Introduction
Sam:
On this week's episode of the pRETTy happy. podcast we sit down with the co-founder of Rett University, Susan Norwell, where we learn about some great things coming from RettU this year.
Sarah:
But first, we talk about the great news regarding trofinetide, or as we now know it - Daybue.
Sam:
All that and much more on today's episode. Today is Monday, April 3rd. Let's start the show!
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Top Three
Sam:
Welcome to the pRETTy happy. podcast. My name is Sam.
Sarah:
And my name is Sarah, and we're the parents of Zoey - a child with Rett syndrome.
Sam:
We are back after taking a week off, but we are so excited to share so much new stuff with you today. I don't think we should take any more time dilly-dallying --
Sarah:
Nope, we should not.
Sam:
-- and just chatting about because there is so much that we need to discuss today. Sarah has our Top Three. So Sarah, tell us about our Top Three.
Sarah:
Our Top Three is from the webinar that Acadia Pharmaceuticals put on for caregivers of individuals with Rett syndrome. They decided that they needed to do a community webinar where individuals could submit questions and they would sit down and answer them they would go through you know what trofinetide is and what Acadia had to offer alongside of trofinetide.
#1 Results from Clinical Study
Sarah:
So number one -- The first thing then they went over in this webinar was the results from the trials. So they kind of went through and explained what those results looked like and basically -- to break it down for you guys -- there were two different evaluations: one evaluation was given to parents or caregivers, the other evaluation was given to the doctors themselves and they were two completely different evaluations, but still similar. Now the parents went through their evaluation and they wrote down I believe it was -- they wrote down zero to three; zero being not true and three being true. So they were given 45 -- or they were given statements about 45 symptoms of Rett syndrome and they would mark whether that symptom was true for their individual or not true or, you know, neutral. And those included their mood, breathing, eye gaze, nighttime behaviors, repetitive behaviors, vocalizations, hand movements, and facial expressions, and many, many more because there were 45 symptoms that they were looking at to see if trofinetide affected them or not. Overall, trofinetide -- from the beginning to the end -- the difference was on average a -4.9. So essentially from being true to not true -- so from having symptoms to not having symptoms -- they, on average, went down by 5 points almost. Which is a big improvement, I feel. Especially if there's only 0, 1, 2, 3.
Sam:
Yeah, that's definitely a large improvement compared to what it was. And even though some of it -- because I've heard some people say that it was just incremental improvements and, yes, I think you may be able to say that because this is such a new drug that in some instances those incremental improvements could have been other factors -- the fact that these improvements happened across the board with those who were taking the actual drug versus those taking the placebo, that means a lot.
Sarah:
Yeah, they did compare it. They had two groups: one taking the medication, one not taking the medication. And then this was a 12-week period that's it for this final trial was a 12-week period and then in afterward individuals who wanted to stay on could stay on for 40 weeks and then they continued to monitor, but these evaluations were given several times during that 12-week period. So potentially it was incremental just for that 12 weeks, but if you were on it for longer, you potentially could see that those incremental things build into bigger things.
Sam:
Yeah and as I've been sifting through the many posts and comments on various social media sites about the the approval for trofinetide, also known as Daybue, there's been -- it's interesting to see so much hesitancy especially considering the fact that so many people are so eager about a cure. We don't have a cure right now; what we have is a therapeutic that can assist. And it's funny to see some people specifically point out that those incremental improvements, for them is not enough to justify taking the drug. Now, there may be other factors involved yeah and that's fine I can't judge somebody's situation -- I can't judge it effectively based off of a single social media post.
Sarah:
Yeah.
Sam:
But to simply say, "Well the results were only slightly positive." Umm... oh okay? But that's better than nothing.
Sarah:
Yeah. I mean --
Sam:
And we literally have nothing else, there is nothing else! We have plenty of drugs to be able to take care of a headache. We're headed into allergy season here in the US and spring is kicking my butt and I have plenty of options of things to take. And you and I actually had this conversation yesterday about how there are certain medications that for me just do not work. They just don't work and that's fine! And we're gonna see that with trofinetide, there are -- it's not going to work for some people and for others it is going to be they are going to have adverse reactions, but that's that's pharmaceuticals.
Sarah:
In general, yeah.
Sam:
Yeah, yeah. I shared with Sarah that I have taken and opioid once and I can't remember which surgery it was after I've had multiple surgeries throughout my life, but I took it once I was a teenager and I was so sick to my stomach. I cannot take any sort of medication for pain relief if it's an opioid-based. I can do ibuprofen, Tylenol does little to nothing, morphine is my favorite. There's -- but that's not the case for everybody
Sarah:
No
Sam:
And so, you know, an incremental Improvement across the board could mean drastic improvements for others. It could be mean negative symptoms for others. You just don't know, and the great thing is about this is if you take it and it doesn't work and or maybe it causes problems, you go off of it.
Sarah:
Yep, yep. It's only effective as long as you continue to take it.
Sam:
Exactly, exactly. So it is just like a Claritin or an ibuprofen or whatever it is: so long as the medication is in their system it will be impactful. This is not a permanent change
Sarah:
No.
