A new medical treatment is giving sight to blind patients and so much more. It's called gene therapy and, though costly, there's no doubt that it’s changing lives. Learn more about this new health care advancement from pharmacist Jay Weaver, AVP of Pharmacy Sales Operations at Blue Cross and Blue Shield of Texas (BCBSTX).
Listen to Part 1 of Can changing your genes change your life?
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Blue Promise is a podcast and online video blog that aims to address complicated health issues with candid conversations from subject matter experts. New editions are published regularly and are hosted by Dr. Dan McCoy, President of Blue Cross and Blue Shield of Texas, and his co-host, Ross Blackstone, Director of Strategic Influence.
DAN:An innovative new class of therapies may cure some very sick patients who previously had little hope but their six figure costs could render them inaccessible. Unless health care stakeholders can come up with equally innovative ways to pay for them. We're talking about gene therapy.
DAN:Thanks for joining us for this edition of Blue Promise. I'm Dr. Dan McCoy and I'm the President of Blue Cross and Blue Shield of Texas. I'm here with MY co-host Ross Blackstone.
ROSS:Thank you Dr. McCoy and we have Jay Weaver here he's Associate Vice President of Pharmacy Sale Solutions for Blue Cross Blue Shield of Texas. Thanks for being here.
ROSS:Okay. So, we were talking earlier in our previous segment about how gene therapy is getting some notoriety I heard recently on NPR a story about how a group of researchers are conducting a trial the 660 volunteers with early symptoms of Huntington's disease. Apparently, this disease affects about 30,000 people in the United States. And if just one of your parents has Huntington's disease there's a 50/50 chance that you could get it too and it is deadly. So. It's a little concerning yet a little exciting that's something called gene therapy might be able to help. So, Jay let's start off this segment by saying by just kind of. Letting people know. If I've heard about gene therapy and I'm I'd like to know a little bit more how do I figure out if I might be eligible for gene therapy how do I go about accessing it. Where do people learn more information.
JAY:It's a great question. So, one of the key places is there's a rather large repository of public information since much of this research and much of the work that's been done around these therapies has been is actually we've been funded by you and I,by tax payers because it's a really important development and improvement technology and you know human innovation. There are some public sites some government Web sites that actually share, what are the ongoing trials the trials that are that are being being conducted for these kinds of therapies and how do you apply to become part of that. So there's a lot of great information out on the NIH web site and some other public information along those lines. So I would start there and then certainly there's there's just a lot of information on the Internet around these therapies. But I would say you know like like anything we'll be very careful as we go to you know I call it Dr. Google and start looking for information. You know you have to really look at the source of the information and was the bias of the person publishing or the organization publishing the information and really you know push that through the lens of of you know. Trusted intermediaries or learned intermediaries that can help you digest all of it.
DAN:Seems like one of the best ways to learn about treatments for these unique diseases and unusual diseases are support groups for like-minded patients or their families because often they're there really have someone researching and looking and promoting that drug out there.
DAN:So one of the things that happens on these therapies is that let's just we'll just kind of cut to the chase and say there we talked to previous that they're very unique. They're a very small set of people they cost incredible amounts of research dollars and take decades to develop to be able to create these drugs to market which. Let's just put it on out there. They're pretty expensive drugs but there's more to the story about evaluating these drugs and just the cost.
JAY:Yeah absolutely. So. It might be good to just take a moment and talk a little bit about that development process and then how that get pushed out and then kind of think about those costs in context. I think to your point which is think about the care that we provide to people with very rare and serious conditions and then what is it that we need to consider when consider the cost of something like that such as inpatient care for folks and all sorts of other medications that may or may not work for their condition.
DAN:So you used Cystic Fibrosis for example before, but I mean, untreated, Cystic Fibrosis is an incredibly expensive disease and not only that risky because there's multiple hospitalizations, multiple treatments, often Intermittent pretty dangerous drug use, to try to manage some of the complications of that disease..
DAN:Over years, Is that fair? Plus, the impact on the patient and their family’s ability to function in their job or school and to enjoy their activities of daily living, pretty fair right?
