Ep. 1: Putting Patients First: Patient-Centered Evidence & PROs in Biopharma

You're listening to a sponsored episode of The Top Line.

Welcome everyone. You're listening to the top line brought to you by Fierce Pharma and Fierce Biotech. I'm your host, Irena Lol.

Today's episode is sponsored by RTI Health Solutions, a global research partner delivering the evidence and insights life science leaders need to make better decisions. RTI Health Solutions brings together specialists in health economics, real-world evidence, medical affairs, and scientific communications, integrating rigorous research with clear, compelling storytelling to help clients demonstrate value across the product lifecycle.

We're talking about what it really means to put patients first, not just as a buzzword or slogan, but as a strategy that's reshaping evidence generation, market access, and medical communication. I'm joined by two guests who bring deep complementary perspectives on this topic. First, Ash, Senior Medical Affairs Lead at RTI Health Solutions. Ash has driven global medical strategy and scientific engagement across Europe.

Supporting product launches, developing medical education programs, and training international field teams. Her background spans immunoncology research, medical advisory roles, and hands-on experience building medical materials and patient-facing education. She brings a clear view of how patients' insights translate into real-world medical practice and communication.

I'm also joined by Mark, who brings more than 25 years of experience designing, collecting, and interpreting health data across therapeutic areas. Mark is an expert in qualitative and quantitative research, everything from Delphi panels and stakeholder interviews to multinational payer and HTA insights.

He's led large-scale studies across oncology, neurology, cardiovascular disease, psychiatry, and more, and has authored multiple papers on research methodology and patient-centered evidence. He spent his career helping companies understand what truly matters to patients, payers, and clinicians. Welcome, Ash and Mark. Thanks for joining me today. Thanks, Karen. Thank you for having us.

Today we're gonna explore how patient voices are reshaping the way therapies are developed, evaluated, and communicated, and why listening earlier and more intentionally can make the difference between approval and access. So let's just jump right in. And something I like to say is that I think something we all have in common is that we have all been patients at one point in our lives. So when we talk about putting patients first, what does that really mean today? And how is the patient voice?

Shaping evidence and decision making in ways it didn't before? Yeah, that's such an important question, Irina. I mean, we're bombarded with these words, right? Patient engagement, patient voice empowerment, patient centricity, patient focus approach. Buzzwords, there are many, but do we, as scientists, pharma industry leaders, regulators, policy makers as a society, do we really understand what this means? I think today putting patients first is no longer a nice to have. The patient

Voice is no longer anecdotal or symbolic. The patient voice is a proactive catalyst, a catalyst that improves how clinical evidence is generated, communicated, assessed, and a key factor that ensures that healthcare products, devices, and innovations reach their maximum potential public health benefit. And as someone working in medical strategy and scientific communications, for me, for us, putting patients first involves building a proactive

Inclusive, and symbiotic partnership with patients. You know, patients have to be involved in co-developing, co-crafting strategies to generate and communicate a value in ways that generally improve patient lives. This could take a few different forms. Pragmatic clinical trials that include telemedicine and flexible participation option for patients.

It could take the form of publications that are co-authored by patients that forego jargon and include plain language summaries. It could involve the development of patient journeys and patient advisory boards that often reveal insights that are often you know contradictory to what clinicians have reported. And all of these activities are just a glimpse of what traditionally has been in the nice to have category in the pharma space, right? Today, patient participation in such activities.

is essential. It helps inform robust medical strategy, audience appropriate communications, holistic, multidimensional regulatory decision-making processes, and eventually leads to faster approval timelines and a sustainable and egalitarian model of access to healthcare innovations. And you know, more than that, I think on a more practical note, putting patients first

Is also an intentional exercise and cross-functional collaboration. When patient engagement sits with one team, it often becomes optional. I think when it's shared, it's shared across RD, medical, regulatory, market access, commercial. I think it that's when it truly becomes transformational in the pharma setting. In my opinion, we need to call for like structural and even cultural change. We need to build clear frames that empower teams.

To stay curious and also have the confidence to boldly ask, right? What would this initiative look like if patients helped develop it, co-develop it from the beginning? And would it look the same if they didn't? Ash, I agree with everything that you just said. You know, you can have two pharmacological treatments that work identically to treat various aspects and clinical signs of a condition. They're doing the same thing in the body at the chemical level.

