A discussion of where cell and gene therapies are currently doing, what's on the horizon, personalization, and much more.
This is an area that is evolving rapidly, a lot of things we're starting to see coming out in this space, really starting out with the first car-T cell therapies in 2017. This is where the market began with the two first products Kymriah, and yes, car-T launching right around the same time in late 2017. This was a really amazing time, having been involved very closely with one of those products, seeing patients essentially cured from childhood and other types of cancers. It was really a game changer for patients and for the overall healthcare system. In the cell therapy space, we now have therapies for multiple myeloma, follicular lymphoma, acute lymphocytic leukemia, B cell lymphomas, and the list kind of goes on and on. It's really, really exciting for these patients who really had no other options prior to these cell therapies emerging. We also sell cell therapies approved for the first time and endocrinology conditions with diabetes type one treatment, among others. So really an exciting time for cell therapies.
Then most recently, of course, we've seen some gene therapy and therapies emerge that I'll talk about in a moment. But this area has really evolved very drastically over the past several years really exciting time for gene therapies. The first student gene therapy followed the first cell therapies in 2017, with Lux Turner's approval and retina disease, which I think it was an interesting place for gene therapies to begin, right and retinal disease versus some of the other conditions that we saw very prevalently with cell therapies, so very different area treatment. And then several additions, additional therapies in the gene therapy space have followed impacting conditions like hemophilia, beta thalassemia, Duchenne muscular dystrophy, among others. Most of these are relatively rare conditions. So, the trends that we're seeing in this space are really impactful one-time treatments that are intended to treat relatively small populations of patients anywhere from we've heard clinical trials, having a hard time recruiting even 10 patients, up to maybe a few 1000 patients. The most recent news, obviously, with new approvals in the gene therapy space for sickle cell disease. This is a much more common condition, which affects approximately 100,000 people in the US. There are two different therapies that have been improved in this space, one being like Genia and the other being past Jimmy, and the first that I mentioned there being for CRISPR therapy, which is a gene editing mechanism of action, first approval in the US for that product as well or for that mechanism as well. So really, I think some of the trends we're seeing is that we're broadening the breadth of conditions that these treatments can impact. And we're also seeing new mechanisms of action emerging even within the cell and gene therapy, typical mechanisms that we've seen. It's a very exciting time.
Car-Ts I view as kind of being halfway pavers for selling gene or innovative therapies across the world. And they've been highly beneficial to patients who have received them, as I mentioned previously, really, curing conditions, in some cases for patients are really life changing. They've had response rates and significant durability. So, some of these patients are seeing long term benefit from the treatments, even if not cured months, if not years out remission from the cancer that they've been treated for. Most car-T, as we discussed also are for hematologic cancers, or blood cancers, with a number of indications and those growing every day. If you look at the pipeline, though, very exciting three at my last count, this is an estimate, there are around 300 car-T in the pipeline, and various phases of research and development. There, we're starting to see more of the allogeneic products coming closer to commercialization, meaning that maybe you will see the cost decrease a little bit more patients can be treated, and possibly the logistics won't be quite as complex as some of those autologous products that we're seeing more prevalently in the market right now. So, some of the interesting indications that we're seeing in these pipeline products are more broad cancer types, such as solid tumors. So, we see some indications for pancreatic, colorectal and breast cancers, among others. And we're also seeing some interesting neurological treatments immunology as and, of course, additional blood cancers or hematologic conditions as well. So very broad again, on the car-T cell therapy space is again, kind of was the first to come to market. And we'll continue to see an increased innovation in this area. So excited to see what will continue to come with parties.
This is a really interesting area. And I agree cell and gene therapies are about as personal as far as our current medicine options come. These treatments are designed for one individual patient, when we're thinking about the autologous treatments, they really are using that one patient's own cells as the starting material to manufacture the medicine. So, these patient cells are actually being shipped to a manufacturing site and turned into the medicine to treat these debilitating conditions. So that is a very personalized treatment, obviously, however, it does create some challenges and some barriers in this space. And something that I think is really exciting when you think about the future, even what we're seeing now, again, with that new CRISPR therapy being approved, some of these gene therapies are, are really addressing a very small part of the genome. And really making edits to that small part of the genome, either by removing it or changing it or modifying that faulty part of the genome in some way. And these gene therapies right now are incredibly specific to these genomic mutations are faults, and really, therefore can only address a very small population of patients, which is why we mentioned previously, a lot of these indications for these cell and gene therapies are very, very niche, and very small populations can be impacted by them, because especially in the case of gene therapies are indicated to treat sometimes very, very specific mutations that are really impacting a very small part of the overall genome. And therefore, a small population, if you look at what is developing is what we're calling gene editing platforms that may really change that narrow focus. So, if you think about more of a platform application to a treatment that can almost modify different parts of the gene based on how it's kind of programmed to go into that cell and modify the genome. So, it's almost thinking about it from a much broader perspective. You're looking at an approved maybe an approved platform that can be modified for one patient versus another patient versus another patient to really impact a very specific, maybe different part of the genome for one population of patients versus another, but the mechanisms still being very much the same. So when I look at kind of the science that's emerging, and some of the educational sessions I'm listening to and hearing, I'm very excited by the thought of a much broader application that gene therapies can potentially be used for in a much broader population of patients who could benefit from these treatments, if we can look at it from a slightly different perspective, than how we do today in really thinking about the platform versus the individual gene therapy, treatment kind of perspective. So very exciting times, again, evolving and still read and research a lot of these areas and these ideas, but certainly something that I think will continue to develop and eventually come to market.