JAY:Absolutely. I mean the costs of just you know the background cost of that disease are immense.
DAN:And the risk of treating it
DAN:Right there so. So, I guess what I'm trying to go is there's not going to be. I think people think the first thing that pops up on these gene therapies is number one is that oh my gosh they're a lot safer than the old therapies. Well not necessarily. So there has to be some evaluation on safety. And then number two they're incredibly expensive. Which they are but these diseases are often pretty expensive to begin with. Right. When you look at the totality of a patient's care so you're really using the same kind of lens that we use is just being focused a different way.
JAY:Yeah that's a fair point. And you know like other you know expensive and potentially dangerous therapies. We do a lot of evaluation to say okay what does what does the evidence out there that says you know that this works and then what are the what are the safety considerations which I think are are critical and sometimes overlooked. We're talking about delivering these therapies with a virus. And we talked to earlier in the first segment about even a common cold which is caused by virus has different effects, our body reacts to a virus any time you put something in our body that's foreign our body wants to react to and says hey this this isn't supposed to be there. You know we've evolved our immune systems over time to respond and react to viruses you know through. We intentionally give people vaccines to respond to viruses right. So, it's not, It's not, questionable that we would, you know potentially worry about someone's immune system attacking a virus that's intentionally put in the body. So, as we begin to evaluate the therapies we have to can take that in consideration. Are you someone who's going to respond to the virus, negatively or is your immune system pretty much okay with the virus being put there and then depending on that we could consider you as a as a candidate for that therapy.
DAN:So let's kind of disect what you say there because I think that's pretty important. So the first thing is is that to make sure that. You're competent you're not going to get sick from the virus. All right one and then the second thing is you mentioned you'll make antibodies to it. And many of these gene therapies both now and in the future they often rely on a fairly similar virus to get it in your body. So. You what you don't want to do also is get a treatment that didn't work the best for you. Only to have one two years from now come out and then you can't use it because you had the virus before that.
JAY:So that's a great point and I think that's probably one of the bigger concerns we look at advocacy groups that are looking at these therapies and folks are saying OK well when when do I enter this system when is it that it's the right time for this treatment.
DAN:Now I'm the doctor here someone tell you if you hold the hand of a patient that's got a horrible disease a lot of times they're going to want treatment for the disease even if it's not perfect. So one of the arts that going to be here to solve is the fact that when these drugs first start coming out everybody's going to want them because their child is sick and you can understand that the challenge of managing that kind of discussion around safety and appropriateness of that particular vector and that drug, is that fair?
JAY:Yeah that's I think you're spot on I mean you think about all of us who went into health care are here because we want to help people want to help people with these terrible diseases. Number one Number two it just as humans right and we see the human suffering our inclination is to want to help them as well. So I think that there's going to be a lot of driving forces between both patient groups but also physicians who are wanting to do a great job and help these folks with very terrible conditions. So how do we balance that with, you know, when is when is the right time for that treatment for that person, is the evidence there for that treatment.
DAN:And that's really the second part of it right. I see you get through that viral discussion that we talked about and the next one really is is that gene going to fix the problem that the patient has.
JAY:Yeah. And I think that that's also is a question given that as we talked about in the first segment there are there are certain diseases that are multi genetic. And we don't know always if if you've got the specific form that is going to be responding this gene. So there's a lot of testing ahead of time to say do you have the form of this disease that is that is sensitive. So we'll use we'll go back to a different example for the moment we'll go back to diabetes. We know there's different forms of diabetes there are some people who just simply don't make insulin. We know there are people who make insulin their body cannot use the insulin to bring down their blood sugar. If you were to go in and give a gene therapy to someone who can't use their insulin right because their body is just destroying it you went in and sent this viral vector in and snipped out the piece of insulin information and put a new gene and you say hey we're done and then we still have the same problem because the problem isn't that their body doesn't make insulin the problem is they can't use it. So. So I think that having a realistic expectation using the science. To kind of backstop. Who are the right candidate for this is important because you know as we point out earlier these things are not without risk. Right. I mean. You know we always look at risk benefit sort of weighing. When we make medical decisions and you know we do that from a policy standpoint that's what FDA’s job is really is just to help look at benefit and risk and say is the benefit to society worth the risk. Or in specific patient population is benefit outweigh the risk and I think that you know at a macro level that it's important but at a micro level you know to to your point is if you're a practicing physician as you look at that way you'd say well. Is this. Is this likely to work. Right. And is it worth the risk of the therapy. And he said you know I'm not sure that they've got the right form of this disease It may not be likely to work.
