Patient Advocacy in Oncology Research
Host Jason Eger, ERT’s Director of Operational Readiness, is joined by oncology patient advocate Deborah Collyar. Together, they discuss what patients are really looking for and how sponsors and the clinical research industry at large can adapt to discover the real-world answers patients need.
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How did you end up in patient advocacy? [1:23]
At the time of her first diagnosis, Deb was a computer company executive. Along with her husband (also a cancer survivor), she got involved with an oncology patient advocacy group. This was in the early 1990s when very little was known about effective cancer treatments, and she felt that this was important to change
Can you explain the role of a patient advocate? [2:17]
A patient advocate contributes in a dramatic way to improve the experience of patients they don’t know through direct patient support, political advocacy, fundraising, and watchdog advocacy. A research advocate takes a long-term partnership approach. They focus on improving and speeding research results and ensure patient results and things that are important to the patient community, not just the scientific community, are a major focus.
Why do you believe it’s so important to speak up for the patient in clinical research? [3:28]
Typically research has been all about science, product development, and regulatory compliance. No one has truly focused on the patient experience or the fact that the patient is the true end user. This needs to change, and clinical research should set goals for finding better treatments, not just more, that give patients the improvements they’re looking for.
Do you find there’s usually a difference between the answers patients are looking for vs. answers sponsors are looking for? [4:57]
While they are different, they can be considered together so we can get to the answer we’re all looking for. Companies may be focused on getting label approval from regulatory agencies, rather than the usefulness of this product and how it can fit into the rest of a patient’s life. This is where the patient voice is crucial, because it helps us complete the picture and cover aspects that are actually important to the patient community.
What are the risks of a lack of patient focus in clinical trial design, or in the industry at large? [7:30]
Not recognizing the patient as the end user can negatively impact the results patients get from a treatment. By involving patient advocates in the trial design process, sponsors can avoid or eliminate barriers that prevent individuals from enrolling in a clinical trial. Sponsors can also turn to adaptive trial design, which celebrated its 50th anniversary in 2019, as a potential way to improve the patient experience.
Does the method of incorporating patient centricity vary based on therapeutic area or indication? [9:30]
It does vary. In oncology, for example, cancer is very multi-faceted; it can be chronic, life threatening, acute, and so on). The type of cancer or patient situation can make a big difference in terms of what will be effective.
Currently, the industry approach to “patient centricity” still views the patient as a target to get data from. To address this, we need to start thinking “what’s in it for patients?” instead of “what’s in it for me (as a sponsor)?” Focus on patient needs ensures that regulatory and product concerns will also naturally be addressed.
Are there any advancements that you’ve seen improve the patient experience in the last few years, or any you anticipate to come? How can the industry implement these changes? [12:18]
Things are definitely improving, and more people are understanding the difference in perspectives among trial stakeholders. However, the scientific and medical communities need to be more in line with the patient perspective of a “cure”. There also needs to be an increased recognition of the fact that clinical research is often an endurance test for patients, particularly when sponsors are looking to address retention or compliance issues.
Can you share an example of a sponsor who experienced some benefit after proactively choosing to keep the patient at the center of their trial? [15:02]
A breast cancer study funded in part by the National Cancer Institute was the first trial that explored neoadjuvant therapy. Patient advocates were involved in the trial development process from the beginning. As a result, the study recruited patients quicker, required fewer amendments, cost less, and was completed faster. Bringing in patient advocates early was crucial to the trial’s success..
Do you have any final thoughts you’d like to share? [18:28]
We’ve all heard the terms “real-world data” and “real-world evidence.” The concept is important and has a lot of promise, but we have to think about what’s in it for patients. A shift from real-world data (raw material), to real-world evidence (analytics) to real-world answers is key to addressing what patients, families, and doctors really need and want.
Deborah Collyar: Because treatment is an endurance test and you are enduring treatment, whether you’re on a clinical trial or whether that you’re on an approved drug or device, you’re having to endure something that you really didn’t ask for.
[Intro]: Welcome back to the Trial Better podcast. In this episode, we’re focusing on patients in oncology research and how sponsors can better enable trial participation and compliance by adapting more patient centric trial designs, helping navigate this discussion is Deborah Collyar, the founder and president of PARE, Patients Advocates and Research. In this episode Deborah will go over the role of the patient advocate and key areas that will not only improve trial design, but also trial data. So let’s not delay this conversation and dive into this edition of Trial Better.
