It’s a pleasure to be here. It’s a pleasure to see all of you here. I want to echo our gratitude for coming to the eCOA Forum and for engaging in some pretty important discussions over the next couple of days. I’m really looking forward to what comes out of the discussions themselves. I’m really excited to introduce our first speaker. I don’t think there’s any better way to kick off the discussion than talking about the patient perspective. So TJ, I’ll encourage you as Bill mentioned, there’s bios of all your speakers in your folders, I encourage you to read that in detail. I won’t embarrass TJ, but as you can see it’s a harrowing story. I have no doubt that after his discussion you’re going to feel compelled to visit his site and hear the story a little bit more. But thank you to TJ for joining us.
I’ll just do a brief introduction. TJ is a writer, speaker, advocate, and patient advisor to the biopharma and clinical research industries. TJ Sharpe will share his personal journey as a stage 4 melanoma survivor. So thanks TJ. Please welcome him to the stage.
All right. Thank you. Good morning. I have 29 minutes and 57 seconds to give you a patient perspective. And at the end of this we’ll do a couple Q&A and I’ll be on a panel later, so feel free to ask any questions you need. My HIPAA rights have long gone by the wayside, so I’m here to talk about what it’s like to be a patient in a trial, and how being involved in a trial, what patient-reported outcomes, what COAs are, to us and how when you become a patient on a clinical trial it’s more than just the stats and the ability to put something in a PalmPilot, which I did have. It’s understanding the journey a patient and their family takes and what it means to them. So without further ado we’ll get right into it.
I’ll talk to you about the calm before the storm, as we call it. And I’ll skip ahead. This is me now. That’s my family up there. And a quote from one of the presentations I had. Not to give away the ending, but I am still here, which is a great thing. But to get from where I was to here took a lot of hurdling, challenges that I faced as a patient that are very typical of what many patients encounter when they look to find a treatment or try to find a clinical trial that will give them an outcome that they want. And that’s kind of the key word that you’re going to hear over and over again, over the next two days. And my outcome was not measured on a graph. It wasn’t measured by a median lifespan. It was measured in time that I was going to get. And you’ll see that the time I wanted to have to watch two little kids grow up.
So I’ll start with a timeline real quick and tell you sort of how I got to where I was. When I was 25, I was diagnosed with stage 1B melanoma. The dermatologist took a little slice out of my clavicle and said, you need to go to NYU Medical Center and don’t believe everything you read on the internet about melanoma. So when I went home, on my palm pilot prompted Google melanoma, I realized I had become a young adult cancer patient. I was an IT project manager, I lived in northern New Jersey, and I didn’t know what it meant to be a cancer survivor.
So I did what every cancer patient would do. After the surgery, I wore long-sleeve shirts, bucket hats, lots of sunscreen, and then I moved to Florida, which is where I met Jen. And we got married in 2008. Had this little baby girl in 2010. And 2012, had our second child, our son, Tom. That’s all a pretty good timeline and it’s a pretty happy story at that point. Survived cancer, find the love of your life, get married, have kids, sort of the American dream.
Sixteen days after he was born, I want into hospital with what I thought was a slight fever. The ER doctor took a look at the chest X-rays, my blood work, and say, you have history of melanoma, I think you have a recurrence of your cancer. I said, Doc, I just had a little baby boy, I think you switched my chart with somebody else. He said, no I’m pretty sure you have cancer. So I checked into the hospital.
And when I left 16 days later, I had a stage 4 melanoma diagnosis with an 8 cm tumor removed from my small bowel, and four other tumors spread across my lungs, my liver, and my spleen. But when I left there, I had a few more things. When I left that hospital, I knew how bad my diagnosis was. In 2012 when I was diagnosed, the median lifespan for someone with stage 4 cancer was about 18 months, and that nine out of ten people with my diagnosis would not be alive in five years.