As with any new innovation, or anything that's new to market, whether it be technology, or fashion, or food or anything, there are challenges to overcome, if you think about something new people don't know what to do with it, they don't know they need it, they don't know how to handle it, they don't know how to treat it, they don't know how to process it. And a lot of this is education and just becoming educated about what it is that we're working with here and what the opportunities are. When we think about these, again, these amazingly life changing products. So, when I think about the challenges and how we overcome them, I kind of think about the beginning kind of to the end. And the first thing is patient diagnosis. And identification is a real challenge. When you think about sickle cell disease, this is considered a disease that impacts a somewhat underserved population of people, which makes it really challenging for those patients to have access to the appropriate level of care that they might need in order to even just get diagnosed with their condition. So not only being diagnosed but being identified for a clinical trial is another challenge. So really thinking about how a patient getting rolled in a clinical trial when they can't even possibly get to a physician who is able to diagnose and treat their condition in the first place. So huge challenges that patients face that, not a lot of us think about every day, but it's very, very real in the lives of some of these patients who really don't have resources that would give them access to the type of healthcare that they need. So, I think patient advocacy groups come into play here. And innovation with technology, I think is really important. There's a lot of I know, AI is a big term out here. And I think that there is a huge opportunity to use artificial intelligence within EMRs to identify patients who could potentially be candidates for clinical trials. So again, this is an area of evolving innovation as well. AI itself, how do we use it? How do we optimize it, but I do think we're making some progress with AI and clinical trial recruitment and patient identification? So that's one thing. And kind of one of the ways are a couple of ways where we might start to kind of overcome or jump over that that hurdle.
The second one that I'm seeing a lot of is manufacturing and scalability. So, I mentioned previously that right now, most of the products or many of the products require the patient cells to be extracted from their body in a pretty complex process and shipped off to the manufacturer to be made into medicine. That's a very time intensive, complex process that requires a number of steps and a number of costs. So, if you think about that, being a barrier, I think, when we think about future products will be more off the shelf an off the shelf is kind of a word for a cell or gene therapy that does not require the patient cells, but that can be made from either donor cells or other biologic products. So those products could potentially be manufactured much easier or more efficiently because they don't require the patient to donate the cells to the process. So that's another area where we can start thinking about overcoming the hurdle of manufacturing and scalability. And then also just efforts to improve the manufacturing efficiency overall. So right now, these processes being very new to manufacturers, there are a number of outsourcing models that are being employed to improve manufacturing and really that chemistry, manufacturing, and controls that are very important to making sure that the product gets manufactured in the correct and most safe way and improving those processes and creating efficiencies around those processes are a huge, huge initiative and a huge focus for the manufacturers and developers of these products. So, I think we have a long way to go there too. But I think we are headed in the right direction, knowing that that is an area where we need to focus and continue to innovate and improve.
The next thing that I would mention is, like I mentioned, kind of the broad application of the science moving kind of from that single product to more of a platform-based product offering, just the science and the research is moving really quickly in this space. And I think we're just going to continue to see a table before our eyes where we're going to see right now single product per single patient. And we'll continue to see that science moving way beyond that. And hopefully, in the future, we'll be able to be so much more efficient in address so many more patients and people with debilitating conditions through this technology.
One more thing, the patient experience. So, I mentioned some patients don't have access to the level of care that they need or deserve. So limited access to treatment, not only diagnosis, but also many of these products are available through what we call certified treatment centers. And many of these certified treatment centers are in metropolitan areas where there are large academic centers and experts in in these conditions in treating these conditions. Many people don't live near those centers. So actually, accessing a treatment center that a certified to administer these treatments can be a very significant challenge as well. Patients often have to travel to get to these treatment centers. And oftentimes in the case where they're required to donate the cells and go through other processes prior to receiving the infusion or the administration of the product, they can be required to stay in the hospital for a number of weeks, or even once and then next also about adverse event management. As these treatments become more and more sophisticated, I think we'll see less adverse events. But with the parties themselves. For example, we're very used to seeing a certain set of adverse events that physicians thankfully are very good at treating and managing at this point. But I think as these treatments evolve and emerge, we'll see fewer and fewer adverse events as we home in on more sophisticated and more precise treatments.
