The impact of disease burden: Understanding patient priorities in aRCC
Dena Battle – President KCCure, Alexandria, USA
Prof Tom Powles – Barts Cancer Institute, London, UK
DB: Hi, I’m Dena Battle, President of KCCure and today I’m here with Dr Tom Powles and we’re going to be talking about the impact of disease burden and understanding patient priorities in advanced RCC. Tom, if you want to introduce yourself, say hello.
TP: Hi, I’m Tom Powles, I’m an oncologist.
DB: These are our disclosures. So, Tom, here we are, it’s 2026 and this is probably a brief thing but there are a lot of systemic therapies now for advanced RCC. I think it’s changed a lot, certainly in the last ten years, we have a lot more treatment options. So I don’t know if you want to comment on that?
TP: I think it’s good, I think outcomes of patients have really improved and I think that systemic therapy has made a big difference. I think surgery is similar over the last… there have been some changes but in the end we are operating on similar patients with similar outcomes. And local therapies, while they’ve become more popular, there’s not a huge amount of data around those at the moment. It has really been systemic therapy that’s driven this and we lack biomarkers, of course.
DB: With all those options now, thinking about patient experience and their preferences, because what doctors think is important and what patients think are important aren’t always the same, the patient experience and what they’re going through, doctors have a lens and a very keen observation but they might not always have a full picture of what patients are living with at home. So, as you probably know, KCCure, we’re a patient advocacy organisation but we’re an evidence-based driven organisation, we do a lot of surveys. We did a survey, this is from our 2022 survey, we had a thousand patients who responded, around 400 had metastatic disease. We asked this question, we’ve asked this question on all of our surveys: when thinking about therapy for metastatic cancer what is the most important desired outcome you want to see from treatment? This is a rank file question and what we see repeatedly is that patients want a chance of eliminating all evidence of disease, this is their number one goal or hope from their treatment, and then durability is second. We’ve seen this in both our 2022 and our 2024 survey. Anything surprise you about this? Probably not.
TP: It’s fairly obvious that people want to get rid of their cancer. One of the challenges as systemic therapy has evolved is that’s become a realistic goal. If you went back ten years that wasn’t really a realistic goal. So while it may be the priority, I’d like to be a popstar and that would have been mine but that’s never going to happen. And that was true when we go back ten years or even 20 years in this disease. So I think that’s different now, I think we are seeing cures of patients. So I think patients are right to come in saying, ‘I want to be one of those patients that goes into a long-term remission without evidence of cancer.’ I think it’s a realistic goal and it’s one that we need to drive towards.
DB: Then this was another question we asked: how would you define long-term response to treatment? Interestingly enough, when this question came up it was a doctor had asked us to add this. The doctor’s perspective was that two years would be considered a long-term response to treatment and what we see is patients have a much longer perspective of what is considered long-term response.
TP: That comes back to that previous point where actually the progression-free survival for a lot of these systemic therapies is about this time. So we’d like patients to be above average, of course, but we are realistic around that. The reality is that we do really well with a subgroup of patients but it’s less than 50% and therefore when you look at averages doctors are always going to come in with average figures. Clearly I don’t think there’s any cancer that I can think of where patients are going to be satisfied with anything less than two years. I don’t see anyone, even in pancreas cancer where we know the outcomes are really poor, the aspiration for everyone must be longer than that. So I think this is entirely reasonable.
DB: We developed this concept knowing that patients want curative outcomes, this is what we call disease burden. There are some of those patients who are going to make their way to a curative outcome but it is still really small. In the meantime can we think about ways to get more patients away, not away from death but away from being so consumed by their disease? That’s where our 2024 survey we really delved into this concept and this was, again, over a thousand patients, 492 had metastatic disease in this case. So if we ask patients, these are questions that we ask patients on cure, so this was the percentage of metastatic patients who can be cured based on all available treatment. So this isn’t ‘Do you think you can be cured?’ or ‘Are you cured?’, this was ‘What percentage of patients do you think can be cured?’ 20% said none, 17% said ‘I don’t know’. It was interesting because when we asked this question in a provider survey none of the doctors said, ‘I don’t know’, which I thought was interesting. But the takeaway here that I thought was interesting is 55% believed that at least 10% can be cured. So there is an understanding that, you talked about this too, some patients can be cured. That seems about right, around 10% maybe can have a curative outcome.
TP: I think that’s reasonable, yes. Our aspiration is more like 30% and I think we’re probably getting closer to 30% than 10%.
