Print BCTV: Impatient Patients -- How patients are driving transformational therapeutics in MS

Impatient Patients

Transcript of BioCentury This Week TV Episode 163



Scott Johnson, CEO, Myelin Repair Foundation

Gail Maderis, President and CEO, Bay Bio

Dan Mendelson, President and CEO, Avalere Health

Ken Thorpe, Ph.D., Professor and Chair, Department Health Policy and Research, Emory University




Centers for Medicare and Medicaid Services

National Institutes of Health

Food & Drug Administration



Steve Usdin, Senior Editor




STEVE USDIN: Repairing the brain to treat multiple sclerosis. This week, we'll speak with patients who are taking the search for new medicines into their own hands.


And we'll hear what the Affordable Care Act is doing, and what it isn't doing, for patients with chronic diseases like MS.


I'm Steve Usdin. Welcome to BioCentury This Week.


NARRATOR: Your trusted source for biotechnology information and analysis. BioCentury This Week.


NARRATOR: More than two million people around the world are affected by multiple sclerosis, a debilitating autoimmune disease. MS is caused by the destruction of myelin -- a fatty protective coating that surrounds nerve fibers.


Without myelin, signals can't move through nerves, causing loss of muscle control and other symptoms. Immunosuppressive drugs slow and prevent myelin destruction, slowing progression and helping patients manage their disease.


But even though patients have more therapies than ever, nothing repairs damaged myelin, so they can't reverse the loss of muscle function.


Since 2004, the Myelin Repair Foundation has been funding, conducting, and coordinating research with a goal of getting a drug to patients by 2019 that can restore damaged myelin.


The goal is ambitious. Success will require cutting in half the time it usually takes to go from a concept to a new drug. If it works, the Myelin Repair Foundation will not only change the lives of MS patients, it will also create a model that could be applied to other diseases.


In the meantime, patients living with MS are wondering how the Affordable Care Act will affect their ability to access the expensive drugs they depend on. We'll address that in this week's Affordable Care update.


STEVE USDIN: We're joined today by Scott Johnson, CEO of the Myelin Repair Foundation, and Gail Maderis, President and CEO of BayBio.


And I should start by saying that you're both MS patients, as well as being actively involved in trying to drive the development of new drugs.


I want to start with you, Scott. You said that the model for developing new drugs is broken. There have been a lot of new drugs for MS developed and come on the market in the last few years. Do you still think it's broken?


SCOTT JOHNSON: Yeah. When I say that the system's broken, I think that's for all diseases, not just MS in particular.


And there are several drugs on the market now for MS, all of them are to suppress the immune system. None of them are about repairing damage, and that's really what we're focused on, is how can we repair past damage?


STEVE USDIN: And Gail, you've kind of seen it from both sides, from being CEO of a biotech company, now leading a trade association. You're on the board of a company that's developing an MS drug.


Do you think that there's an opportunity here for patient organizations like the Myelin Repair Foundation to make a difference in the speed of which -- of drug development?


GAIL MADERIS: Absolutely. The disease foundations have been instrumental in investing in both high-risk drugs that really are breakthrough drugs, and also in developing knowledge base tools, access to patients and thought leaders, that can help advance the whole field.


So we see the disease foundations playing a role both in developing specific therapies, but also really raising the tide of the knowledge base and providing tools, such as biomarkers, animal models, and assays that allow multiple companies to more rapidly advance their drugs or discover their drugs, and also to improve the odds of success by having the best state-of-the-art tools.


STEVE USDIN: Scott, you've said, I've heard you say, that your goal is to be able to shave 50% off of the time that it takes to go from discovery to a drug.


How are you going to do that? How does a group like the Myelin Repair Foundation have that kind of dramatic result?


SCOTT JOHNSON: Well, and that is a dramatic result. And then, you're absolutely right. We're not shooting for just 5% or 10%. We really are looking for actually 50% to 70% of the time. And you asked about the, kind of why I say that the system is broken, it's because it does take a long, long time to get all the way from discovery biology to FDA approval.


And there's a lot of different participants in that value chain. And I think that one of the things that we do to try and speed that up is be an overall coach, or manager, and think strategically about the entire value chain.


And I think that's one thing that really does set us apart, is that we try and work with the academics to build collaborations there in a way that I don't think companies could do very well.