So when we left, we had a few things. We had goals. I wanted to give myself the best chance of getting a durable response. I knew what the statistics were because I had spent 16 days in the hospital continuing to Google melanoma. I also knew that there were medicines out there, this immunotherapy world, that was really on the forefront of cancer research, but were so new that there wasn’t really much evidence around it, certainly there wasn’t any approvals. So I wanted to give myself a chance to get a durable response. And when I did that I wanted to get healthy. I wanted to do things like go to my cousin’s wedding. I wanted to take my kids to Disneyworld. And I set all these goals when I was in the hospital. Because my purpose wasn’t just to be a cancer survivor, wasn’t to live months longer. I had a purpose. My purpose was to be a dad, and to be a husband, to be a brother and an uncle, to be a son.
And so when I left that hospital, this time it was different. With this cancer diagnosis, I had a different outcome. It wasn’t just get over cancer; it wasn’t just make this a chapter of my life. I had a goal and a purpose to achieve a response, to give me the chance to do all of these things that I kind of took for granted. To get there, there was a number of things that we had to overcome.
The first thing was finding the right treatment. I mentioned before the statistics in stage 4 melanoma in 2012. So while I was in the hospital trying to overcome these barriers and finding myself a treatment that would work for me, I did all my research and I realized that there were these immunotherapies that were just in Phase I trials. That sounds like a pretty good option for me. So I left the hospital and I went to talk to the oncologist. And I did it in a general hospital, general oncologist, he was not a melanoma specialist, didn’t know much about clinical research or at least didn’t tell me much about it. So we went in, and Jen asked the doctor, all right we know this is bad, what are we looking at here, Doc, how much time do we have. And he gave her a very honest and very blunt response, said, I’ll be surprised if your husband’s here in two years. I had a four-week old son and a two-year-old daughter, and I had that goal of getting a durable response, I had that purpose of being a dad, being a husband. That wasn’t enough.
So I bit my tongue, when we left the doctor’s office. I said, all right, thank you for setting my first goal for me. And we left. And that night we started researching. Okay. Yeah that was a tough day. But there is an opportunity out there for us to find something that might give us a chance to achieve that goal of a durable response, to get healthy. And I went on four second opinions before I finally found someone who was willing to offer me a clinical trial. She gave me the choice of two different trials. Option A was anti-PD-1 drugs, which I’m sure many of you have heard, have been in the news a lot recently. We have a Phase I anti-PD-1trial. Or option B, there is this procedure called TIL, so you take a tumor out of you and find the T cell of it, that’s your cancer, and then we’ll reinfuse them back into you, and we use [unclear 09:52] to supercharge these T cells and hopefully let your body do what it’s supposed to do. I said, that sounds great, that sounds like Star Trek type medicine right there. Let’s give it a shot.
By the way, Doc, how have other patients done on this? She said, as far as we know, you’re going to be the first person to try these treatments in this sequence. Oh. Okay. So what you’re telling me is that no one has ever failed this before. I think we should give it a shot. I don’t know how many patients would go on four second opinions before they’ve gotten a clinical trial. I don’t know how many patients would hear those words and think, maybe I should be the second person to try this and not the first. But I knew I had that goal of getting a durable response, I knew I had the purpose of not just being a cancer survivor, but being all those other things that you saw up there. When do we start? Let’s sign us up.
Too many clinical trials go unfilled because patients don’t know about them. Too many patients don’t know all their options because they don’t know the clinical trials exist and they don’t know the right treatment for them. I was fortunate. I was able to navigate to a doctor who finally gave me a chance at starting treatment.
Speaking of navigating—this is my favorite new slide—we moved our family from Fort Lauderdale to the Tampa area, and spent the holidays in 2012 over by Moffitt Cancer Center, with an amazing doctor who is a world famous melanoma researcher. Got over there, and we got my informed consent signed, the drug was there, we are ready to go. And this combination, I’m like, all right, let’s get this going, I’m jumping in, we’re going to save TJ’s life. Doctor said, yeah, we’re pretty much ready, there’s just one little problem. The contract. The pharma companies and the site, IRB, they’re just crossing Ts and dotting Is. Oh. It should be done soon. Okay. So I waited a week. Waited two weeks. Waited four weeks, and now after a month I’m still waiting. And I called the oncologist and asked him, what is taking so long? I am three months into these 24 months that I was given and I’m waiting for a contract to be signed. And he said, I can’t really help you, it’s out of my hands, and I think you need to move on to Plan B.
