DEE-P Connections’ Post

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

New: Compass Pathways Pushes Back Phase 3 Readouts, Lays Off 30% of Staff Here's my initial take... This news is sure to knock the confidence of the psychedelic drug development field, just months after Lykos Therapeutics’ FDA approval miss plunged us into what felt like a nadir. Since then, other mid- and late-stage psychedelic drug developers have sought to distance themselves from the issues that seemed to have scuppered the MDMA new drug application (NDA), emphasising how their programs are designed with the agency’s expectations top of mind. Compass is walking a fine line when it comes to the messaging around these delays. In emphasising the logistical challenges of finding and scheduling both patients and therapists, the company risks stoking scepticism around its product’s commercial viability. Compass execs have tried to get ahead of these concerns by emphasising the particularly onerous nature of clinical trials, and their hope that healthcare professionals won’t need to rely on therapists if COMP360 is ultimately approved and rolled out. Instead, Compass hopes that healthcare professionals like nurse practitioners would be eligible to deliver the drug in that case, given its ‘psychological support’—and not ‘-assisted psychotherapy’—model. But Compass’ punt on its second phase 3 (COMP006) suggests that, on top of these recruitment and logistical difficulties, the company is also feeling nervous about its ability to adequately address the agency’s concerns around functional unblinding; or at least acutely aware of the need to meet the FDA’s expectations in this realm. Regardless, the company is in a tough spot in that it must assuage the concerns of both the agency, who will be focused on whether blinding was preserved throughout the studies (and particularly in COMP006), as well as investors, who would have preferred the earlier read-out. One thing’s certain, the company has a mammoth task on its hands, here. Its execs emphasised several times, today, that it is the first company to conduct psychedelics trials of this scale, and that it has a lot of learning to do.

Sharing on the counselling guide when dispensing oral chemotherapy medication to patients The use of oral cancer drugs has significantly increased in recent years and it is anticipated to continue to grow. It has gained much popularity among patients due to its convenience. Unfortunately, it is often wrongly perceived to be more tolerable and less toxic compared to conventional chemotherapy. The shift of treatment responsibility from healthcare facilities to patients' home raises significant concerns about drug safety. Therefore, effective counselling is vital to educate and empower patients to engage in their own drug management for a safe and optimal treatment outcome. Patients and carer need to be well-informed about the treatment and be provided with written guides for drug administration, the importance of early recognition of side effects and its prompt management. In order to achieve optimal treatment outcome without compromising patient safety, the importance of treatment adherence shall be emphasised upon. Link below 👇🏻👇🏻 https://lnkd.in/gc_YCXMM

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 

The development of therapeutics for the treatment of rare diseases can present several challenges to drug developers. Nazim Kanji and Huw Jones explore how #CRO & #CDMO partners can lend a consultative approach to help their clients across all areas of drug development in this article with Pharmafile: https://bit.ly/3wHjSBC

Treatment Access: PHARMAC Proposes Expanded Access to Urinary Tract Infections Treatment and New Epilepsy Medication Funding PHARMAC has opened a consultation to expand access to treatments for urinary tract infections and certain types of epilepsy, aiming to benefit over 2,900 New Zealanders in the first year. Alexandra Compton, Senior Therapeutic Group Manager at PHARMAC, noted that the proposals include community provision of fosfomycin for UTIs, which could reduce hospital-treated cases by 80%, and funding zonisamide for epilepsy. Supported by new government funding, this initiative seeks feedback from those affected, with the consultation open until August 1. If approved, these changes will enhance treatment access and reduce healthcare burdens in New Zealand. For more details please click the link! https://lnkd.in/gqTeCide #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

