The Tao of Excellence的动态

?? Attending the #PatientsAsPartners conference in London this week was a remarkable experience. The event brought together diverse stakeholders, primarily from the pharmaceutical industry and contract research organizations (#CROs), to discuss and showcase advancements in patient involvement within the drug development process. ?? Despite notable progress in patient engagement and involvement activities compared to previous years, there remains significant room for improvement. A particular area of concern is the inclusion of pediatric patients in the design and execution of clinical trials. Currently, these young patients are often not considered a standard part of the process, which is a gap that needs addressing. ?? ?? ?? Advocates for patient involvement emphasize the importance of integrating patient perspectives whenever they can add value, making it a mandatory part of clinical research to ensure it aligns with the needs and preferences of patients. While it is understood that involving patients directly in clinical studies may not always be feasible, caregivers—who possess extensive experience and knowledge from managing specific conditions around the clock—can provide crucial insights and should be included. For those looking to incorporate patient involvement in their projects, it is essential to recognize that there are always avenues and opportunities to do so. Engaging the right experts who can ensure that the involvement is grounded in solid scientific principles is crucial for delivering meaningful outcomes. Therefore, never hesitate to reach out to those with expertise in patient engagement to guide you in this process. At the European level, the network representing the expertise of professionals working in PPI in children's hospitals is eYPAGnet: www.eypagnet.eu If you need our advice or our services, we will be happy to serve! Joana Claverol Naomi Litchfield Michelle Gunn Patients As Partners in Clinical Research Community News #PatientInvolvement #PatientEngagement #ClinicalTrials #ClinicalResearch #ChildrensRights #DrugDevelopment #PatientEmpowerment #PatientEducation

??? To Attend an Upcoming Symposium on Advanced Therapy Medicinal Products (ATMPs) I am thrilled to be part of a special symposium that marks a significant milestone-15 years of groundbreaking advancements in Advanced Therapy Medicinal Products (ATMPs). This event will not only celebrate this remarkable journey but also highlight how the work of the Committee for Advanced Therapies (CAT) has been instrumental in shaping the regulatory framework in Europe. Their efforts have led to the approval of 27 innovative gene- and cell-based therapies, offering new hope for patients with unmet medical needs. This symposium will give a unique opportunity to gain valuable insights into the evolution of CAT, discuss regulatory developments, and explore upcoming innovations that could revolutionize patient care in the next 15 years. - Learn about the evolution of CAT and how it has adapted to meet the challenges of emerging ATMP technologies. - Discuss with experts the regulatory developments that continue to shape the future of ATMPs. - Explore the upcoming innovations that have the potential to revolutionize patient care in the next 15 years. I’m looking forward to engaging with Frontier to develop drugs and other industry leaders, scientists, and regulatory experts. These conversations are essential as we work together to ensure that high-quality, safe, and effective therapies continue transforming the healthcare landscape. I will share insights from the symposium with my network and continue building collaborations that will help advance these revolutionary treatments. I encourage you to do the same and look forward to staying connected as we collectively shape the future of medicine! #ATMPs #GeneTherapy #CellTherapy #HealthcareInnovation #ClinicalTrials #RegulatoryAffairs #CAT #EMA #MedicalInnovation #CNRResearch

Unveiling the Potential: The Attractiveness of R&D in the Netherlands Today I had the pleasure to participate in a panel discussion about "Research & Development: How Attractive is the Netherlands?" organized by Axon Healthcare and hosted by J&J Innovative Medicine Nederland. This meeting brought together over 180 participants from the healthcare and pharmaceutical sectors, both in-person and online, to delve into the opportunities and challenges within the Dutch R&D landscape. The event was kicked off by Michel van Agthoven, who emphasized the Netherlands' commitment to fostering an innovative ecosystem for drug development. Speakers such as Wiesje van der Flier and Jos GW Kosterink highlighted the Dutch academic excellence, the synergy between academic institutions and industry, underscoring the pivotal role of this collaboration in maintaining the Netherlands' position at the forefront of pharmaceutical innovation. Discussions centered on several compelling statements, facilitated by Hans Schikan of Health~Holland, including the enhancement of clinical development through better understanding of multinational study complexities and the critical nature of quality data delivery. The dialogue extended to potential risks, such as the impact of focusing solely on cost reduction which could lead to a decline in local R&D activities. Notable contributions came from Annemiek Verkamman and Benien Vingerhoed - van Aken, who discussed the strategic importance of clear regulatory frameworks but also partnerships between universities, hospitals, and the industry to secure the Netherlands as a leader in patient access to innovative treatments. The collective panel insights showed a robust infrastructure well-suited to the rapid advancements in medicine, making the Netherlands a prime location for international studies. Looking forward, it is clear that the Netherlands is set to not only continue but to enhance its role in the global R&D arena. With the Dutch Clinical Research Foundation soon to be launched National Actionplan Clinical Research we look forward to accelerating the next steps. Thank you to all the participants and speakers who made this event so full of insights and ideas. Look foreatd to continue to engage and expand the network to further the advancements in sustainable healthcare. Your thoughts and participation in upcoming discussions are highly valued as well so please engage! #clinicalresearch #healthcareinnovation #sustainablehealthcare #clinicaltrials