I stopped him there, I said, wait a minute, Doc. You told me last month that my best chance of seeing this little boy go to college or walking this little girl down the aisle was this trial. And now you’re telling me that because the piece of paper isn’t signed, that I’m going to lose that best chance. I’m going to lose my best opportunity to fulfill my goals, to fulfill my purpose. He said, I’m sorry, I can’t do anything for you. Okay. Let me get back to you, figure out what we’re going to do.
So we got home and the first thing I did was get on LinkedIn and found two people I knew that worked at one of the sponsor companies for the trial. And I emailed them and said, hey, can you get in touch with your friend in Legal and go have them go look for this piece of paper, get it signed?
And I figured that wasn’t enough, so I channeled my mother’s Italian side of me and I decided I was going to call one pharma company myself. Picked up the phone and dialed the global 800 number. They said, hello this is Big Pharma. Hi, I’m a cancer patient, and I have a concern about one of your clinical trial drugs. And that’s the fastest way to get to the legal department of any pharma company in America. And I was transferred over and the summary of what they said was, I’m sorry but the document is not processed. I heard about processes earlier, trying to report. Well this was a process for them. I told him, for you this is a document, a piece of paper. But to me it’s my life. And I’m not giving up that chance to watch those kids grow up.
And then I proceeded to give them a little more colorful explanation of what I thought about their documents. If you’ve seen the movie Office Space, you’ll get this. If you haven’t, go watch it and you’ll understand a bit better. Couple days later my oncologist called and said, I don’t know what you did but we just got a call from a pharma company, start the trial on Monday.
From what I’m told, the average clinical trial contact takes about 13 weeks to execute. In those three months, 150,000 cancer patients die, more die of heart disease. If you take the top six disease states in America, over 400,000 people will die in the time it takes a lawyer to sign a piece of paper. If you want, you can raise your hand if you have to deal with lawyers in this room. I’m sure most of you have had to do some kind of contract negotiation, budget, whatever it may be, where you are going through a process, you are negotiating, you are trying to push something along in a supply chain. If you don’t remember anything else from the morning, at least from this part of the morning, remember this: that while you’re doing your job and while you’re doing it well, there is somebody out there who is waiting, there’s a family out there who is waiting for a chance to live a longer life, a healthier life, a better life, if you can save their life.
And that’s where I’d like to say this story ends. We’re all happy, but I have another 13 minutes, so I’m going to extend it a little farther because it is clinical research, and we knew when we signed up that this might not work.
So we took our family and we moved them over. I have to adjust the timeline here. And I got in this really intense trial, I was in the hospital for about another week and a half. I ended up having to get a colostomy surgery because of the diverticulitis I had. So that put us back, and by the time I finally started the trial, it was January of 2013. We did this intense therapy, and it didn’t work. There is me at the end of the trial. I’ve now lost 50 pounds. I’m six months into this timeline. And I have just failed this what I thought was my best chance of seeing those kids grow up.
So I talked to the doctor, said all right, what do we do now? He said, all right, well hopefully when you’re done your washout period, we’ll get you on one of the anti-PD-1 drugs. I said, that all sounds great except the “hopefully” part. Is there something I don’t know about? He said, well, the contract’s not signed yet, so. Oh, fantastic.
Fortunately, one of the melanoma advocacy organizations contacted me that week, they asked how I was doing, I was writing a blog here for the Philadelphia newspaper. I said, this is how I’m doing, I just failed my trial, and I can’t make it through my daughter’s third birthday party without throwing up and passing out. Catherine called me back, goes, let me look into something for you. Called me back four hours later instead of four weeks later, and said, there is a clinical trial very similar to the one you want, another anti-PB1 drug. And it’s right down the street from your house, it’s like ten minutes away. I said, that’s great, do they have a signed contract? He said yeah.
So we moved our family back to Fort Lauderdale, and I went to this very small—there it is—went to this very small cancer center where I started the second-ever trial for a drug that was called MK3475. Some of you may have heard of it, it’s now approved for melanoma and many other cancers as Keytruda.
