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2. Biopharmaceutical Section Best Contributed Presentation Award – 2019 JSM
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6. Joint Statistical Meetings Denver Panel
Patient-Focused Clinical Trials: Challenges and Considerations for Trial Design, Endpoints, and Analysis A practical guide from a sponsor’s perspective
Stephanie Manson
Novartis
Joint Statistical Meetings Denver Panel
Monday August 1, 8:30-10:20am

7. Disclaimer
The opinions expressed in this presentation and on the following slides are solely those of the presenter and not necessarily those of  the Novartis Group companies (“Novartis”). Novartis does not guarantee the accuracy or reliability of the information provided herein.

8. Patients as trial partners
We have adopted a deeply held belief that the patient is an important part of trial design but the biggest barrier is now practical. Trial teams often move so quickly and have relatively little experience engaging patients that logistics can be a real issue
The Ideal
The Reality

9. Which patient to engage?
Design Implement Interpret

10. Patient Engagement Logistics – make it automatic
Plan contracts and recruitment in advance
Don’t let ‘great’ get in the way of ‘good’
Systematize in protocol review

11. Thoughtful PRO Endpoints
Which domains have largest response? Which are important to patients?
Where
Is change expected across all/responders/specific responders?
Who
What is a clinically relevant change/MID?
What
When is the best time to measure to maximize the expected response?
When
What is the relationship between the drug’s mechanism of action and PRO endpoints?
Why

12. We will look at HRQoL differences between the two arms
Endpoint Examples
We will look at HRQoL differences between the two arms
We will test change in pain from baseline to 6 months in patients with pain at baseline, with clinically relevant pain reduction defined as a change of X points in question Y.
Earlier vs later phase trial – how do endpoints differ?

13. Linking design, analysis, interpretation
Create hierarchy of specific PRO endpoints
Analysis plan written specifically around endpoints
Specify contingent descriptive analyses
Useful tool when no a priori data supports hypothesis
E.g. time to deterioration analyses with and without confirmation, analyzing related symptoms
Analysis + Interpretation
Review key PRO results with investigators and patients

14. Example: PRO-CTCAE
Sample Items
Clear signal from FDA about desire to use instrument to measure AE impact on cancer patients
Finding the right trial: Differences in AE profile
Build hypothesis supported by questions

15. Example: Computer Adaptive Tests (CATs)
Items selected from validated item banks (e.g. PROMIS, EORTC)
Based on initial responses, future questions are tailored to specific symptoms experienced by patients
PHYSICAL FUNCTION
30min walk
House work
NAUSEA
PAIN
How often
Impact on Activities
Improved by NSAIDs?
DEPRESSION
In this simplified example, the symptoms we are most concerned about are pain, nausea, physical function and depression. We can set up the CAT so that only if the patient answers yes to these symptoms are they asked about the details of what they have experienced. Benefits of CAT including more relevant questions which waste less of the patient’s time, questions are more precise and can cover a wide range of symptoms and functions.

16. ‘By failing to prepare, you are preparing to fail’ - Benjamin Franklin