lupa-f
Forum Replies Created
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Thanks.
I’m trying to get a permobil M3. I’ve been using regular mobility scooters from Pride up until now (I’ve used like 4 different types), but now that I haven’t walked at all in years the scooter seat has been causing me some problems with more contractures in my knees and some pressure soreness on my butt. I’m always sitting in the same position in the scooter so I can’t really adjust myself much to alleviate those issues which supposedly the wheelchair can help with. So I think I have at least a decent medical reason to switch. My new doctor I saw last year yelled at me for still using a scooter :).
I’ve got good doctors and therapists that I think can help, but I haven’t gotten to the part where medical necessity has even been considered. I’ve been blocked by paperwork and made up rules.
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I guess I should clarify about my Medicare experience. I have a Medicare Advantage plan, so we’re dealing with a regular insurance company, not Medicare directly, that’s where the problems are happening.
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My injections and labs for each dose end up costing about $4000 or so I think on top of the Spinraza cost, but I don’t have to have anything special done since I don’t have any spinal fusions or rods. To make that $4 million over 5 yeas number correct, each dose would need about an extra $100,000 added on for the procedure which even in the worst case I doubt it’s anywhere near that.
A lot of these articles seem to be pretty sloppy when recapping this stuff. I find it’s best to just go off the official company press releases because they have to be accurate since they are public companies.
edit: oops meant to do a reply to DeAnn, but hopefully it’s obvious what I’m responding to.
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There’s an error in the article when it’s comparing costs. It says “Novartis pointed out that chronic injections of Spinraza cost more than $4 million over five years” which we know isn’t true. Even counting the most expensive first year, it adds up to $2.25 million. Either the author misquoted Novartis or Novartis is using different numbers, but that $4 million number should be for over 10 years.
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I was actually surprised, in a good way, at the price. I was expecting it to be much closer to the $4-5 million range that had been first suggested. At $425,000 per year for 5 years, it’s close to Spinraza’s price when you factor in the costs of the procedures every 4 months, and then there’s nothing beyond that which makes it significantly more cost effective over the long run.
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Just the name Zolgensma has been approved, the treatment itself has not been yet.
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This reminds me of a bit of controversy about some dialogue in a recent Game of Thrones episode. One of the female characters said she’s a stronger, better person because of some horrible things that happened to her. It was rightly called out that that is a terrible message to send, that these awful things were necessary to make her as capable as she became.
When I see people talking about how SMA is what makes them a special person today, I sort of feel the same way, that it’s a terrible message, that we needed this terrible thing to happen to us to make us good, strong people. I know I had to make different choices because of SMA, but I can’t say any of them necessarily made me a better person than if I had done things differently if I didn’t have SMA. I know I still would have been a smart, interesting, and capable person if I never had SMA and I’m sure all of you would have been too.
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That would have been the age range when I first started showing symptoms. I always had to plan my future where I knew I’d be losing abilities, but maybe these kids won’t have to experience that.
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One other thing about these results. They’re using the HFMSE to evaluate type 3s. If it’s the same test that they have me do during my 6 month evaluations, it doesn’t really work well for type 3. Even before I started Spinraza I was able to complete about 80% of the tasks, with my stronger arm able to do everything they asked. It doesn’t leave much room for showing improvement when you’re already at the maximum so I wonder if that’s why the change showed for type 3s was lower than type 2.
My left arm was has always been able to lift about 2-3 pounds, but the maximum in the tests I did was 500g or about 1.1 pounds, and even then it wasn’t lifting the weight, but sliding it, which I could do easily. So if my left arm is improving, there’s no way to determine that using this method which of course makes it looks like nothing is changing when the results are reported.