Jason Eger: Hi everyone and welcome to another exciting episode of Trial Better. I’m your host, Jason Eger, and I’m here with our guest a well-known and long-time patient advocate, Deborah Collyar. Deborah, welcome to the show.
Deborah Collyar: Thank you, Jason. I appreciate it. This is going to be an interesting talk.
Jason Eger: And we’re very excited to have you. So Deborah let’s get right into it. Why don’t you tell everybody, because I know that it’s very interesting, but how did you get into patient advocacy?
Deborah Collyar: Well, I’ll give you the short version. I was an executive in a computer company when I was diagnosed with my first cancer, I’ve had more than one and my husband and I actually both got involved in patient advocacy because at the time, which was in the early nineties we found out how little had actually changed as far as treatments and how little was actually known and we felt that that was important to change. So there was a group that was getting started in San Francisco, the Bay area where I live and we joined them. They’ve actually celebrated their 30th anniversary this year. We haven’t been involved quite that long, but almost, and started to look at the different types of patient advocacy that are out there. Traditionally there’s direct patient support and fundraising and political advocacy. We actually learned from the AIDS activists about kind of what we call watchdog advocacy, but there wasn’t a lot going on in research.
So I actually was involved in starting PARE, Patient Advocates and Research in the mid-nineties and a research patient advocate is a little bit different type because it’s a long-term partnership approach rather than a onetime event or activity. What we really focus on is improving and speeding research results to people. Another way to think about us is that we can be a useful thorn to make sure that patient results are a major focus and that includes things that are important to patient communities, not just to the scientific end points of a study.
Jason Eger: And so well, I’m so glad you joined us. I mean, it’s so nice for us to talk to someone with such passion and you’re a survivor. So I mean, you speak the truth here. So as we talk about this, why do you think, and why have you felt so passionate about it being so important to speak up for patients in clinical research?
Deborah Collyar: Right. Well, research traditionally, no matter where it’s done, whether it’s academia or in companies, or even in governments and foundations has been all about the science and the development of product and the regulatory focus, no one has really focused on the experiential side of research, what it’s like to actually go through it, even like the daily diaries or things like that have always been from a scientific standpoint and that’s fine, but that’s not the whole picture. I think the idea here is that for a long time, I don’t think that industry realized that patients are the actual true end user. Everyone was focused on the healthcare professionals and medical providers, but there’s another audience there that we really need to pay attention to because ultimately it’s whether or not patients get better treatment, not just more and if they actually feel better that their medical conditions are actually improved by the products that we want them to use.
Jason Eger: So as you have you explained it and we’re kind of morphing, I would say, I guess the question I have for you is, is there a difference between what sponsors are looking for in the clinical trial versus what a patient is looking for out of a clinical trial?
Deborah Collyar: Yes, absolutely. They are different. They can be considered together so that we ultimately get to the answer that we’re all looking for. But companies traditionally have been focused on getting a label approval from the regulatory agencies. And if you look at it, that’s actually a minimum bar. Whatever we can do to get that label and not do anything more so that we can get it out to the market and make money is kind of where the mindset has been. It hasn’t been focused on the usefulness of that product. That’s either an after fact or an afterthought or possibly something would be done after market approval, but not always. So I think the way to think about that is a person who gets a medical condition or serious medical condition especially, whether that’s chronic or acute or life threatening or not. That’s one aspect of their life and it’s an important aspect. They have to make very important decisions sometimes at a very short period of time, but they’re actually living an entire life and their medical condition and treatments have to be able to fit in to the rest of their life.
Now, their life changes, but there are still other aspects that they have to work in to continue to have a full life. So one example of that is that medical, functional tests that are medical tests are what are typically looked at in research and in clinical trials, because they’re measurable but they don’t necessarily tell us if that person is actually experiencing improvements in their medical condition. So by bringing in the patient voice and answers and experiences, then we’re able to actually complete a more complete picture so that we’re covering aspects that are important to the patient communities, as well as answering scientific questions.
Jason Eger: So Deb, as you bring forward the voice of the patient, what can sponsors expect or what are the risks to whether it be in a clinical trial itself or just in the industry in general, what are the risks to sponsors when they don’t focus on the patient?
