Patient Advocacy Experts from PTC Therapeutics, Terumo BCT and Foundation Medicine

We were recently invited by Frank Dolan, founding member of Takeda, Amylin, and ACADIA, and currently CEO of Arsenal Advisors, to host a panel on patient advocacy, a key and growing issue in the industry. His panelists were Anne Bruns, Aurora Flores and Brian Tomlinson. We additionally invited Kasia Hein-Peters to add her wisdom. Here is an edited version of those conversations.

GREENBERG: What do you find in your interactions with patient advocacy organizations and with patients themselves that are the most important topics that are being addressed within the realm of patient advocacy? HEIN-PETERS: During the pandemic we all realized how social, economic and healthcare inequalities translate into higher death rates in underserved populations. Patient advocacy needs to focus on addressing these inequalities urgently. Also, more proactive approach to fighting disinformation is needed, to ensure that patients understand when they are being misled.

BRUNS: I think when we work with families in these organizations, they’re just really wanting to know what’s out there. And I think for a family who has had this extreme diagnosis coming into their lives and completely changed their lives, not only for the patient, but the entire family as well, they’re just looking for something. And I think our role in industry.

We can help offer that little bit of hope for them, whether it’s something that’s in early stages, or maybe it’s just listening to them, I think we can really just be that listening ear and an extension of support for this journey that they have unwillingly found themselves to be on.

TOMLINSON: I think for Foundation Medicine, what we certainly hear is they want and really want access. They want access to our testing. They want access to information which will ultimately help them to inform their decisions that they’re making and that that cancer treatment journey plan in conjunction with their health care provider. They want help. They want support. They really want to be included early on in the process to really help inform the strategy, whether it’s helping with clinical trial design or identifying and addressing unmet needs within a community. I also find they really want access to expertise. Folks to talk to, the medical team, our reimbursement team, our government affairs team, to really help them understand the information that’s in the Foundation Medicine report that they receive after they’ve had testing.

FLORES: And if I may share from the perspective of the Hispanic community, what we are finding with that community is that there’s really a lack of information that is in language that they’re able to understand, that they’re able to relate to. There’s a lot of cultural barriers. There’s language issues, also issues of documentation that people are having some challenges accessing medical care because they’re afraid that they will be stopped, that they will be questioned.

So really tailoring programs that are going to address their specific needs is some of the things that we have been listening to as well.

GREENBERG: Who are some of the most vulnerable populations? Who are some of the most marginalized populations? Who are the demographics that need the most help that you find yourself having to support? Do you have to do outreach or do you have to do remediation to some extent with the populations that aren’t getting the help that they need? HEIN-PETERS: People at the frontlines, who help to keep the society going and yet are the most exposed to the virus. The exposure, combined with financial hardship, lack of paid sick leave, inadequate or no insurance, and other access issues create a vicious circle, worsening the pandemic on one hand and increasing the disease burden on the other.

FLORES: The Hispanic community is very diverse. We have all kinds of populations from the very highly educated to those that are lower socioeconomic status, lower knowledge, education. And I tend to find those individuals who are of lower education are the ones with the highest need. They’re the ones that are not accessing care necessarily. They’re the ones that are not very compliant when it comes to their child’s treatment, because sometimes they don’t even understand how to administer a therapy.

They tend to not answer their calls when they’ve got somebody on the other line that speaks English, speaking their language, they are intimidated by some. Some of them are illiterate. So we think that, oh, let’s just translate a material so that they can read it, but not everybody actually can even read in their own language. So you find who those individuals are and then tailor programs and tailor educational brochures and materials to them.

GREENBERG: What about elderly populations, what about African-American populations? What about the poor? Are you in touch with some of them and the issues that come up for them?

TOMLINSON: Through the partnerships that we have with patient advocacy organizations, some do have, and have had for many years, educational resources and programs that specifically target those audiences.

