Gate One’s Post

Day 2 Highlight at World Orphan Drug Congress in Boston 🌐 Industry insights on partnering in the rare disease space struck a chord with us. Panelists discussed the advantages of partnerships between large and small biopharma and pharma companies. While established organizations can bring more resources to the development of medical treatments, smaller companies often have a deeper understanding of rare diseases and innovative approaches to problem-solving. 💡 Our agile team at Alethium Health has designed our clinical trial platform to anticipate nuanced needs and challenges before they happen. We’ve optimized trials by adjusting for patient or caregiver struggles, such as incorporating televisits and providing additional translations, to make clinical trials globally accessible at exceptional speed, reduced costs, and minimal risk. 🚀 #ClinicalTrials #OrphanDrugCongressUSA #DigitalClinicalTrials #RareDisease

I am proud to be conducting research and partnering with clients with products targeting rare diseases for over 10 years! Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Jack Scannell's perspective articles are rich in insights into drug development, challenging assumptions about disease models and highlighting their pitfalls. He stresses that many long-standing pre-clinical models in academia are long-standing because they lack predictive validity, revealing long-standing unmet clinical needs. Jack points out the lack of incentives for statistical hygiene and model validation in pre-clinical research. Without independent assessment based on standardized criteria, the validity of most pre-clinical models relies solely on author assertions, with peer-review as the closest form of evaluation. For instance, the ischemic stroke model leading to several publications has failed to translate experimental strategies into clinical success. Models lacking correlation with clinical utility yield unsuccessful high throughput screenings. Low throughput screening in well-validated correlative models, led to early antibiotics with higher discovery rates than those achieved today. Takeaway: Independent assessment based on standardized criteria not set by the author of the pre-clinical model is crucial for improving predictive value and reducing false positives, leading to better commercialization outcomes. #DrugDevelopment #PredictiveValue #ModelValidation

📢 𝐖𝐨𝐫𝐥𝐝 𝐄𝐏𝐀 𝐂𝐨𝐧𝐠𝐫𝐞𝐬𝐬 𝟐𝟎𝟐𝟒 𝐑𝐞𝐩𝐨𝐫𝐭 𝐢𝐬 𝐡𝐞𝐫𝐞! 👇 Remap Consulting joined over 1,500 other delegates from pharma, biotech and Payers/HTA's, in attending Europe's World Evidence, Pricing and Access Congress in Amsterdam, Netherlands from 12th - 13th March 2024. Our report summarises the key plenary sessions and parallel tracks that took place during the conference, and key takeaways from Dr. Graham Foxon's talk on the #EUHTA. Click to read the PDF report ➡ https://lnkd.in/ejKYkCT7 We'd love to hear your thoughts about #EPAconf , please share them below! 💬 #healthcare #marketaccess #pharma #hta #rarediseases #realworldevidence #digitaltransformation

Exciting news from Bristol Myers Squibb on #mezigdomide (#CC92480) combined with tazemetostat and dexamethasone for multiple myeloma! Mezigdomide, a potent CELMoD under investigation, has shown promising preclinical results, proving to be significantly more potent than #iberdomide and other #IMiDs. In the CA057-003 trial (#NCT05372354), 13 patients who had undergone a median of 5 prior lines of therapy received #mezigdomide, #tazemetostat, and #dexamethasone. The overall response rate (ORR) was 53.8%, with higher mezigdomide doses yielding an ORR of 71.4%. The combination demonstrated a manageable safety profile, with grade 3/4 TEAEs occurring in 69.2% of patients. Notably, mezigdomide rapidly degrades Ikaros and Aiolos proteins, inducing apoptosis in multiple myeloma cells and stimulating the immune system. The future of CELMoDs like mezigdomide looks promising as they continue to show high synergy with traditional partners and advanced treatments. Get detailed analysis of the abstract here @ https://lnkd.in/gd-xFFqy Stay tuned for more updates! #MultipleMyeloma #ClinicalTrials #Mezigdomide #BMS #CancerResearch #CELMoDs #healthcare #business #clinicalstudies #results #oncology #pharmaceutical #hematology