Sam:
The drug itself is not intended to create a permanent change.
Sarah:
No, it is not.
#2 Acadia Connect
Sarah:
Number two - they presented a resource, we're going to call it a resource, called Acadia Connect. So this resource gives families access to nurse care coordinators and family access managers. Do you know what that means?
Sam:
I'm gonna guess that the intention is to help families and caregivers and guardians and so on and so forth figure out how to navigate getting access to trofinetide.
Sarah:
Yes.
Sam:
Yeah because, I mean, I don't think I've ever been in a position where I personally or anybody that I know personally has been taking a drug so early in its lifetime so yeah.
Sarah:
So essentially these connections the Acadia Connect, the nurse care coordinators, and the family access managers - their job is to help everyone get access to the medication. So they are going to help with insurance and cost, they're going to help with filling your prescription, and they are going to provide support and education. And they will actually -- so once you talk to your doctor and say you want to get on the medication. if you go to the Daybue.com, I believe, website they have a -- they have information where you can sign up to get a nurse care coordinator or a family access manager and they will help you with your insurance. So they'll actually take a look at your insurance for you, they'll find out if you need pre-approvals, they'll help you get all of that submitted, they're going to help you get the medication. If you don't have insurance, they have support plans and whatnot to be able to help with that. They want you to be able to get the medication if that is what you're wanting, which I thought was awesome because most medications, they don't have somebody that's like, "Oh yeah, you need a pre-approval for this. We're going to help you get that so that you can get your medication." Most people are just like, "Here's your prescription; figure it out."
Sam:
Yeah, yeah. Well and it being so new and, at least here in the United States, it is going to be a heavily controlled substance it makes sense. Do you know if they have made any sort of recommendations about like using a compound pharmacy versus just kind of a stand-- compounding, excuse me, pharmacy versus a standard pharmacy?
Sarah:
So that was actually going to be my number three.
#3 Specialty Pharmacies
Sarah:
So we'll jump into number three.
Sam:
Number three!
Sarah:
Number three was that they are going to be -- this medication is going to be made at specialty pharmacies. Now what that means is that your medication -- the medication will be shipped to you. So you're not going to go to the pharmacy and say you need more; you will just contact -- you'll work with Acadia Connect and they will help you make sure that you're getting your deliveries on time -- you're getting your medication. But it is from a specialty pharmacy so you're not going to just go to your local pharmacy and be like, "Hey, I need Daybue."
Sam:
Gotcha. That does make sense and especially considering the number of people that are going to be eligible to be able to use it...
Sarah:
Right, compared to how many people need medication in general.
Sam:
Yeah exactly. This does make more sense to have it shipped versus having it stocked. Obviously, there are some downsides to that, but let's be honest folks, there are downsides to having stuff within brick-and-mortar stores.
Sarah:
It's true, yep.
Sam:
So I think this is fine -- a fine option, yeah.
Sarah:
Yep. So they, you know, I thought they did a great job answering questions and, you know, a lot of the questions that were coming in were questions that they couldn't answer. They were specific to the Rett individual and they were questions that needed to be discussed with that individual's doctor because they obviously, on this panel, they're not going to give out medical advice; they can give you the facts about Daybue, not you know tell you what exactly you need to do or whatnot.
Sam:
Yeah exactly. I imagine there were conversations about, you know, "My child has a G-tube and you know struggling with weight and there's a potential with Daybue for a side effect of diarrhea. How does that impact?" and all that stuff. That's not their job. Their job is to deliver a safe and effective drug, which they have done.
Sarah:
They have shared the information with healthcare providers about the side effects: the pros, the cons, everything about it. And then it's up to you to have a discussion with either primary care, your neurologist, your Rett care personnel, whoever it is, and decide and -- preferably -- alongside your individual who has Rett, is this something you want to give a try on.
Sam:
And they did suggest when they were talking about side effects they did suggest things that you could talk to your doctor about like getting on an anti-diuretic or getting off laxatives before starting because it could cause diarrhea. But in the end, they kept saying you know you just need to talk to your loved one's doctor because they are going to be able to give you the best advice not the panel that was, you know, in this webinar. But I think they did a great job overall and they answered a lot of questions, and, you know, at this point it's just a waiting game to see when or what happens once people get on the medication so...
Sam:
Yeah. It's going to take time, like we were mentioning before. You know, it appears like there's a real possibility that if there's positive effects of trofinetide within a 12-week trial period that if you continue to stay on it that it could do look even better and better and better and better over time. For example, I -- if I go off -- I use usually use Zyrtec as my allergy medication, if I go off of it for like two or three days if my allergies are just horrible and it takes a couple more days to build back up, to have that medication in my system and to help fight off the horrible side effects of allergies. So I mean, it's definitely possible that that's the case, but as a reminder, this is not a cure. This is not something that's going to stay in the system forever. And so if it doesn't work, okay, it doesn't work. You know, and you go back to what you were doing before and you handle it that way until we have something else. So the fact that we have something is better than nothing at all.
Sarah:
It's awesome.
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Interview with Rett University's Susan Norwell
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