Mike Hyungkyu Park’s Post

🚨 Therapeutic adherence is a major public health challenge. To date, more than half of all patients do not take their medications exactly as prescribed. Our teams have launched a prospective international and non-interventional study in cardiology. The aim is to assess a medicine’s real-world effectiveness and impact on quality of life. The BEAMER questionnaire has been incorporated into this study and proposed to all patients. Real-world patient data collected relating to therapeutic adherence will be used to enhance the BEAMER database. As Valérie Lehner, RWE Global Medical & Patient Affairs at Servier stated: 🗣“Placing patients at the heart of the development of this behavioral model reflects Servier's commitment to therapeutic progress to meet patient needs.” Congratulations 👏 to all the teams involved in this project! Innovative Health Initiative (IHI) EFPIA - European Federation of Pharmaceutical Industries and Associations #adherence #patient #WeAreServier #MovedByYou

🧠 𝗔𝗹𝘇𝗵𝗲𝗶𝗺𝗲𝗿'𝘀 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁: 𝗔 𝗣𝗿𝗼𝗺𝗶𝘀𝗶𝗻𝗴 𝗦𝘁𝗲𝗽 𝗙𝗼𝗿𝘄𝗮𝗿𝗱   The race to find effective treatments for Alzheimer's disease has produced mixed results. While the FDA has approved Aducanumab (Aduhelm®), Lecanemab (Leqmbi®), and Donanemab (Kisunla®), the European Medicines Agency (EMA) is taking a more cautious approach. ⚖️   Lecanemab (Leqembi®), approved by the FDA in July 2024, has demonstrated potential in slowing cognitive decline in Alzheimer's patients. However, the European Medicines Agency (EMA) rejected its application, citing concerns about the balance between the observed benefits and the risks of serious side effects, such as Amyloid-related imaging abnormalities (ARIA). 🔬   𝗜𝗺𝗽𝗼𝗿𝘁𝗮𝗻𝘁 𝗨𝗽𝗱𝗮𝘁𝗲 👍 MHRA Approval: The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for Lecanemab, making it available to patients in the UK. 🔄 EMA Re-examination: The company behind Leqembi has requested a re-examination of the EMA's July 2024 decision. This could potentially lead to a different outcome.   𝗡𝗲𝗲𝗱 𝗟𝗲𝗰𝗮𝗻𝗲𝗺𝗮𝗯 𝗳𝗼𝗿 𝗬𝗼𝘂𝗿 𝗥𝗲𝘀𝗲𝗮𝗿𝗰𝗵? 🛒 Evidentic is your sourcing specialist for licensed Lecanemab , sourced directly from the original Leqembi®, and other top-quality clinical-grade molecules. 🧬 Check our online shop (link in the comments) and discover the potential of clinical-grade molecules to improve research outcomes!   #Alzheimers #Lecanemab #FDA #EMA #MHRA #Neuroscience #Evidentic #MonoclonalAntibodies #MABs #ClinicalGrade #DrugDevelopment #DrugDiscovery #MedicalResearch #Pharmaceuticals #ResearchandDevelopment

🙋♂️ "We are prepared to go a long way." This quote from one of the most renowned systemic mastocytosis (SM) experts resonated with me at the closing session of the ECNM Congress. The congress provided a platform for collaboration and learning in the field of mastocytosis, uniting SM experts and patient advocates across Europe. It was also a valuable opportunity to strengthen the bond between the scientific and patient communities. Key highlights from the event: 👉 Characterization of SM patients is an ongoing process, emphasizing the importance of collaboration and consensus on classification and diagnosis. 👉 Understanding the disease's pathogenesis remains a priority for researchers. 👉 Identifying novel therapeutic targets holds promise for enhancing the quality of life for individuals living with SM. Witnessing the potential of these exchanges underscores how collective efforts from scientific societies, patient organizations, and pharmaceutical companies can significantly impact people's lives! #patientadvocacy #innovations #pharma Blueprint Medicines

