‘The Worst Disease You’ve Never Heard Of’ - Brett Kopelan, Executive Director of debra of America


Imagine 60% of your skin having open wounds every day. That's the grim reality of those with Epidermolysis Bullosa, or EB, a rare genetic connective tissue disorder which results in blisters and tears to the skin being created from even minor contact or friction. The range of complications this causes for almost every normal activity – from eating to bathing to sleeping – is daunting, leading our guest today, Brett Kopelan, to call EB “the worst disease you’ve never heard of.” He should know. Brett and his wife have the equivalent of fulltime jobs just managing daily care for their daughter Rafi, who suffers from a severe form of EB. Advocating for Rafi led to Brett becoming Executive Director of debra of America, the only national not-for-profit providing all-inclusive support for patients battling the disease. In this revealing discussion with host Shiv Gaglani, Brett opens up about his frustrations with the health insurance system, the challenges of raising money for rare diseases, and why he feels there is a need for more doctors to specialize in rare disorders for patients over twenty-three. Brett shares some positive news as well about several promising gene therapies on the horizon. “I can say that the past two years has really been the first time that I've felt I'm working for my child's life, not for beyond her. So that's an exciting thing.” Mentioned in this episode: https://www.debra.org




Shiv Gaglani: Hi, I'm Shiv Gaglani. It's been called ‘the worst disease you've never heard of,’ as coined by our guest today. Epidermolysis Bullosa is a rare genetic connective tissue disorder found in one out of every 20,000 births in the U.S. We're going to learn all about it on this episode from Brett Kopelan, Executive Director of debra of America, the only national not-for-profit providing all-inclusive support to the EB community. More importantly, as he would tell you, he's also the father of Rafi, who was born with a severe form of recessive dystrophic epidermolysis bullosa, rdEB. Prior to his rare disease work, Brett was an accomplished entrepreneur starting three companies raising more than thirty million in venture financing, where he led business development and marketing. He's also chair of the board of directors at the Foundation for Cell & Gene Medicine, is a member of the Alliance for Regenerative Medicine or ARM, and past chair of the board at the National Organization for Rare Disorders. 


Before we get started, I'd like to thank Elsevier CEO Kumsal Bayazit for recommending Brett for the podcast. Her daughter and Rafi were classmates together some years ago. So, Brett, thanks so much for taking the time to be with us today.


Brett Kopelan: Thanks for having me, Shiv. It's a real pleasure to be here.


Shiv Gaglani: So, I obviously know a lot about your background and Kumsal filled me in a bit. I'd like to start with you telling us about you and your family's rare disease journey.


Brett Kopelan: Sure. The patient journey is always a big thing we talk about, especially in biotech and in pharmaceuticals. I like to understand the burden of living with a disease like this. So, I was indoctrinated into the world of rare diseases just about fifteen years ago, back in November 2007, Upper West Side of Manhattan. My daughter was born two weeks late. She was really great, except she was missing skin, bilaterally, on both feet, and on the back of one hand, and at that point, the official diagnosis was she was just overcooked. That was the official diagnosis by two physicians. So that's what I now call a total waste of a half a million dollars in education. 


About six hours later, while I was out somewhere on the West Side with some friends working on a hangover to celebrate the birth of our daughter, I got a call from my wife. She said, "Well, they just took her to the NICU and I don't know why I'm kind of freaking out." And I said, "I would not be so concerned at this point, because she did have some open wounds on her feet and the back of her hand. They're probably concerned with the concept of infection and a newborn, so they just wanted to protect her." 


So, the next morning, I showed up early at the hospital. My wife and I went into the NICU and the only way I can actually describe it was that the world fell out from underneath my feet at that point. I looked at my little baby girl who had blisters all over her body. Generalized blistering, stuff that we hadn't seen, popped up. They had no idea what was going on and we were being told at that point that they weren't sure if she would ever leave the hospital if she made it through the next twenty-four hours. That led to the next ten days of trying to rule everything out. Every god awful disorder that you could think of was tested for and then we got lucky in that there happened to be a pediatric dermatologist who happened to be walking on the floor of the NICU who happened to hear them talk about blisters. She said, "Well, I think it might be this. Let's test for it." So, they did a punch biopsy and it's called immunofluorescence to see if there was a lack of protein. They do protein staining, and it came back about a day later saying it is indicative of Recessive Dystrophic Epidermolysis Bullosa. Let's just say EB going forward because it's a mouthful.


