Empowered Patient Podcast

Whitney Stewart, the Director of Clinical Project Management at Curebase, and Megan Coder, the Chief Policy Officer of the Digital Therapeutics Alliance, have worked together on a white paper Setting the Stage for a Fit-For-Purpose DTx Evidentiary Standard. The goal is to outline foundational principles that, in some cases, are unique to the digital therapeutics category. Their focus is on baseline expectations related to procedures and the type, quality, and timing of clinical trials necessary to evaluate and widely implement DTx therapies. Megan explains, "Given some of their development pathways and processes, given how they're regulated as a medical device and not a drug, we've really started talking with Curebase and some of our other members. We are looking at, and we recognize that, these digital therapeutic products have great value to patient care. They can be used alongside or sometimes even in place of some of these traditional therapies. But what became evident to us pretty quickly is that these therapies really don't sit in line with all of the evidentiary standards that exist for the current existing treatments and therapies." "We wanted to talk about this idea of a fit-for-purpose standard that can pull in some of the aspects from traditional therapies into this evaluation framework. This is so a patient, clinician, payer, and policymaker can understand, "Oh, this is what this therapy does, this is how I know it's going to be successful, and this is how I know what the impact is going to be for my patient population." Whitney elaborates, "The future implications include a better understanding of the clinical evidence dossier, which just means more effective and fair evaluations of the technologies. So, do they need to conduct the same trials and generate the same evidence as pharmaceuticals? No, I think we all are aligned with that. Do they need to go through the same processes as high-risk medical devices? No, they probably don't. Most of them are fairly low-risk. So we've been talking about it just to get everybody on the same page and with the same kind of evaluation criteria so that everyone knows exactly what sufficient looks like." #DigitalTherapeuticsAlliance #Curebase #DTx #DigitalTherapeutics #PrescriptionDigitalTherapeutics #DTxTrials #ClinicalResearch #ClinicalTrials DTxAlliance.org curebase.com Download the transcript here

Karen Smith is the Founder of Catie's Cause, a nonprofit charity dedicated to building awareness about the safe use of over-the-counter pain medications. After the death of her daughter Catie from liver failure due to overuse of Tylenol, Karen founded this organization to shine a light on the unintended and intentional side effects of easy access to these medicines. Karen explains, "The research shows that there are four main groups of individuals who are impacted by acetaminophen poisoning. Young adults definitely are a large group, oftentimes because they're on their own and they don't realize the dangers of medication. Some of them do begin to abuse it and they take it way too regularly. Sadly, about 50% of the deaths are from suicide, so obviously intended, but 50% of them are unintended. So young adults are a big group." "Another group is young children because parents tend to freak out when their little ones get a headache, and they overmedicate. The third group impacted are the elderly because they have difficulty reading the warning labels or are taking a lot of other medications, and their liver may already be compromised by that." "And then the last group are those with limited access to healthcare. That's primarily because they don't have someone to warn them about what could happen if they overuse that medication. Instead of going to a doctor, they tend to go to the pharmacy and pick up whatever's on the shelf." #CatiesCause #AcetaminophenPoisoning #AcetaminophenDangers #KnowYourMedications #LiverSafety #DrugSafety #Tylenol #SouthRampartPharma #DoIt4Catie #PaintTherapy catiescause.org Download the transcript here

Dr. Jing Wang is the Dean and Professor at Florida State University College of Nursing and is focused on bringing a high-tech, high-touch approach to patient care in the education of nurses. The idea is to leverage technology and digital health tools to support nurses so that they can spend more quality time with patients. The newest generation of students are digital natives, and one goal is to help them use that knowledge to smooth the transition for the less technology savvy. Jing explains, "I think it's all about how technology is introduced into the learning in our current nursing education and health profession education. That is critically important. That's what we are doing here at Florida State University College of Nursing. We would like to do it in the right way, to introduce technology as a tool to enhance care and facilitate care versus this is a fancy technology, we must use it. I think that's core, and that's the attitude that we are hoping our graduating students will have." "We have launched a new Institute on Digital Health and Innovation. It's very exciting that we are launching our first design sprint. We're bringing students all together for them to work 24 non-stop to focus on solutions with people who build homes, people who design homes, people who take care of seniors, and people who focus on wellness. This includes doctors, nurses, and social workers. How can we design this home that is a future aging-in-place home that would consider all of the aspects for our seniors or adults who would like age-in-place?" #FSUResearch #FSUNursing #DigitalHealth #AginginPlace #DigitalPrecisionHealth #Nurses Nursing.FSU.edu Download the transcript here