We also can work with many companies at one time, and partner with them. And we also work hard to bridge the valley of death and to validate discoveries that are made in academic research so that they're positioned well for pharma to pick up. And that's kind of how we try and address that.


And what we find is, all those different elements all kind of work together and build on each other in terms of taking time out.


STEVE USDIN: Can you give some examples of some of the specific things that the Myelin Repair Foundation has done to try to move the science ahead?


SCOTT JOHNSON: Sure. So if we start at the front end, on the discovery biology side, one thing that we do that I think is very different is, to do really great science today, you've got to be really narrowly focused.


But when you're talking about any particular disease, you need a multi-disciplinary team to really attack that. And there really aren't mechanisms or avenues in academia for true, deep collaborations.


So what we did was actually build a collaborative team that includes now about 45 full-time equivalent scientists in academia that we fund.


And we manage that process in terms of, we worked with them to put together a research plan. And that might sound kind of just like a basic thing to do, but most disease organizations do not have a research plan. And having a real road map for where you want to go that you constantly update is really key.


STEVE USDIN: And Gail, you did a study that's come out recently about collaborations between patient groups and industry.


One of the things that I found interesting in it was that there's a kind of misalignment in some cases between their expectations of what they're going to get from these collaborations.


GAIL MADERIS: Well, I think one of our findings was that there needs to be good alignment. And that starts with understanding what the goals of the project are and what the outcomes of the project would be.


We actually saw a very good alignment in a number of areas -- understanding patient needs, caregiver needs, access to clinical trial designs and centers, and clinical trial enrollment.


So in a lot of areas, there was very good alignment. There were a few areas that were misaligned, and those tend to be a lot on pricing, market research.


But I think some of that is just terminology. That in business, we oftentimes talk about understanding the market as a euphemism for understanding patient needs.


And yet, disease foundations may interpret market research as increased drug sales, not understand patients or understand the fit of a drug.


SCOTT JOHNSON: Thanks. We're going to talk more about that. And later in the show, we're going to talk about what the Affordable Care means for patients with chronic diseases like MS.


NARRATOR: You're watching BioCentury This Week.




STEVE USDIN: We're talking with two MS advocates who are also patients -- Scott Johnson and Gail Maderis.


I want to ask both of you, most of the drugs that are on -- all the drugs that are on the market today-- work on the immune system. They don't actually prevent the MS from progressing, they certainly don't give anybody function back.


You've got an idea, in the Myelin Repair Foundation, that you can address the fundamental causes of MS. How's that going to work?


SCOTT JOHNSON: Well, if you think about it, myelin forms in the body as you grow, and I think it continues to form until the late teens, typically, they see the brain myelinating.


So we know that myelination is a natural process. Also, most likely, healthy people who don't have MS, like other cells in your body, there's probably myelination taking place.


And myelin is actually the coding on your axons, it's almost like an insulation, and so it's probably being remodeled constantly.


And so likely, in MS, the real problem is that when it gets damaged, that repair process is being prevented. So we see this as a doable target, because essentially, we're not trying to convince the body to do something that's foreign or different. We're just trying to allow it to do what it's been blocked.


And so the focus has always been on the immune system in MS because it was considered an autoimmune disease, and also, people really didn't think you could repair the brain. And so that's really what we're talking about is repairing the brain.


STEVE USDIN: So Gail, NIH spends $116 million a year on MS. The market for MS drugs is $10 billion a year. Why is something like the Myelin Repair Foundation even necessary? Why isn't this happening from the public sector, from the private sector, already?


GAIL MADERIS: Well, there's been a lot of treatments and advances for MS. Most of those are -- all of those right now -- are attacking and suppressing the immune system.


So there hasn't been a lot of research into the repair of myelin, which has long been the Holy Grail. But it's a tough, high risk approach, and a lot of the pharmaceutical companies and biotech companies just haven't had the tenacity to work on that.


STEVE USDIN: Well Scott, you've certainly got the tenacity to work on it. What are the milestones? What have you achieved so far, and what are the milestones we might see coming up in the next few years?


SCOTT JOHNSON: It's a really good question, because actually, when we started, as Gail pointed out, no one was interested in myelin repair. No one really thought it was possible or really therapeutically relevant.