Covid has certainly brought to light new health care disparities. And I’ve seen a number of large national cancer nonprofits actually start to develop additional diversity programing. But there’s lots of folks that don’t reach out to them for information or support. So how do you find those patients that are the most vulnerable and really need a lot of help?

BRUNS: In addition, we have so many patients that live in areas where there’s not a center of excellence, they might be the first patient their physician has ever treated with this disease. And especially when you look at rare [diseases]. And depending on economics, if they don’t have those resources and know who to go to, they’re not getting the information. And so reaching out to families, getting them connected with advocacy groups, social media support, whatever we can do to help connect them. I mean, that changes everything.

I remember going to a very small conference in Texas for my son’s rare disease. A nationally known doctor there who was one of the KOLs in this field spoke to a family that was blown away. and the mother is like, my son can go to school because it’s all like compromise. [They’d] never seen that before. The information they were getting was not 100 percent accurate. So that opened up a whole new world for her and that helped us create kind of a physician to physician directory so that when we had these physicians who were treating it for the first time, making their first time diagnosis, we could connect them with doctors in the field that had been seen, that were part of the research teams so that they can understand it just to make some of that information flow a lot smoother to some of these communities. HEIN-PETERS: The most urgent issue to address is a paid sick leave and access to quality healthcare. We need to stop accepting the current norm that some patient groups are simply left behind. Sickle cell disease is a good example, where patients, mostly African-Americans, requiring a transfusion therapy have issues accessing the best care, what results in poor disease management and suboptimal outcomes. Only recently has the access to red blood cell exchange therapy improved through changes in coding, but there are many other access issues to solve for this population.

GREENBERG: I’m wondering what you see in other companies that use the term patient centricity and what they really mean by it. It’s related to patient advocacy, but do you see what is being done or what isn’t being done in order to affect the patient centricity model?

HEIN-PETERS: Interestingly, I have not seen yet companies stepping in to address inequality issues. It seems that we are still trying to figure out what we can do above and beyond our usual support for market access.

TOMLINSON: I think there are companies that talk about it. And then I think there are companies that actually do it. We really work across the entire ecosystem as we lead our external engagement efforts with both the patient community as well as the professional communities, patient advocacy organizations.

We work with patients and caregivers. We work with grass roots communities. We work with professional societies, medical institutions, consortia. And I think to really be patient centric, you can’t do that without talking to patients and engaging with those stakeholders.

We’ve formed an incredible group of individuals called our Patient Community Council, which consists of six people who have had comprehensive genomic profiling done on their cancer. And we work with them throughout the year on many, many different projects where they give feedback, they challenge us, and they really help us to create what we’re doing for patients. Our singular goal as a company is to help patients get access to information that can help them make an informed decision about their cancer treatment plan.

BRUNS: I’m fortunate that I found the company that I mean, I’ll be biased. I love our company and our expectations for working with patients. We have the ability to bring in organizations and patients on a regular basis to talk to the entire company. It’s global. Everyone from finance to regulatory, when they come here and put a face and a story with a product that we’re talking about on a daily basis, it’s huge. We always say we work for a cause, not a company. And that supports us in everything we want to do on the patient advocacy side. I can tell you all about my son’s disease, for one thing, but I don’t know all the diseases that I work with. And so I rely on our really great partnerships and our relationships with our families to understand that so that we can make better informed decisions.

FLORES: I’m biased as well, but one thing that I noticed, I was brought on board about a year ago and my colleagues shared with me that when they were out in the field, they felt frustrated that they were unable to support some of the Spanish speaking families because of the language barrier. They went to management and said, this is what we need. And until we get someone that speaks their language and that can connect with them, we’re not going to be able to reach these families. So they heard them. They hired me. I came on board and I’ve been amazed because even translating some materials, they will always ask for the feedback and really take it to heart. Other companies say, oh, but this is the way that it’s translated. This is the correct way to say it. It may be grammatically correct, but it’s not culturally translated. So the fact that my company really takes to heart my feedback, my insight is a big deal because some of those cultural nuances [are important].

