The Effective Statistician - in association with PSI

Keynote by Prof. Tim Friede | The Effective Statistician Conference 2024 What You’ll Learn: ✔ When and how to combine RCTs with real-world data (RWD) ✔ The CJD study: lessons from combining registry and trial data ✔ Hierarchical Bayesian meta-analysis and shrinkage estimators ✔ Robustness of these approaches in the face of heterogeneity ✔ Practical coding tips using the bayesmeta R package ✔ Design strategies for prospective data integration ✔ Regulatory perspectives on RWD-supported evidence Why You Should Listen: If you’re working in rare diseases, pediatrics, or situations where large-scale RCTs are not feasible, this episode offers practical tools and methodological clarity. Tim’s approach helps statisticians create more informative and reliable evidence from limited data—crucial for both research impact and regulatory engagement. Resources & Links: 🔗 Bayesmeta R package 🔗 The Effective Statistician Academy – I offer free and premium resources to help you become a more effective statistician. 🔗 Medical Data Leaders Community – Join my network of statisticians and data leaders to enhance your influencing skills. 🔗 My New Book: How to Be an Effective Statistician - Volume 1 – It’s packed with insights to help statisticians, data scientists, and quantitative professionals excel as leaders, collaborators, and change-makers in healthcare and medicine. 🔗 PSI (Statistical Community in Healthcare) – Access webinars, training, and networking opportunities. If you’re working on evidence generation plans or preparing for joint clinical advice, this episode is packed with insights you don’t want to miss. Join the Conversation:Did you find this episode helpful? Share it with your colleagues and let me know your thoughts! Connect with me on LinkedIn and be part of the discussion. Subscribe & Stay Updated:Never miss an episode! Subscribe to The Effective Statistician on your favorite podcast platform and continue growing your influence as a statistician.

Sebastian Schneeweiss, a Harvard Medical School professor specializing in medicine and epidemiology, shares insights on emulating randomized trials with real-world data. He discusses the motivation behind this approach, emphasizing its significance for regulatory-grade evidence. Topics include the challenges of data quality and adherence, use cases for real-world evidence in expanding treatment indications, and the innovative ‘Benchmark-Calibrate-Extrapolate’ strategy. This conversation is a must-listen for those in biostatistics, epidemiology, and healthcare innovation.

Discover the transformative power of genuine networking, driven by curiosity rather than small talk. Learn to set clear intentions for building meaningful connections at events. Uncover myths that may hold you back and develop strategies to overcome them. Get practical advice specifically for conference attendees, ensuring you network with confidence. Embrace the idea that authentic relationships with peers and mentors can unlock unexpected career opportunities and foster personal growth.

Emma Crawford, an autism advocate and leader in workplace inclusion, shares personal insights into navigating autism in professional settings. She emphasizes the critical need for inclusive work cultures and proactive leadership that seeks feedback. Topics include embracing neurodiversity, the role of technology in accessibility, and the importance of kindness in leadership. Emma calls for a shift in attitudes towards disability, encouraging workplaces to become environments where everyone feels empowered to express their needs.

Anna Forsythe, a Pharmacologist and healthcare economist, is the founder of Oncoscope, which automates systematic literature reviews to enhance cancer treatment decisions. In this conversation, she explores how daily updates in literature reviews can revolutionize oncology. Anna discusses the balance between innovation and clinical responsibility, and the introduction of AI in improving SLR accuracy. She shares insights on making cancer data accessible to patients and her commitment to altruistic ventures that prioritize patient care over profit.

Join Pavel Mozgunov, a group leader at the MRC Biostatistics Unit, and Julia Geronimi, a principal translational statistician at Servier, as they delve into innovative methodologies for biomarker analysis. They discuss how to effectively pre-specify analysis strategies, enhance predictive modeling, and tackle the challenges of small sample sizes. The conversation unpacks the complex distinctions between prognostic and predictive biomarkers while advocating for structured approaches to optimize clinical trial outcomes. Get ready for fresh insights into the future of biomarker-based trials!

Yannis Jemiai, Chief Scientific Officer at CITEL with 20 years in the field, and Flaminia Chiesa, a statistician with a focus on leadership and innovation, delve into the evolving role of statisticians. They discuss how AI and real-world data are transforming clinical trials and the necessary skills statisticians must develop to remain relevant. The conversation highlights the importance of interdisciplinary collaboration and offers insights on building confidence in leadership roles as data science becomes more strategic.

