Rare Disease Drug Shortage During COVID-19 Effort Creates Dilemma

Mark Dlugoss: Hello, this is Mark Dlugoss, Senior Contributing Editor for Ophthalmology 360. Welcome to the Ophthalmic Project powered by Ophthalmology 360. The Ophthalmic Project is a series of blogs and podcasts that address issues facing ophthalmology today by offering observations and interviews with shakers and movers, in the ophthalmic field. Today, the Ophthalmic Project will look at a dilemma where the increased production of the COVID-19 vaccine has temporarily suspended the manufacturing of the only drug approved to treat patients suffering from Thyroid Eye Disease. Joining me now to discuss the situation is Jeff Todd, President, and CEO of Prevent Blindness, and Sarah Brown, Director of Government Affairs for Prevent Blindness. Welcome to the Ophthalmic Project.

Jeff Todd: Thank you.

Sarah Brown: Thank you. Happy to be here.

Mark Dlugoss: I would like to get some information about the Prevent Blindness organization. It’s been around for 200 years now. Can you tell us about the organization?

Jeff Todd: Sure. Yeah, we were founded in 1908, so we’ve been around for about 112 years now. We’re a very old patient advocacy organization. We were found very specifically to eliminate a form of infant blindness that was rampant in the early 1900s. But we’ve certainly grown since then to do everything eye care related from birth to death, every eye condition you can imagine. Our work focuses really around three pillars; public policy advocacy, public and professional education, and public health expansion.

Mark Dlugoss: What role does advocacy play in your organization’s mission to Prevent Blindness and preserve sight?

Sarah Brown: So advocacy is a pretty major part of Prevent Blindness as a mission, as Jeff said, it’s one of the reasons why our organization was founded. I’d say that one thing that makes advocacy at Prevent Blindness, particularly unique, kind of sets us apart, is that our advocacy encompasses several different kinds. So of course, we’re always trying to build our relationships with lawmakers in Washington to affect change in public policy. We also work to equip patients with advocacy skills that are needed when they interact with their eye care providers, with insurance plans, or with their community networks. So part of our mission to Prevent Blindness and preserve sight is to empower patients to advocate for themselves on behalf of their own vision health while also focusing on policies that allow for this to happen.

Mark Dlugoss: Let me add a question here. It’s about the Congress. With the new Congress in, is Prevent Blindness optimistic about the way things may go and how things benefit not only for the organization but for people suffering from blinding diseases.

Sarah Brown: I think there is quite a bit to be optimistic about with this new Congress and speaking of that, we have our 16th annual Eyes on Capitol Hill patient advocacy event that’s coming up in February. So this is a great opportunity for patients to be able to visit with their lawmakers, to share their stories about vision and eye health, and raise awareness about some of the need for federal investment in programmatic efforts that can help prevent vision loss and eye disease. In terms of the climate here in DC, and you know there’s always a reason to hope that our lawmakers will understand that there’s this need that’s ready for them to work on something that we think is a bipartisan issue. I think things are still coming together in terms of what the agenda is going to look like for the 117th Congress. And considering we’re just about a week into the new administration, I would say the same thing, but I think that there’s going to be a lot of momentum this year.

Mark Dlugoss: That’s good to hear. That’s good to hear. I know that a lot of the ophthalmic organizations have been fighting, not saying fighting, but they get their message across to Congress. Sometimes they’re cooperative and sometimes they’re not. So it sounds like you got a handle on this and hopefully, we’ll see some progress in the years at least in the next four years. Tell me how you work with patients who have been impacted by vision loss.

Jeff Todd: Well, you know as Sarah suggested, one of the biggest ways that we work with them is by engaging them in our advocacy efforts. We find that patients are certainly the best voice, whether it’s on Capitol Hill or at their local state houses, that patients are the best voice to make change as it relates to public policy. So we certainly do a lot of education to educate patients and caregivers about various eye conditions. But we also like to engage them from an advocacy perspective, it’s a way of empowering them to take control of their own eye health and their own condition as well as advancing the mission and empowering others.

Mark Dlugoss: So you cover all eye diseases, every aspect involving problems with the eye?

Jeff Todd: Yeah, we sure do. I mean, I’ve been with the organization 20 years now and, and it seems like every year there’s another condition where there are new treatments out. So there’s a need to educate and inform folks. We do spend the bulk of our time on those conditions that have a broad impact, that impact a lot of people, and have a broad population health impact, but we certainly provide information on pretty much every eye condition out there.

Mark Dlugoss: Let’s start moving into our discussion regarding Thyroid Eye Disease. And first of all, let’s just start with a simple medical definition of what thyroid eye disease or commonly known among ophthalmologists and optometrists, Graves’ eye disease. Basically. What is it?