There is me on my first dose. And there is me four and a half years later on my 75th, and hopefully final dose, which was last August. And the reason for the stopping of the trial, or at least my participation in it, was a scan report that came through that summer, that said, “malignant disease is not suspected on the basis of this examination”. For the first time in almost five years, someone told me I didn’t have cancer. That was a great moment.
That’s not where it ends. I had these goals. I set them when I was in the hospital, on my smartphone, my BYOD. I wanted to get a durable response. I wanted to get healthy. I wanted to go to my cousin’s wedding, and I wanted to take my kids to vacation with them and to be a dad. And it’s that purpose part. Here’s me. I became a youth soccer coach.
I did a triathlon, that was a goal since when I was a kid. My dad used to tell me, always set your goals, put them where you can see them. And I had a goal as a kid to do a triathlon. I thought it would be me swimming across the island in Hawaii. Didn’t quite get there. But I did have a goal. And I reset that goal when I started to get healthy. Because the person who set that goal with me is in this picture. It’s not just another participant. That is the ER doctor who diagnosed me. He’s become a friend and an occasional training partner, and we did this triathlon together. He was much better at it than I was.
I got to be an usher at my cousin’s wedding. She told me I couldn’t come to the wedding, I told her she was out of her mind. She thought I wasn’t coming to her wedding while I was sick. As long as the doctor gave me the okay, I was going to fulfill that goal. I was going to have that purpose of being a part of her life.
I was going to take my kids to Disneyworld. This is us on the log flume, and as you can tell they both enjoyed it very much. Anyone could have taken them to Disneyworld for the first time, but they’ll always remember their dad did it. Not just a goal, that was a purpose.
It was to be that person who is able to go out there and coach. It was to be the dance dad. Every Thursday I take Julie to her dance class, and she’s got The Nutcracker this weekend and she’s so excited, and I’m the one that gets to sit there for two hours every Thursday. Did not put that on my goals, I’m going to be honest and say that was not my initial goal. And it wasn’t until someone told me I wasn’t going to be here to do it.
I got to do a Tough Mudder. Any of you ever done one of these Tough Mudder type runs, it’s 12 miles in the mud, you go over obstacles and at the end you get a beer. And my brother-in-law and I did one, so this is about two years into my five-year battle. I remember he called me and said, hey, did you sign the release form they give us? I said, yeah why? He said, did you read that? No, why, you actually read those things? He said, you can’t do this if you have a serious medical condition. I said, yeah. He’s like, you have cancer, you have a colostomy bag, how are you going to do that? Don’t worry about it, it’s just paperwork, don’t worry.
I had a purpose. I had a purpose to make a difference in those lives. I started writing about this. And I met young cancer survivors. And one inspired me to do a 500-mile bike ride, you see up there, for young adult cancer survivors for a group called First Descents that gives, they’re called Out Living It projects. They go surfing, they go mountain biking. And her story struck me, and I had that goal of not just being someone who made it, but being able to affect the rest of the people who were going to come after me. They’re going to look for hope, they want to have a goal and a purpose, and to be a survivor, to be a dad or a mom or a sister or a brother, son or a daughter.
So this is what I do. I get to write about this. I still share, not just my story, but to be a patient voice. I get to speak about it and try to inspire those of you who can do something about this. I certainly can’t cure cancer, as my college grades will show. But I can bring a perspective to this industry. I work as a patient voice for a couple different pharmaceutical companies. I work with a group called TransCelerate, which is a non-profit that’s funded by a number of the bigger pharma companies, one of which I called. And I am their patient advisor to what they try to do. They try to make clinical trial experience better. They have an eConsent, and they have a patient protocol engagement toolkit, and they have [unclear 25:26] I’m that patient voice to them.
And so I ask, as a wrap up and going into Q&A, that you think of what you can do. This is a very dramatic example, but it’s not unique. The patients who are out there, that are struggling with any kind of disease, not just a terminal one but anything chronic that affects them, that gives them a lower quality of life or a shorter lifespan, that causes them anxiety, how can you change that?