But one treatment may have a more positive effect on function or health-related quality of life than the other one. And you can only learn that by talking to patients themselves. It's really fantastic that regulators, HTA experts, reimbursement decision makers, and clinicians are recognizing this more and more. Over the course of my 25-year career, we've seen a change in the development of patient reported outcomes measures. And

really solid codified methods have emerged and continue to improve to assure that the questions are valid and reliable and to use an FDA term fit for purpose with the patient populations that they're being designed for. But we still have a ways to go in ensuring that we capture the patient voice and that decision makers not only review these messages but prioritize them alongside

clinical efficacy and safety data and emphasizing endpoints that matter the most to patients, like does the treatment reduce my pain? Does it improve my mobility? Does it make it so that I don't have to go to so many clinic or or hospital visits? So you both really mentioned and hit on the point that, you know, putting patients first and patients is so crucial.

So what does meaningful patient engagement look like in practice? And how can initiatives like patient advisory boards or direct patient input genuinely shape product development and market access strategies? Yeah. You you're really speaking my language. To fulfill the obligation to incorporate a robust patient voice into a product, pharmaceutical manufacturers should create formal, ongoing patient advisory boards.

early on and throughout development. And they need to be made up of diverse patients and caregivers. We should consult the patient advisory boards early on to test the clarity and relevance of the value proposition for the product and ensure it addresses patient priorities and concerns, especially around access and affordability. And doing this as early as possible helps us to harmonize patient input with that of other decision makers.

Like I said before, we have to ensure that clinical trials use validated PROs that capture endpoints that are relevant to daily patient function and health related quality of life. But in addition, HTA bodies and payers increasingly are requiring these data to understand the true value of a therapy beyond efficacy and safety.

More and more we have to include research in patient burden with quantitative and qualitative studies that detail the burden of illness, you know, including things like time lost from work, the costs of caregivers, emotional impact. All of these provide a crucial context that justifies the cost of a treatment. We should also include patient advocacy groups.

And have them to oversee research in HTA submissions because that ensures a living human perspective is part of the formal submission and deliberation process that can result in independent high-quality submissions to HTA bodies. These kinds of patient-centered submissions carry significant weight because they're perceived as unbiased. In addition,

We should train and support patient experts to participate directly to provide testimony in oral hearings or committee meetings with HTA and reimbursement authorities. Because you know, a brief articulate in-person testimony from a patient or a group of patients often has a greater impact than pages and pages of text data that's in the written submission. We should structure market access dossiers.

To explicitly link the product's clinical benefits to improvements in patient quality of life and functional status. We can use patient quotes, direct quotes, or case studies within the narrative to illustrate the data points. We can include a robust section in the submission dossiers detailing how the manufacturer is going to address patient access and affordability barriers. They might

Put in you know patient assistance programs or training support, things like that. And when they do that, what they're demonstrating to the decision makers is a commitment to the patient population. And that appeals to payer concerns about equity, proper administration, adherence, wastage of a product, so on. Yeah.

Absolutely, Mark. Very insightful. And I think, you know, all these process, everyone has to be involved, right? All cross-functional groups have to be involved. And I think we have to be creative in how we communicate this value across the product life cycle. And you know, since we also live in the age of AI and digital transformation, I think many of our clients have also enjoyed integrating insights from compliance-approved social media listening tools, right?

These are very powerful tools that allow us to access large amounts of data and distill insight from previously unknown demographics or hard to read geographies and in multiple languages. And not only that, you know, these tools allow us to study trends and opinions over time. So there are also some state of the art and budget friendly strategic insight collections exercises, such as social media listening, in addition to patient advocacy groups and patient advisory boards that you mentioned.

that we can use to build patient journeys, medical education programs, and also solutions for impactful conference engagement. And these are just a few, I'm sure we'll come up with more examples as we speak further. Yeah. And Ashmark, you both emphasize the valuable insights that patients have and it's really crucial. So when it comes to communicating value and how can industry do better job of speaking that patient language?

both in the materials that we create and ensuring the value really resonate with different stakeholders like payers, clinicians, and HTA bodies. Yeah, absolutely. That is another important aspect of what Mark, I and the rest of the team we try to really improve. When it comes to communicating value, I think the industry needs to speak the patient's language while still meeting the needs of the clinicians, payers and HTA bodies, right?

And this starts with understanding how everyone frames value. They frame it very differently. Patients care about real life impact. Are they able to move, eat, work, enjoy social life, reduce caregiver burden? Patients want therapies that improves quality of life, restores normal routines, and therapies that are accessible and personalized. Clinicians, on the other hand, are hardwired to look at efficacy and safety from a large data set.