DB: So you’re more optimistic. But when we asked patients whether they believed their disease was cured, to describe their own situation, only 2% said that they believed they were cured and 46% said that they were incurable or terminal, 29% not yet cured. And then we had an ‘Other’ with comments and most of those comments came along the line of ‘I’m not sure if I can be cured.’ A lot of uncertainty surrounding this terminology about cure.
TP: I think doctors are very uncomfortable with the word cure and they don’t like talking to patients about that. Because if you’re cured why do we need to follow you up? If you’re cured does that mean I can plan the rest of my life? And if you’re cured and the cancer comes back does that mean you’ve let me down? So that’s quite a complicated issue. I use the word cure sometimes in this disease but I wouldn’t do it until a patient had been a few years down the line.
DB: So that comes to our next question. When we ask patients how has your doctor described your disease and here, to your point, it was even less – only 1% said their doctor had told them that they were cured. 41% said they were incurable or terminal, 38% just didn’t talk about the term at all. So I think that reflects this discomfort with uncertainty, especially knowing we’re just not aware of what the long-term outcomes are, risk of relapse, things like that. So, coming back to this concept of disease burden, if we think of these patients who are cured but then we think about patients who are on systemic therapy for a long period of time, over time they become consumed by their disease. We wanted to look at four categories.
DB: Coming back to this concept of disease burden, what we wanted to do was measure this concept of the opposite of cured. As patients are on systemic therapies for longer, as these therapies are being sequenced, over time they become really consumed by their cancer. There were four areas that we wanted to look at, one was uncertainty – how confident do you feel making plans about your life, short term and long term? Time toxicity – how much time are you spending at the doctor’s office, in clinic, getting blood draws and how often are you hospitalised, things like that? And then emotional burden – how much of your day is spent thinking about cancer? Then daily disruptions, and we define that as just three things – eating, sleeping and interacting with family and friends. So this is just to go through those results that we found in our survey. So how confident do you feel making decisions, making plans, next week, next month and in six months? As you can see, if we compare the patients who are on treatment and have evidence of disease, compared to patients who describe themselves as off treatment and NED, it’s a really big difference of how confident they feel making plans for their life.
TP: I think that makes sense as well. You would expect making long-term plans is quite difficult in this situation. Even if you’re confident your cancer is under control I don’t know how comfortable you’d feel tempting fate, making five-year plans. I just feel the people who I see will trust their luck a little bit but not so much that they… Many say, ‘I’d like to make my daughter’s wedding in two years’ time’ or ‘I’d like to reach this event’ but I don’t think many people are planning holidays that far in advance because it just tempts fate a little.
DB: You can see just in the next six months you would hope that people would feel sort of confident to plan a trip or something like that but just over a quarter of patients felt confident, those patients who were on treatment with evidence of disease. It just shows this high degree of uncertainty that people are living with and that’s just one element of the burden. Then this question: in the last three months how many clinic visits have you had? This was really high, it was higher than what I would have thought – 75% have had three or more. So if you think about normal circumstance, most patients are going every three months, right? Over a quarter of patients have had clinic visits more than five visits. So from a time perspective that’s a lot. Does that surprise you?
TP: I think it’s a reflection of some of the complexities of systemic therapy. I don’t think this is the easiest journey and I think at times, even in the longer-term follow-up, we’re still seeing patients quite regularly. We see patients monthly, very few patients once every two months who are on systemic therapy.
DB: Yes, so they’re coming in pretty regularly. If you look at the data on this, generally a clinic visit is a full day for a patient, it’s a full day off work if they measure the time associated with that. So this is pretty significant time toxicity that patients are dealing with. Over a quarter said that they had been hospitalised in the last three months and, out of those patients who were hospitalised, more than half of them were in the hospital for more than five days.
TP: Those numbers are quite high I think. My experience in my clinic is that at any time probably only 5% rather than 26% of the patients on systemic therapy are in hospital. So this is probably a little bit different from our experience. We have very few kidney cancer patients who are in hospital for more than five days. But, having said that, the data is the data.