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Here’s a link to the spinraza website with the data: https://www.spinraza.com/en_us/home/results/later-onset.html?cid=eml-adhoc-spzus1698-hp-spz_web_refresh-con_s12_segabc-clin_data_&mdmid=LH1882525
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Type IV shouldn’t have any symptoms other than some general weakness. I have type 3 and that’s the only thing I’ve really ever had wrong with me. I don’t experience most of the other problems many of the others on here with type 2 (and type 1) have had. I had weakness showing up as early as age 5 but walked till I was about 30, so you shouldn’t have too many issues if you’ve not noticed anything until recently. Many of the symptoms are caused by the lack of muscles, not SMA itself.
Genetic testing should be able to tell you clearly if you have SMA. The types (0-IV) of SMA aren’t really any different from each other except for severity.
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There is (or maybe was) a place like that near Chicago. When I was considering moving a few years ago, I talked to them at the Abilities Expo, but I can’t remember what the name of it was and it doesn’t seem like they’re on the exhibitor’s list for this year’s expo. The big thing for me that ruled it out was that for “rent” they’d take all your income for the month, regardless of how much, they would provide food and pay the bills, and then give you a $200 stipend per month for spending money. I make a decent amount of money so that would have never worked for me, it was definitely designed for low incomes, but it was interesting to consider.
If I can find the brochures they gave me I’ll try to update.
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It mostly depends on what type of place I’m going to and where it is. Generally if it’s in the suburbs and some type of chain store or something I don’t bother saying anything. If it’s a hospital or medical place or government building I don’t bother either. If it’s in the city and/or I know the building is pretty old (even if it’s a new name or management), I’ll make sure to say something and even then it hasn’t always worked out even though they assured me it’d be okay. There’s one concert venue in the city I’ve been calling for years and they’re still “working” on the new elevator.
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The big difference for me between Spinraza and Zolgensma is that Zolgensma (at least so far) is a one time injection, no maintenance doses every 4 months. Not having to do an intrathecal injection every 4 months is worth it if everything else was the same. Every time you do one there’s a chance for complications that have nothing to do with the drugs themselves. I’ve had good and not so good injections already and would like to not have to worry about doing them again. Zolgensma might even be able to be injected just into a vein, no intrathecal injection at all, since it can cross the blood-brain barrier.
Also, Zolgensma, if I’m understanding it right, doesn’t seem to depend on how many copies of SMN2 you have whereas Spinraza works better the more you have. In the trials, Spinraza patients will 3 copies did considerably better than those with 2. I have 3 copies so Spinraza works pretty good for me, but if you have 2 or less, Zolgensma will probably work better.
But ultimately it’ll come down to whatever our insurance decides for us.
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Has anybody looked into this? Typing with your eye movement: https://www.digitaltrends.com/cool-tech/optikey-enables-people-with-neurological-disorders-to-type-using-just-their-eyes/
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I’m glad someone is pointing out how ridiculous the pricing of these drugs are. Biogen essentially paid $225 million for Spinraza and has made several $billion in profit off of it already in about 2 years (Biogen did not develop Spinraza which it seems a lot of people believe, they licensed it from another company). And since Biogen got away with it, we get to see Novartis set their price off Spinraza’s price and not off anything else. It’s a vicious cycle.
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How much temperature affects my strength. If my hands get cold for a while I can barely use them and it takes a really long time for them to get back to normal. Like trying to use my keys to open my door is nearly impossible since I can’t grasp the key tightly enough. And “cold” for me means about 60 degrees outside or below. I can mitigate it a bit with gloves, but then wearing gloves restricts my movement anyway so it’s not really much better. If I’m out in really cold, like below freezing, it can start affecting my shoulders, which makes it a bit interesting when I have to drive and I end up steering with T-Rex arms (this only lasts a little bit until the heat in my van warms up enough).
I used to mention this to my doctors and they’d just be “hmmm that’s interesting”, and give no advice or thoughts. Finally I had one doctor tell me it was a symptom of SMA and really common and I’ve since had a few therapists and doctors confirm it. The only solution I’ve ever been given is to wear heavier clothes or winter coats but those make things even worse so it doesn’t really work for me.