TOMLINSON: I guess we’re all biased. Foundation meaningfully works to support patients. And I think one of the pieces that’s key is what you both said, bringing patients in to every town, every all-staff meeting we have, featuring them in our all-staff emails, featuring them in our department team meetings, letting them meet the employees, because there’s so many people that work at these companies that never have the opportunity to really interact with patients that are benefiting from the work that they do, whether they work in finance or accounting or facilities or lab operations, they never actually see that patient and they never get to hear from them. And we take a concerted effort to spend a considerable amount of time at our all staff meetings listening to patients, listening to caregivers, hearing about their experiences, featuring our patient advocacy partners. And you can see how captivated the audience is when they’re listening to those stories. One piece that I think is critical to the success of our types of roles is really buy-in from our executive teams in the value and the importance of investing in positions like the ones that we have.

BRUNS: We’re all passionate. And we do have executive teams that support us in that. We’re consistently thinking about families. I wonder how so-and-so is doing. It’s a 24/7 job just because we we’ve become so invested and we think that that shows the families that we actually really care. It makes a huge difference.

TOMLINSON: It’s so disheartening to me when we hear from patients that call us and say they’re demanding to have their cancer tested and their physicians are refusing to do it. And how do we help them with that? The reality is they may have to go to a different physician. A patient had reached out to one of the patient organizations that we work with. She is well-educated. She’s a nurse. She was recently diagnosed with ovarian cancer and she received Foundation Medicine testing. Her physician wouldn’t give her a copy of her report, which she is entitled to, and told her that the report didn’t have any useful information on it for her. And she was really frustrated. So she reached out to the patient organization and we arranged for her to be able to talk to one of our medical science liaisons, who was able to walk her through her report. And there were actionable items for her that now she has that information, she understands it, and she can take that to her next appointment with what is now a different physician. We have to be there for these people. And it’s such a challenging time in their lives.

GREENBERG: I’m wondering how you and the organizations you work with are able to affect and improve the physician patient relationship, because I know that’s also a big and related topic. Some physicians don’t communicate well, don’t know what is expected of them in terms of communication. What is your role in in helping to facilitate the doctor patient relationship?

HEIN-PETERS: Doctors and nurses are natural patient advocates and yet they do not always have time to explain the disease and treatments. They may not understand relationships between social, economic, cultural and environmental determinants of health, so their recommendations for patients may not take into consideration the realities of their life. First, doctors need more time with patients and less spent on bureaucracy. Secondly, we need a more 360 approach to disease management, considering all determinants of health.

BRUNS: I’ll say it’s aimed at providing them with resources, whether it’s a patient advocacy organization or where to find information so that they can in turn take that to their physician and have a higher level of discussion. When I talk as a parent, when you get this diagnosis and you’re sitting there in the hospital room and you have your physicians in white coats and they seem so overwhelming and they seem so smart, and you as a parent are like, I don’t know what they’re talking about. Once you start to become educated and aware of options or just the terminology, just being able to understand the terminology, it opens up a whole new level of discussion with your physician on care treatment options. Are there clinical trials? No. Brand new family is going to go to ClinicalTrials.gov and look for trials.

GREENBERG: A friend of mine is a relatively young woman but has a number of comorbidities, some fairly serious, and her research has led her to educate and provoke some of the physicians she’s been engaged with so that they become better at what they’re doing. Is that your experience?

BRUNS: You have to look at this diagnosis and find the right doctor. It’s a partnership and you’re creating a care team and you’re part of that care team because ultimately you’re going to learn from each other. And we don’t want to discount any physician’s experience. They have a wealth of knowledge. But it may be their first time seeing this information, and we have to learn together and be able to build that relationship. And I think our role is to just help provide that information or just where to find that information.