Deborah Collyar: Well I kind of feel like maybe we’ve been answering that for decades in product development. A lot of times there is, especially now, reimbursement is the goal. It’s not just regulatory approval. The end goal is actually getting reimbursed for the product and what I like to remind people about is that patients and families are payers as well. It’s not just the quote payer community, or insurers or whatever, as the true end user we have to feel like there is some value that’s being given to that patient in order to feel like that’s a worthwhile treatment or product. So when you’re talking about clinical trial design, I wanted to cover that a little bit. Having some of us involved in the design process itself can actually help eliminate or avoid barriers that are often set up in clinical trials without anyone thinking about it again, because they’re not thinking about what would this be like to actually enroll in this study? Adaptive design is very important and believe it or not in 2019, that was the 50 year anniversary of our birthday so to speak of adaptive design and adaptive design is important because it means that you’re pre-specifying and planning in the protocol when we’re going to look at the data and how we can actually learn from the trial as we go and adapt it for what seems to be working in real people.
Jason Eger: So Deb, as people are working on so many different indications, and I know your experience is a lot within oncology itself. Is there a difference between various indications; so can a sponsor promote patient centricity in oncology designs differently than they would for let’s say another indication.
Deborah Collyar: Yes, I think so. So I also work in some infectious disease and auto-immune and some rare diseases and there are differences. And of course, when we talk about oncology, we act like cancer is one thing, but it isn’t, it’s like the 800 pound gorilla because it has everything from chronic to life threatening to acute, to a never ending thing. So depending on the type of cancer that you’re talking about and the type of situation that those patients are in, that that medical field has to deal with can make a big difference. All you have to do, I do want to cover two things here. One is the term patient centricity or engagement or whatever the latest buzzword is, all you have to do is go on Google and click on the images for that term patient centricity and what you’ll find there is the industry’s approach and industry I’m meaning academia as well to patients and what I have not been able to find an image that does not include a patient that’s there as a target. And the message that comes through is that we’re using patients.
There’s more data we can always get from patients. It’s not about supporting patients and it’s not about what I call wiifm. So you’ve probably heard of wiifm, which is what’s in it for me and that’s what everybody does. We need to switch the paradigm that exists and the mindset within drug development and device development to wiifp first and foremost, if we’re really going to focus on patients, then we need to first think about what’s in it for patients. Why in the world would they be interested in what we have to offer? What is their situation; this is like market research 101, what is your audience? What are their situations and conditions? And then you automatically will take care of the regulatory focus and the product focus because that’s what’s in it for me as far as a company. So that I think is an important concept that we need to get down. The other one…
Jason Eger: Oh, I’m sorry, keep going. I was just going to say, have you actually seen that shift or the start? If you will, I’m assuming clearly we’re not there yet as an industry.
Deborah Collyar: Well, I think things are definitely improving. There are more and more people who are starting to understand that it’s actually different for patients than it is from the company perspective or the investigator perspective. So here’s an example. I’ll give you a couple of examples. Okay. One is when you’re being diagnosed with something and I’ll use cancer as an example, just because that’s what, I’m more familiar with. The word, the C word is not cancer. It’s cure. We overuse that term. We hype that term all the time and what the scientific and medical communities mean by that can be as simple as a five year survival rate, especially for oncology, but that’s not what people and the public and patients think about when they hear that word, that means, oh, good. I’m not going to have to worry about this again. Never again, it will not affect the rest of my life. So they’re very different meanings to that word.
Another word is, you know, we talk about retention. Now, the word used to be compliance and adherence, and I still hear people using those words. But again, from a patient perspective, those words don’t mean anything because treatment is an endurance test and you are enduring treatment, whether you’re on a clinical trial or whether that you’re on an approved drug or device, your having to endure something that you really didn’t ask for. And so if we start to think about clinical trials and treatments more in that term of it being an endurance test, rather than why aren’t they complying with us or adhering to our protocol, then that can change the dynamics of what we do and how we do it so that it is more patient oriented.
Jason Eger: So summarizing the patient is really the number one stakeholder, right? If we had to prioritize it, that’s who the focus should be on in research. And that’s what you draw for every day?
Deborah Collyar: Yes. And so there’s an example of a study. I’ve been in many, many clinical trials from the development and concept, development and then protocol, development all the way through the activation approval recruitment retention, and then actually giving a public study results summaries as well. So I do have one example that I could share if you’d like.
Jason Eger: We’d love it.