FDA's Office of New Drugs (OND) Research Outcomes Report for FY2022-2023! This comprehensive report highlights the significant strides made in regulatory science, aimed at improving new drug approval processes and addressing unmet medical needs. Key Highlights: 1.Research Contributions: Over 80 external presentations and 94 peer-reviewed publications showcasing groundbreaking work in various therapeutic areas. 2.Innovative Projects: a.Development of clinical outcome assessments for rare diseases. b.Physiologically-based pharmacokinetic modeling for predicting fetal exposure to maternal drugs. c.Advancements in pediatric oncology and acute pain treatment strategies. 3.Regulatory Impacts: 33 outcomes that directly facilitated regulatory decision-making, including new drug development tools and internal policies. Read the full report #FDA #DrugDevelopment #ClinicalResearch #RegulatoryScience #Innovation #Healthcare

We will participate in multiple important congresses and meetings and very much look forward to connect with participants on site interested in data innovations in hematology. Make sure to check our calendar regularly - it would be great to meet up in person and discuss what the value of working with the HARMONY Big Data Platform could mean for you: https://bit.ly/3yfKM0h Contact via LinkedIn with Ellen P.B. de Waal European Hematology Association (EHA) or email communications@harmony-alliance.eu to arrange an appointment. Please share our posts. ____ Public−Private Partnership for #bigdata in #hematology 128 Partners and Associated Partner from 28 countries: including Academic Institutions, Hospitals, Medical associations, HTA agencies, regulators, data scientists, 9 European Patient Organizations, 9 Pharma Companies. Funded by Innovative Health Initiative (IHI) Supported by European Commission EFPIA - European Federation of Pharmaceutical Industries and Associations

FDA/CDER’s Quantitative Medicine Center of Excellence Announces the Availability of New Resources The U.S. Food and Drug Administration’s (FDA’s) Center for Drug Evaluation and Research (CDER) Quantitative Medicine Center of Excellence (QM CoE) was founded on March 25, 2024 to foster integration of quantitative methods across CDER and help advance therapeutic medical product development and inform regulatory decision-making. To fulfill, in part, the mission of the QM CoE to: 1) spearhead QM-related policy development and best practices; 2) facilitate systematic outreach; and 3) coordinate QM education and training, the QM CoE is pleased to announce the availability of the following new resources: A quick reference Fact Sheet, providing a high-level overview of the QM CoE The recording and slides for the April 25, 2024 FDA public workshop entitled Streamlining Drug Development and Improving Public Health through Quantitative Medicine: An Introduction to the CDER Quantitative Medicine Center of Excellence A list of Frequently Asked Questions regarding the purpose, scope and upcoming activities of the QM CoE, as well as information on how to get involved An Educational Resources hub that features an assortment of educational content including free, web-based training modules on model-informed drug development (MIDD) developed by the FDA Office of Translational Sciences in collaboration with the Critical Path Institute (C-Path) and experts in the community. The series includes eight modules with 29 presentations and more than 10 hours of content on a variety of MIDD topics. #fda #clinicalpharmacology #centerofexcellence #resources #quantitativemedicine https://lnkd.in/giN6NxZh

Recent update of the European Medicines Agency's Agency's critical medicines list emphasises the importance of plasma-derived therapies for EU healthcare systems and patients. This makes plasma donation and collection even more crucial for the well-being of European patients. How can industry gain visibility into the medicine supply chain and avoid future crises? Governmental experts will dive into these topics at the #IPPC2024 next month: 👉 Cemre Balaban, Junior Trade Policy Analyst, Trade Policy Division, Trade and Agriculture Directorate - OECD - OCDE 👉 Klaus Kruttwig Medicines and Medical Devices Shortages Specialist - European Medicines Agency 👉 Stelios Kympouropoulos, Member of European Parliament, European Parliament 👉 Domenico Di Giorgio, Head of Inspection and Certification Department and of the Pharmaceutical Crime Counteracting -Office - AIFA Agenzia Italiana del farmaco   In the coming months, as part of the Critical Medicines Alliance, PPTA looks forward to collaborate and share our expertise, scientific evidence, and experience to strengthen the frameworks of sustainable and resilient supply chains for plasma-derived medicines. #IPPC2024 https://lnkd.in/gRma-WAB

