Health Guide USA’s Post

Join us for a conversation about a promising clinical trial model for new epilepsy treatments that accelerates access to the wider community of DEEs. A “basket trial” design is used in clinical trials for medicines aiming to treat symptoms of those living with DEEs, including seizures. This broad approach is different than the current standard clinical trial design singling out individual disorders, and would mean increased access to clinical research for a wider range of families interested in participating. With this more inclusive model, treatments approved using this method could include DEEs in the "label" (i.e. approved by the FDA for use), reducing the need for families to have to fight with doctors and insurance companies for the use of drugs off-label. Join us to learn more about this model in our discussion with Longboard Pharmaceuticals who has applied this model in their innovative Phase 2 trial design which was inclusive of all DEEs. DEE-P Connections is actively advocating for this model to improve wider access to trials and approved medications across the #DEEs. All are welcome to listen, learn and ask your questions! April 29th 3:30-4pm ET REGISTER HERE - https://lnkd.in/gxqdDq9C

So excited for this conversation and about the possibility of more #clinicaltrials that are inclusive across the #DEEs, making more treatments available to more families who have children with no medications indicated for their disorders. #equity #access #clinicalresearch #neurology #epilepsy #RareEpilepsy Longboard Pharmaceuticals

Bladder Treatment Breakthrough: NICE - National Institute for Health and Care Excellence Recommends Vibegron for Overactive Bladder Syndrome in Adults NICE - National Institute for Health and Care Excellence has recommended vibegron as a treatment option for adults with overactive bladder syndrome when antimuscarinic medicines are ineffective or unsuitable. Clinical trials show that vibegron effectively reduces symptoms and offers a cost-saving alternative to mirabegron, making it a financially viable option for the NHS. Although direct comparisons between vibegron and mirabegron are limited, evidence suggests similar efficacy. NHS England and integrated care boards are required to implement this treatment option within 30 days of publication, ensuring timely access for patients. Novartis, the manufacturer, has priced vibegron competitively, further supporting its adoption. For more details please click the link! https://lnkd.in/d7BqfF5K #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

PBAC Reviews Endometriosis Treatments for PBS Inclusion, Aiming to Bridge Healthcare Gaps in Australia The Pharmaceutical Benefits Scheme (PBS) review in Australia is examining medication accessibility for individuals with endometriosis. The review aims to expand access to essential treatments and improve healthcare equity for those affected by the condition. The Australian government has allocated $58 million for endometriosis treatment and research as part of the National Action Plan for Endometriosis. The report identified medicines currently listed on the PBS for endometriosis treatment and proposed additional options. Barriers to accessing treatments include limited training among healthcare professionals and a lack of incentive for pharmaceutical companies to pursue PBS listing. The PBAC has acknowledged the need for funding more medicines but also highlighted the need for further research to evaluate the effectiveness of suggested treatments. For more details please click the link! https://lnkd.in/dAZZDuib #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

https://lnkd.in/dG2vfcFv Patel S. Inadvertent administration of intravenous anaesthesia induction agents via the intracerebroventricular, neuraxial or peripheral nerve route – A narrative review. Indian J Anaesth 2024;68:439‐446. I am glad that my review (eight pages) "Inadvertent Administration of Intravenous Anaesthesia Induction Agents Via the Intracerebroventricular, Neuraxial or Peripheral Nerve Route – A Narrative Review" is published in the May 2024 issue of the Indian J of Anaesthesia DOI: 10.4103/ija.ija_1276_23 In line with other neuraxial medication administration error publications, this topic has never been reviewed in the literature. The article contains four tables (clinical analysis, human factors, overview of IV intravenous induction agent errors, and prevention via external ventricular and lumbar drain). I have also prepared a management approach (Figure 1). I prepared the digital image. There are two supplemental files. I am glad I have consolidated my position as the only author to comprehensively investigate neuraxial drug administration errors. I believe this will not change for decades to come. 🙏🙏 My previous work related to neuraxial drug administration errors. Needs updating. https://lnkd.in/dNA8aXKC INDIAN JOURNAL OF ANAESTHESIA Institute for Safe Medication Practices (ISMP) Anesthesia Patient Safety Foundation (APSF)