DB: It’s certainly not representative of all situations but we thought these numbers were at least concerning and worthy of consideration. I think one of the things that we see in our communities at times is when a patient has really advanced metastatic disease and they go into the hospital it can be hard to get out, right? There’s always something, your labs are always a little bit off, there’s always something coming up. I think that can be challenging for patients when they’re in the hospital and they’re not necessarily working directly with their care team, they’re working with the hospitalist or whoever is in the hospital at that time. So I think that can be very frustrating and difficult for patients. Then this emotional burden, this really was heartbreaking to me how much time do you spend per day thinking about cancer. 90% think about it every day, which isn’t surprising, but over 50% say they spend hours thinking about cancer. I thought that was really sad.
TP: For my take on that, this is probably the most troubling of all the slides I’ve seen, particularly after the first six months. I’d like my patients really to only be thinking about cancer when they’re coming to see us on that day, that would be the ideal world. This is very different from that. It doesn’t surprise me and it shows how devastating a cancer diagnosis that requires systemic therapy is. This is a very sobering slide, even for those patients who are doing well.
DB: Yes, this was very heartbreaking for me. But not surprisingly because I have my own experience with cancer and certainly as an advocate working with patients every day the emotional distress is really difficult and it’s managing side effects that you’re dealing with every day but also that existential angst that comes with knowing that you have a disease that, so far, you have not reached that cure goal. Not knowing how long that systemic therapy will work for you, that uncertainty that you’re living with is really unsettling. This was the daily disruptions, how frequently does cancer impact these activities – eating, sleeping, socialising. In the survey we phrased this as interacting with family and friends. 44% of patients said always or often for eating, which this is something that we see in our data. When we ask patients about side effects that are most distressing for them, that are most difficult to manage, fatigue is number one but number two and number three are taste loss and appetite loss. These are things that don’t really get discussed that often with doctors or in trials and things like that. We talk a lot about diarrhoea, we talk a lot about hand-foot syndrome, things like that, but this inability to eat is a real quality of life aspect that we might be missing a little bit.
TP: Yes, there was a study back in the day called the PISCES trial that looked at patient preference and asked patients what side effects troubled them the most. Change in taste came near the top so that’s a reflection of the mechanism of action of some the systemic therapies that we use, so it’s not true of all systemic therapies. But for kidney cancer I think it is a big deal.
DB: Sleeping, interacting with family and friends, these are high percentages for always or often, in my opinion. It just shows how disruptive cancer and living on continuous systemic therapy, how disruptive that is for people’s lives. So if we come back, if we think about these four categories to have a scale, patients who are really thinking about cancer all the time, their daily activities – eating, sleeping, interacting with family and friends – their sense of uncertainty, the time that they’re spending, their life is just really consumed by cancer. All the other things that bring us joy, the things that we look forward to in life just become usurped by the disease over time. So if we think about our goals of treatment, obviously cure is part of that but there is this quality of life aspect that mirrors a curative outcome and the ability to stop getting treatment and to have fewer interactions with doctors, fewer interactions with hospitals. Not that we don’t really always enjoy talking to doctors, Tom, but I think this idea of reducing that as our treatments move along, trying to not just increase overall survival but also reducing disease burden over time.
TP: Should I say something at the end of this?
DB: Yes.
TP: I think this is really interesting. I like different parts of it. I’m not surprised by a lot of it but the bits I’m not surprised about is the uncertainty around cure, it makes a lot of sense on many levels. But it is important to recognise we are curing some patients with kidney cancer and we weren’t doing that before. The time commitment work is really compelling; it’s problematic if patients are spending 20% of their time in a hospital environment. Certainly anything more than 5% after six months of therapy I think would be a problem. The final component of it, and I think the really interesting component, the most interesting, is how patients continue to be troubled by their cancer. It suggests to me that some of the appointments we’re making with patients focussing on symptoms and treatments and perhaps less on expectations and coping mechanisms, there’s probably quite a lot of work to do there. So there’s a lot to be said for this and I like the way you’ve done it.
DB: From the perspective of your own clinic and maybe thinking for other doctors, one thing that I’ve thought about is how aware are you of patient appointments? Obviously if one of your patients is in the hospital you would know that, but there are other appointments. If people are dealing with certain side effects maybe they’re meeting with palliative care as well, sometimes they’re meeting with other specialists and things like that. That time toxicity, I’m not sure doctors aren’t fully aware of that, if that makes sense.
TP: Yes it does. It’s not something we talk about at all. We talk about financial toxicity, we talk about treatment toxicity but we don’t talk much about time toxicity.
DB: This is just an introduction to these concepts and we think there’s a lot more work to be done but hopefully this will just get doctors and patients and all stakeholders to think a little bit holistically on all of these different levels.