𝐑𝐨𝐜𝐡𝐞 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐄𝐌𝐀’𝐬 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐎𝐩𝐢𝐧𝐢𝐨𝐧 𝐨𝐧 𝐏𝐢𝐚𝐒𝐤𝐲® (𝐜𝐫𝐨𝐯𝐚𝐥𝐢𝐦𝐚𝐛) 𝐟𝐨𝐫 𝐏𝐍𝐇 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 Roche (SIX: RO, ROG; OTCQX: RHHBY) Roche announced today that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for PiaSky® (crovalimab) for treating paroxysmal nocturnal haemoglobinuria (PNH). If approved, PiaSky will be the first monthly subcutaneous (SC) treatment for PNH in the EU, offering self-administration after training. This alternative to current intravenous treatments could reduce the treatment burden for PNH patients and caregivers. “PNH patients often face lifelong treatment with frequent IV infusions and clinic visits,” said Levi Garraway, M.D., Ph.D. Levi Garraway, Roche’s Chief Medical Officer. “Self-administering once a month could offer patients and caregivers more freedom in their daily lives.” PNH is a rare, life-threatening blood condition affecting around 20,000 people worldwide. PiaSky is a novel C5 inhibitor that is recycled in the bloodstream, allowing SC administration every four weeks. The CHMP recommendation is based on the Phase III COMMODORE 2 study, showing that PiaSky achieved disease control and was well-tolerated compared to eculizumab, a standard C5 inhibitor. PiaSky is already approved in the US, Japan, and China based on these studies. PiaSky is being investigated in a broad clinical development program for several complement-mediated diseases, including atypical haemolytic uremic syndrome and sickle cell disease. #Roche #Pharmaceuticals #PNH #Biotechnology #HealthcareInnovation #MedicalResearch #ClinicalTrials #EMA #Healthcare #Biotech #MedicalAdvancements

We catch up with Shahid Hanif to learn what the GetReal Institute has achieved over the past year and what attendees can expect from the GetReal Institute 2024 Annual Conference. Read at The Evidence Base® #heor #healtheconomics #outcomesresearch #rwe #rwd #realworldevidence #realworlddata #marketaccess #pharma #biopharma #medicalaffairs #regulation #regulatoryaffairs #hta #healthtechnologyassessment #healthdata #healthequity #healthpolicy

Registration for the next PARASOL Scientific Workshop is now open! Mark your calendars for Monday, October 7th, from 12:00 PM to 6:00 PM, and Tuesday, October 8th, from 8:00 AM to 12:00 PM in Bethesda, Maryland. Can't make it in person? Join us virtually from anywhere in the world. https://lnkd.in/e_9q3QXU    The FDA and NephCure, International Society of Glomerular Disease, Kidney Health Initiative, and National Kidney Foundation have expressed a willingness to leverage their combined strengths for the joint development of the substantive actions of this workshop to facilitate the development of safe and effective treatments for focal segmental glomerulosclerosis (FSGS). The goal of this scientific workshop is to advance the understanding of proteinuria and eGFR-based endpoints as surrogate endpoints for accelerated and traditional approval of new treatments for FSGS. The workshop will be used to discuss the results of the aforementioned analyses and engage in a data-driven discussion with the larger community about the use of these endpoints as surrogate endpoints for the approval of treatments for FSGS.   The primary audience includes leading academic experts, interested pharmaceutical companies, regulatory agencies, patient representatives and advocacy groups, clinicians, researchers and individuals affiliated with medical professional societies, and other government agencies and members of the healthcare sector (e.g., payers).