🌟Ardelyx's, Xphozah (tenapanor), has earned the coveted #orphandrug designation from the US Food and Drug Administration for the treatment of paediatric hyperphosphatemia. 🏆 Originally approved last month as an add-on therapy for adult chronic kidney disease patients on dialysis, Xphozah is an oral phosphate absorption inhibitor. Its #innovative mechanism involves inhibiting the sodium hydrogen exchanger 3 (NHE3), thereby reducing phosphate absorption through the paracellular pathway—the primary route of phosphate absorption. 👉 Key Achievements: ✨ Tax Credits and Market Exclusivity: With this designation, #Ardelyx is set to receive tax credits for US-based clinical trials and, upon approval, potentially enjoy seven years of market exclusivity specifically in the #paediatric hyperphosphatemia domain. This recognition reflects the FDA's acknowledgment of Xphozah's potential impact on improving the lives of young patients. 🌊 Addressing Hyperphosphatemia Challenges: #Hyperphosphatemia, characterized by elevated phosphate levels in the blood, poses significant challenges for patients with chronic kidney disease. Ardelyx's Xphozah provides a crucial solution, especially for those unresponsive or intolerant to conventional phosphate binder therapy. 📈 Next Steps: As Ardelyx moves forward with Xphozah's potential application in paediatric hyperphosphatemia, the focus remains on advancing #clinicaltrials and contributing to the evolving landscape of therapeutic options for young patients. The recognition by the #fda not only signifies a significant step for Ardelyx but also reinforces the #commitment to addressing unmet medical needs on a global scale. #ArdelyxInnovation #FDAApproval #PediatricHyperphosphatemia #MedicalAdvancements #HealthcareTransformation #SymmetricTraining

Major Bleeding With Concomitant SSRI, OAC Prescriptions - Pharmacists and other healthcare professionals might want to raise some questions about prescriptions for selective serotonin reuptake inhibitors (SSRIs) in patients on oral anticoagulants (OACs) and vice-versa. That is based on the findings of a new Canadian study, led by McGill University in Montreal... Read More Here - https://lnkd.in/dYVek5C5 #pharmacist #USPWeeklyNews #communitypharmacist #PharmacyTech #Pharmacystudent #specialtypharmacy #longtermcarepharmacy

Subcutaneous Ocrelizumab Provides New Administration Opportunities for Multiple Sclerosis: Subcutaneous ocrelizumab can be delivered in approximately 10 minutes and deliver comparable clinical benefit and safety to the IV formulation. #finance #pharmacy #lifesciences

NICE Approves Etrasimod for Ulcerative Colitis: A New Hope for Patients Aged 16 and Over The National Institute for Health and Care Excellence has endorsed the use of Etrasimod for ulcerative colitis patients who have not responded well to conventional treatments. Etrasimod, sold under the name Velsipity by Pfizer, has shown promising results in clinical trials and is a cost-effective option. The guidance includes a commercial arrangement with the NHS to offer discounts on the drug. NHS England and integrated care boards are required to ensure availability of Etrasimod within a specified timeframe. For more details please click the link! https://lnkd.in/d85SBxXY #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

Depressive disorders are highly prevalent and disabling medical conditions worldwide. Medicinal products with better efficacy, improved safety and more personalised profiles are much needed. Our colleagues André Elferink and Taina Mattila cooperated with other experts of the medicines authorities of Germany, Poland and France in the CNS working party drafting a new updated EMA guideline for drug researchers and developers working on antidepressants. The revised guideline on clinical investigation, to replace the previous guideline that came into force in 2013. The document is available for external consultation until March 31, 2024. The new draft is the third revision of the guideline, which incorporates the latest insights in requirements for clinical trials in this field. Updates have been made for example in requirements for clinical trials in difficult to treat patient populations (treatment resistant and partial responders), and for the first time, antidepressants with a rapid onset of effect, psychedelics and targeted therapies to address symptom clusters are addressed in the guideline. More details on the proposed changes in the guideline are summarized in an article https://lnkd.in/eZq5eAqG Please note that there is also a new draft Guideline on clinical investigation of medicinal products in the treatment of epileptic disorders up for consultation on the website of the European Medicines Agency: https://lnkd.in/esR7GQ2b