IDDI - Regulatory Statistics & Clinical Data Science Experts’ Post

Synergy Healthcare is thrilled to share groundbreaking developments in the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The Food and Drug Administration (FDA) is on the verge of approving the first-ever medicine specifically designed to combat this serious liver disease. Developed by Madrigal Pharmaceuticals, the pill, known as resmetirom, targets a protein in the liver, effectively reducing liver fat, inflammation, and scarring associated with MASH. If approved, resmetirom will mark a significant milestone in healthcare, offering hope to patients with advanced stages of MASH where current treatments have shown limited effectiveness. At Synergy Healthcare, we pride ourselves on being at the forefront of clinical research, especially in metabolic and neurological diseases. The potential approval of resmetirom represents a significant step forward in addressing the unmet medical needs of patients suffering from MASH. The addressable market for drugs to treat advanced MASH is substantial, with an estimated 315,000 individuals in need of effective treatment. Resmetirom's innovative approach and promising clinical trial results position it as a potential game-changer in the field of liver disease treatment. We commend the dedication and perseverance of researchers and scientists, including those at Madrigal Pharmaceuticals, for their tireless efforts in bringing new therapeutic options to patients in need. Stay tuned for more updates as we eagerly await the FDA's decision on resmetirom, and let's continue our collective efforts to advance healthcare and improve patient outcomes! 💊💪 #SynergyHealthcare #ClinicalResearch #MASHTreatment #Resmetirom #HealthcareInnovation #LiverDiseaseTreatment #FDAApproval #MedicalBreakthroughs 🔗 https://lnkd.in/ekjzn4uy

A New Hope in Alzheimer’s Treatment: Kisunla Approved🔬🧪 Something near and dear to me and my family. The FDA has approved Kisunla (donanemab-azbt) for early-stage Alzheimer’s disease. This drug targets amyloid plaques (abnormal thick proteins found in Alzheimer’s patients) in the brain, believed to contribute to the disease's progression. Kisunla is an antibody that helps clear harmful amyloid-beta proteins linked to cognitive decline. Administered intravenously every four weeks, some patients may stop treatment after their amyloid levels become undetectable. Approval is based on Phase III trial results with over 2,700 participants, showing significant cognitive improvements compared to a placebo. About half were able to stop treatment after a year due to low amyloid levels which is unlike many drugs commonly used for these patients. Kisunla carries risks and doctors monitor patients with regular MRIs. The estimated cost for six months of treatment is $12,522, with each vial around $696 🙁 HOWEVER…. Medicare will cover the drug, and Eli Lilly provides support programs for patients!! 👏🏼👏🏼👏🏼👏🏼👏🏼 Kisunla's approval represents a significant advance in early Alzheimer’s treatment, with an emphasis on the importance of early diagnosis and improving quality of life while paving the way for further research into Alzheimer’s therapies. If someone you love is in early stage of cognitive decline, it may be worth discussing the benefits and risks with your doctors. (More information can be found in the NYT, Reuters, NBC and other news organizations, including clinical journals.) #KISUNLA #ENDALZ #CLINICALRESEARCH

By joining a clinical trial, patient will be a part of groundbreaking research that can lead to new treatments and cures, improve patient care, and help shape the future of medicine. Patients' participation makes a difference! #ctd2024⁠ #ctd2024my⁠ #getinvolved⁠ #iamaware⁠ #clinicaltrialsday2024⁠ #clinicaltrialawareness⁠ #malaysia⁠ #kualalumpur⁠ #clinicalresearchmalaysia⁠ #clinicaltrialfacts⁠ #clinicaltrialsjourney⁠ #oncology⁠ #cancercare⁠ #healthcare⁠ #patientjourney⁠ #clinicaltrialsvolunteer⁠ #clinicalresearch⁠ #clinicalstudies⁠ #pharmaceuticals⁠ #medicine⁠ #drugdevelopment⁠ #patientsafety⁠ #globalhealth⁠ #clinicalethics⁠ #patientawareness

We may be in the "#DTx winter" but at least in Europe there's a way for #digitaltherapeutics to reach the people who need them and a way for companies to get reimbursed. It's worth checking this article out on the evolution of Germany’s Digital Therapeutics reimbursement program over the last 3 years. ✅ Since its start in September 2020, six provisionally listed DiGAs have been removed from the directory, either due to failure in providing the required evidence or at the manufacturer’s request. ✅ The majority of DiGAs are available as mobile applications (34), often paired with a web application (9) while standalone web applications are less common (19). ✅ Over the three years since the program’s inception until the end of September 2023, approximately 374,000 #DiGA prescriptions have been activated for patient use. ✅ The directory predominantly features DTx for mental health conditions (25), followed by musculoskeletal disorders (8), neurological diseases (6), and endocrinological conditions (6). ✅ Less common, yet represented, indication fields currently featured are gynecological diseases (4), oncology (3), diseases of the auditory system (2), cardiovascular diseases (2), respiratory diseases (1), urological diseases (1), lifestyle conditions (1), and diseases of the gastrointestinal tract (1). Silver linings... #digitalhealth