So we actually set out a 15 year plan. I mean, when we started, people said well, maybe in 50 years there'd be a myelin repair. And we said well, we want to do it in 15.


And so really, our first interim goal, we said well, in five years, we want to have done enough great discovery biology that many companies begin to view it as an area that they should start thinking about.


So we were really pleased that within five years of the time we started funding research, I'd say there were at least a dozen companies at that point then that really saw myelin repair as the next generation of MS treatment. So that was our first five year goal.


Our 10 year goal was to have our first clinical trial. And we actually -- the ten year point won't come until next year, but we actually beat that goal by about 30 months.


We had our first clinical trial began at year eight.


STEVE USDIN: And that was for an autologous --


SCOTT JOHNSON: Right. That's exactly right. Mesenchymal stem cells. Yes. So our ultimate goal was to have a treatment to patients within 15 years. And so we've really made a lot of progress on all those fronts, and I think that there are, like I said, many companies now that view it as really the area they should focus on.


The bar is actually set relatively high in terms of immunosuppressants. And I think the body -- we've been able to pull together such a great team. We've got about 80 scientists now working on myelin repair. And this is a worldwide team, and it truly is a team effort and it's made tremendous strides.


STEVE USDIN: So the goal, I mean, you said 15 years, so that's what, 2019 that your goal is to have a drug on the market that's doing myelin repair by that time?


SCOTT JOHNSON: That's right. Yep.


STEVE USDIN: And I imagine that myelin repair is not limited just to MS. There are probably going to be applications, if you're successful, in other kinds of diseases.


GAIL MADERIS: Absolutely. So the learning that is taking place at Myelin Repair Foundation could impact other diseases of the brain, including dementia and --


SCOTT JOHNSON: Alzheimer's.


GAIL MADERIS: Alzheimer's, yeah.


SCOTT JOHNSON: Parkinson's.


STEVE USDIN: And do you think that this approach of having a foundation that's kind of trying to take an overarching view, and basically patients taking control of their destiny, or you're trying to. Is that something that's applicable outside of the MS space?


SCOTT JOHNSON: I think absolutely. And it's funny, I don't really think of myself as a patient advocate, which I think is the label. But I really see us as pioneering a new way for non-profits to be the overall coordinator of research.


And I think that there's a lot of different participants in that value chain. As I said, there's certain constraints that all of them have. And actually, non-profits have more freedom to operate than anyone else in terms of what we can do and how we can work with others.


And I think that's really the key role we're trying to demonstrate, and demonstrate that you could use the same process that we're pioneering and demonstrating on myelin repair for any disease.


STEVE USDIN: Gail, we've been talking about the discovery side of turning science into drugs, but there's also a kind of a translational effort.


Where do you see the role of patient organizations, non-profits, in this kind of translational effort?


GAIL MADERIS: Well, I think the disease foundations can play a role throughout the entire chain, from discovery all the way through market and post market.


In the translational side, they can bring a set of tools such as animal models and other tools in terms of clinical protocols, that can help de-risk the process and provide access to the best practices and cutting edge knowledge to numerous companies that are developing different therapies.


STEVE USDIN: And that's something that you've actually with the Myelin Repair Foundation is that you've created some animal models that the companies are using. You've done a lot of translational work.


SCOTT JOHNSON: That's right. I mean, I think it's interesting. If you talk to an academic scientist, because there's such a disconnect between most academics and the pharma industry, they think when they make a discovery, it's going to magically turn into a treatment.


And what they don't realize is that what it takes to get published in a journal is very than what pharma needs to decide whether they want to begin to invest in a program.


So what we've spend a lot of time and effort -- is we've developed something we call our Translational Medicine Platform.


And the idea behind that is, it's to de-risk and validate the targets that we've identified in our academic labs, and put together data packages in the kind of format that pharma is used to seeing for their internal programs, so they can really decide whether this is something they should begin to invest in.


STEVE USDIN: So you're kind of packaging it up and giving them the opportunity to decide whether something they're going to move forward.


I want to switch to another topic, which is, at the beginning of the conversation, we talked about halving the development time for drugs.


And I'm wondering, if you think that, if you halve the development time for MS drugs, is that going to have any effect on the pricing? Because the pricing on the MS drugs is quite high.