DAN:And I think the issue here too is the stakes are a little higher right because these are permanent in many cases we think they're permanent and they typically when you give that virus to a person that's got the gene it didn't targeted necessarily just a lung cells right. It's going to affect all the cells and so there are some, It's not like you can go back in and turn it off, I mean this is one and done. I mean people I think today people think about medications and they think well if if I have an adverse reaction of high blood pressure medicine has got to get through it and all of a sudden it'll get out of my system. But these. These drugs I'm using that word a little bit loose here. They don't really get out of the system.
JAY:And that's that's true. I mean our our goal is really to fix the problem in the cells and you know some cell lines are more durable than others and in your skin cells turn over a fair amount so if you were to do a gene therapy on the skin cells it may may not last all that long but you know other cells in our body last a very long time. So we want to be able to fix those but I think this whole idea of this one and done we used this example earlier the cystic fibrosis where there's a whole lot of health care costs that occur over decades of someone's life and then and then you know potentially some really big things that happen towards the end of their lives sets up a conversation about this cause the backdrop of this cost conversation is that it's one and done treatment really presents a health care finance question more than a cost of care. Right. It's moving from taking all those costs and paying for them over time. It's kind of like if you had to pay to go buy a house and you had to pay for your whole house at one time. I mean you know if you're independently wealthy that's great. Most of us work and we pay for our house with the mortgage we pay over time that would be challenging to say OK well you're going to get your house you've got to pay for it today with cash is very much the same with these therapies taking all those costs that occur over decades and having to cram into a one-time treatment is difficult and I don't think we've planned for that well and in our current way that we pay for health care.
DAN:Now in fact it's kinda of a health care economics discussion but it's so relevant to this conversation that historically many of these diseases that have had these horrible downstream consequences we haven't had good treatments for whereas a lot of these diseases that have very slow downstream consequences like heart disease and hypertension and even diabetes to some degree. But they affect millions of people. We've really been immune from having to worry about how to finance them because there's so many people in their diffused over such a long period of time. But the challenge of these new genetic disease treatments is that it's a much smaller group of people. The development costs are incredibly high but the payoff could be really big. So the question is how do you It's really bringing that health care financing question to the clear and present danger today. We need to figure it out.
JAY:Yeah and I think that there are there are certain people in the think tanks around the country and around the world that are there begin to wrestle with this is there a different way to new ties that. That value and that cost over time. There are different ways to think about. The way to value those therapies and you know do we do we do it the same way do we do we pay for it like a you know like a mortgage and do we do have a warranty on it. And I think that brings up something else that is an
DAN:A whole new area of thought.
JAY: Right. Value based contracts for example you know as we move from a fee for service environment and health care where it's just like OK well there's a service and we pay for it I know near and dear to some of your guys hearts is. You know how do we begin to share risk and align incentives with other organizations and in this case we're looking at that with the manufacturers of these therapies. OK well. There's a chance they don't work and then what's the warranty if it doesn't work and should there be some sharing in the risk if there's there's sharing in the reward. Right is that we've had a society work to to bring these therapies to market and we're going to incentivize and pay back the folks that did that great work and brought it to market. And on the backside of it do we say if if it fails do we share in the risk.
DAN:Which of all of that helps patients, we had this conversation today because this is an exciting area of science but I think getting around and solving those healthcare financing challenges is really important to discovery because if that didn't get solved its going to be really hard to figure out a way to to put additional money. Into these in this research which. I think If you look at the diseases that could potentially be affected there's a lot of them out there. Jay thanks for being here today and Ross thanks for being here and thanks for joining us for this edition of Blue Promise.
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