The IgG4ward! Foundation is delighted to announce to publication of the MITIGATE trial in the New England Journal of Medicine this week. MITIGATE, the first world-wide clinical trial ever performed in people living with IgG4-RD, confirmed the effectiveness of B cell depletion treatment in this condition. Treatment with Inebilizumab in the trial reduced the risk of disease flare in the investigational treatment group by a remarkable 87%. The Foundation is deeply grateful to the people living with IgG4-RD who volunteered to participate in this trial. Through their efforts, we anticipate people across the world may benefit from the use of this medication to treat IgG4-RD (following the hoped-for drug approval by regulatory authorities). We are also grateful to the group of investigators - doctors, nurses, clinical trial coordinators, statisticians and others - who performed the trial at sites all over the world. Finally, we are grateful to Amgen, the trial's sponsor, for its vision and investments in pursuit of this therapy for people with our disease. Please join us in celebrating this achievement for people living with IgG4-RD. #IgG4relateddisease #IgG4RD #IgG4wardFoundation #NEJM #MITIGATETrial #MedicalResearch #ClinicalTrial #Inebilizumab #AmgenRare #Amgen #PatientAdvocacy 

# Only put off until tomorrow what you are willing to die having left undone \*\*Disc Medicine's DISC-0974 shows positive hematologic responses in myelofibrosis anemia, leading to a Phase 2 trial initiation.\*\* ## 🌟 Quiver AI Summary Disc Medicine, Inc. announced positive results from a Phase 1b trial of its investigational drug, DISC-0974, aimed at treating myelofibrosis \(MF\) anemia. The data, presented at the 2024 ASH annual meeting, showcased significant hematologic responses, paving the way for the initiation of a Phase 2 study. This breakthrough brings hope to patients suffering from MF and underscores the potential of DISC-0974 as a treatment option. Investing with a Health Savings Account \(HSA\) can be an empowering strategy to secure your financial future while prioritizing your health and well-being. By seizing opportunities like Disc Medicine's promising advancement in myelofibrosis anemia treatment, you can align your investments with the advancement of healthcare solutions. Don't miss out on the chance to invest in groundbreaking medical breakthroughs through your HSA. #ActNow #HSAInvesting #Healthcare #Health #Family #Wellness 💪💰💼📈🩺✨

🥼 Dr Anjay Rastogi discusses the impact of decreasing or discontinuing #RASSI treatment within three months after a hyperkalaemia episode, highlighting this strategy as "depriving patients of life-saving, organ-saving therapy." 🌟 Watch our video series to learn how potassium binder can help optimise RAASi therapy 🔗 https://ow.ly/1wXI50TqLS3 This programme is supported by an unrestricted educational grant from AstraZeneca. #hyperkalaemia #PotassiumBinders #HeartFailure

✨ In Just 5 Years: STXBP1 Therapeutic Programs Soar from 0 to 16 ✨ 🔍 What if the cure for STXBP1 disorder was just a few years away? With the rapid progress in therapeutic programs in recent years, this future might be closer than we think. In 2019, there were no industry-supported therapeutic programs for STXBP1 disorders. Just 5 years later, that number has grown to over 16 therapies in development, with multiple programs progressing into advanced stages, including clinical trials. The STXBP1 Foundation’s Fast Forward 3-Year Plan (2023–2025) has been instrumental in accelerating these developments. With four key pillars—Propelling Precision Therapies, Preparing for Clinical Trials, Building Biopharma Interest, and Supporting Patients Today—the plan ensures that every aspect of research, development, and patient care moves forward cohesively. Rafa's Moonshot is currently involved in advancing three innovative programs, including:  ✅ Drug repurposing ✅ Gene-targeted therapies like Antisense oligonucleotides (ASOs) ✅ A novel compound With this momentum, the potential for transformative treatments feels closer than ever. The coming years will pave the way for therapies that could redefine possibilities for those living with STXBP1 💫 #STXBP1Awareness #RareDiseaseResearch #InnovativeMedicine #RafasMoonshot

🥼 Dr Anjay Rastogi discusses the impact of decreasing or discontinuing #RASSI treatment within three months after a hyperkalaemia episode, highlighting this strategy as "depriving patients of life-saving, organ-saving therapy." 🌟 Watch our video series to learn how potassium binder can help optimise RAASi therapy 🔗 https://ow.ly/1wXI50TqLS3 This programme is supported by an unrestricted educational grant from AstraZeneca. #hyperkalaemia #PotassiumBinders #HeartFailure

Paediatric Patients Benefit from FDA Approval of NexoBrid for Severe Burns Treatment The FDA has approved NexoBrid by MediWound and Vericel® Corporation for pediatric burn patients, expanding its previous use for adults. The approval was based on positive results from the Phase III Children Innovative Debridement Study, which demonstrated NexoBrid's effectiveness in treating 145 pediatric patients. This breakthrough represents a significant advancement in burn care, offering a safer and less invasive treatment option for young patients. It also highlights the potential of proteolytic enzyme therapies in wound care, with MediWound continuing to innovate with products like EscharEx for chronic wounds. NexoBrid's approval not only transforms pediatric burn care but also solidifies MediWound's role in the evolving wound care industry, which is expected to grow to $38.8 billion by 2030. For more details please click the link! https://lnkd.in/dFUaps5g #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical