Practical Patient Care’s Post

As the #pharma industry navigates the impact of the Inflation Reduction Act on drug pricing and #innovation; real-world evidence, propelled by the Cures Act, is becoming increasingly vital for driving strategy and drug development. I wanted to share this older paper we wrote before RWE became a thing, where we discuss the creation of a large real-world data asset focused on the neuroscience therapeutic area. #RealWorldData #RealWorldEvidence #InflationReducionAct #21CCA #CuresAct #DrugDevelopment #Pharmaceuticals #Neuroscience #DataScience #Innovation #BeforeItWasCool Regenstrief Institute, Inc. Brian E Dixon, PhD

𝗥𝗲𝘃𝗼𝗹𝘂𝘁𝗶𝗼𝗻𝗶𝘇𝗶𝗻𝗴 𝗥𝗔 𝘁𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝘄𝗶𝘁𝗵 𝗴𝗿𝗼𝘂𝗻𝗱𝗯𝗿𝗲𝗮𝗸𝗶𝗻𝗴 𝗻𝗲𝘂𝗿𝗼𝘀𝘁𝗶𝗺𝘂𝗹𝗮𝘁𝗶𝗼𝗻 𝗿𝗲𝘀𝘂𝗹𝘁𝘀! Huge congratulations to Murthy Simhambhatla and the entire SetPoint Medical team for achieving positive results in their RESET-RA clinical study! 🌟 Their innovative neuroimmune modulation treatment for rheumatoid arthritis (RA) has shown remarkable promise, offering new hope for patients who haven't responded well to traditional therapies. The statistically significant results, with no safety concerns and low adverse event rates, highlight the potential of their rechargeable neurostimulation device as a safe and effective alternative to current RA treatments. Wishing SetPoint Medical all the best as the team prepares to present this groundbreaking data at upcoming medical meetings and completes the premarket approval submission to the FDA. #MedTech #RheumatoidArthritis #Neurostimulation #HealthcareInnovation #MedicalDevices

MCP-Mod in dose finding studies – current standard and advanced approaches The AGAH symposium at the German PharmToxSummit in Munich this year focused on MCP-Mod. Gabriele Bleckert (Staburo GmbH) gave a very condensed talk about the overall principles and significant advatages of this methodology which in the meantime is an established standard and well accepted for phase-II-dose finding trials by regulatory agencies – FDA as well as EMA. The combination of Multiple Comparison Procedure and Modelling was illustrated with 2 examples, one with a “classical” pharmacodynamic endpoint HbA1C and the other one with a psychometric score for cognitive impairment demonstrating impressively that the method can even be used in trials with psychiatric disorders as long as the endpoint presents as suitable surrogate for efficacy. Sebastian Bossert (Boehringer Ingelheim) explained the challenges during the planning phase especially in terms of selecting the right dose. And he gave insight into how modern drug development introduced the principles of MCP Mod into adaptive trial designs which finally allow combination of Proof-of-Concept and dose finding within one trial eg by first including the highest dose with an interim futility analysis before continuing with the dose finding procedure. The concept was illustrated by the BALANCE-CF 1 trial in cystic fibrosis with ppFEV1 as surrogate endpoint. He also demonstrated that even sophisticated trial designs do not guarantee success if the active principle is not convincing. The speakers were honoured with a very vivid discussion and the organisers from AGAH, Ruwen Böhm and Barbara Schug (both SocraTec R&D) were impressed by the intense interaction with the auditorium. #MCPMOD, #EarlyPhase, #DoseFinding  

Kudos to the The National Institute on Drug Abuse (NIDA) and FDA on their published correspondence in Springer Nature Group / Nature Medicine on recent progress in the development of novel medical devices to combat the opioid epidemic. Neurotype Inc. was honored to participate in the 2022 FDA workshop discussed in the article. I am particularly excited to see the article discussing the potential of brain electrophysiology as a tool for Opioid Use Disorder treatments, as we are developing with our brain biomarker platform. Their article underscores the importance of innovative, evidence-based, translational approaches to improve patient outcomes! Publisher's article here: https://lnkd.in/gCDUs7t8 #opioidcrisis #medtech #healthcareinnovation #electrophysiology

A recent study by Gobrecht et al. sheds light on a promising path forward in nerve regeneration drug development. Researchers have been struggling with the slow pace of axon regeneration, which crawls at a maximum rate of 1-2 mm per day. This challenge is further compounded by the limited support period provided by Schwann cells in the peripheral nervous system, ending around three months after nerve injury. Failure to achieve reinnervation within this timeframe can result in lifelong disabilities and neuropathic pain for patients. The findings of this study could be a game-changer in the field of nerve regeneration, potentially leading to more effective treatments and improved quality of life for patients. #nerveregeneration #medicalresearch #healthcare

