The Effective Statistician - in association with PSI

The Effective Statistician - in association with PSI

The Effective Statistician - in association with PSI

Combining RCT and RWD – applications in rare diseases and practical recommendations

Download it: MP3 | AAC | OGG | OPUS

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.

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About this podcast

The podcast from statisticians for statisticians to have a bigger impact at work. This podcast is set up in association with PSI - Promoting Statistical Insight. This podcast helps you to grow your leadership skills, learn about ongoing discussions in the scientific community, build you knowledge about the health sector and be more efficient at work. This podcast helps statisticians at all levels with and without management experience. It is targeted towards the health, but lots of topics will be important for the wider data scientists community.

by Alexander Schacht and Benjamin Piske, biometricians, statisticians and leaders in the pharma industry

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