What that means is my daughter doesn't have the ability to produce type VII collagen. We've all heard of collagen. This is a specific type of collagen that is the building block of something called anchoring fibrils which are what hold the two layers of skin together. If you think about those two layers of skin as Velcro, being held together by latches, she doesn't have any of the latches, which are the anchoring fibrils. So, any friction or trauma will create a blister or just a skin tear. 


Given the rules of New York and getting on Medicaid, that's when I first spoke to debra of America. Their nurse was incredibly helpful, telling me what we would need, what to expect, what our insurance needs would be, what wound care supplies we would look at, and so we spent the next thirty days in the NICU so that you would automatically qualify for Medicaid. What I will say is the second we got that diagnosis the first thing I did was run to the phone to call my brother who was a surgeon at that time. I said, "Adam, they think it's something called dep-o-derm-omolsis-belosoa." And he said, "Oh, four letter word," hung up the phone, ran to the nearest computer, and started googling it. And I think we can all figure out that Google was not your friend at that point in time. I saw that she would have a very short life, that was really going to be about pain, disfigurement -- it’s an incredibly painful disorder that is financially taxing, emotionally taxing on top of physically -- and that there was no treatment or cure, not even on the horizon, at that point. 


That's when I contacted debra of America. The chairman of the board at that time, said, "Given your background, would you be interested in joining the board of directors of debra.” The formal title is the Dystrophic Epidermolysis Bullosa Research Association of America. That's a terrible email address, so we go by debra. Talking to my wife about it, we felt that we were lucky in that I was at that time choosing the insurance coverage for the company I was working at. We felt that we had more means than others, and knowing what this disorder does to people, and how it attacks them -- physically, financially, emotionally -- we felt we wanted to give back. So, that was one aspect. 


The other aspect was, I wanted to know every international researcher on the planet. I wanted to know what was going on, if anything would be considered promising, wanted to funnel money there, and should there ever be a clinical trial, or more than one, we wanted to be in the top of the list at that point. So, the strategy paid off. I joined the board of directors in January 2008. By the end of that year, my daughter was the eighth kid in the world to undergo stem cell transplant in hopes to treat or cure. We moved to Minneapolis where she underwent the procedure at the University of Minnesota. Two years later, came back to New York. I was a little disillusioned with the organization and was having financial difficulties and I said to my wife, "We're gonna go out and start a foundation and put them out of business." She looked at me and said, "Don't be stupid. Take it over. It has name cache, it has a board of directors, it has money in the bank. Do something constructive with it." 


So, in January 2011, I became the Executive Director and at that time, there was no translational research. There was academic research, but nothing to be considered translational. Since then, we have our first product approved in the European Union.  Now we have our first gene therapy -- what they call in vivo gene therapy -- scheduled for approval in February of 2023 and we have two other gene therapies currently in phase three clinical trials now. There are multiple avenues being looked at and I can say that I guess the past year, past two years, has really been the first time that I've said, “I'm working for my child's life, not for beyond her.” So that's, that's an exciting thing.


Shiv Gaglani: That's an incredible background and progress. I know for the rare disease parents, families, and patients I've spoken to, it's never fast enough. But even given that time timescale, you and the team have made some pretty amazing progress. Before we go more in-depth on the policy stuff -- which I know you're pretty expert at -- what is life like for Rafi and your family on a day-to-day basis these days?


Brett Kopelan: Well, you know, it's a difficult journey. More so for her than me. The best way I can start it off, is you remember the last time you had a paper cut, and you put Purell on it? That sting and that burn? Now imagine 60% of your body being an open wound and you just sat in a vat of Purell. That's what life is like on a daily basis. There are some good drugs, oxycodone, other stuff out there, that don't quite take enough of the pain away. So, you're looking at anticipatory anxiety for bath and bandage changes which is every other day. These things can take four hours to undergo between removing all the bandages, doing the actual routine or the procedure, and then rebandage them, and that doesn't include setting up and cleaning up. So it is, in essence, a full-time job just in managing her care. 


The only thing not affected are brains and lung. Her entire GI tract is in fact as affected from her mouth straight down to her buttocks and so going to the bathroom is incredibly painful. Eating is difficult. She has to have a surgery every eight weeks to widen her throat. It's called an esophageal dilatation. They really just stick a balloon down there, pop open scar tissue, and then she's good for seven weeks, until, you know, we need the next one. But life is challenging. She wakes up very early. She's in a wheelchair, but she goes to school. She has a group of friends. She hates when people say ‘you're an inspiration’ because she's like, “I'm just living. Everyone has issues, it's just a question of degrees.” She says, "I don't know why I'm inspirational, because I just I wake up, I go to sleep, and do everything in between." So, she's pretty resilient. She's also a pretty special kid. She had a Make A Wish opportunity -- the Make A Wish Foundation is an incredible organization. Most kids her age would want to see Selena Gomez, something like that. Her choice was Jane Goodall. So…


Shiv Gaglani: That's awesome.


Brett Kopelan: It really is. It was great. I think my wife and I got more out of it than my daughter did. But it was a great day. 


I'll tell you what our journey looks like from a parental point of view. Start about 4:45 a.m. in the morning, when we get up and start giving her the medications that she needs. Everything from growth hormones to pain medication to levothyroxine, she gets about ten minutes, every morning, you got to space it out over time. She gets fed overnight through a G tube and a pump. We give medicine, make the bandages, wake her up about 6:30 a.m. get her to go to school by 9:00 a.m., so it's a long process. We change about half the bandages. Sleeping through the night is not a reality. I haven't had a good night's sleep since I don't even remember, maybe grad school. Your body gets used to it and you certainly live on a lot of coffee, but it's a difficult slog and the only thing that's really helped has been the hope for treatments given the number of avenues. I know we'll get back into that. But now that something is really working very well, it's helping a lot. Hope is now turned into an expectation of a better life.


Shiv Gaglani: That's awesome. Well, thanks for breaking that down. I will say two touchpoints based on what you mentioned about Rafi. You know this because you know John Crowley who we had on the podcast…he was on the board of the Make a Wish Foundation and now his daughter Megan is now working at the Make A Wish Foundation. That's a more recent update. And the second is about Jane Goodall. I'm sure you have pictures. Maybe even there's some footage of that. Would love to see it. So, I'm talking to you right now from New York. I just got here because one of our other podcast guests, Chelsea Clinton, she and her dad have taken a good interest in rare diseases. She's involved with some rare disease groups. She's having this reception at the Clinton Global Initiative about global health equity and invited me over to talk to her a bit about rare disease work. She and her mom just came out with this Apple TV series I recommend called “Gutsy” and in one of the episodes, they're with Jane Goodall. So, there are all sorts of interesting connections. If I see her, I'll mention that to her.


Brett Kopelan: I'd love to talk equity and access with them. I mean, that's a huge topic when it comes to any disease, but rare disease in particular.


Shiv Gaglani: Let's get into that. You're in Boulder, Colorado, and you moved there from New York, largely because of insurance coverage. Maybe you can go into the policies and legislation you're working on to get a guaranteed insurance coverage parity for those with rare diseases, and then from there move to some other policies that are currently being discussed.


Brett Kopelan: I'll tell you the reason why we moved to Colorado. You know, I run debra of America. I provide insurance to my employees, and I think we can all say we hate insurance unless you absolutely need it, right? Well, a few years ago, Blue Cross Blue Shield raised our rates 100%. So it became expensive. But we were lucky, and were able to get a plan with Aetna which then raise the plan 465%. So, we went from $2,400 a month to insure a family to $11,500. And that's not affordable for the Clintons, or Microsoft…certainly not for a rare disease organization. So, we knew that she had to be Medicaid dependent, and New York Medicaid is woefully insufficient. It wouldn't even pay for the bandages which cost us about $100,000 a month, and there's only a handful of states that actually provide adequate coverage from a Medicaid perspective. 


Colorado has one of the best children's Medicaid programs out there for chronic diseases and they also have arguably one of the best EB centers in the country. There's a handful of EB centers out there, and here in Colorado is really one of them. So, it was an easy decision, partly because I grew up skiing out here and I always said in college, I want to live in Colorado. So one could almost say it's a dream come true. Right? It was a great move. We used to fly to Cincinnati every three months to Cincinnati Children's for her care. Now we drive forty minutes. So, a huge difference in that. 


