Making market access decisions about rare disease therapies can be challenging due to limited clinical trial data, particularly when dealing with smaller patient populations. Dr. Belinda Yap, Director of InnomarConsulting™, HEOR, discusses how patient support programs can leverage real-world evidence (RWE) strategies to supplement the data for rare disease therapies to inform decision-making. Discover how RWE can bridge the data gap from pre- to post-launch across the product lifecycle in this video: https://ow.ly/BA0F50T3iVV
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🔬 Optimizing rare disease clinical trials comes with unique challenges due to small patient populations, limited natural history data, and heterogeneous manifestations. At #IDDI, we are not only experts in statistical methodologies but also deeply understand the complex statistical regulatory environment hence able to focus on the key considerations to enhance the efficiency and success of rare disease clinical trials. Curious to discover our expert insights on optimizing rare disease trials? Read our blog: https://lnkd.in/esfRcCpA #rarediseases #rarediseaseresearch #clinicaltrialdesign #orphandiseases #biometricscro #biostatistics #clinicaltrials
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Next Tuesday at #PatientasPartnersEU, Marisa Minetti and I will discuss how Chiesi Group and Chiesi Global Rare Diseases are working towards valuable patient-centric research. THIS IS US THAT generates innovation and value putting the patients first. #patientaspartners #patientengagement #clinicalresearch #innovation #collaboration #chiesigroup #chiesiglobalrarediseases
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Rare diseases bring several challenges to clinical trials. Read our blog below to see how IDDI can help you with optimize your study.
🔬 Optimizing rare disease clinical trials comes with unique challenges due to small patient populations, limited natural history data, and heterogeneous manifestations. At #IDDI, we are not only experts in statistical methodologies but also deeply understand the complex statistical regulatory environment hence able to focus on the key considerations to enhance the efficiency and success of rare disease clinical trials. Curious to discover our expert insights on optimizing rare disease trials? Read our blog: https://lnkd.in/esfRcCpA #rarediseases #rarediseaseresearch #clinicaltrialdesign #orphandiseases #biometricscro #biostatistics #clinicaltrials
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📈 Mesothelioma Survival Rates Show Gradual Improvement Encouraging news from the latest AIHW report: survival rates for mesothelioma, while still challenging, are gradually improving over time. The report shows that between 1991–1995 and 2016–2020, the 1-year survival rate increased from 36.2% to 48.5%. Although mesothelioma remains a difficult diagnosis, advancements in diagnostics, treatment and early detection are making a tangible difference in patient outcomes. This progress underscores the importance of ongoing research, better diagnostics, and innovative treatments to improve survival and quality of life for those affected by mesothelioma. To learn more, download the full report here: https://lnkd.in/gH5GNPaK Reference: Mesothelioma in Australia 2023 Report (AIHW), p. 9 and data table
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🙌 We are thrilled with the incredible turnout for this CME-accredited symposium led by Prof Julian Gillmore. If you miss the live session or want to revisit the valuable research shared, you can now watch the entire series online 🔗 https://ow.ly/KzLZ50TpNfg Enhance your diagnostic skills in ATTR-CM by: 🌟 Understanding recent epidemiological data 🌟 Identifying patients for detailed diagnosis 🌟 Exploring emerging therapeutic agents #CardioVascular #ATTRCM #MedicalEducation #CME
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Rare diseases pose unique challenges for treatment development. Small patient pools, evolving disease knowledge, and the urgent need for therapies demand innovative approaches. Optimizing clinical trial designs is key. Researchers are employing several strategies. Adaptive trials offer flexibility, allowing adjustments based on new data. This streamlines the process by focusing resources on promising options. Enriched enrollment targets specific patient groups, leading to stronger data. Master protocols test multiple therapies within a single trial, saving time and resources. Real-world data integration provides a more comprehensive picture of the disease and treatment response. Optimizing trials offers several benefits. Faster development timelines expedite new therapies. Streamlined trials and a focus on promising approaches improve efficiency and reduce costs. Targeted enrollment and flexible designs lead to cleaner data, informing the development of more effective therapies. The future of rare disease treatment development relies on continued innovation in clinical trial design. Collaboration among researchers, regulators, and patient advocacy groups is crucial to accelerate progress and bring hope to patients. #Planimeter #AdaptiveTrials #CleanerData #EffectiveTherapy #ReducedCosts
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Rare disease trials come with specific challenges. Read the blog below to find out more, and reach out to IDDI to help support you in your rare disease clinical trial.
🔬 Optimizing rare disease clinical trials comes with unique challenges due to small patient populations, limited natural history data, and heterogeneous manifestations. At #IDDI, we are not only experts in statistical methodologies but also deeply understand the complex statistical regulatory environment hence able to focus on the key considerations to enhance the efficiency and success of rare disease clinical trials. Curious to discover our expert insights on optimizing rare disease trials? Read our blog: https://lnkd.in/esfRcCpA #rarediseases #rarediseaseresearch #clinicaltrialdesign #orphandiseases #biometricscro #biostatistics #clinicaltrials
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🔬 Precision medicine is poised to transform healthcare by enhancing early disease detection, refining diagnoses and tailoring treatments. To harness this potential, various governments around the world have launched national #genomics programmes each designed with unique goals. In the first of this two-part infographic series, we take a look at five genomics projects in Asia, Australia and South Africa. Discover how these global initiatives are driving medical innovation and shaping the future of healthcare. Read more via https://lnkd.in/gKhHAqmN Stay tuned for Part Two! #npm_sg #precisionmedicine #globalhealth
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🌟 Live from the Regeneron's Digital Biomarkers Summit 🌟 During the session "DHTs in Chronic Pain Studies: Collaborative Development between Eli Lilly and Company and Empatica," Brian Winger, Associate VP of Digital Health R&D at Eli Lily, reminds us that #dBMs can potentially eliminate placebo responses. 💊 A good reminder that strong scientific rationales for innovation are often the quickest path to adoption. 💪🏽 #Regeneron #DigitalBiomakersSummit #ClinicalOperations #ClinicalResearch #ClinicalDevelopment
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Part 2 - Snapshot of MTRC activities (HEOR) Our goal at MTRC is to inform the decision-making of our clients and to support their market access activities. Below is a snapshot of our planned activities for the next two weeks. This can help our existing and prospective clients to better understand the potential scope of our support. From the week of October 21st, we are planning to work on 16 projects, including: HEOR 1. Working on the development of an HTA national submission dossier in Sweden 2. Working on the development of a health economic model for neurovascular technology in Belgium, England, France, Germany, Italy, and Spain 3. Delivering the European value dossier for a wound care product in Europe 4. Working on the Evidence Gap Analysis for a wound care product in France 5. Working on the update of adaptation of the global value dossiers for several technologies in cardio-renal and infectious disease space to European settings 6. Working on adaptations of the health economic model for in-vitro diagnostic tests in infectious disease areas in France and Spain 7. Working on the development of a health economic model for neuromonitoring technology in England Let us know if you need any market access or HEOR support for your medical technology in Europe: https://lnkd.in/eZrwqVe2
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1moAmazing! 🙏