Sarah Brown: Sure. Well, Graves’ eye disease, broadly, is a disorder where your immune system attacks the thyroid and causes it to produce more thyroid hormone than your body needs. In many cases, not all but many cases, this can lead to inflammation and swelling that stimulates the production of muscle tissue and fat behind the eye, which can cause the eyeballs to bulge out and that’s thyroid eye disease.

Mark Dlugoss: Okay. How many people are affected by it?

Jeff Todd: That’s somewhat hard to determine because thyroid eye disease can easily be diagnosed, but it’s estimated that approximately 15,000 to 20,000 people each year in the U.S Are diagnosed with thyroid eye disease.

Mark Dlugoss: What are the available treatments for the disease as it stands.

Jeff Todd: Well up until Tepezza, which was a treatment that came out about a year ago, there really were no treatments to directly treat the disease. There were some treatments that are some treatments to mitigate some of the symptoms such as steroids, surgery, eye drops, et cetera, but Tepezza is certainly the first treatment available to address the disease.

Mark Dlugoss: So recently, Horizon Therapeutics, who’s the manufacturer of Tepezza, actually had to suspend its operation in order to turn around, well they didn’t, but the contract manufacturer organizations had to suspend in order to produce vaccines for COVID-19. How’d you hear about this issue?

Sarah Brown: Prevent Blindness was initially notified of this shortage from the manufacturer themselves. But patients also started to reach out to Prevent Blindness once they began to learn that their treatments would be disrupted. So since patient access is a pretty major aspect of our mission, that really underscores the need for us to address the shortage through appropriate advocacy channels.

Mark Dlugoss: What have the patients you’ve talked to directly, experienced with this treatment disruption?

Sarah Brown: So since the FDA approved Tepezza in early 2020, Prevent Blindness previously, had heard stories from people whose lives had really been changed for the better as a result of this treatment. So we also continue to hear stories from people who were diagnosed with TED before the approval came out, as such, they have also endured irreversible damage. As well patients who are now stuck with lifelong consequences of this disease, which include multiple surgeries, facial disfigurement, in a lot of cases. And that also comes with the emotional turmoil that it causes for people who have to get acquainted with their new appearance. To put it rather colloquially, a patient described it to me as, think about the worst haircut that you ever had, except that worst haircut is permanent, and think about how emotionally traumatizing it is when you have to live with that every single day.

It’s kind of like that when your face is disfigured by proptosis that this disease causes and along with that the damage to vision that is also irreversible. Those are some of the effects of this disease that patients have had to live with before the approval of this drug. Since the impact of this shortage has already started to impact patients, many providers don’t have a backlog of this treatment to endure through any sort of shortage. So the disruption is happening rather immediately, and it’s happening right now. So some of the patients who have reached out to us we’re actually nearing the end of their treatment cycle, right, as this shortage occurred. And so they’re starting to see the symptoms of their TED re-emerging. Also comes with that, the psychological impact as one patient described to me particularly, and that’s the fear that this disease will return and not knowing if what they’re seeing in the mirror or through their eyes is in their head, or if it’s actually happening.

I think it’s also important to note that the clinical trials did not study what would happen if patients experienced a disruption in the course of their treatment or what would happen if they do not complete their treatment cycles. So all of that has caused patients to really wonder, where do they start when this shortage is over? Do they start the treatment cycle over again, or do they pick up where they left off? Will their insurance company even pay for all eight treatments again, if they do have to start over? Will it be as effective as if they’d just gone straight through the treatment cycles without stopping. And I think in the long-term without continued access to these treatments, TED patients may experience substantial burden due to managing that emotional toll. Of course, there’s a financial strain that’s associated with managing the disease once it’s not treated, as well as the impacts to their overall health and wellbeing because of this disease. And all of that on top of the stress that a lot of people right now are experiencing because of the pandemic.

Mark Dlugoss: Do we know how many patients with TED are caught in the cycle and they can’t get out? Like if it goes over 24 weeks, do we have an idea of how many are actually caught in that vacuum? I guess you want to call it?

Sarah Brown: I don’t know that specific number to that Mark.

Mark Dlugoss: Okay. That’s good and incredible. When can patients expect to receive Tepezza again? I mean, I guess that’s the next question because obviously patients are caught. And then have we gotten any word as to what’s going to happen when the drug is to be back in manufacturing again?

Jeff Todd: Yeah. Again, it’s a little hard to determine because the rollout of the vaccine changes and we’re still trying to understand how that’s evolving. But, what we’re hearing is that absent in the intervention that Tepezza will probably be rolling back out again around the end of the first quarter of this year. So late March, early April.