Deborah Collyar: Okay. This was a breast cancer study that was funded in part by the National Cancer Institute in the US which I will call NCI and it was one of the first studies that actually explored what’s called neoadjuvant therapy. What that means is giving treatment to shrink a tumor before it’s removed at surgery. Because this concept was new it was not known how this was actually going to work out what kind of results patients would actually have. Now you notice I’m using the word results instead of outcomes, because results are what people get and outcomes is what research does. So that’s another example, but this trial was really interesting. We were involved from the very beginning of it from the concept, and then into the protocol development, there were several things that we changed regarding the design. We wanted more answers to more questions that were important for patients as well. So we were able to get some patient reported outcomes, PROs, added to it. We looked at the functionality of the study for patients, think of it as sometimes we can be a sanity check, for example, if nothing more. We opened up eligibility when we could, so that it would lead to larger patient markets, larger populations that could work on that. We helped streamline the process for it. We were very involved in how recruitment could happen and what issues that patient population was facing because it was for triple negative breast cancer. And so we developed a whole communication plan and program that was implemented.
The results of that actually were that even though everyone was worried that the study was not even going to be able to finish it accrued much faster than anyone thought it would because this involved many different fields within oncology and it was because of a lot of the communication approaches that we had taken. There were fewer amendments than their traditional studies because they actually listened to us in the development of the protocol rather than waiting until there was a problem with the study and then what do we do when we have more amendments we have to do. So, you know, those things can cost a lot of money obviously. The trial was faster, it went more smoothly and we actually got answers that were published in ways that did start to lead to a definitive trial. That changed practice.
Jason Eger: That’s clearly a success story, and I’m sure that you are repeating that as much as possible. Do you…
Deborah Collyar: As much as possible? Yes, I do have to say that there are many times where we don’t have those successes, but that’s often because we’re not brought in early enough.
Jason Eger: Understood. Understood. And so I guess before we wrap up here, Deborah, I’m curious if you have any final thoughts, what you think, I know what I think you would like to see the industry do, but what trends do you think you’ll see in 2020 when it comes to patient advocacy and whether it be technology or the industry in general?
Deborah Collyar: Well, you know, I think we’ve all heard the term real world, right? So real world data and evidence. I think it’s really important for everyone to understand that that’s important. It has a lot of promise, but again, we have to think about this in what’s in it for patients, and it’s not just an app that can give you more data. It has to be more than that. What I’d like to see is where we go from real world data, which is a raw material to real world evidence, which is as far as we might go now, which is the analytics side of it to RWA, which has real world answers. That’s what patients, their families and their doctors want. So if we can start to figure out in our trials, how can we actually address some of the issues that are there for those patient communities, then we’ll have better market acceptance, better reimbursement and better value for patients too.
Jason Eger: Again, continued success, Deborah. I’m sure we could continue talking. I want to personally thank you for joining us on the Trial Better podcast. Thank you so much for what you do and sharing your story and your passion for bringing patients to the center of clinical research. I know personally, and I’m sure that this message will stick with all of our listeners as we work to this patient being the center of the trial and center of the industry. So Deborah again, thank you so much for joining us today. We really appreciate it.
Deborah Collyar: Well, thank you. I appreciate talking with you and I look forward to more dialogue with everyone who is trying to make better treatments for patients and remember that’s better treatments, not just more.
Jason Eger: Absolutely. So listeners, thanks again for tuning in. Don’t forget to submit us any feedback you may have at [email protected] And if you could leave a rating or review at your favorite podcast streaming channel for us, we’ll see you next time on Trial Better. Thanks everyone.
[Outro]: Special thanks to Deborah and Jason for today’s episode. What an insightful discussion on patient centric trial design, and the tangible benefits for both patients and sponsors alike. Before we go, thanks for tuning in and be sure to join us for our next Trial Better briefing an interactive webinars series, where we dig deeper into the topics we cover on the podcast. Find information to register and upcoming briefings at trialbetter.com. Finally, please remember to comment, rate or review Trial Better on Apple podcasts or your favorite podcast channel. You can also reach us [email protected] Thanks for listening and stay safe, everyone.
Deborah Collyar has been a patient engagement leader since her first cancer diagnosis. She founded Patient Advocates in Research (PAIR) international communication network in 1996, \”where research meets reality.\” Deborah infuses hundreds of patient advocates into research programs, and delivers innovative ways to gather input from thousands of patients. Her work encompasses many diseases, programs and policies at grassroots, national and international levels and emphasizes patient issues throughout development, clinical trials, and health literate communication with providers and patients. She is a speaker, blogger, author, team member, trainer, and faculty at professional workshops.