They like looking at longitudinal studies, randomized clinical trials that showcase credible, scientifically validated evidence that can be generalized to broader populations. And payers primarily speak in, well, dollars, right? Demonstrating value to them requires data on economic benefits, reduction in hospital visits, reduced healthcare resource utilization, and an overall reduced burden on healthcare systems.

So many ways of defining value. How do we align this? How do we optimize value communication to all these groups? We need to make sure that scientific messaging is tailored. And perhaps putting patients first in this context might be the answer to this. Initiatives like patient-led medical writing, co-authorship in scientific publications, patient ambassador programs are just a few examples where we can really leverage.

co-creation with patients in a formal setting. These communications tool allow us to capture insights from the patients and integrate them in this pipeline right from when the evidence is generation, right? We can do it consistently and make sure that we replicate it across communication portals so that it resonates not just with patients, but also with clinicians, payers,

And the society at large, medical education programs, public awareness campaigns, social media interactions, and niche conferences spotlights that empower the patient voice, I think should again be consistently leveraged across scientific sectors and geographies. And lastly, there are also some novel interdisciplinary approaches like implementation science that allow us to inform medical, clinical, commercial strategy to proactively facilitate product use.

adoption, adherence by anticipating barriers that patients' healthcare providers and healthcare systems might face. So these were just a glimpse of some measures that we can utilize early and across product life cycles and therapy areas that'll allow us to speak the patient's language in a way that all decision makers can understand. And I think this is even more important in today's day where

Misinformation is so rampant, right? I think Mart can tell us a little bit more, but I think there's a heightened need to underscore for the right information to reach the right audience at the right time. Misinformation is a good segue to what I want to say. Ash, as you were speaking, I kept thinking about the Tuskegee syphilis study, which as we know was a notorious forty year medical research project that involved unethical experimentation on African American men.

And it highlights some of the most severe violations of medical ethics and informed consent that we've ever seen. The so-called study continued unchecked for forty years from nineteen thirty-two until nineteen seventy-two, which really wasn't that long ago. That's in my lifetime. I did a qualitative study a couple of years back, and I spoke to the regional lead from a sickle cell anemia patient advocacy group in the US Midwest, and she really impressed upon us.

how important it is for the black community to have people that they trust and who look like them to explain why they ought to receive a treatment or even more so why they ought to participate in a clinical trial. This population hasn't had a very good history from trusting the medical and and public health powers that be. So that was an example where it really kind of hit home that the patient and patient advocacy voice

can literally make the difference between deciding to undergo a therapy that can remove such agony and suffering and disability in children and adults. Yeah, absolutely. I think this is a perfect example for showcasing why medical communications is much more than just disseminating scientific results, right? It goes beyond the science notebook and it takes into account cultural, geographical, societal cues that

make the messaging complete and holistic and appropriate. So yeah, thanks for sharing, Mark. Absolutely. Yeah, and just on that note, every patient have a different story to share. Not every community has the same story. So it's often said that patients tell that different story than everyone else in that system. So how can embracing these differences, even when they contrast with clinicians or payers, help companies strengthen their strategies? Yeah.

We're all doing a better job than we ever have before in terms of listening to the patient voice, but there's still plenty of room for improvement among decision makers who don't have the full story. I'm thinking about a joke that that says that you can ask a patient, their spouse, and the GP how well the patient's hypertension is controlled. So the patient says, it's fine. I I measure it at home, I've got a cuff, I write down the measures.

I bring it in for checkups and I discuss it with the doctor. The GP says, Well, y your blood pressure's a little high today here in the clinic, but you probably got a little white coat going on. And the measures you wrote down and brought in, those all look great. So I think you're well controlled. And then finally the spouse speaks up and says, You know, he's red in the face all the time. He only writes down the good measures for the doctor to see and not the bad ones. So, you know

It it is classic and it's so it's so realistic. It you know it's tongue in cheek, but it shows that we need to have all the pieces of the jigsaw puzzle in place to be able to see the full picture of a person's healthcare journey. Stories told from everybody's perspective, all perspectives have a way of triangulating or even correcting each other. And this is what all decision makers need to strive for in healthcare.