Mark Dlugoss: Now in that estimation, are you basing that potential rollout, again, based on Horizon taking their drug to another contract manufacturer, or is this just something that they’re just throwing out there? Cause I know they have to go through the FDA, they’ve got to get approval from them to move the drug to another company, which I really think is something like this. You would think that’d be an easy thing to do, but we’re dealing with the FDA.

Jeff Todd: No, I think that’s based on some general discussion that the manufacturer has had with the company itself, that they feel that they’ll be able to produce normal levels around that time. But again, you know, as with Coronavirus itself in the vaccine, there’s so many unknowns about it. And certainly, I think it’s important to just note that, of course, Prevent Blindness is very supportive of getting that vaccine out there. It’s very important. So we’re really trying to balance supporting that vaccine rollout with ensuring that the Tepezza patient isn’t negatively affected by this.

Mark Dlugoss: Yeah. I just hope that, from the sounds of everything, the process to get this worked out with the FDA is quick and fast. I mean, like I said, there’s in ophthalmology alone, there are at least 75 new ones in the last 20 years. I mean, somebody has got to have available slots within the drug. So it’s amazing. I didn’t know, in my research and trying to develop this podcast, was how this whole cycle worked and never realized that these contract manufacturing companies reserve slots for all these drugs, and what happens is they assign each of these. When I first heard this I go, why is Horizon being punished? Because they’re not making this drug. And I found out that everybody works through these contract manufacturing and I’m sure the FDA and the government with the Protection Act is doing everything it can to get the vaccine out and leveling putting everything on a back burner. So to speak.

Jeff Todd: I think, you know, along those lines, as I understand it, these companies, they have the capacity to produce Tepezza. They would just have to increase the level of their activity and in order to do that, they need FDA approval. And that’s where we’re seeing some challenges.

Mark Dlugoss: Now we know so much about this. This question just popped into my head. We know about thyroid eye disease, but I know of at least 10 to 15 other rare eye diseases that are out there. Are they facing the same situation? I assume?

Jeff Todd: Well, so far we haven’t heard of any. We’re definitely keeping our ears open for that. I think one of the reasons is that Tepezza is a relatively new drug. And so they’re in the process of just putting this drug out. So the supply to begin with may have been smaller than many of these other conditions, but we’ve not yet heard of anyone, either within the eye space or outside of it, that that has had a similar situation. But I do suspect that there are others out there that if they haven’t already, they may be experiencing it shortly.

Mark Dlugoss: Yeah. It’s really interesting. It’s just a matter of showing us, cause that’s when just talking to you now and I just go, Oh my God, I know at least 10 to 15 situations that can be a major problem because a lot of these same drugs have to go through a routine of 24 injections, or like in Tepezza’s case, there may be some extent. I mean, even the retina drugs that are on the market now, they still have to go through a cycle of over six months. So it’s interesting to see where it’s all going to unfold. I know several ophthalmic and vision care organizations that like, Prevent Blindness and the American Academy of Ophthalmology have petitioned the government for some easement in the situation. What is Prevent Blindness’s stance on the situation? What is your organization doing in this fight?

Sarah Brown: Well, aside from this particular situation, Prevent Blindness has consistently been supportive of policies that really enable patient access to safe and affordable, and effective treatments. So we really want the FDA to use authority that has been granted by Congress to prevent drug shortages during periods of increased vaccine production, to also ensure that patients who need these medically necessary and sight-saving treatments can continue to access them. Right now, Prevent Blindness has partnered with the American Academy of Ophthalmology who like us, is a member of the vision health advocacy coalition to specifically inform patients and providers of this shortage to also give patients the ability to advocate to the FDA and to use their authority to allow production of this treatment, to move forward. But to also tell their stories about how this drug shortage is impacting them. If The FDA continues to allow this shortage to endure, whatever the reason may be, the destruction continues, our options for this coalition may be such that we need to reach out to Congress to inform them of the situation.

Mark Dlugoss: I’m sure that’ll be high on your priority list when you go in February to talk with the congressmen then.

Sarah Brown: We’ll see what happens for sure.

Mark Dlugoss: Is Prevent Blindness working with other vision organizations, both clinical and consumer groups to come up with solutions or a compromise to this situation?