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Zolgensma isn’t going to be any different than Spinraza in that regard, Rachel. Neither one works on the nerves directly, both try to create more SMN1 which stops further destruction of the nerves, they just do it in different ways. The primary issue with Zolgensma and adults is how the virus they’re using to provide it works with an adult’s immune system.
I’m not nearly as optimistic as Michael as far as the time frame for it being approved for adults. Spinraza had trials on older children alongside infants much earlier in the process than Zolgensma and didn’t have the same concerns about its delivery method which made a broader approval simpler. Zolgensma is just starting its trials for older children and adolescents and as a one time treatment they’re going to need data showing it remains effective for a long time.
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From https://www.cnbc.com/2019/05/24/fda-approves-novartis-2-million-spinal-muscular-atrophy-gene-therapy.html: “Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5 million,” ICER President Steven Pearson said in a statement.
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“Do you think it will be approved for older patients with types 2 and 3 this year? Next year?”
Because it’s supposed to be a one-time treatment, I think they’re going to need longer term data on older patients to make sure it’s still effective years down the road so I don’t think it’ll be approved any time soon.
“Does its price ($2.125 million for one-time use) worry you, or has this become a “reasonable” price in the rare disease world?”
The price is ridiculous, but these companies can get away with it because insurance companies are going to pay any price so they’re not accused of letting children die. These companies are putting up ransoms on children’s lives and there’s no choice but to pay them.
As I read elsewhere, you don’t normally pay for drugs or treatments like they’re pricing these SMA treatments. The one example I saw was if you need your appendix out. If you don’t, you’ll die, but you don’t get charged for the surgery based of the extra number of years you’ll now live. The surgery has a fixed cost based on the time and effort required to do it. You don’t get a bill saying you owe $2 million because otherwise you’d be dead, but that’s how they’re pricing these SMA and other rare drug treatments.
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“Living with SMA has helped me become strong, compassionate, resilient, and creative.”
You don’t think you would have had those qualities without SMA? I guess it feels like you’re saying SMA is a good thing because without it you wouldn’t have those qualities and I don’t believe that’s true. I’d never tell a child diagnosed with SMA to not take any of the new treatments because “SMA makes you strong, compassionate, resilient, and creative.”
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It wasn’t confusion. The author is wrong saying it was tested on types 2 and 3. It wasn’t. It’s the same trials they’ve been doing on type 1. So the author mentioning types 2 and 3 could confuse other people into thinking they’d expanded the trial.
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I’m definitely more on Ryan’s side of this, I don’t see the point of “celebrating” having a disease most of us would prefer not to have. But the article didn’t bother me too much until the very end where it says, “What’s so exhilarating about a normal life? Being rare is what makes us interesting.” It feels like it’s saying that SMA and having to deal with it is what makes us interesting. That would be pretty sad, but maybe I’m not interpreting that statement right.
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This is a little bit of a pet peeve of mine regarding Spinraza.
“First I think development costs for medications for rare diseases are very expensive.”
This is only sort of true, and definitely not true in the case of Spinraza. Spinraza (nusinersen) was developed in a by Ionis and Cold Spring Harbor, it was then bought by Biogen for a small amount upfront and about 12% of the revenues per quarter from Spinraza. Biogen is making about an 80% profit margin at the very least off of it. Their CEO even admitted a big reason the cost was so high was because people had no other choice. Biogen did spend the money for the clinical trials, but even that is misleading as the government gives companies up to 50% of the cost of trials for orphan (rare disease) drugs back to the companies as a tax credit (the company’s tax bill is reduced by that amount, it’s a straight cash subsidy). Companies are purposely now trying to limit that amount of people a drug covers by making very many small variations of the same treatment so that each version counts as an orphan drug so they receive those tax credits.
There have been articles on this site from an independent observer stating that Spinraza costs more than 5 to 8 times what it should: https://icer-review.org/announcements/icer-issues-final-report-on-sma/