Highlighting Clinical Trials | Advanced/Metastatic Bladder Cancer

Highlighting Clinical Trials | Advanced/Metastatic Bladder Cancer Part IV: Questions & Answers June 26, 2018 Presented by:

Andrea Apolo, MD National Cancer Institute

Arjun Balar, MD NYU Langone Health

Rick Bangs Patient Advocate

Question 1: Aren't clinical trials just for people who have run out of options? Dr. Apolo: No. Absolutely not. We used to have ... Basically most of the clinical trials were Phase 1, first in human trials and that's a lot of the work that we do at the NCI, and that's when we're testing new drugs. But once we see something is active, it moves from a Phase 1 to a Phase 2 where we have an active dose. And then we're really looking for efficacy. And then we have Phase 3s where we have really large trials, and we have a standard of care arm and then we have a treatment arm. And the trials are not only in patients that don't have options. There's also trials for patients with the standard of care plus. So, you get the standard of care plus an active agent versus the standard of care or not even ... It doesn't have to be versus the standard of care. It depends if it's a Phase 3 randomized trial or if it's a single arm trial. There's a lot of different types of trials so it's not only for patients that have run out of options. It's for patients that are just getting standard of care. They can get standard of care plus a novel agent. Rick Bangs: That's right. So, we don't get better unless we do some clinical trials. No matter what treatment we're getting. Question 2: Will I receive a placebo, which is often called a sugar pill, sometimes saline solution or some other form, will I receive a placebo in a clinical trial? Dr. Balar: This is, you know, one of the most common questions that I get about clinical trials is that will I get a sham infusion or sugar pill or no cancer treatment at all? And I will tell you that my response to patients is that, you know, the field of cancer research in the United States is heavily regulated and it's overseen by institutional review boards that make sure that

Highlighting Clinical Trials | Advanced/Metastatic Bladder Cancer Dr. Andrea Apolo, Dr. Arjun Balar & Rick Bangs

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not only are we treating patients safely, but they're always receiving nothing less than the standard of care. And so, and the only scenario where patients are receiving a placebo pill is where the standard of care could either be a standard treatment on its own plus a placebo or in a scenario where best supportive care on its own would be a standard of care. So for instance, there are some trials where patients are randomized to receive the standard of care treatment with an experimental treatment or they receive the standard of care treatment without any additional treatment and sometimes that is replaced with a placebo pill so that we don't know if the patient is receiving the standard of care treatment plus the experimental treatment or the standard of care treatment on its own. That's occasionally done. But the most important thing is that in any clinical trial this will be clearly disclosed. It will be in the Informed Consent documentation. Your physician who is reviewing the trial with you will have to clearly to spell this out for you, but occasionally we do it, but it’s always in the context of receiving no less than the standard of care. Question 3: Can I select the treatment I prefer from the choices being tested in the trial? Dr. Apolo: Well, if it's a randomized trial, the answer is no. You get randomized at the ... literally a random process done by a computer. But you can also select the trials. If you select a single agent trial that only has one agent or one arm of a combination being tested, you can select the trial that fits you the best. And that's a decision that you discuss with your doctor, that you discuss with your family, and you make it with several people involved. Question 4: Won't a new treatment be better than an existing treatment? Dr. Balar: I mean, that's a great question. I think we always want that the next treatment that we are testing in a clinical trial will be better. But we often don't know that. I do tell my patients that the best treatments in cancer are not from, you know, 10 years ago or 20 years ago. In some instances, not even from five years ago. We would like to believe that our best cancer treatments are now based on the science and the technology that we're developing now, but we don't know that for sure. And that's why we do clinical trials. Because we have to know for sure and that's when trials are involved. It's very unlikely that an existing ... that a new treatment will be worse than an existing treatment, but we don't know yet for sure if it's better, and that's why we do the trials. Question 5: Are clinical trials typically paid for by health insurance providers? Could you speak from the NCI, as well as from the institution side, about how a patient can be in a clinical trial and what would their cost be? Dr. Balar: Sure, I mean, I can speak from, you know, an academic medical center that's outside of the government so it's a private institution. And what I tell my patients and what is outlined specifically in the consent document and what's also in the clinical trial protocol is that what is covered by the clinical trial, which means it's paid for by the sponsor, the funding institution that is supplying funding for the trial, is the experimental treatment itself, if it's a new drug, and any treatments or procedures that are specifically research-related. Which means they're specific for the trial. Unless you were in the trial you wouldn't be getting these specific evaluations or treatments, and so that is all covered by the clinical trial.