Sarah Brown: Aside from the Vision Health Advocacy Coalition, the American Academy of Ophthalmology, that I’ve just alluded to. That’s really the scope that we’re working with simply because the AAO has the provider community working through a coalition who also has some members that represent patients with autoimmune conditions, as well as some other vision organizations really gives us the opportunity to leverage that strength in numbers that is so important in advocacy such as this. If I may offer a differentiating anecdote that I learned through one of our interactions with the national health council who represents a myriad of other patient organizations, back in the spring of 2020, when Hydroxychloroquine was in overwhelming demand, as a cure for, as a possible cure, I should not say that, I cannot speak intelligently about that, but, that particular drug, is especially critical for patients who face lupus, for example. And so Lupus Foundation at that point was really facing what was a supply and demand issue. This specific situation that we’re facing with this particular treatment is more on the manufacturing side. So just to differentiate what those two situations are. We are learning some best practices in how lupus, specifically, went through the FDA to make sure that the drug that was needed for their patient population was also still available.

Mark Dlugoss: Wow. It sounds like you guys got your work cut out for you trying to get to all this moving in the right direction. What can ophthalmologists and optometrists and other vision care specialists do with the disruption situation?

Jeff Todd: I think that the best thing they can, we encourage them to reach out to us. They can follow our advocacy section of our website, learn more about the problem, become advocates on behalf of the problem. They can also keep us informed if they have patients who are experiencing this and when those patients are comfortable, have them reach out to us. One of the most powerful advocacy tools that we have is patient stories. And so the more that we can tell the stories of those individuals who’ve been affected the better.

Mark Dlugoss: Cool. If a patient or a caregiver wants more information on the thyroid eye disease or any other eye vision problems, especially if it involves a disruption on drugs, where can they go?

Jeff Todd: They can go to our website, PreventBlindness.org, and simply search for thyroid eye disease. Or they can email us at [email protected] or call us at +1 800-331-2020. We also have a number of social media accounts, Facebook, Twitter, LinkedIn, et cetera, where information is often delivered. We’ve really built out our website to provide a lot of information, both on thyroid eye disease, as well as on this particular issue.

Mark Dlugoss: If a concerned citizen wants more information on Tepezza manufacturing disruption and how to contact representatives, what can they do?

Sarah Brown: So I’d encourage them to visit our website at preventblindness.org/advocacy. Through the Vision Health Advocacy Coalition, we have established what’s called a letter desk where patients can reach out directly to the FDA, specifically to the contacts there who are working on this situation. Even as there’s been a change over in the administration, some career staff have moved on or reshuffled positions, we are still able to connect those patients to the folks who have authority over these decisions. We encourage patients to contact the FDA, to let them know that they’re currently experiencing this disruption. It’s not something that’s going to happen in the future it’s currently happening right now. But to also stress the importance of why this particular treatment is so important to meeting the needs that they are facing with their disease.

Mark Dlugoss: Just out of curiosity, I’m not sure if it affects anything, but obviously you’re familiar with Wiley Chambers who handles the ophthalmic side of things in the FDA. Is that the person you guys go to to talk about these things from the FDA, or do you have to go to the director?

Sarah Brown: We have not. I think the folks that we are talking to, or that we’re directing patients to at least is with the FDA Ombudsman’s office and the opposite of consumer complaints.

Jeff Todd: Dr. Chambers has certainly been made aware of this issue and is someone that is on our list to potentially reach out to

Mark Dlugoss: Yeah, he’s been around a long time. He knows the ropes of trying to get through Congress and he’s done an excellent job for ophthalmology and it helps him being an ophthalmologist too. We talked about this whole situation. Are there any other points of discussion you want to address in regard to Tepezza or any other things that we may have overlooked that you want to present while we’re on this topic of talking about this?

Jeff Todd: Well, I think I would just like to end by saying that, you know, absolutely the Coronavirus is the major public health pandemic that we’re currently facing. The challenge is that other healthcare conditions don’t just stop because we have this other condition that we’re dealing with. And while this particular issue about the Tepezza disruption may not impact huge numbers of individuals. For those thousands that it does impact, it really matters. And so those are the folks that we’re looking out for right now.

Mark Dlugoss: When you look at, what they call orphan drugs, for orphan diseases, I mean, I know I can’t remember the name of the company, but that’s all they do. And I just can’t imagine, I mean, sometimes they don’t even make hardly any money off their drugs and you get not only just ophthalmology but look at the whole gambit of medicine. I mean, there’s a lot of disruption going on here and it’s really sad, but at the same time, like you said Jeff, we’ve got to take care of this Coronavirus or we’re not going anywhere. Sarah and Jeff, I want to thank you so very much for joining me today and have talking about the Tepezza disruption and hopefully Horizon and the FDA and everybody could come to some kind of agreement as to how we get this thing up and running again and maybe solve some of these problems for the patients who are really in need. So I thank you for joining me today in discussing it.

Jeff Todd: Thank you for having us.

Sarah Brown: Of course, thank you, Mark.

Mark Dlugoss: You guys have a great day.

Jeff Todd: You too,

Sarah Brown: You too.