
00:08:33
Speaker
in some part, they're either, maybe it's not data that's collected for the, you know, the the main arm of the study, but maybe as data is collected, we can, you know, there can be ways by which to incorporate data from a broader swath, from a broader population of patients, and then, you know, provide that potential benefit where, especially in, you know, rare diseases,
00:09:01
Speaker
pediatric diseases where there is a significant, you know, these are life limiting conditions where there is not a lot of research and and treatments and that exist. And so there's a very, very high need. And it not only is potentially, you know, an improved, could improve quality of life, of course, if if the patient, and know, is able to be on on drug in the trial, but it just really expands that knowledge base for when the treatment does come to market. And there is already that data that's collected from really a broad population within any a disease area.
00:09:42
Speaker
And then of course that supports, um, with reimbursement and, and, you know, in the, in the post-marketing, um, you know, phase of, of the drug itself too.
Designing Patient-Friendly Trials
00:09:53
Speaker
ah think we have the ability to, um, you know bring about these innovations and and changes in the design of trials between collaborating so closely between FDA, between sponsors and bringing in the patient perspective into these early conversations to really help to facilitate you know this awareness from even the regulatory side of how more inclusive data could really benefit you know obviously the population, but then also ah the the understanding of how this the the effect of this treatment, any given treatment as well.
00:10:31
Speaker
Yeah, yeah, I fully agree. and It's interesting because there's ah obviously there's a benefit for the patient. So from your point of view, having not being able to enroll in in a trial all means that you don't benefit from the treatment early.
00:10:49
Speaker
but I want to go back to something you said earlier on, which is that essentially there's a benefit for the sponsor as well because they gather more data, which with an open mind may ah show something that they were not expecting. um But i feel like we're in ah in a situation where we, well, and I think that's true, that we want to we know exactly what we want to demonstrate when we start a trial.
00:11:25
Speaker
um But going back to a point that you you raised earlier as well, there's so much that patients may not be able to accommodate that it feels like we're, we've written something and we're we're trying to make it fit the patient population rather than working with the patient population to know what, what could work and what they would benefit from.
00:11:53
Speaker
um So from your point of view, ah what do do you Do you have an appreciation for what patients are able to accommodate? What what will a patient do to take part in a trial and where will it be too much for them?
00:12:19
Speaker
You know, it's it's hard it's hard to answer that from you know just one patient perspective myself. And of course, it varies very widely. based on the the community and you know the the burden of the condition itself and how much it would take for a patient to you know be a participant in the trial on top of the management of their care on a day-to-day basis.
00:12:46
Speaker
um But you know I would say that when it comes to, for example, with patients that have mobility problems or pediatric populations where you need, where that person needs a caregiver to assist them in, you know, getting dressed, getting into a wheelchair, getting out of the home, getting to a clinical site, you know,
00:13:13
Speaker
all every Basically, every step of the way, there needs to be somebody present and supporting that participant and that patient. um these can be you know These are very high burdens to you know accommodate in order to participate in the trial that's going to require you know in-person visits multiple, you know once or twice a week even for any given length of time. um And so i really, you know, strongly advocate for how to make trials provide the optionality for each participant. And even within, you know, even within the same disease area and within the same trial, there can be a wide range of a patient's ability to either travel to a site or to, you know,
00:14:10
Speaker
perform any given procedures or tests. And there can be some patients that prefer to you know go into a site or versus have at home care where there can be monitored and get you know data that's collected seamlessly. And you know that can also be more of a social, kind of like a personal preference as well.
00:14:39
Speaker
do they want people in their homes or not who are strangers or you know providers or nurses and how that might affect or disrupt how it could either provide you know seamless care or disrupt maybe their day-to-day lives if they are they're working in person and they may have to then go home to receive you know from a home health care provider and you know get blood tests done. So then that may actually be an extra burden or an extra step, you know, maybe then at home care wouldn't necessarily be feasible or or make the most sense if the person is already out of the home and, you know, on their lunch break or something can can go to a site. So to say is that dependent on the disease population and just even within the same clinical trial,
Patient Involvement and Technology Integration
00:15:30
Speaker
building into the trial design, the option for patient for the the flexibility for, you know, any given
00:15:38
Speaker
participant to have the trial seamlessly integrate into their day-to-day lives in the easiest way possible. So that participation you know supports this instead of becoming an increasing burden or hindrance.