Dr. Deb Ondrasik is a pediatrician based in Massachusetts, and the topic today is the CACNA1A Foundation and its work on raising awareness about developmental disabilities. Deb's daughter was diagnosed in 2015 as one of eight children worldwide with a CACNA1A gene mutation causing epileptic encephalopathy. This is a story about the need for whole genome sequencing to determine the root cause of childhood developmental delays and the value of this information for developing a treatment plan. Deb explains, "CACNA1A is a genetic mutation that's a group of diseases that can present in a number of different ways. It's mutations along a gene that affects the calcium channels and the neurons, and it's on chromosome 19. And so, there are numerous hundreds of different point mutations that could present a little differently. But kiddos can have anything from developmental delays to autism to epileptic encephalopathy, which my daughter has. Also, there are other conditions like hemiplegic migraines. And so, it is a variety of neurodevelopmental disorders that can be presenting." "I work as a general pediatrician. I see a lot of kids that have delays and significant delays or autism or epilepsy. And oftentimes, the families are told that that's the diagnosis. Your child has epilepsy, and we're going to give them some meds and see if we can control it. Or, if your child has autism, we're going to get them into therapy, which is partly correct. Medication is super helpful, and therapy is super helpful. But I think what's really important is to try to find the root cause of the symptoms, which are epilepsy, autism delays. And oftentimes, for some reason, pediatricians and even neurologists don't always go ahead and do the genetic testing." #CACNA1AFoundation #CureCACNA1A #RareDiseases #RareDiseaseAwareness #CACNA1AAwareness #PatientAdvocacy #CACNA1AResearch #Epilepsy #IntellectualDisabilities #CerebellarAtrophy #CongenitalAtaxia #CaregiverSupport #WholeGenomeSequencing CACNA1A.org Download the transcript here

Heather Ridenoure, Center of Excellence Segment Leader, and Maeve Ruggieri, the Director of Product at Contigo Health, shine a light on the challenge of employee substance use disorder and the resources available for care. This chronic disease can be caused by adverse childhood events, genetic predisposition, stress, and environmental factors. Contigo addresses any substance that can be abused through their Centers for Excellence program with virtual care and in-person care options where participation is voluntary and can be accessed 24/7. Heather explains, "As far as how frequently this occurs, the newest statistics from the Centers for Disease Control said one in seven people have a substance use disorder. That's new data, and that's been increasing by 30% since the pandemic, which is pretty significant. So, this is one of the reasons we struck out on a journey to try to address some of these issues." Maeve elaborates, "As Heather mentioned, substance use disorder being a chronic disease, is something that causes clinically significant impairment. So that may or may not be visible to an employer in the workplace. Their employees may seem like they are high performers or be high performers and not be showing the stereotypical signs of substance use disorder, things that we may see from TV or movies, like erratic behavior, things even down to missing deadlines." "But what's so important is having the member be ready to participate themselves. This program is rooted in shared decision-making between the member and the providers, and our team at Contigo, who's doing a lot of care and case management and support. And if the member is not ready to participate, to explore recovery and manage their substance use disorder, then it's not time yet." #ContigoHealth #SubstanceUseDisorder #HealthPlans #HealthBenefits ContigoHealth.com Download the transcript here

Kyle Filkins and Kris Napper were both born with SMA, spinal muscular atrophy. Meeting as kids at The Muscular Dystrophy Association summer camp, they have developed a friendship and a documentary. After beginning the use of the drug Evrysdi, their rare genetic disease stopped progressing, and the oral form of this medication allowed them to avoid spinal injections. Now in their thirties, Kris and Kyle are advocating for the community of those with SMA, working on their documentary, and looking forward to more adventures. Kris explains, "Just shortly before Evrysdi was released and made available, there was another drug called Spinraza that was the first treatment ever for our disability. It was a spinal injection, which poses some complications as far as it's a procedure with some risk. It can be painful, and there's the risk of hitting nerves and causing damage. It's not the most fun thing to go through, but it was beneficial. So I was doing that." "So for young children, just being diagnosed can be life-changing because you can stop that progression before it really hits. For us, we've already had significant progression. Still, hopefully, this will stop it where it is, and we'll be able to continue our lives for the foreseeable future, a lot better and longer than originally anticipated." Kyle elaborates, "Before I was on any type of therapy, I would go to bed one day able to do something, and I'd wake up the day after not being able to do that. And it would never come back. And it's pretty great to be able to go to bed and sleep securely knowing that what I did today I can, for the most part, probably do tomorrow. It's definitely life-changing. That's for sure." "There's been a lot of people interested in this story. Kind of piggybacking here on what Napper said, at the outset, it was really kind of just a budget film and like, "Oh, maybe they'll do cool activities and make jokes and stuff." But then with the advent of this therapy and specifically Evrysdi, that's really changed the outlook of the scope of the film, and for the better, as Napper said." @KKtheFilm #SMA #SpinalMuscularAtrophy #MuscularDystrophy #RareDisease #Evrysdi KrisandKyletheFilm.com SnappyClothing.com Evrysdi.com Download the transcript here