SCOTT JOHNSON: Well, I come out of the business world, and in the business world, you always talk about, competition drives prices down. And so my natural assumption is that that would be true.


I mean, you hear numbers being bandied about now that, you know, on average, it's maybe up to $5 billion to develop a drug. And so clearly, companies have to recoup those costs through high prices in order to be able to stay in business.


So we think if you could really cut the time substantially, and the cost, and increase the rate at which you have successes, then that's huge and should bring down cost.


And our idea is that, we want to try -- as a non-profit, we're kind of agnostic. We want to get as many potential myelin repair therapeutics moving through that pipeline.


So our hope would actually be that there might be two or three that hit the market. And so that would, we hope, cause pricing pressure and result in more affordable therapeutics.


STEVE USDIN: Great. Well thank you. We'll continue our discussion about chronic diseases like MS in this week's Affordable Care Update.


NARRATOR: Healthcare is changing, and we're changing, too. Each week, watch BioCentury's Affordable Care Update -- a special part of every show dedicated to keeping you informed about this unprecedented transition.


And watch all of the weekly updates in one place at any time, only at




STEVE USDIN: I'm pleased to be joined by Ken Thorpe with the Partnership to Fight Chronic Disease and Dan Mendelson, CEO of Avalere Health. Both of you, we started out this show talking about developing new drugs for MS. But patients who have MS, they have to get access to drugs and to care now. How's that going to work under the ACA? What are the issues that they're going to have to think about?


DAN MENDELSON: Well, when patients go to sign up for products under the exchanges, they're going to see their premiums. What they really need to be thinking about, though, is the fact that deductibles in these plans are very high. $2,500 on average for the silver plans, and $5,000 for the bronze plans. And in addition, the co-payments that they will have to pay specifically to their drugs are also going to be very high -- typically a percentage of the cost of the drug.


STEVE USDIN: So, Ken, what does that mean for people? What's that going to mean for access, for people's quality of care if they're going to have these very high co-pays, for example, for drugs? They're going to have really high co-pays when they go to specialists.


KEN THORPE: Well, it's going to be a problem, because many of the new people signing up on the exchanges are people that have pre-existing conditions and perhaps in their state couldn't get access to health insurance coverage. And so they're going to be coming on, buying insurance now with the expectations that they're actually going to get their prescription drugs and other services almost fully covered for. So the concern that I would have is, if you have a patient with three, four, five chronic healthcare conditions --


STEVE USDIN: Which isn't uncommon.


KEN THORPE: Not uncommon at all -- that they're not going to fill and refill their prescriptions because of the expense. You could look at a plan where you're paying 40% of the cost of providing or getting prescription drugs in addition to the $2,500, $2,600 deductible. So that's a real heavy lift for a lot of people.


DAN MENDELSON: Now, some of the lowest income people will have premium and copay assistance, so that will help a little bit. It's kind of the moderate low income. And one of my concerns about this construct is that it looks like pharmaceutical companies are not going to be allowed to offer copay assistance for this population, and that's something that could really help a lot if they're able to do that.


STEVE USDIN: That's a huge deal. And that was something that was in flux. Is it really a done deal that it's known that they're not going to be able to do that?


DAN MENDELSON: It hasn't been decided yet, but those are the indications. And again, I think that the question really is whether they're allowed to, and if not, then perhaps through some kind of a foundation structure.


STEVE USDIN: So that's something that drug companies have done in the past. When patients are on Medicare and they can't give co-pay assistance, they give money to foundations, to patient groups, and those groups in turn give money to patients to help them with co-pays. But it's not really a very efficiently way to do things, is it?


DAN MENDELSON: No. I mean, again, it's problematic. I think that it really gives a major issue back to the policymakers. And it's going to really be incumbent on the Administration and the Congress to think about access and to make sure that people who need these medications are actually getting them.


STEVE USDIN: So Ken, one of the other elements of the Affordable Care Act, besides the exchange structure itself, was setting up so-called ACOs, Affordable Care Organizations. In theory they could be really good for people with chronic diseases like MS. How's that working out?


KEN THORPE: Well, the Accountable Care Organizations could potentially be very effective. The real key is whether they really change how they practice medicine. The focus has really got to be on preventing the growth in chronic disease in the first place and more effective engagement and management of patients with chronic healthcare conditions. And that's not something that we do very well.