00:15:55
Speaker
Yeah, yeah, yeah. um And it's interesting because there's a lot more talk, I believe, about including the patient's perspective in trial or design.
00:16:09
Speaker
i don't know i don't know if it's just talk or if it's action as well. ah But from your point of view and your experience working with the industry, How would you qualify meaningful patient involvement in trial design? i agree that, well, currently, I would say there is fairly limited information
00:16:40
Speaker
integrated involvement of patients into the design, early design of trials. And even before, you know, even before the design process begins and really understanding that patient population from what are their needs, their burdens? What does the disease landscape look like? What are the patient preferences? um And really understanding that from not just broadly the the disease area, but actually connecting directly with specific patients, you know certain patients, disease advocacy organizations as well,
00:17:25
Speaker
And then bringing those those and that involvement throughout the design through with the conversations of in the design of, you know, determining primary endpoints and what is data that's going to meaningfully represent or or cap capturing what is meaningful to that community. So, you know, get taking The example of cystic fibrosis, again, um the FEV1, which is the measure of lung function, is always considered the the primary endpoint in cystic fibrosis trials, which, you know from a high level, makes sense. I mean, you're you're measuring how much lung function improves, therefore you assume, okay, they're having a person who have better quality of life, um you know more ability to exercise, less you know less tired, being less tired, et cetera, et cetera.
00:18:19
Speaker
um But in, you know, populations where there's a lot of lung damage that already exists, like in the CF, for my own health, for example, even any treatment really doesn't improve that lung function value, that number, because of the structural existing damage that the lungs have. And so, you know, with any given treatment, there may not be this significant improvement in the lung function itself, but that you know there can be significant changes in day-to-day levels of coughing, tiredness, the clearance of the airways, and reduced need for um antibiotic use to treat lung infections and so forth. and so But all of this you know
00:19:12
Speaker
it's it's If we have the primary endpoint of lung function, well, then this isn't really being captured. And so that's also why to go back to the point of inclusion and exclusion criteria, this population of patients isn't included into the trials because they may not see that benefit in the lung function improvement. So just coming full circle there. But all that to say is, you know this is this needs to be closely understood by sponsors.
00:19:41
Speaker
and you know bring in these patient voices directly early on before the design, before you know you have to go through and make amendments and such, so that we're designing trials together with patients that, a we're gonna be inclusive of a broader population, and we're gonna be measuring what is most meaningful for their improvement in their day-to-day lives. And you know this includes also in those early conversations with the FDA, Because of course regulators are, you know in any given disease area, they're used to getting data from you know a certain ah indication where, as far as you know if there's been trials that exist in the past in that indication, then that's the way that they're used to seeing, well, why is, you know if we're gonna be changing up endpoints or if we're gonna be being more inclusive in these designs, there needs to be some of that um community backing. And so this is a way by which you can, sponsors can collaboratively
00:20:40
Speaker
you know, it's holding hands with patients, with the patient community together and bringing this forth, you know, through the regulatory process as well. And it and it helps to, um you know, get more, i would think, support for for this kind of like innovative designs and such. So I think this kind of true partnership is still quite rare. um you know, I see it, I do see it increasingly more so and um and hopeful for the future in that with the, you know, the use of these types of of even technologies like creating digital endpoints and, um you know, for example, there's a, in cystic fibrosis, a a device that's measuring pretty accurately measuring cough levels. And that's potentially, you know, these types of wearables and technologies can really complement and and integrate into, and already are,
00:21:40
Speaker
into these designs and making sure that we're capturing what is most impactful for patients.
00:21:48
Speaker
I love what you're saying. i love
Benefits of Patient Involvement in Trial Design
00:21:52
Speaker
it. but It's interesting because essentially what you're saying is everyone should work together so that we understand patients and and their day-to-day life better so that then we understand what is more meaningful to them. But I really like that you also mentioned talking to regulatory agencies about it because obviously if they're not used to seeing other endpoints than FEV1, then they're not necessarily going to understand and that can delay trials. So it makes sense. But you also talked about the link with technology then.
00:22:34
Speaker
a which needs to be taken into account because if you can measure things differently, especially at home, ah then that can even ah that that can help even further with're with understanding the results that you're getting and and the improvements.