Katrin Kupas, a seasoned Director for Real-World Evidence with a decade of expertise in Health Technology Assessment, shares fascinating insights on integrating RWE with Joint Clinical Assessments. She emphasizes the importance of early evidence planning and when RCTs might fall short, making room for RWE. The conversation reveals the intricacies of indirect comparisons and the risks of non-randomized studies. Katrin also highlights the ROBINS-I tool for effective bias assessment, offering listeners actionable strategies to enhance their evidence generation plans.

Interview with Emma Crawford and Alex Spiers ✔ Why STEM outreach is so important for the future of our industry ✔ How your company, community, and even your own career can benefit from getting involved ✔ The challenges students face (especially girls and those from underserved areas) in seeing themselves in STEM ✔ How PSI’s Schools Outreach team can support you—with ready-made materials, training, and volunteer opportunities ✔ What it's really like to walk into a classroom and talk about your job Why You Should Listen: You don’t need to be a teacher to make an impact. If you’ve ever wished more people knew how rewarding our work is, this episode will show you how to be part of the solution. Whether you join a virtual session or speak at a local school, your story might be the spark that changes a student’s life. Get Involved – Here's How Whether you're based in the UK or elsewhere, there are so many ways to participate: Visit the PSI Schools Outreach HubIncludes career talk templates, outreach resources, and volunteering info Contact the PSI team: careers@psiweb.org Join platforms like: STEM Learning I'm a Scientist Maths4Girls If you're a PSI member, sign up for the Schools Outreach Newsletter in your Glue Up profile ("Outreach Champions Distribution List") You can also download editable school letter templates, contribute your own materials, or join our resource-sharing library to support other members doing outreach. Other Links: 🔗 The Effective Statistician Academy – I offer free and premium resources to help you become a more effective statistician. 🔗 Medical Data Leaders Community – Join my network of statisticians and data leaders to enhance your influencing skills. 🔗 My New Book: How to Be an Effective Statistician - Volume 1 – It’s packed with insights to help statisticians, data scientists, and quantitative professionals excel as leaders, collaborators, and change-makers in healthcare and medicine. 🔗 PSI (Statistical Community in Healthcare) – Access webinars, training, and networking opportunities. Join the Conversation:Did you find this episode helpful? Share it with your colleagues and let me know your thoughts! Connect with me on LinkedIn and be part of the discussion. Subscribe & Stay Updated:Never miss an episode! Subscribe to The Effective Statistician on your favorite podcast platform and continue growing your influence as a statistician.

Interview with Kaspar Rufibach What You’ll Learn in This Episode: ✔ The true definitions of interim, final, primary, confirmatory, and updated analyses ✔ Why a “final analysis” doesn’t always mean “last analysis” ✔ How language choice can impact stakeholder trust and regulatory interpretation ✔ The operational vs. statistical meaning of “stopping a study” ✔ When to use which data cuts and how to define your analysis set ✔ The nuances of estimation vs. hypothesis testing after stopping a trial ✔ Why clarity in communication is just as critical as technical accuracy Why You Should Listen: Even experienced statisticians often use terms like “final” or “interim” inconsistently, which can cause confusion not just within study teams, but also with regulators, clinicians, and the public. Kaspar provides much-needed clarity on how we as statisticians can: Use more precise terminology when describing study analyses, Align our language with stakeholders, Avoid miscommunication that can erode public and regulatory trust, and Educate others on the importance of estimating vs. testing. Resources & Links: 🔗 LinkedIn article by Kaspar Rufibach on clinical trial 🔗 Preprint Paper (with industry and regulatory co-authors) 🔗 The Effective Statistician Academy – I offer free and premium resources to help you become a more effective statistician. 🔗 Medical Data Leaders Community – Join my network of statisticians and data leaders to enhance your influencing skills. 🔗 My New Book: How to Be an Effective Statistician - Volume 1 – It’s packed with insights to help statisticians, data scientists, and quantitative professionals excel as leaders, collaborators, and change-makers in healthcare and medicine. 🔗 PSI (Statistical Community in Healthcare) – Access webinars, training, and networking opportunities. Join the Conversation:Did you find this episode helpful? Share it with your colleagues and let me know your thoughts! Connect with me on LinkedIn and be part of the discussion. Subscribe & Stay Updated:Never miss an episode! Subscribe to The Effective Statistician on your favorite podcast platform and continue growing your influence as a statistician.