Marlena Del Hierro VP of partnerships and commercial strategy at Seniorly, which is a resource for families looking for senior living options and seniors planning ahead. The Best of Senior Living Awards determined by Seniorly highlights the best 150 communities around the country based on feedback from families, senior living advisors, licensing reviews, and media searches that might reveal something problematic or terrific about a facility. Marlena explains, "We have an algorithm that then shares the top five to seven options for your loved one. So that's a unique piece, because of the data and the technology that we're constantly gathering from a variety of sources to help these families make the best decision for their loved ones." "The reason why we're doing these awards is that we should be pushing the envelope. The Baby Boomers are fundamentally challenging these senior living communities. And the awards are just a sense of preparing us for more of a modern era in senior living." @Seniorly #SeniorLiving #BabyBoomers #IndependentLiving #AssistedLiving #FinancialPlanning Seniorly.com Download the transcript here

Dr. Steve Marcus, CEO of Cantex Pharmaceuticals, is developing drugs based on Azeliragon that might slow the metastasis of cancer and extend the survival rate for patients with brain and other cancers. This drug has been shown to suppress the RAGE receptor, which is expressed on the surface of cancer cells and some immune cells. When RAGE is over-activated, as it is in several cancers and inflammatory diseases, it can cause pathology. Steve elaborates, "Azeliragon inhibits something abbreviated as RAGE, the receptor for advanced glycation end products. Originally, it was thought that RAGE is very important for the progression of Alzheimer's disease. And because of that, a company called vTv Therapeutics initiated large-scale clinical trials of Azeliragon for Alzheimer's disease. Unfortunately, those trials failed to show effectiveness of the drug in Alzheimer's disease, but did demonstrate very robust safety. The drug is given once a day and was given to patients for periods up to 18 months, and it was very well tolerated." "At the same time, over the last several years, there has been extensive literature indicating that this receptor, RAGE, plays a role in the progression of cancer, both brain cancers, as well as cancers outside of the central nervous system. And with that information, I approached vTv Therapeutics after their clinical trial failed to show efficacy, and I proposed licensing the product and taking it into cancer indications." #CantexPharma #GBM #Glioblastoma #BrainMetastasis #RAGEReceptor #Cancer Cantex.com Download the transcript here

Dr. Spyros Papapetropoulos is the President and CEO of Bionomics, a biotech company focused on central nervous system disorders, particularly social anxiety, and post-traumatic stress disorders. Social anxiety is a disease primarily of adolescents and young adults, and the social isolation during COVID contributed to a rise in the number of people experiencing this condition. The Bionomics drug in development is BNC210, a therapeutic that can be taken on schedule an hour before conducting an activity likely to induce anxiety. Spyros elaborates, "Social anxiety disorder is actually known as social phobia, and it's very different, because everyday interactions cause significant anxieties. And they're very situational, so you become very self-conscious. You feel embarrassed and have a fear of being scrutinized or judged negatively by others. It is considered a very, very important chronic mental health condition that, if left untreated, leads to comorbid depression, even suicidality, and addiction down the road." "BNC210 acts on nicotinic receptors. These are receptors that are expressed or found in areas of the brain. The alpha-7 nicotinic receptors are found primarily in areas that control emotions, so in areas that are considered brain anxiety generators. I don't know how much your audience has heard about the amygdala, but the amygdala is considered one of the most important areas, also responsible for fight or flight, for instance. And it's an area of the brain that has received a lot of attention. So BNC210 binds to the alpha-7 receptors found in the amygdala and reduces the activity or the activation of the amygdala, which is abnormally high in anxiety-related disorders." #Bionomics #SocialAnxietyDisorder #SocialPhobia #CNSDisorders #PTSD #COVID bionomics.com Download the transcript here

Matt D'Onofrio, Co-Founder, President, and Chief Operating Officer at Evoke Pharma, is developing a treatment to help patients with diabetic gastroparesis. This little-known disease is thought to be due to damage to the vagus nerve, interrupting the communication between the brain and gut, stopping the stomach from functioning normally. Current treatments are a pill required to move through the stomach. Evoke has developed a nasal spray to treat the condition that bypasses the stomach and provides predictable relief. Matt explains, "We developed a product called GIMOTI, or nasal metoclopramide. The interest behind doing so is to completely bypass the stomach to get a dose on board for patients. The unpredictable nature of the stomach makes dosing with pills very unpredictable. We know that a number of patients who take oral treatments don't find efficacy. We believe that's, in part, because of the stomach's problems itself." "By developing the nasal format, or GIMOTI, we are able to avoid that unpredictable issue and get the product on board directly by spraying it into the nose and getting absorption straight through the thin membrane in your nostrils, getting the product into the bloodstream from there. We found that this is very helpful for patients who do have issues with any absorption problems. We also found it helpful for patients who are nauseated and vomiting because you can't vomit up a nasal spray. There have been a number of different things we found as the product has come on the market that's been helpful for it." #EVOK #Evoke #FightGastroparesis #DGPAwareness #DiabeticGastroparesis evokepharma.com Download the transcript here