FLORES: We build these educational programs for families where we cover these topics, where we empower that patient with knowledge. Before they can have these conversations with their doctors, they need to know what is out there, what is available to them, what they should be requesting. They become more knowledgeable. They feel more confident having these conversations with their doctors. But we do that by providing these educational programs, by creating trust with the community, by having conversations about what is it that you’re going to discuss with your doctor. A mom walked in with the information that was given to her and she felt so great after this appointment about her son’s dosage. And she’s like, just by taking this information that I learned about from this conversation we had, the doctor made a switch to give the child what he had been previously taking. Patient empowerment is certainly a key to being able to get these families to really talk to their doctors about their needs.

GREENBERG: What I hear very often is that executives say we want to imagine a patient in the room at every discussion we have, and wonder what that patient is thinking about how we’re talking about them. Do you see that in reality? Somebody told me recently that he was with one of the big pharma companies, and he said their reps never talk to patients. That doesn’t seem to be a very good path to success. They’re talking to the doctors, but they have to know what the patient’s point of view is, too. TOMLINSON: Bringing patients in for topic-specific discussions or projects or planning is so key. We do it at Foundation Medicine all the time. We’ll hold what I call insight, meaning so many advocates with a particular cancer focused number of groups to really get their direct insight about what they’re hearing from their constituents, so that we can then bring that back to the broader team to share that perspective. When I think back to my start in oncology, I’m not even sure that these positions existed. GREENBERG: There is so much bad information out there, and mediocre information, and of course good information. Is that a lot of your activity, helping the advocacy organizations sift one from the other and really understand what the good information is?

TOMLINSON: I would say if you’re if you’re a patient and you’re going to an advocacy community, an advocacy organization and a reputable one, that’s really your best source of information and support. And we have material on our website that patients can use to understand comprehensive genomic profiling, to understand the report that they have to ask questions of their physician. But I think we would all agree that really the main source of that information and where patients would go to look for it would be a nonprofit patient organization as a trusted, reliable, credible source of information. I think the challenge we have is not every patient goes there or sometimes patients reach out, but they reach out.

HEIN-PETERS: First, communication cannot be an afterthought, like it is for example with Operation Warp Speed. Well-intended and helping vaccine companies to move really fast, the OWS ignores public opinion and doesn’t address concerns related to the “rushed vaccine development.” As a society, we will pay for this by slower vaccine adoption, hence a longer pandemic in the U.S. Complaining about anti-vaxxers or conspiracy theorists does not help. Instead, transparent, understandable, empathetic communication from a trusted source should be developed from the start of any new vaccine, drug or medical device development, especially when brand new technologies are used. Secondly, we should educate the public how to differentiate good from bad. There are some simple ways to do it, which I described in this short article on LinkedIn: https://www.linkedin.com/pulse/immune-infodemic-kasia-hein-peters-md/. And, last but not least, I applaud the recent WHO project of educating communication professionals how to fight misinformation and disinformation: https://www.who.int/news/item/30-10-2020-start-of-1st-who-infodemic-management-training.

GREENBERG: Are there situations that come to mind, specific anecdotes or just general situations that you can think of in which you realize that this is what success looks like? What are some of the things that have happened in your activity that say to you, we’re doing it right?

BRUNS: I was at a national hematology conference as a nonprofit, and we had a patient with us and we were looking at this poster on her condition. And the researcher came over and was talking to us about it. And I said, well, she actually has this disease. And the researcher was just like, what? And he had never met a patient before and researched this for years. I think they were there for a couple hours, just chatting. That connection was huge because he going to take that back to the lab, to whatever he does with a different perspective. We all meet these families and you look at them and you can see you can see the fear in their eyes. You can see the stress. You can just see that weight on their shoulders, that they are so overwhelmed, they’re so tired. And I think for me, after having a conversation with a parent or a patient, being able to kind of just see that weight and just kind of that relax, even if it’s just for a split moment, is success to me, because just being able to take some of that pressure off is like that success is just helping them feel listened to, that they were heard and that they were supported.