Those are the three things that I had to overcome. I had to find a good trial or treatment that would work for me. I had to get it started. And I had to find the right treatment for me. And the right treatment, right person, right time. Mantra, it’s one that I continually try to bang in the heads of people in the industry. Things like patient-reported outcomes and patient-focused drug development speak to this example and to the journey that all patients take.
So, on behalf of the patients whose lives you’ll touch, the names you’ll never know and faces you’ll never see, I want to end this by saying thank you. Too often you don’t hear what you do makes a difference. And it’s not just the one person or one family. But it’s all those lives, all those families that you will touch every day when you do your job well. So for them I humbly say thank you.
And we have three minutes for questions. Thank you very much.
[Q&A Section 27:30]
Any questions? I know we’ll have a panel after the next speech. So if you don’t get to it now, ask me in a minute. Just shout it out.
AUDIENCE MEMBER 1
[inaudible 27:44-47] —when you were initially diagnosed, what would be one thing you would do differently from then to now?
I would leverage social media and patient support much more than I did. Because as my dad and aunt will tell you, I think I know everything. And you learn very quickly that you don’t know what you don’t know, and as you start acclimating to this is my life now, you want to absorb information and you just don’t know where to—there’s so much information you don’t know where to get it from. And I’ve found, as an advocate, that social media, support groups, anyone you can connect with, someone who’s walked the path before or is walking it now. I would have done that more than just try to blindly find my way through. Go to Google search which is what most patients do. And that’s why I do what I do now, because I want to be able to help those patients who are looking for answers.
Anyone else? I’ll be here all morning, through lunch at least.
AUDIENCE MEMBER 2
Yes, the first trial especially. Having to relocate. There were a number of things in the protocol that really became difficult with trying—I laid it out, I was a project manager, so I laid it all out in a timeline and one thing would change and I’d shift everything down. I could tell because, what I know now, I could tell that it was just—a scientist put it together. And I don’t think a patient had any input in it. So what I have continually pushed industry to do is say, look, co-design these things. There were too many times that I had to get a blood draw and we had to go back to Tampa, when we moved back. Before my trial, the TIL part I couldn’t do anything about. I’ve seen in my experience, I’ve actually seen more other people who have experienced, how either inconvenient it is or how people don’t sign up for trials or adhere to the protocols because they just have other things in life that they deem more important. For me there was nothing more important than trying to beat cancer. But when we start talking on less serious, for lack of a better word, trial design, if it’s not done with a patient in mind, with a patient voice, you just assume that, oh we know what we’re talking about, and you don’t have the reality of maybe a single mom who has to drop two kids off at school and then go to work, she can’t take time to be able to go and get a blood draw or measure something where you could have some kind of a medicine or wearable that does a good job of getting the data you need for the clinical trial while giving her the freedom to do what she wants to do.
AUDIENCE MEMBER 3
Yes and yes. The first trial, they did a really good job on the first trial of walking me through it. The second one, much less to it, and it’s probably my first trial example is probably more the aberration, because it was such a new thing, because it was very detailed, it took us two hours to go through that informed consent. And they brought out the protocol and walked me through a number of things, and I don’t think that’s going to happen for a wild majority of clinical trials. Even for my second one, I had to pull information out. Had I not gone through that first experience, I probably on my second trial would have just said, okay, yeah just tell me when to come in, my infusion is every three weeks, that’s great. And I would have learned, hey, you’re going to have to get a blood draw. You’re going to have to get seen by the doctor before you get approved. You’re going to be here for half a day just getting an infusion. That wasn’t explained very well. I knew to ask those questions but most patients I would say don’t have that insight.