As scientists and decision makers, we need to translate the patient's lived experience into data and insights that resonate with the clinicians and the payers' perspective, back to the question you posed, Irena, on clinical relevance, practical care, and healthcare use and costs. And we're able to achieve this by moving beyond simple qualitative anecdotes to integrating robust

patient-centric evidence into the core HEOR strategy. We can use health and medical communication best practices to translate the patient experienced payers and clinicians in a language that they understand. As I said before, payers need to see how a new product is going to save the plan money. Clinicians are hardwired and trained to think in terms of diagnosis and treatment pathways, decision making, objective measurement,

all those things. So the patient voice has to be translated into those frameworks. Pharmaceutical manufacturers can engage clinicians to understand how a product profile that includes PRO data could influence shared decision making with their patients. The ATOR strategy should explore which patient relevant benefits would prompt the clinician to choose the new treatment.

or product over an existing therapy. We can use patient input to highlight symptoms or burdens that are invisible to providers or what might show up in standard labs or physical exams, like chronic fatigue or anxiety or pain. Then we can demonstrate how the PRO data captured those burdens and how improvement in those areas could reduce healthcare utilization.

So that's going to make the payer sit up and take notice if there's going to be fewer healthcare visits or reduced reliance on pain medication, for example. In advisory board and clinician thought leader Delphi panels, we can present clinicians with patient vignettes that are directly tied to the HUR economic model inputs. For example, if the drug improves a patient's mobility score.

By 15%, as measured by the way that they respond to a set of PRO questions. How would that change the patient's prognosis or the follow-up schedule that would get tailored with them by the clinician? We can invite patient advocates to participate in ad boards and Delphi panels with clinicians, or at least give them the opportunity to provide input on the design and analysis of information from.

those expert interactions. And that can create a powerful dialogue where the patient can explain the real world meaning of the data points that the clinicians are reviewing. And this can foster empathy and a deeper understanding of the functional value. So in our research, we can focus on helping manufacturers to ensure that the patient voice moves from being a soft narrative to a hard narrative

decision making data point that clinicians and payers can ignore. I love that. And, you know, we talk about the patient's voice and the lived experience. It's all unique. You know, one lived experience is going to be the same over the other. So looking ahead, what are some tangible ways pharma and design products, services, and support programs with patients in mind and not just for them? All right. How much time do you have, Irena?

well pretty long, but here are our top three recommendations. Some practical advice, some strategic advice on how to put patients first. It's been my personal mission to encourage more patient involvement and large clinical conferences. So our first piece of advice is to include the patient voice at clinical conferences too. I think one of my core memories as a scientist and a medical communicator is when I once attended a colloquium.

between a patient and their physician, where they both were going through the diagnostic and treatment journey that they were part of, right? And you know, both of them were offering their personalized insights and perspectives. And the clinical narrative could have just ended with the physician saying, the patient received a novel cutting edge therapy and had a complete response rate. But I think the true story came to life when the patient translated the message for us, right?

How taking this novel cutting edge therapy allowed them to go back to their normal life, how now they could play football with their child, how they regained their independence by being able to drive again, how they found financial stability once again by being able to go back to work. And it was one of those academically stimulating yet heartfelt moments for me as a scientist. And

It's been my passion ever since to try and encourage this educational endeavor at as many conferences as possible. And it's precisely these types of projects that Mark and I and our colleagues really enjoy putting together for our clients. Number two, patient inclusion programs, patient initiatives should not have an expiry date. And what I mean by this is

You know, when we design patient inclusion programs, we really encourage our clients to think about long-term sustainability and applicability. You know, how can these programs be kept alive after the drug has been approved or launched? How can this program be kept alive even if the patient voice leader at that organization moves companies, right? How can we use patient journey algorithms for another asset? How can we use this medical education tool?

For another therapeutic area, we really need to make the best out of limited resources, time, energy that we have, and also extend, you know, these initiatives cross-functionally, institutionally, and also in geographic areas. So, you know, that really comes down to an operational shift, right? We need to adopt a one team mindset, share resources, be transparent and work together to make the most out of all these investments we put into making

to putting the patient voice first. And lastly, I think there's also a need to make the patient voice more inclusive, right? The loudest patient voice may not accurately describe the entire population. So we really need to work together to make this exercise more inclusive, to truly reflect the real world population we aim to serve. When developing a product, let's make sure all demographics, geographies, cultures

All levels of health literacy, age, gender, ethnicity, socioeconomic status, disability, language, digital access, and even caregiving roles are represented appropriately in the patient population. Let's be proactive, inclusive, and co-create mutually beneficial ways of thinking about science, testing innovation, communicating value, not just for improving patient lives, but also for healing us all as one united society.

And that's how I think we at RTI Health Solutions can help co-create a new paradigm for not just commercial success, but also patient success. Thank you, Ash, for those insight. I'm Mark for this discussion today. And thank you, everyone, for joining us on this episode of the top line. I'm your host, Irina Lowell. For more information, you can learn more in our show notes at Fierce Pharma dot com. And that's the bottom line from the top line.