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However, procedures or evaluations that we would be doing anyway in the course of a patient's care for cancer, those are traditionally billed directly to insurance. And in fact, actually Medicare mandates this. And so, things like routine blood draws, routine blood work, imaging assessments to see if the cancer treatment is working or not, the actual infusion of the medication. So, the procedure involved in, let's say placing an I.V. line or the administration of the drug. Those things are things that we would be doing for you anyway, and therefore those things are billed to issuance. Dr. Apolo: So, from the government's perspective, I work at the NCI in Bethesda, Maryland, and we are a very unique institute. Really there is no other place like that in the world, but we're a research hospital only. So, we only run clinical trials, and we only see patients in clinical trials. And there is no cost to the patient that are participating in our clinical trials. This is very unique. We're the only place that does this. But if you were in a hospital, a normal hospital, then generally the cost for the standard of care like Dr. Balar said is covered by your insurance, but the experimental agent is covered by the pharmaceutical company. Question 6: A participant mentioned that their doctor had not mentioned clinical trials. Is this something they should be bringing up with their doctor? Dr. Apolo: Yes. I would say absolutely. Bring it up. You know, sometimes the doctor isn't aware that there's a clinical trial for this particular setting. And sometimes it takes a little extra work from the patient to look it up or for the patient's family to look up what's available. Beacon is a great help in this setting because I know you guys have blogs where patients can talk to each other about clinical trials that are ongoing or how to get access to a clinical trial. But, you know, the sad thing is that there's so many clinical trials, and we need patients. And we don't have enough patients enrolling, and I think patients don't know how to get to the clinical trial. That's a big problem. Dr. Balar: Yeah, I would agree. I think there are certain situations where the standard of care treatment is the best choice for an individual patient. But then there are other instances where a clinical trial may be the best choice, even for a patient who's never received any sort of treatment for their cancer. And that discussion really needs to be transparent with the physician. I encourage my patients, you know, that if they ever feel that they want to get a second opinion ... now there are other physicians who may see and treat a cancer that it's always worthwhile to even get a second set of eyes to look at your case. And then lastly, you know, as Dr. Apolo mentioned, because we have so many trials out there, not all of them are created equal. And some trials may be a better fit for an individual patient compared to others. So, it's really important to, you know, arm yourself with as much knowledge, not only about your cancer, your diagnosis, but also, you know, treatment options that are out there that may include both standard as well as a clinical trial treatment. Dr. Apolo: I encourage patients always to get a second opinion. I'm a tertiary center, so often patients come to me for a second, third or fourth opinion, but I think it's really important to be informed and just to talk to different doctors treating your particular disease who have access to clinical trials. think it is possible for non-US patients to come to the US and participate in trials. Question 7: If somebody has participated in a clinical trial and they have a favorable response to the treatment that is being evaluated, the trial finishes, will the treatment be able to be continued? How


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does that patient stay on the treatment if it's indicated that it was working for them? Is there something they need to be able to do? Dr. Apolo: Well, this is generally pre-defined in the protocol itself. Most treatments, if it's for metastatic disease, go on indefinitely while the patient is responding as long as they can tolerate it. But some protocols state there will be only one year of therapy provided and not because of financial reasons, but just because we don't know if there's any benefit to giving it for a longer period of time. So, this is always pre-defined in the study itself. So this is something that the patient will be informed at the time that they enroll in the trial. Stephanie: Okay. Most excellent. And again, it never hurts to ask questions about it. Usually on the trial dashboard you'll see the PI's contact information. The PI is the primary investigator. They often have a phone number or at least some kind of a contact. You can certainly ask questions about it. And again, bring it up with your doctors. So, Rick, would you like to just talk a little bit about this further reading before we shut the program down? Rick Bangs: Sure. So, what you're seeing on the screen is an article that Tony Crispino, which is one of my counterparts, a patient advocate and I coauthored, and if you're interested in understanding about some of the issues that we would work with the researchers on relative to clinical trials, feel free to send me an email. I'd be happy to share this article with you cause it talks about how clinical trials are designed and developed from a patient perspective. So feel free to reach out to me using the email address you see on the screen.

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