In a recent article by Cytel, the indispensable role of key opinion leaders in the realm of rare disease clinical trials is illuminated. Rare diseases present unique challenges in drug development, from limited patient populations to complex regulatory pathways. Here are some key takeaways: 1. Expert guidance: KOLs, with their deep expertise and insights, play a pivotal role in guiding clinical trial design and execution in the context of rare diseases. Their involvement ensures that trials are tailored to address the specific needs and challenges of these conditions. 2. Patient advocacy: Beyond their scientific acumen, KOLs often serve as advocates for patients with rare diseases. Their advocacy can influence trial protocols, ensure patient-centric approaches, and ultimately improve the chances of success in clinical development. 3. Navigating regulatory hurdles: Regulatory bodies have unique considerations for rare disease therapies. KOLs, well-versed in both scientific advancements and regulatory landscapes, can help navigate these complexities, accelerating the path to regulatory approval. 4. Collaborative networks: Establishing collaborative networks involving KOLs, researchers, industry stakeholders, and patient communities fosters a synergistic approach to tackling rare diseases. Such partnerships facilitate knowledge exchange, resource sharing, and innovative solutions. The collaboration between pharmaceutical companies and Key Opinion Leaders is paramount in advancing rare disease therapies. Their expertise, advocacy, and collaborative efforts are instrumental in overcoming the challenges inherent in rare disease clinical trials. Read the full insightful article here: https://bit.ly/44Y1jWi #RareDisease #ClinicalTrials #KeyOpinionLeaders #PatientAdvocacy

At Symbiance, we celebrated this special day by reflecting on over 30 years of dedication to advancing clinical research through our premier services in #StatisticalProgramming, #Biostatistics, #DataManagement, #Pharmacovigilance, and #RealWorldData and have supported numerous clients in achieving their research goals. To all pharmaceutical, biotechnology, and medical device organizations: if you are seeking a specialized partner with expert, highly skilled professionals offering cost-effective Biometrics and PV services, we invite you to connect with us. Discover how our comprehensive services and capabilities can benefit your projects. Our team holds extensive experience working with over 50 pharmaceutical companies globally, including many top 20 sponsors, and has supported various regulatory submissions. Let's discuss how our Biometrics services can elevate your clinical trials to the next level. #InternationalClinicalTrialDay #ClinicalTrials #HealthcareInnovation #Symbiance #CommitmentToCare #AdvancingMedicine #Biometrics #DataManagement #StatisticalProgramming #Biostatistics #CDISC #RWE #RealWorldEvidence #DatabaseProgramming #ClinicalProgramming #SDTM #ADaM #AnalysisDataset #TLF"

??♂? "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

At the Duke-Margolis Institute for Health Policy conference, the Director of CDER at FDA discussed the future of clinical trials, showcasing significant strides towards regulatory modernization and efficiency. The FDA's dedication to integrating innovative methodologies in drug evaluation and research was emphasized, with key takeaways including the integration of adaptive trial designs and digital health technologies to streamline drug approval processes. The establishment of a translational medicine team within the Office of New Drugs highlights the FDA's focus on accelerating therapy approvals, especially for rare diseases. Additionally, the FDA's modernization of inspection protocols and enhancement of cross-sector collaboration are among its strategies to align with the dynamic landscape of clinical trials. The groundbreaking approval for a drug targeting Friedreich's ataxia underlines the FDA's embrace of real-world evidence and adaptive regulatory approaches. Furthermore, future FDA strategies include enhancing collaborations with NIH and CMS to leverage real-world data for better drug monitoring post-approval. For professionals and stakeholders in the clinical trials and regulatory sectors, these developments signify a pivotal era of innovation and collaboration. Stay up-to-date with the latest in #ClinicalTrials, #FDA, #RegulatoryInnovation, #DrugApproval, #RealWorldEvidence, and #TranslationalMedicine by visiting the full article here.

?? Exciting News for Advanced Therapy Medicinal Products (ATMPs) Enthusiasts! On 30 December 2008, the Advanced Therapy Medicinal Products Regulation (EC No 1394/2007) came into effect, marking the beginning of a new era for ATMPs. Since then, these therapies have grown significantly, offering revolutionary treatments for unmet medical needs. To celebrate 15 years of progress, the European Medicines Agency (EMA) is hosting a symposium on 10 October 2024, at their headquarters in Amsterdam (also available remotely). ?? This event will highlight: How the Committee for Advanced Therapies (CAT) has shaped the regulatory framework for ATMPs in Europe. The approval of 27 groundbreaking gene- and cell/tissue-based medicines that are now offering transformative treatment options for conditions previously considered untreatable. These therapies address critical unmet medical needs, providing hope for patients with rare and serious diseases. ?? Who should attend? CAT members EMA committees & regulatory authority members Industry & academia representatives Healthcare professionals & patients Policymakers Anyone interested in the future of ATMPs ??? Important Dates: In-person registration deadline: 24 September 2024 Remote attendance registration deadline: 1 October 2024 Register your interest and join the conversation on the future of ATMPs! ?? #ATMP #RegulatoryAffairs #InnovativeTherapies #EMA #Healthcare #AdvancedTherapies

Pharma Clinical - Your Bridge to Israel In the ever-evolving landscape of clinical research, the collaborative efforts between CROs across different countries are instrumental in advancing scientific discoveries and improving patient care worldwide. At the heart of this cooperation lies the importance of selecting a CRO partner you can trust. Pharma Clinical is the leading CRO in Israel with more than 35 years of hands on experience in managing and supervising clinical trials across a wide variety of medical indications. In short, this is how we can help you: ? Our experience in dealing with all of the medical institutions in Israel saves you valuable time ? The abillity to speed up trial approvals saves you money (and time)? ? We got you covered A-Z expertise that will get you more accurate trial results getting you closer to your end goal The Israeli healthcare system proved its efficiency most during the pandemic, being the first country in which vaccines were tested - successfully. Conducting your next medical trial in Israel can make a real difference. Pharma Clinical - your bridge to Israel ??

?? 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