AUDIENCE MEMBER 4
I would say that Moffitt Cancer Center is one of the best in the world. And they are excellent. You’re a part of a machine, for lack of a better word. So they’ll take an hour for a blood draw. And Holy Cross, bless their hearts, it’s a really small center, and they have two research people there instead of two dozen. I want to go get my blood draw and I just text my nurse. But the first clinical trial team that we had there, the first team wasn’t very patient-friendly, to say the least. I now have a second oncologist there, who I love, and who is amazing. They’ve gotten better, but there is sort of the small center feel. It’s great, but there is also a lack of manpower. They are trying to do other stuff too, so things like having to re-consent and understand what it means, they don’t always have the time for. I think that if you’re going to be a cancer center, they have to have the resources to be able to put a doctor in a room with you for an hour and a half and go through a consent. And the smaller center didn’t really have that. And they have other—too many healthcare facilities run on a very tight budget and a tight timeline, and patients feel like they are kind of getting rushed through. And I had that a little bit at Holy Cross, I just knew enough to say I don’t understand this, walk me through this again. Or, I want to come back and let’s go over what my next steps in the protocol are going to be. So it certainly was different in the facility, and it’s not a knock on Holy Cross, just there is resource constraints that I’m sure all of you are familiar with that happen in smaller research facilities.
We’re probably out of time but let’s do one more and then we’ll move on. I’ll be around so feel free to ask any questions.
AUDIENCE MEMBER 5
You participated in a trial that has a design that gets a little controversy nowadays because it had a crossover. So everyone in your study got access to the investigational drug. A lot of academics in oncology think that that design does a disservice to patients because it doesn’t give the industry an opportunity or the FDA an opportunity to really compare chemotherapy versus what drug you got. But patients tend to like it because they always get a chance of getting the drug. What do you think about that sort of thing? Would you never participate in a trial where you had a 30% chance of getting chemotherapy?
That’s a long question for the last one. I will make this as quick as I can. To put it in perspective, there’s the two anti-PD-1drugs that are out there now. I went with one and I use this as a comparison for decision scientists. I say okay, I call it Pepsi and Coke, the drugs are fairly similar. But the trial design for the one that I was on did have a crossover. If you were randomized, there was a third chemotherapy arm, a third high dose, a third low dose of study drug. So if you were the one-third that got randomized to chemo arm and you got the short straw, if you progress at 12 weeks then you can cross over to the study drug. That sounds great and it’s probably the biggest reason I chose that trial, other than the contract was signed, was that I didn’t want to leave myself without a chair when the music stopped. It is imperative that trials are designed with patient outcome in mind. And I actually think I made the wrong choice. I think I should have signed up for the other trial, and if I got randomized to chemo arm just dropped out of that trial and signed up for the other one. I think I actually made the wrong decision. I wanted to give myself the best chance of getting the anti-PD-1 drug. And that’s the mindset that patients go through, that too often industry and researchers don’t think of. They just say, all right, you’re a patient, you can get randomized. No, I have these goals and purposes, I don’t want chemotherapy, I knew how bad those odds were. And I didn’t know if those 12 weeks would be the difference in my body being able to beat melanoma or it not. And there’s too many people that I know that didn’t get the option that I did, and aren’t here to tell their story. So to answer the question, I don’t know that you can definitively say that a crossover arm will make it less likely that the FDA is going to approve your drug. I can say that what the FDA and what Scott Gottlieb has done with patient-focused drug development, if you come to him and say, we consulted patients and this is what they want and they want the ability to be able to cross over, that is looked on favorably. At least from everything I’m told and what I’ve heard from the FDA, they said, when you are designing a trial with patient outcomes in mind, that is something that is a favorable thing, not a disservice, and not well you can’t report overall survival now. Well we kind of know what overall survival is for stage 4 melanoma patients. And if you’re using one of the surrogate endpoints, that that’s okay, if the patient feels like it’s okay to use progression-free survival if they can also access study drug. So from a patient standpoint, from an advocate standpoint, when a trial is designed in a way that says that makes patients want to get on it, if there are slight hurdles to clear regulatory barriers, then that’s up to the industry to work with regulatory agencies to understand that, hey, it might not be the perfect measure, but patients want this as their outcome measure, this is what patients want and we are ultimately here to serve them. I think it makes a very compelling argument when you submit that as your submission to the FDA. Did that answer the question?
AUDIENCE MEMBER 5
All right. Moving along?
[END AT 39:01]