But in that, one of the things I knew that debra was missing early on when I became the executive director, was a legislative side. Really, we didn't have much of a footprint on Capitol Hill or in the states.  Clearly, from an insurance coverage point of view for Medicaid coverage, we needed to ensure that our community was being adequately covered with insurance. Right now, only about 32-33% of our population has adequate coverage. One of the things that debra does is give out free bandages. But when you're only looking at about seven or eight states that provide adequate coverage, it's really difficult. Not everybody's in the position that I am where I can move, I can leave my family and friends behind. My job follows me. So, we're again, lucky. 


We started shaping up a piece of legislation called the EB Care Act, which at that point would have guaranteed that genetic testing was done because most insurance companies don't consider it medically necessary. Many Medicaid programs don't. They consider it experimental. Clearly, genetic testing is not experimental. But if there is no treatment or no cure, it's not considered medically necessary to have a genetic test, and at that time, our genetic test was $3,400 and they are still. A whole genome screening is expensive, rapid genome screening is expensive. There's a whole exome and then there's now something called panels as well, which can be $250, but, it's important to get these things covered. 


The EB Care Act would have guaranteed that insurance companies and Medicaid pay for the bandages, for any medication that are necessary and out-of-state coverage because no one really knows about Epidermolysis Bullosa, and there's only like, five or six really good clinics out there, and they're always cross state borders. So, getting that paid for is also difficult. 


We had bipartisan support, but doing anything in government is difficult. We found that we needed more data, because we could say, “Hey, it costs $100,000 a month to cover” and they said, "Show me the details." It's actually one of the things we're doing now is developing an economic burden of illness model looking at adjudicated insurance claims, and building what does it actually cost? We need more than just my ad hoc analysis of two years of explanation of benefits, which are $1.3 million a year to take care of my daughter. So, we need that from a policy perspective, and then there's another side. I do a lot of work with other rare disease organizations -- Global Genes, RDLA I used to work with NORD, Foundation for Gene & Medicine, ARM -- strength in numbers, right? When you really want to get something passed, you need a tremendous number of co-sponsors on bills, and you need the Christmas tree bill to attach it to, and all that kind of stuff. So, when you have greater numbers, you're able to put something together that can make more sense. 


One of the things we're working on is called Epic RD. It is Equity and Parity in Insurance Coverage. There's a lot to it so I don't want to go too deep into the woods because it's a deep, deep rabbit hole. But, it would guarantee that if there is an FDA-approved medication for a rare disease, that Medicaid/Medicare would pay for it, that it would pay for durable medical equipment like wheelchairs, bandages -- which is problematic with many insurance companies now -- that appeals and prior authorizations would be streamlined. So, it's very deep and there are other pieces of legislation that are just as important. 


The Precision Medicine Answers for Kids Today Act which guarantees that Medicaid will pay for genetic testing. I'll tell you as someone that works in biotechnology and pharmaceutical companies, now they start asking, what is the specific mutation that is there, right? It's not just "Oh, it's a genetic mutation," because we're beyond small molecule drugs at this point. We're at base editing and prime editing and read-throughs and exon skipping. So, you need to know specifically what they are. So genetic testing is required. In this particular bill, Medicaid will be required to pay for the genetic testing and mutation analysis, when indicated. But more importantly, because it's taxpayer dollars, that you don't just go to the most expensive test that will give you the most information, but it is important to allow the geneticist or whoever is in charge of the case to choose which testing is most appropriate. These days with EB, you can just select ten genes to look at or eighteen genes or you can do a panel, which could cost about $600 or $700 depending on the number of genes you're looking at, as compared to $3,000 or  $4,000. There’s the STAT Act…there's a new piece of legislation every other day.


Shiv Gaglani: It’s amazing. Hopefully, this one gets passed. One of our previous guests is Philippe Pakter -- who Kumsal also introduced me to -- and he had an issue of cross-border care when his daughter, Lysiane, was born in France, but the center for excellence for Pierre Robin Sequence was in Germany. Being able to get all of that reimbursed by the EU became a whole issue like going from New York to Colorado. Making sure that you're getting coverage for these rare disease patients and families is critical. 