Finally, I guess one more thing, you cannot speak about these products without talking about the price tags, right? I think the most recent we looked at these products on average for at about $1.5 million per gene therapy, again, on average that could go up or down depending on the day and what gets approved, cell therapies are a little bit lower being in the hundreds of 1000s versus the million-dollar range. But regardless very high-cost products. I think when you think about the price, there are a number of pieces that go into that. Number one being kind of that payer access and making sure that the payer is going to pay for these medications, that your insurance provider is going to have a policy that accepts the payment of these products and the administration of these products through their benefits. And the patient still may have financial barriers. So, making sure those patients can access any programs that are available to them that might support them financially, whether that be through foundations, or through the manufacturer themselves. And then I think one of the most interesting areas that is really important when we think about the value that these medicines deliver is keeping track of the outcomes, right measuring, did the patient have the outcome that we expected? Did the patient experience the durability that was seen in the clinical trial? Was that patient experiencing the same type of durability in the same type of outcome and potentially, the cure that many, many patients hope for that these medications are we hope that they will deliver and really measuring those outcomes and elevating the story around the value. So, if these patients have been experiencing hospitalizations every month for the last three years, and this product might cost $1.5 million? Is this product actually a much better deal than what they were experiencing financially previously? And many of us believe that these products are actually very, very much worth it from a financial perspective and a patient outcomes perspective. So, although the price tags seem high, when you compare those to the costs that these patients are incurring on a regular daily basis, treating their diseases, so that price tag might not seem too high after you evaluate some of those other factors. So, I give you a long list there. There are a number of challenges, but I also feel like so many opportunities, and I know there are so many of us working together to overcome those challenges every single day.
Patient advocacy groups themselves have evolved so much over time, advocacy groups used to be really a resource for patients to find kind of camaraderie and commonalities with others who shared their condition and who shared kind of what they were going through some of their challenges in daily life. And those advocacy groups have really evolved into very highly sophisticated organizations that support these patients, not only through that personal experience, but through the financial aspect through clinical trial opportunities, and also through impacting even Healthcare Policy and payer policy. So, I think that advocacy groups are an excellent resource, not only for patients, but for healthcare providers of all kinds to be aware of what advocacy groups are there and to help refer patients to those advocacy groups to help them find whatever type of support it is that they need. There are a number of advocacy groups that are really, really excelling in that clinical trial support, sometimes it is a key offering that those patient advocacy groups really exist around is getting involved in clinical trials. So, there are a number of different apps that have been developed where patients can go to the advocacy website, learn about the app, download the app, put in their personal information, and actually receive alerts when potential clinical trials that they might qualify for, are available. So really leaps and bounds beyond what clinical trials used to be an enrollment used to look like, even five or 10 years ago. So really exciting areas where advocacy groups are getting involved in them access to these patients that other healthcare providers may not have, because advocacy groups are free to join their online patients don't have to go anywhere, they don't have to pay anything, they don't have to be intimidated by a physician or a healthcare provider, they don't have to really take any risk, they can really join these advocacy groups or take part in some of the work that the advocacy groups do without feeling any kind of pressure to reciprocate or go to the doctor or spend money or really anything in these advocacy groups help connect them with the level of support that they need or that they're looking for at what point in time that they're, they're looking for that support. So, I think those are some really important areas where advocacy groups get involved.
I think one of the things that I love about advocacy groups, and that we've seen them really come to rise to the occasion on with cell and gene is that they kind of call it all the patients opinions and voices, and they serve as one much louder voice that can really influence again, healthcare policy payer policy, and really help the overall political and healthcare environment take notice of this patient population, and that they they deserve attention. They deserve resources, and they deserve the treatments that they need. So, I think advocacy groups again, have evolved leaps and bounds since even five or 10 years ago. And I think they'll continue to evolve to support patients and support the healthcare system in help improve efficiencies that we see on patients accessing the care they need. The other thing that's interesting about advocacy groups is they some of them work with the FDA, and they help get patient voices included in FDA advisory boards or other FDA meetings where the FDA could be potentially making a decision regarding approval of a product or not approval of a product, and they like to hear from patients and patient voices are often kind of sponsored by these advocacy groups and can really help when the FDA is reviewing those products, they really do want to know how the patient views the treatment, what their experience has been. And they do take that into account when approving these products for very rare diseases. So, a lot of different areas where advocacy groups can really help elevate these patient populations that mean these products and treatments.