Recent trials of Alzheimer’s drugs lecanemab and donanemab show some promise as indicated by the 27% and 35% relative slowing of cognitive decline compared to placebo. However, the question remains: Are these effects meaningful for patients? Here’s what all the fuss is about: • Lecanemab: Slowed cognitive decline by 27%. After 18 months, patients had a 0.45-point slower decline on the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) compared to placebo. However, this change is less than half of what patients typically perceive as meaningful. The smallest change in a treatment that patients perceive as meaningful and impactful is termed the minimal clinically important difference (MCID) and for CDR-SB, this is 1.0 point for mild cognitive impairment (MCI) and 1.6 points for mild dementia. • Donanemab: Slowed cognitive decline by 35%. While seemingly more effective, the changes in cognitive scales were still below the threshold that patients, families, or physicians can notice as determined by the MCID. On the integrated Alzheimer's Disease Rating Scale (iADRS), donanemab showed an average improvement of ~ 3 points, but the MCID for iARDS is a 5-point change for MCI and a 9-point change for mild dementia. While group level results are statistically significant, will this appear to be noticeable to most patients and their families? Even marginal improvements in slowing congitive decline are welcome, providing the drug in use is safe. For concerns about efficacy, safety and other reasons, donanemab has come under scrutiny #Alzheimers #Pharma #CognitiveHealth #PatientCare

Spinal Muscular Atrophy Treatment Expanded: Medicinrådet Recommends Risdiplam for Patients Over 25 Following New Pricing Agreement The Danish Medicines Council (Medicinrådet) has expanded its recommendation for the SMA drug risdiplam, marketed by Roche as Evrysdi, to include patients over 25 years old, following a new pricing agreement with Roche that balances cost with expected treatment benefits. This expansion marks a significant advancement in SMA treatment, offering a non-invasive, oral therapy option for all ages. While patient advocacy groups have welcomed this broader access, the decision also underscores the ongoing challenges of ensuring equitable and sustainable access to expensive therapies for rare diseases. The new pricing model reflects the varying levels of clinical evidence for different age groups, ensuring that the treatment remains accessible while protecting healthcare budgets. For more details please click the link! https://lnkd.in/dkNSaG8u #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

Common Drug Interactions During Chemotherapy: How Pharmacists Safeguard Your Treatment Chemotherapy patients often take multiple medications, which increases the risk of drug interactions. These interactions can reduce the effectiveness of treatment or cause harmful side effects. Here's how pharmacists help prevent these risks: 1. Comprehensive Medication Review: Pharmacists review all your medications, including over-the-counter drugs and supplements, to identify potential interactions with chemotherapy. 2. Adjusting Treatment Plans: If a risk is detected, pharmacists collaborate with your healthcare team to adjust doses or suggest alternatives, ensuring optimal treatment outcomes. 3. Patient Education: Pharmacists provide essential guidance on how to safely take medications during chemotherapy, helping you avoid interactions that could affect your health. Pharmacists are key partners in your cancer care, working to ensure that your chemotherapy regimen is both safe and effective. #Chemotherapy #PharmacistRole #DrugInteractions #CancerCare #PatientSafety #OncologyPharmacy #MedicationSafety #HealthcareProfessionals

We've reported further significant clinical improvements in Rett Syndrome patients after 20 weeks of daily treatment with our cannabinoid drug therapy, NTI164. All 14 patients showed enhanced clinical outcomes across four core Rett-domain anchors, with 100% improvement (p<0.001) compared to 93% at 12 weeks. Dr Thomas Duthy, Executive Director of Neurotech International, said “We are pleased to see these patients continue to do very well on extended treatment with NTI164, with zero safety events recorded relating to diarrhoea and nausea/vomiting from week 12 to week 20 with zero weight loss recorded. Our safety and efficacy to 20 weeks is trending favourably when compared to the current FDA approved treatment, DAYBUE™ (trofinetide). Rett Syndrome represents an attractive market opportunity for NTI164, with a potential annual market opportunity of approximately US$2.0 billion. We are currently working with our clinical advisors and Professor Ellaway (our newly appointed Chief Medical Officer) on the design of a registration directed Phase III clinical trial.” A caregiver of a patient in the NTIRTT1 trial commented after 20 weeks of treatment with NTI164 “Increased use of eye gaze technology and increased ability to use the eye gaze accurately when making choices.” Read the full ASX Announcement here: https://lnkd.in/gx_hG54p #NTI #Neurotech #RettsSyndrome #ASX #ASXNews International Rett Syndrome Foundation Ontario Rett Syndrome Association