One thing we talked about before on our first call after Kumsal introduced us, was the fortieth anniversary of the Orphan Drug Act, which is coming up in 2023. It was signed January 14, 1983. That obviously was a massive piece of legislation. I know you know the person who helped write a lot of it. I'm curious, what kind of legislation is working, and/or what are you most excited about for the next ten years to make sure that we create a lot more treatments so it isn't just palliative care for Rafi. I mean, you have some good news on that front, hopefully, in the next couple of years.


Brett Kopelan: Look the Orphan Drug Act is something you have to continue to protect and it's an evolving piece of legislation. It is not in itself static. One of the things that the Orphan Drug Act did was provide some incentives for drug development within rare disease, because if you have, say, a thousand people that could be eligible for treatment, what drug company in their right mind is going to go out there and develop for them?  It's just not cost-effective. So, you're constantly making sure that those incentives stay intact. A great example is something called the pediatric rare disease priority review voucher, and it gets set to what they call ‘sunset,’ or go away, every few years. It's probably one of the most interesting and successful incentives out there. 


Let's say you're developing a drug for a rare disease, for EB, and you get that drug approved. You have then applied for this review voucher and if you are eligible for it and you get it, your next drug gets expedited review -- six months instead of twelve months, right? But the most important thing about it is that it is a sellable asset. So, a small single-asset company can go to Pfizer and go to Novartis and sell that expedited review for their next blockbuster drug and so when you're looking at hundreds of millions a month, then it's pretty significant and the going rate right now is $110 million for that and so you're able to recoup a lot of your research spent in that. A few years ago, it was set to expire. Myself and a few other people did a lot of advocating on Capitol Hill. I wrote a piece for Politico along those lines, and thankfully, Congress saw it our way and it was extended, and then it was extended again now through, I think, 2024. So it's important to keep that going. 


And the other ones are PDUFA, which is user fee laws. It basically funds the FDA. The FDA is not only funded by taxpayer dollars through Health and Human Services. Anytime a drug is approved or applied for, biotechnology and pharmaceutical companies pay a user fee, and that funds the FDA. But here's the more important part: drugs used to take forever to get through the regulatory pathway, but because of PDUFA, that time has shrunk to six or seven months, potentially. So, it's incredibly important and it's a must-pass piece of legislation that is right now being batted around in government. They’re going to use a continuing resolution, and now we're going to have an omnibus package and it'll get authorized in the omnibus for sure. Mostly because people that I work with are pounding the pavement and the telephones and making sure that senators and congressmen vote for it.


Shiv Gaglani: That's incredible. This reminds me of how important it is to understand these details, because it’s something we certainly don't learn in med school. That actually brings me to a question I love to ask. Ultimately, Osmosis is a teaching company. We like to take complex things, whether it's EB or diabetes, and explain it in a very enjoyable, efficient, visual manner.


Brett Kopelan: Sure.


Shiv Gaglani: If you could snap your fingers and teach any audience -- be they legislators, be they healthcare professionals, med students, researchers, the general public -- any subject, like design a video or a course, what would it be, and to whom and why?


Brett Kopelan: Well, from med school perspective, I know that while many of you may go into academia, and academic research, know that you're going to come up with the science, but you're not necessarily the ones going to commercialize it. So, what you want to also understand is what they call the regulatory pathway, how to get your invention from the bench to the bedside, how to translate it through that whole progress. The regulatory pathway is incredibly difficult. It's certainly much more than just saying, “Oh, my God, it works in a mouse model.” Because a lot of things don't translate from mouse model, over. 


Understanding the regulatory pathway and then being able to work with patient organizations because it's most of the time, it’s the patient organizations that are funding the work that you're either doing or going to do. You want to get the patient perspective in drug development and even in the early stages. You want to understand what a clinical trial participant or a guinea pig will endure. "Oh, I've got this great small molecule or I can do this editing, but they have to get seventeen injections every day for six weeks." To an academic researcher, you may understand that it'd be difficult, but it's not gonna stop your research, righ? But there may be a better way to deliver the medicine. So, engage in the patient population, engage in the organization to find out what is clinically meaningful to them in this case, because that will guide some of your research and how you define a therapy over time. 