I spoke a little bit to that with the CRISPR cell and the CRISPR gene therapy that was approved for sickle cell disease. And one of the things that I always say, being a pharmacist in the innovative therapy space, whether it be 20 years ago or today is, it's amazing when these products come to market, or you're researching them in the pipeline, some of these conditions you never knew existed. So as a pharmacist, or a physician or healthcare provider, you have to figure out what that condition is and learn about it, learn about the treatment, learn about the medication, the mechanism, and the science behind it, so that you can help educate others. So, you really get to see firsthand how quickly these things are evolving and how, how little we know about the healthcare environment that we serve as healthcare providers. So, it really is humbling when you think about some of these products that have been approved in the past and will be approved in the future for conditions that affect extremely small populations of patients. And very, very rare genetic mutations that are being addressed by these gene editing or other gene therapies, and how much we have to learn. So, I think when you look at the progress we've made today, some of these medications that have been approved, again, for hemophilia and specific mutations in muscular dystrophy, Duchenne muscular dystrophy, very, very specific populations of patients are being impacted by those. I
f you look at the pipeline, there are over 2000 cell and Gene and other innovative therapies were waiting for patients are waiting for. So many previously untreated conditions will be evolving in that pipeline as well. My role at Cardinal Health and being a healthcare provider in general, is to make sure that I understand what's in that pipeline, what conditions are coming? How can we prepare the market? And how can we prepare payers, providers, physicians, nurses, and even patients for these products that are coming in these conditions that really possibly no one's ever heard of. So, I think there's a really unique opportunity to take advantage of the learnings that that we can see and really prepare the market for what's to come. So, I think it's a really exciting time in that pipeline. We think about cell and Gene, as you know, oncology a lot of people affiliate cell and gene therapies with oncology or hematologic conditions. But when you look in down into that pipeline, we see treatments in the cardiovascular space, endocrinology, retinal conditions, as well as neurological conditions like Parkinson's, epilepsy, multiple sclerosis, and others. So, the future is bright right now, it is very, very small market, it's pretty niche. But I think we're here to see this market really grow and expand into some areas that are going to be extremely impactful for large populations of patients.
I think we'll be surprised no matter what we think we'll see, I'm sure we'll see innovation that will just absolutely surprise us and continue to amaze us and allow us as we move into the future. But right now, if I had to kind of think about what I would expect, is I think we'll see continuing focus on products that are more off the shelf. And when I say off the shelf, meaning they don't need those patients, individual cells for manufacturing, so either allogeneic products or products that are gene therapy products that can kind of be that platform-based product that can impact more segments of the genome or a larger population of patients. So fewer products that require patient cells and that patient kind of done intensive patient process going through donating cells and preparing them for infusion, moving on to more of an off the shelf type of product and manufacturing.
Second thing may be more focused on manufacturing scalability and efficiencies. I think we all are seeing the manufacturing processes are extremely complex, extremely challenging. And really one by one, manufacturing is very difficult to scale. I think we'll see more focus on manufacturing scalability and efficiency. Hopefully that will lead to prices potentially becoming more manageable or a little bit lower due to those efficiencies and larger manufacturing scalability so that we can really ensure that the patients who need these treatments can access them.
The third thing I would say more attention on those platforms that I mentioned versus the individual gene being altered by One therapy. So potential changes. And I would not promise this, but I think some of what I'm hearing is just more openness to the way that the FDA views products in the gene therapy space and evaluating products maybe on a broader scale. And again, thinking about those platforms versus individual treatments for individual gene editing or gene modification. So again, I will claim not to have a crystal ball at all in that, but I'm just hearing so much in that space, that it seems inevitable that we'll move towards something more efficient in that area.
Finally, I think one of the most interesting things from a clinical perspective is I think we'll see additional focus on the outcomes and the financial risk sharing between the parties involved. So again, going back to that patient experience, and did they did they get the outcome that they expected from this treatment? And clinical trials, we see certain response rates, we see certain durability rates are the same rates that we see in clinical trials translating into real life for these patients in the real world. Does real world evidence support that? And what is the value associated with that? And we call this risk sharing or outcomes or value-based agreements between providers and manufacturers, or payers and manufacturers, I think we'll continue to see a push for some financial risk sharing between some of the parties involved in cell and gene therapy and these treatments in the future as well. So those will be my key, my key things if I were to kind of think about what I would expect to see in the future. And again, I'm sure I'll be humbled and surprised with things as they continue to evolve.