So I think the real key is not just linking providers together in an organization. The real key is, are we really changing the way that we both prevent and deliver healthcare services to chronically ill patients?


STEVE USDIN: And are the ACOs going to be able to do that?


KEN THORPE: The successful ones are going to have to figure out a way.




KEN THORPE: The only way for them to really save money is by keeping their populations healthy, cutting down on readmission rates, making sure that preventable hospitalizations don't happen. That's going to be a change in the style of how those services are delivered.


STEVE USDIN: And you said the successful ones. Another way to look at it is that some of them are going to be basically looking at a capitation model where they're going to cut back on services in order to hit their numbers, right?


KEN THORPE: Some of them are going to focus on it that way. But again, to the extent that we're really measuring quality and we have metrics that are looking at performance, they may not come out so well on the quality side. So I think, again, to really do this right, it's not just cutting services. But it's really engaging patients in new ways of delivering service.


STEVE USDIN: You know, I want to talk about that when we come back, and talk more about the things other than the exchanges that are important to happen for patients with MS and other chronic diseases. We'll be right back.




STEVE USDIN: We're back with Dan Mendelson and Ken Thorpe talking about the Affordable Care Act and chronic diseases. Ken, the Affordable Care Act, the issues we've been talking about have all been around the exchanges. But there's actually a lot bigger issue, particularly around Medicare, for patients with chronic diseases. Is the Affordable Care Act getting us to where we need to be?


KEN THORPE: No. I think that's a real problem. The Affordable Care Act largely dealt with expanding health insurance, a critically important component of reform. But what it didn't do is really deal with some of the long term financial issues facing the Medicare program. So for example, since 1985, about 80% of the growth in Medicare spending is due to an increase in the prevalence of chronic disease.


So today about a quarter of Medicare patients are diabetic. That's double since the mid-1980s. Another 25% are pre-diabetic. Yet there's nothing in the Medicare program that's really designed to effectively prevent the progression towards diabetes, and there's certainly no care coordination in the original Medicare program designed to keep diabetic and other chronically ill patients healthy. So I think that those are two major holes that we need to fill in the discussion about entitlement reform.


DAN MENDELSON: You know, agreeing completely that the Affordable Care Act does not solve all the ills of the healthcare system, I do think it had some provisions that did move things along a little bit. So for example, the quality provisions that are now used to pay Medicare Advantage plans were all passed in the Affordable Care Act, and they do include diabetes metrics. And so you have now Medicare health plans that are more focused on chronic disease. And that's a positive thing. It hasn't fixed everything, but it is positive.


STEVE USDIN: One of the things, when you look at the press releases coming out of CMS, the agency that runs Medicare, there's so many pilot programs, it looks like the Federal Aviation Administration rather than Medicare. Do we have too many pilots? Is it time to move from pilots into action?


KEN THORPE: I think we have too many pilots. I mean, I think we need to have a balance of implementing things that we know that work -- and we have a long history of data on interventions that we know are very effective, programs like the Diabetes Prevention Program, a very intensive lifestyle intervention program that works. Elements of care coordination, health coaching, transitional care, team-based care. We know it can be very effective.


So I think that we need to have a transition towards implementing best practice programs. And then do targeted pilots on areas that we don't have enough information on. So for me, personally, I have pilot fatigue. We're not one pilot away from a miracle here. We need to have a balance of implementation and targeted pilots.


STEVE USDIN: So Dan, one of the issues is Congress isn't going to pass new healthcare legislation any time in the foreseeable future. Does CMS, Medicare, do they have the authority to translate some of these pilots into real programs that are nationwide now?


DAN MENDELSON: It's interesting, Steve. While the payments are coming down in fee for service medicine, more and more Medicare beneficiaries are voting with their feet and leaving that system and going to Medicare Advantage. So we now have more than 30% of Medicare patients opting into health plans that actually do have an incentive to care for patients with chronic illness that is more significant than what we see in fee for service.


STEVE USDIN: Well, thanks. That's this week's show. I'd like to thank my guests, Scott Johnson, Gail Maderis, Dan Mendelson, and Ken Thorpe. Remember to share your thoughts about today's show on Twitter. Join the conversation by using the hashtag #BioCenturyTV. I'm Steve Usdin. Thanks for watching.