I think it's important to always keep your eye down the road. Keep your eye on what, from a regulatory perspective, you need to do in order to do what you want to do in the earliest stages of thought to help people because you can come up with an innovation, but if you don't think six steps down the line -- you got to play some chess, not checkers, in this case -- it may never get to where it needs to be into the patient's hands into their bodies, and eventually help them. It's really important to look down the line. There are just some incredible rare disease leaders out there. They know the science in some cases better than the academic research. So, pick up the phone, give a call, engage.


Shiv Gaglani: Those are all great pieces of advice and you're clearly among them. And you remind me a bit of recent guests we also had on the podcast. Nick Sireau, whose kids were born with a one in five hundred thousand condition called Alkaptonuria, AKU. Like you, he got really involved in the AKU society and fortunately, there's a therapy that's keeping his kids in very good health, relative to where other people with AKU could be. But he's also very involved in Beacon, which helps rare disease patient groups, and Orchard, which is doing OCD therapy. Orchard has funded two clinical trials on OCD that are starting. 


Similarly, when I read your bio, it's very impressive, because while you're involved with debra, you're also involved in launching several other nonprofits helping other companies. I'd just love to hear briefly from you about that pathway. Obviously, you were involved in startups before and raised venture capital, etc. You have that background in business development and marketing and organizational management expertise. But how do you pick the other things you're working on and what are you hoping to achieve with some of them? Maybe you can give us a punch list?


Brett Kopelan: Sure. Well, look, what I will tell you is that back in the days when I was raising venture capital, it was easier than doing it for a rare disease company, let me tell you that. Charitable giving is always the same percentage of GDP. Each household has a certain percentage of their money that they're gonna give to charity, and they're gonna give it to breast cancer, they're gonna give it to their grad school or their undergrad and I have to go out and say, why my organization -- that's trying to help a disease that you've never heard of that you'll probably never see in your life -- why am I more deserving of your money. So, it's actually a much more difficult raise for this. 


But as I said, I joined a lot of other larger advocacy organizations. Some like Foundation for Cell & Gene Medicine get information out to patients and communities so that they understand what is involved being in a clinical trial. I can tell you that before we jumped into that clinical trial I mentioned earlier -- the stem cell trial -- I think I spoke to almost every pediatric bone marrow transplanter in the country. I wanted to know what their thoughts were. I wanted to understand what the potential pitfalls were and what I learned from clinical trials in general is you can get better, you can stay the same, you can get worse, or you can die. Those are four options, and you have to be willing to accept each one of them. Not all clinical trials will say you're going to die, or that is not always one of the options, but you have to understand what the clinical trial is. So, that kind of educational material…we've put together some shorts, some infographics, discussing what you should talk to your physician about if you're considering going into clinical trials, stuff like that. 


Other organizations that I’ve been involved in from an advocating side are really developing policies that help drug development in general. It's really easy to demonize biotechnology and with medicines, they charge too much. It's really easy, rioght? But in the end, you have to think they're the only ones that are going to help you in the long run. So, it doesn't mean you should let them take advantage of you by any means, but putting in policies like incentives for rare disease, like policies about consistencies across the regulatory pathway at the FDA these things that support drug development, that make it more efficient, and more timely, less costly, in the long term means you will get help faster. So, that's really why I started going to some of these larger organizations because you know, those policies that helped biotechnology eventually helped me and more importantly they helped my daughter.


But as you mentioned, I'm also in the process right now starting up another foundation through a venture capitalist that I know. I think we've all heard of BRCA mutations. It's mostly known within women, that if you do have this BRCA mutation, the first thing you do is either have a hysterectomy, or you can have radical mastectomy. And it also affects males for prostate cancer or pancreatic cancer. He has a BRCA1 mutation, so he's had prostate cancer. He's very well off, so he has committed to funding the beginning this organization with $20 million to make a big difference in BRCA now. Here's another reason really to invest in rare disease research, because you are showing that certain platforms and certain ways of addressing diseases work, and you can do it in an expedited method and then you can take that to more prevalent diseases, like diabetes, like cardiovascular, like other types of wounds and stuff. 


So, take advantage of the shorter road and eventually, it'll play out. But to do it, it's a very complicated pathway. You can't ignore the legislative side, you can't ignore the academic side, you can't ignore the commercial side. You have to really have your hands in as many areas or fingers and pies -- whatever you want to say -- as you possibly can. For those that ever start up a company what I can tell you is hire for your weakness, not your strength. Don't hire the ‘yes man.’ Hire someone that will argue with you until the sun goes down and knows what they're talking about because in the end, you'll be able to achieve more in a shorter period of time.