FLORES: Some of the families that I’ve worked with, when I have conversations with them, I can tell I’m the only person that they’ve really opened up to. They tend to isolate themselves. They rarely have support groups. It’s not something that’s very big in our community, whether it’s because they’re ashamed, because they feel weak, by asking for help, by relying on others. Whatever it is when we bring programs to them and we bring other families to them and they connect this way. They say, I’ve seen this mom or I have seen another patient like mine at a clinic, but we don’t really talk. Well, when we bring them into an intimate setting like this, they somehow open up and they start sharing stories and they start exchanging contact information and then they stay in touch. For me, that is success because we are providing a service. We are bridging the gaps for these individuals.

TOMLINSON: I think you also have the small successes, connecting that patient to someone who can help them understand their disease. And while that’s not helping the masses, you’ve helped that one person. And sometimes if it’s that one person I helped that day, I feel I feel good. And then there’s the larger successes when you can work with multiple organizations on initiatives for education information. You can work with organizations to advocate for access to treatments for patients that don’t have access to them, access for testing. And those are the big successes.

GREENBERG: And so part of what you’re talking about, Aurora, is not just that you deliver information to patient advocacy organizations and to the patients themselves, but that the interaction between the patients is also a very important part of this, that they share information and that that helps them both in the course of their therapy and in feeling understood about other people getting what was going on with it.

FLORES: Yes, absolutely. A lot of these families, they feel very alone is what I hear a lot I feel like I’m the only one going through this. So when they meet another family that is going through the same thing, they’ve been through that journey and they can support each other and help each other. It’s really a wonderful thing to see. I had a mom at a program saying you need to come to our support group. We meet once a month. And that family had never been to a support group. She started to attend. So those are successes.

TOMLINSON: That piece is so critical. There’s so much focus on the therapeutic and the treatment that that oftentimes folks forget to address the emotional need that that family is going through. It’s not just the patient. It’s everyone in their circle of connectivity. And they may not even recognize it at first, but they need help. And it used to be so rewarding for me when I worked at a nonprofit and we would bring together a bunch of cancer patients and they would have lunch together and they would network and they would form these longstanding bonds and you would see them coming back year after year. I think it’s so critical and so important.

BRUNS: We have a program for just teens, all with the same disease, and there’s a bunch of them sitting at a table, and this one young woman started sharing and she’s like, “Well, I can’t because I have – “ And then she stopped and she’s like, “Wait — you all have the same thing!” And that group of young adults is in college now and they are still strong. And to just have that instant connection of somebody else gets it. I think the mental aspect is can be just as important as any type of therapeutic. They need that emotional support, I’m sure.

GREENBERG: I want to give each of you an opportunity to list a few bullet points. If the people listening to this or reading it want to imitate the kind of success and the kind of interaction that you’ve had, what are a few of the bullet points that you think are important for them to institute in their own companies?

HEIN-PETERS: Focus on what matters to patients and not only what matters to companies, engage in long-term relationships, not short-term opportunities.

TOMLINSON: When you think about your external or internal stakeholder environment, really think about it broadly, right? Don’t just think about patient advocacy organizations. Think about organizations, individual patients, grassroots communities, influencers in that sphere, because they’re all part of that broader ecosystem. I would strongly suggest external stakeholder engagement early and throughout the process. I always like to say, ask, listen, learn and then act, do something with the information, engage throughout, throughout the years, the projects, keep them informed. They really want to know what’s going on.

BRUNS: I would just add, you have to be out in the field, you have to be in those conference rooms. You have to be talking to patients and then bring that back. And then that needs to be incorporated into every department within the company to be fully patient-centric, to actually be a company that cares about their patients. Every company needs to be aware of how these families are dealing with this journey.

GREENBERG: So that even the people who are not customer facing or patient facing within the company need to have that awareness because that will affect the whole activity of what everybody does?