00:22:53
Speaker
Well, and and yeah, and to add to that exactly, it's you know often some of these measures or you know patient reported outcomes um are just that. they're right They're reported from the patient, so they're often not you know used as primary or secondary endpoints, right? Sometimes, or maybe sometimes secondary endpoints, but it's really the data is maybe not as qualitative. And so for For the example of you know using a device that accurately measures rates of coughing for you know any given day for a patient and you know a pulmonary type of condition, this is really um you know much stronger data than ah you know a patient reported outcome measure. and
00:23:38
Speaker
that can also better support you know the use of this kind of data um you know in as ah as a primary endpoint. So this is of a value in the way way by which technology can be really interwoven.
00:23:52
Speaker
So I don't know if you have experience with that yet because clinical cool trials are very long, but what do you think a sponsor can achieve in terms of clinical trial results or clinical trial execution by following your advice of working with patients even before trial design?
00:24:16
Speaker
Well, you know, I think it's it's multifold. I think first you are establishing that early relationship with the patient communities. So you already have, you know, coming in with a strong relationship being built, that trust factor, patients, you know, the broader population,
00:24:38
Speaker
having probably learned about this type of collaborative effort and work in design the design and in creating this type of you know novel way by which this treatment can be capturing and you know data from patients. So you're you're really collaborating, you're bringing you building that relationship early on. That then in turn provides more, as we said at the beginning, the problem of awareness of the trials itself, um of you know, patients being more
00:25:11
Speaker
eager potentially to participate as it is more meaningfully, um you know, capturing or or integrating into their lives, maybe reducing those burdens, those excessive burdens of participation. So then they're more likely to want to participate and then also less likely to fall off the trial and, you know, terminate early. um And, and then when it comes, you know, so then this can then shorten timelines potentially. for the trial getting to the market and actually helping the entire population that it's intended for. So, and then of course, you know, just from the from the sponsor side too, shorter timelines, um less you know cost later on down the line and in recruitment. and And I don't know the the numbers here, but obviously I know like each month that a trial is delayed adds like X amount of dollars to the budget. So sure, it requires, I think,
00:26:11
Speaker
bit more upfront planning, collaboration, even monetary commitment. But I think, you know, down the line, it's a strong benefit for all parties, for patients, for sponsors, um and really in just improving, hopefully, patients' quality of life for every any disease area.
00:26:36
Speaker
Yes, yeah. So there's no reason not to do it, essentially. um So hopefully, I'm really hopeful. Every time I talk to you, actually, i'm very hopeful and positive for the future because and I feel like it's progressing and going in the right direction. So hopefully we see change, even more change coming up in the in the coming years.
00:27:04
Speaker
I have one last question for you. What's the best piece of advice you've received that you apply at work?
Advice on Advocacy and Closing Remarks
00:27:14
Speaker
I think my passion drives my work, obviously, giving Given the you know health experiences and just navigating my own care and wanting to see improvement for not only my own health in my life, but on behalf of my community, the cystic fibrosis community, and more largely to um just patients who struggle day to day and um you know in navigating their care and in clinical trials and um
00:27:47
Speaker
So I would say the advice that I received fairly early on from other advocates and other you know patients who i saw were doing in getting involved in these capacities and in raising their voices is exactly that, is to be a voice for your mission and your community and your passion. um and to not be deterred or not not be afraid. There's, you know, not saying like doors open all the time and opportunities to, you know, for example, early on, I was asking to be a, you know, patient involved in the review of clinical trial protocols. And i you know, created published articles on the problem of, you know, minimal minimal inclusion into trials and the design of novel endpoints and um the problem that that is for patients. And i wasn't deterred by, you know, maybe not being able to publish this work or what the backlash or or what people might think about my perspective based on, you know, at the time, somewhat limited experience. um I've learned a lot as I, the more years that I've been involved in this space and and and as I've learned
00:29:11
Speaker
the, generally the the healthcare care ecosystem. And um I think it's, To summarize, you know, kind of going back, it's just to say that i want to encourage others to be voices for their for their passions, for their communities, and for what they want to see in changing the future of healthcare and clinical research for patients.
00:29:44
Speaker
Amazing. I love it. And you can feel the passion. So i I don't doubt at all that that's something you apply day in, day out.
00:29:55
Speaker
oh Thank you so much, Ella, for joining me on this podcast. It's been an absolute pleasure, as always. And as I said earlier, it really makes me happy to hear you and makes me positive for the future of clinical trials and how inclusive they're going to be.
00:30:17
Speaker
Thank you, Sylvain. Thanks for the opportunity. It was a pleasure. And thank you everyone for listening to us today. You can find more pis episodes of the podcast on the CRS Cube website.