Shiv Gaglani: That's some really good advice and again, I’m very impressed with the depth and breadth of what you're doing. Wishing you the best on that launch and the impact that I'm sure it'll have. We're coming up on time. So speaking of advice, what advice would you give to our audience, primarily current and future healthcare professionals?


Brett Kopelan: Well, you know, my disease really has been in EB. It's really been under the dermatology umbrella as the coordinators of care and it's been in pediatric dermatology and a lot of these rare diseases are pediatric in nature. With more therapies coming down the road, we need more adult physicians in these rare diseases, because as an adult, you can't get treated in a pediatric hospital. Adult physicians have to think about more things than pediatric physicians. We actually opened the one and only adult clinic in Philadelphia -- Jefferson University -- it is the only one in the country. 


Once you get past the age of twenty-three, you don't have any specialists. And dermatologists generally get a bad rap -- cosmetics and all that. I have more respect for dermatologists than most other physicians, mostly because just think about the number of things that manifest in the skin. It's the largest organ. So, I would say for those that are thinking about dermatology, pay attention to EB because we now have -- or we will have in the very near future -- medicines and therapies that are going to extend lifespan beyond pediatrics and it's going to be imperative that these people get taken care of as adults. Not just in EB, but in rare diseases in general across the board. I think you'll find that they are generally coordinators of care and those that have specialists in it are all pediatric. We need adult physicians for rare diseases and geneticists. Study your genes.


Shiv Gaglani: That's a novel piece of advice. We've never heard that in over three hundred episodes. That's really interesting to know that there's a lack and the need there. My last question -- we could talk for hours, I'm sure -- but is there any major salient point you want to get across to our learners before we let you go for the day,


Brett Kopelan: As you go through your stages of life, here's what I'm going to ask you to do: if you are parents now or you become parents, teach your kids inclusion, okay? Rare diseases in general -- EB in particular, but rare diseases in general -- are isolating. You will see a rare disease kid eating lunch at school by themself. That's not right. That's not right. They are, in many cases, no different than any other kid. They love Harry Potter. Maybe it's friends and artwork. You'll find a lot of commonalities, because in the end, they're just kids, or they're just adults. It’s really not about bullying or anything like that. It's about inclusion. 


Don't be afraid to go up to that kid that looks different. Don't stare. Ask questions. Don't be afraid. Because when I look back at my childhood and I ask myself if I would have been inclusive, it's a very painful thing for me to say, I wouldn't. I was your typical kid growing up in New York City in the suburbs of New Jersey, throwing footballs around and playing video games and all that kind of stuff. If there was a kid like Rafi at the lunch table, what would I have done? It's a very painful, introspective look at myself to say -- and by the way, my first career was in clinical psychology -- so to look back and to say, I know that I wouldn't have been. So, one of the most important things I can say is teach your kids to be inclusive. They will be better individuals for it in the long term and that is the best way to help those that need help.


Shiv Gaglani: That's powerful. That's a very powerful lesson. Thank you for sharing that. I very much agree, especially in this age where there's so much focus on diversity, equity, and inclusion. It's important, and we'd like to get this across to our audience that inclusion also means disability, specific rare diseases, and that's one reason I think this area appeals so much to Osmosis and us is because there's a great need. There's a need for education and awareness. There are so many out there, and our hope is as a company dedicated to being approachable -- one of our values is "open your arms” -- we can help at least a subset of our audience, and think about becoming more inclusive with the rare disease community. 


So, Brett, it's been an absolute pleasure getting to know you and having you on the podcast. As I said, we could talk for hours. I really appreciate you taking the time to be with us, but more importantly, for the work that you've done for Rafi and obviously for many, many other people you probably will never meet.


Brett Kopelan: Well, I appreciate the opportunity to be here and to let people know about EB and, really, congratulations on Osmosis and Raise the Line.


Shiv Gaglani: Thanks so much. And with that, I'm Shiv Gaglani. Thank you to our audience for checking out today's show and remember to do your part to Raise the Line and strengthen our healthcare system. We're all in this together. Take care.