BRUNS: I think it’s inspiring. We had someone from finance kind of want to know what we did, and we had a whole conversation. We showed photos, and she wanted to start coming to our meetings because she mentioned I’m so inspired by these families. And so no matter where patient advocacy sits in a company, it has to be part of every department in some capacity.

TOMLINSON: Patient advocacy is a point of connectivity across the entire company, not one piece, everybody: marketing, commercial, research and development, any team that you’re working with, payer teams. And some of your key partners, executive team engagement and what that looks like. And that doesn’t mean every day or every week, but the executive team touch points with some of your key stakeholders that you’re working with. It means a lot to them.

GREENBERG: So if they’re talking to audiences that don’t necessarily speak English as a first language for audiences that have trouble understanding the medical terminology, that’s important to see people as different kinds of groups and even different kinds of individuals.

FLORES: You actually took the words right out of my mouth because that’s exactly what I was going to say, is that depending on who your audience is, right, your target audience, you want somebody that is representing that community to go out and talk to that community. They love it when someone can speak their language. But if you’re trying to reach the African-American community, or Asian-Pacific Islanders, make sure your staff is representative of them, or somebody with that disability or that condition. Because we only understand those cultural nuances. It’s not just that we want to give them what we think is best for them, but what do they want? We actually took the time to speak to the families and ask them, what do you want to know about? What are your concerns? And I heard things like, how do I get support for my child at school and nutrition? My child is gaining too much weight. So we identify somebody to talk about higher education or through AP programs with children who have disabilities. And mental health services identified a Spanish speaking psychologist that can be able to talk to these families. We had a program where there were moms that said, this is the first time I’ve ever even been able to talk to psychologists. I’ve never spoken to a psychologist before, a therapist. And then another important point that was made to is staying in touch with the families. So after we have brought these programs to them and these services stay in touch, let’s not just go to the community and check the box and say, OK, we did that, but maintain that relationship, check in with them. How’s it going? Is there anything else you need or how can we support you?

Anne Bruns - Director, Clinical Patient Advocacy

PTC Therapeutics

Anne has served as Executive Director of the Atypical HUS Foundation, Executive Consultant and Rare Disease Advocacy and Patient Communication Expert for Optio Biopharma Solutions before coming to PTC, where she was previously Associate Director, U.S. Patient Engagement.

PTC Therapeutics, since 1998, has been a biopharmaceutical company focused on innovative small molecule and gene therapies for rare genetic disorders. They operate offices and/or research labs in more than 20 countries and occupy a business footprint in more than 50 countries around the world. Recently, they expanded into oncology and virology therapeutic research.

Aurora Flores - Associate Director, Patient Engagement

PTC Therapeutics

Aurora was Health Educator, Every Woman Counts for the California Health Collaborative, Project Manager for ADEPT, TEAM Lab Evaluator at the University of Southern California, Community Educator at Quintiles, and Enrollment Development Specialist I at AltaMed Health Services before joining PTC.

Kasia Hein-Peters - VP Value and Health Outcomes

Terumo BCT

Kasia has been a Global Brand Director for Novartis Vaccines and Diagnostics, head of Blue Impact Consulting, a board member of Epidemico, Vaxelis Marketing Head at Sanofi Pasteur, and previously VP Global Marketing and Communications at Terumo BCT before assuming her current role. As VP Value and Health Outcomes, she addresses the trend toward stronger evidence-based medicine models for payers, providers and regulators.

Terumo BCT is a global leader in blood component, therapeutic apheresis and cellular technologies, believing in the potential of blood to do even more for patients than it does today.

Brian Tomlinson - Director, Patient and Professional Partnerships

Foundation Medicine

Brian was Chief Program Officer at the Lymphoma Research Foundation, then served in the same role for CancerCare, and moved on to become Director of Patient and Professional Partnerships at Foundation Medicine.

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient’s unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patient’s cancer and match them with relevant targeted therapies, immunotherapies and clinical trials.