The nonprofit association, established in 1985, is active in promoting research into ALS, providing assistance to patients through its network of 39 state chapters, and in coordinating care though its certified ALS clinical care centers, now numbering 50.
An in-depth article on this telephone interview, exploring Balas’ views on Radicava, the “hopeful” approach taken by the FDA in approving MT Pharma’s therapy without requiring a U.S.-based trial, and other issues can be found here.
Q: Radicava was approved by the FDA in early May, the first new treatment for ALS in 22 years. Can you please talk about the importance of this approval, both in terms of this systemic treatment itself and in terms of advancing research and work into new therapies?
To the best of your knowledge, how has Radicava been received by the ALS community?
A: As far as the [systemic] treatment of ALS — I think a lot of that is yet to be determined. It shows potential effect for functional ability in ALS and preserving functional ability by 33 percent. That can have significant impact in individuals from a numbers perspective. If it is effective in those individuals, it could certainly impact somebody’s quality of life significantly.
It doesn’t necessarily stop the disease or increase overall survival. But I think it might be very useful for some people, in order to preserve function, and function like being able to speak, or swallow, or move — breathe — are very important aspects of anybody’s life. So, it’s a nice first step.
The FDA showed that it is anxious to move forward with therapies that may have impact for an unbelievably unmet need in this disease. This is only the second [approved ALS] therapy and neither are curative by any means. So I think, to me and to many others, it signals an appetite from the FDA to move therapies forward if they are positive. I think that’s going to drive some more research and development. I certainly hope so.
Q: Is the ALS Association aware of patients already using edaravone, perhaps having gained access to Radicut, as the treatment is known in Japan and Korea, where it was approved in 2015?
If so, is the ALS Association in contact with these patients, and monitoring their progress under the therapy? Are there particular benefits or problems that you have seen?
A: I think that people are excited. I don’t want to say they’re scared, but they’re anxious. There’s a lot of anxious folks out there, wanting to know if they’re going to be able to get it, will it work for them — all of these very large questions — will the insurance companies cover it?
It’s a very new thing for this group to have a treatment. So I think there are a lot of questions and maybe a little bit of anxiety, but a great amount of excitement. I think, if anything, this approval has brought true hope to this community.
We know that there are other things that are very close in the pipeline, Phase 3 studies that are supposed to ring out in the very near future, and the sense of real potential and real hope is probably the overwhelming feeling.
I have heard that to be true [patients in U.S. using edaravone]. I have not had any personal communication with any individuals who have told me they have received it in some alternative way in the United States.
I know there are some organizations promoting import through a third-party source, which honestly we are very concerned about, always concerned about safety of any kind of drug importation allowed. Our system has patient safety in mind as a primary focus. But I have not had any conversation, one on one, with any patient or individual who has told me they received it outside, on a black market or something.
Q: Trial evidence seemed to suggest that this therapy provided most benefit to those in earlier disease stages; for instance, patients in the trial had ALS onset of less than 2 years at the study’s start.
Still, the FDA approval notice states the treatment is for all ALS patients, making no distinction between disease stages. Are you concerned about the emphasis the trial placed on early stage patients, perhaps in regard to insurance coverage?
For instance, one policy we saw online (United Healthcare) seemed to imply targeting certain ALS groups, specifying that for initial treatment a patient must have “a % forced vital capacity (%FVC) ≥ 80% at the start of treatment” and for continued coverage (subsequent years) “NOT [be] dependent on invasive ventilation or tracheostomy.” [Emphasis part of UH policy statement, under Coverage Rationale]
Have you heard of any restrictions being placed on coverage for Radicava for this or other reasons?
A: I think it’s important to consider that this is about clinical trial design — and how the trial was designed. [It] focused on the FVC, forced vital capacity … and if you talked to MT Pharma they told you the methodology, the reasoning behind that trial design, and also some of their own limitations … in which they were doing that trial in Japan.
So, I don’t have concerns around that [pivotal trial’s focus on early stage patients]; it’s a small cohort, it certainly showed statistical significance. The FDA decided to move with a wider label, and actually that’s right in line with what the patient community had asked the FDA to do. We had submitted a patient guidance document, which they are reviewing right now, and within that it specifically asks for those parameters in which safety is less of a concern. Here we have a drug that’s been on the market for quite some time.
I am concerned about the coverage decision by United Healthcare. I think it sets very bad precedent [in discounting FDA label] not only for this drug, but for any future therapy that might come out.
A board member of mine said: Just because you don’t have a 34-year-old in your trial design, does that mean then that a 34-year-old should not get that drug, if you had people who were over the age of 40? Trials are designed for a variety of reasons, and for United Healthcare to make a decision based only on the clinical trial design, as opposed to what the FDA has asked — in a population that has pretty much no therapies — is gravely concerning to me.
I think it sets not only a bad precedent for this, but we have drug manufacturers who are looking at investing in R&D. And their [UHC’s] decision is discouraging for future investment. This a terrible disease, with not a large population. So to discount the FDA decision in a disease that has NOTHING for patients to look at toward a therapy, I frankly think it’s slightly immoral.
We are not aware of any yet (other companies placing similar restrictions on insurance coverage). We are requesting meetings with insurance companies as well as CMS [Centers for Medicare and Medicaid Services] to have this conversation.