A double-blind clinical trial of 120 patients showed that topical use of Nigella Sativa has significantly better effects than synthetic drugs and placebo. Symptoms such as numbness, burning, and muscle cramps were significantly reduced. This natural option could be an effective solution without the side effects of conventional drugs. The results are promising for improving the quality of life of diabetic patients. Learn more about this topic on our website: https://lnkd.in/dGb_mUC3 If your company would like to create or manufacture a product indicated for neuropathic pain and neurological disorders in Diabetic Neuropathy, please contact us: info@farmaimpresa.it #DiabeticNeuropathy #NigellaSativa #TopicalTreatment #DiabeticHealth #ClinicalStudy #NaturalMedicine

Pain is a universal human experience, affecting around 20% of the population. It is complex and can be initiated by several distinct conditions, posing unique therapeutic challenges that require a tailored treatment approach. Current pain management options have limitations and there is a need for better therapies. We've created an infographic tracking progress in the discovery of therapies for neuropathic pain, outlining new drug targets and candidates in preclinical development and clinical trials. See the full infographic "Tackling the Hidden Epidemic: Advances in Pain Management Drug Discovery" at https://okt.to/YOZG4b #Infographic #PainManagement #NeurologyResearch #ResearchChemicals

📢 Exciting News! 🎉 We're thrilled to announce the publication of our research in the esteemed Journal of Association of Physicians of India (JAPI)! Our study underscores the critical need for Clinical Pharmacology Clinics in our country to enhance patient care. 👩⚕️ Collaborating with Gynecologist Dr. Avishek Bhadra, and with invaluable guidance from esteemed Prof. Shashank R Joshi, Prof. Jyotirmoy Pal, and Prof. Santanu Tripathi, we delved into the realm of gestational hypertension and its potential association with preeclampsia/eclampsia. 🔬 Our findings? An intriguing insight into sympathetic neurofunction in gestational hypertension! Our study revealed a significant correlation between a rise in diastolic blood pressure post-handgrip exercise and the development of preeclampsia/eclampsia. 💡 This suggests that addressing sympathetic hyperactivity could hold the key to more effective therapeutics for managing gestational hypertension. 🙌 Kudos to our dedicated team members whose hard work made this possible! We're eager to hear your thoughts and feedback on our findings. Let's continue to push the boundaries of medical research together! Link for complete article: https://lnkd.in/gZEJJisA #MedicalResearch #GestationalHypertension #Preeclampsia #clinicalpharmacology

"NTI’s approval strategy looks to parallel the pathway of ‘Epidiolex’ the first and only US FDA-approved cannabinoid drug, with sales now approaching >US$1b in epilepsy." MST Financial has initiated coverage with a $0.60 price target - that’s a 714% upside from the current share price! Their lead drug, NTI164, shows promising results in treating neuroinflammatory disorders like autism, Rett Syndrome, and cerebral palsy. With a large potential market and a clear commercialisation strategy, NTI is positioned for significant growth. Key highlights from the report: - Positive Phase 2/3 trial data in autism - Large addressable market of over A$12 billion - Experienced management team with a proven track record Read the full Report here: https://lnkd.in/gXxgeKMA #NTI #NeurotechInternational #MSTFinancial #biotech #investing #healthcare #autism #RettSyndrome #cerebralpalsy #PANDAS #PANS

A less-explored aspect of the excitement around senolytics for Alzheimer's Disease treatment involves the potential challenges and considerations related to translating promising results from clinical trials into widespread clinical applications. While initial findings may generate enthusiasm, several factors contribute to the complexity of adopting senolytics as a mainstream treatment for Alzheimer's Disease. 1. Long-Term Effects: Senolytics, which target and eliminate senescent cells associated with aging, have shown promise in preclinical and early clinical studies. However, the long-term effects and safety profiles of these drugs need further investigation. Potential side effects and unintended consequences over extended use must be thoroughly understood before widespread adoption. 2. Heterogeneity of Alzheimer's Disease: Alzheimer's Disease is a complex neurodegenerative condition with various subtypes and contributing factors. Senolytics may not be a one-size-fits-all solution, and their efficacy could vary based on the specific characteristics of the patient's Alzheimer's profile. Understanding the heterogeneity of the disease and tailoring treatments accordingly is a crucial consideration. 3. Timing and Disease Progression: The timing of senolytic intervention in the progression of Alzheimer's Disease is a critical factor. It's unclear whether these drugs would be most effective in early stages, during mild cognitive impairment, or even in more advanced stages. Determining the optimal window for intervention requires careful consideration of disease progression and individualized treatment plans. 4. Ethical and Regulatory Challenges: As senolytics and other anti-aging interventions progress, ethical considerations regarding accessibility, affordability, and equitable distribution must be addressed. Regulatory agencies will play a crucial role in ensuring that these treatments meet rigorous standards for safety and efficacy before reaching widespread clinical use. 5. Combination Therapies: Alzheimer's Disease is multifaceted, and a comprehensive approach may involve combining senolytics with other therapeutic strategies. Understanding how these drugs interact with existing treatments or complementary interventions is an area that warrants exploration. While the excitement around senolytics for Alzheimer's Disease is based on promising early results, it's important to approach these developments with a cautious optimism. Further research, including larger and more diverse clinical trials, is necessary to establish the safety, efficacy, and practicality of senolytics as a viable and widespread treatment option for Alzheimer's Disease.