Alzheimer’s disease is among the world’s most pressing health care challenges, affecting more than 6.9 million people in the US and 40 million globally — numbers that are expected to double by 2040. Health and long-term care costs by then are likely to exceed $760 billion in the US. Our analysis indicates that the market for so-called disease-modifying therapies — as opposed to those that treat just symptoms — can jump to $13 billion by 2030 from $250 million this year. Further growth is possible from drugs attacking earlier stages of the disease, with more than 155 million people worldwide believed to have preclinical Alzheimer’s. Given the unmet need and the lack of a cure, there’s ample room for drugmakers including Roche, AbbVie, Alector, Prothena and Novo Nordisk. Access the full report here: https://bloom.bg/3RMdSyr #corporations #healthcare #innovation #pharma #biotech

The rare disease market has grown consistently over the past ten years and is expected to continue to grow, however, recent barriers have emerged, including an increasing pricing pressure and emphasis on cost-effectiveness proven through comparative clinical trials. Our latest white paper delves into the current trends shaping the rare disease landscape, offering insights into emerging therapies, evolving regulatory environments, and the critical role of patient advocacy. Download your copy now to learn more: https://lnkd.in/du7bPnQJ #RareDisease #Pharma #Biotech #HealthcareInnovation #WhitePaper

As the biotech industry continues to evolve, the pursuit of advanced therapies for rare diseases stands at the forefront. These conditions, affecting approximately 300 million people worldwide, present a significant unmet medical need. Here are key considerations for biotech companies venturing into this critical space: 1. Scientific Validation: Rigorous validation of therapeutic targets is paramount. The cautionary tale of Alexion Pharmaceuticals’ acquisition of Wilson Therapeutics underscores the importance of mechanistic trials and copper elimination efficacy. 2. Market Access Strategy: Clinical value alone won’t suffice. A robust market access strategy, aligned with clinical endpoints, is essential. Partnerships with rare disease experts can enhance outcomes and drive commercial success. 3. Patient-Centric Approach: Patient advocacy and engagement are pivotal. Biotechs must prioritize patient needs, ensuring therapies are accessible and affordable. Regulatory approval doesn’t guarantee patient access. 4. Financial Viability: Balancing innovation with financial sustainability is challenging. Biotechs should explore funding avenues, leverage incentives, and strategically manage R&D spending. I eagerly anticipate joining forces with each of our esteemed biotech partners, embarking on a journey to revolutionize lives through groundbreaking therapies! At WEP Clinical, we offer a comprehensive array of Patient Enabling Solutions, meticulously designed to propel your asset from the laboratory to the market. Let’s forge ahead together, making a lasting impact on patients worldwide! #WEPClinical #RareDiseases #BiotechInnovation #PatientImpact #PatientAccess

WEBINAR ALERT: "From Penicillin to digital therapeutics and the pace of change in life sciences: Where are the dollars flowing?" by Dean Erhardt 📅 Thursday, April 4, 2024 at 10am EST | 4pm CET Registration: https://lnkd.in/dSm_MM-S 🔍 Description: The life sciences industry is advancing at an unprecedented pace, from traditional remedies like penicillin to cutting-edge digital therapeutics. Join us for an engaging webinar as we delve into the changing landscape of life sciences investments. 🎯 Key Learning Objectives: ➡️ Understand current trends in life science investments ➡️ Identify high-growth categories expected in the near term (2–5 years) ➡️ Gain insights into anticipated FDA approvals for key disease states ➡️ Don't miss this opportunity to gain valuable insights and stay ahead in the rapidly evolving world of life sciences! Secure your spot now. #LifeSciences #PharmaTrends #DigitalTherapeutics #Investing #Webinar #pharmaindustry #drugdiscovery #drugdevelopment #sciex

Our in-depth full report from #EPAConf is here! Our report consolidates key plenaries and tracks, and includes main takeaway points from Graham Foxon's talk on #EUHTA. 📌 If our summary is helpful, take a moment to repost it to your network ♻ #healthcare #marketaccess #pharma #rarediseases #realworldevidence #digitalhealth #digitaltransformation