It’s important for me to re-emphasize that this decision is one that should be between the patient and the physician. It’s not my position on whether somebody should or should not take this drug, I’m not a doctor. This is a decision between the patient and the physician, not the insurance company
I think the other concern that we noticed was this is for six months, and indicated that then there would have to be an application for any other future coverage. I don’t know if UHC then expects that if someone’s FVC — forced vital capacity — drops below that 80%, then they lose the ability for coverage? I don’t know what their plan is there, but I think it puts people at risk.
Q: Radicava’s list price is about $1,100 per infusion, which is given in 28-day cycles, for an estimated $146,000 each year. How does this US price compare with what you know of the price paid by patients or their families who purchased edaravone directly from Japan? Do you consider the price excessive, relative to list price for other drugs newly entering the market?
A: I know that the price in Japan is lower, [but] I actually don’t know exactly what that price is. I also know that list prices here in United States are very different from the negotiated prices that [drug] companies make between the insurance companies and the pharmacies. So, while on the face value the drug price is high, or it might be high, I’m not sure that’s really the whole story.
My concern is always access — if patients have access to the drug and the drug is being covered, then they’re more happy. If the drug is not accessible because of pricing, then that’s another conversation. I don’t know that to be true yet, so we [the ALS Association] haven’t decided where we come down on the price.
Q: To the best of your knowledge, are programs like MT Pharma’s patient assistance program sufficient in helping patients wanting to use this drug but perhaps without adequate insurance coverage? How useful, in your opinion, are these patient assistance programs, and are there ways they might be improved?
It’s hard to say because the program [patient assistance program by MT Pharma] hasn’t really rolled out. I know they’ve signed people up, it sounds like they have, at least to my knowledge, tried to create a robust information source and group to navigate patients to patient coverage. I think that’s to be determined if that ends up being valuable or completely fruitful.
In my experience — I come from the oncology space and, previous to that, the autoimmune space — the patient programs usually are of great assistance to individuals. I don’t know if they’re — they’re not the entire gap filler for many patients, but for quite a number that I have experienced they’ve been very helpful and usually can get therapy into a patient’s hands in a pretty modest way. I think they’re of great value; at least that’s been my experience. They’re only a piece of the pie, but they’re of great value.
Q: Other parts of pie need to be there?
There are a lot of government programs that can often step in. For our population, we estimate that about 80% of our patients are on a federal program, whether that’s Medicare or VA benefits. So there are those pieces that can come in.
Sometimes, there are organizations that do supplemental funding, if it’s a funding issue; sometimes it’s just a transportation issue, of people actually trying to get to a facility or to an infusion center. There can be community organizations that help to fulfill some of those needs. It depends on what … that gap really is. But it’s often about local access issues as well as federal and state benefits. …
Q: The ALS Association has been working to support patients interested in access to Radicava, such as the May 9 webinar that followed its approval and addressed FDA processes and initial insurance concerns. Are future webinars and other programs being planned? Are there particular topics you are looking to address?
A: We’ll be doing some webinars for the healthcare professional community. This is a space that hasn’t had new therapy for a long time, so there will be a lot of questions, a long learning curve in that regard.
We will more than likely have not only webinars, but then an international conference, and other opportunities, at our clinical care conferences we will create opportunities for Q&A sessions and discussions with physicians and other experts, and some more hotlines as time goes on.
My hope is this is the first of many treatments, and then we’re talking to our constituents about a variety of educational materials on a variety of treatments. We will continue to roll out some things.
Q: Are there specific treatments or approaches now under investigation that the ALS Association is watching with particular interest?
A: I think the one that’s on everybody’s eagle eye is out of Cytokinetics. They’re looking at their data hopefully to read out by the end of the year. It hopefully could have some significant impacts.
What we’re looking at now is more of a cocktail approach, where some are around functions, others around actual disease progression, others around breathing mechanisms.
But Cytokinetics is worth looking up, and tirasemtiv is the name of the drug. They have a few other things in the pipeline, too, but that’s the one that’s closest.
Q: Is there anything you would like to add or discuss? About the FDA’s approval process for Radicava, Radicava as a systemic treatment, and its availability through specialty pharmacies, nature of infusion treatment?
A: I don’t know who approached whom [MT Pharma or FDA], but I certainly think it [approval] certainly is because of the need.
I think this is the story. It’s fantastic that we have a new drug, but I think the story is that the FDA did something they don’t do, and decided to move this drug forward because of such a grave, unmet need in a really terrible disease
I think that it shows their willingness to start thinking in different directions, and really trying to expedite drugs when they think they have something that might have some efficacy and value to the patient. They have less concerns around safety implications, but can still carry out some other trials. There are some trials that have been carried out and will be carried out, are being designed in sort of a Phase 4 rollout. To me, that’s one of the really big highlights here.
[I don’t really see Radicava as systemic treatment.] I see it as a treatment that focuses on preserving functional ability. It doesn’t necessarily treat the underlying cause of the disease. … It’s more about preserving function ability than [targeting] disease progression.
[Specialty pharmacies and concerns]. That’s my understanding [Radicava available through specialty pharmacies]. I don’t now if there are plans for it to be pushed out broadly, but that’s my understanding.
It’s always access, there are always concerns around access, especially for those who live in rural areas or have transportation issues. With our population, by nature of the disease, transportation becomes a major issue. Having to go somewhere becomes a major issue. So, barriers to access is always the concern.
[Infusion treatment] It is no minor feat. It’s a daily infusion — for several days in a row, several days off — it’s intensive. That’s why I really think it’s really a decision between a patient and a physician. Is this appropriate? Does this work for them?