The Effective Statistician - in association with PSI

RWE and JCA - how do they go together

Apr 28, 2025

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.

20:21

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Episode guests

Katrin Kupas

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Quick takeaways

Real-world evidence (RWE) is essential for improving treatment comparisons in healthcare, particularly for Joint Clinical Advice (JCA) when RCT data is lacking.

Addressing potential biases in non-randomized studies requires early planning and integrated evidence strategies to ensure reliable outcomes during drug development.

Deep dives

The Importance of Real-World Evidence in HTA and JCA

Real-world evidence (RWE) plays a crucial role in the healthcare landscape, especially when randomized controlled trial (RCT) data is unavailable. This is particularly significant for the Joint Clinical Advice (JCA) process, where new drugs need effective comparators against existing treatments. RWE can provide valuable insights for these comparators, helping to avoid biases that arise from using outdated RCT data, ensuring that healthcare providers can make informed decisions based on the most current population and treatment standards. The podcast emphasizes the importance of structured workflows that facilitate data collection and analysis to appropriately answer the necessary questions presented in the JCA context, allowing for a comprehensive and evidence-based approach to treatment evaluation.

Intro

2min

Integrating Real-World Evidence in Health Technology Assessment

5min

Navigating Indirect Comparisons in Clinical Research

8min

Assessing Bias with the Robin's Eye Tool

2min

Integrating Real-World Evidence in Clinical Assessments

4min

Interview with Katrin Kupas

What You’ll Learn in This Episode:

✔ What the JCA is and why it changes how we plan evidence generation

✔ When and how RWE can help answer comparator questions in HTA

✔ The risks of non-randomized comparisons—and how to mitigate them

✔ Why we need an integrated evidence plan early in development

✔ How tools like ROBINS-I and quantitative bias analysis can improve credibility

Why You Should Listen:

If you’re a statistician, medical affairs lead, or part of an HEOR or market access team, this episode will help you:

✔ Develop a stronger evidence generation plan across the drug lifecycle

✔ Understand how real world evidence can support JCA submissions

✔ Learn when RCTs aren’t enough—and how RWE can fill the gap

✔ Gain practical advice for designing indirect treatment comparisons

✔ Improve your bias assessment strategies with tools like ROBINS-I

Resources & Links:

🔗 ROBINS-I Tool – for evaluating bias in non-randomized studies

🔗 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.

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