Stefan Herdinius Increasing patient involvment Patient involvement prior to regulatory Scientific Advice

1. Patient involvement
prior to regulatory
Scientific Advice
16 November 2015
EUPATI Webinar
Stefan Herdinius
UCB Biopharma

2. The Scientific Advice process
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High-level description for clinical development

3. Case - Few existing treatment options
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Background
Autoimmune disease
Other concomitant autoimmune diseases very common
Currently symptomatic treatment - no systemic treatment available
Advisory board jointly with clinical experts and patients prior to regulatory
scientific advice focused on new potential systemic treatment to address disease
immunologically

4. Case - Few existing treatment options
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Advisory board with patients and physicians
Questions to patients to inform scientific advice request:
How to address the prevalence of concomitant diseases and the need in the larger patient
population?
What is most important to treat first?
■ The primary autoimmune disease or other concomitant diseases?
Key outcomes of advisory board for supporting scientific advice:
Polypharmacy (the use of four or more medications) due to concomitant diseases
Typical development is in patients without concomitant diseases
Key input to support scientific advice request from regulators
Providing additional data and information for regulators
Better basis for advice

5. Conclusions
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Patient involvement prior to scientific advice
Patient input before initiation of clinical development and scientific advice
requests is of great importance
Makes better plans and informs the scientific advice process before it starts
Gives context to reality of disease and how we can address patients needs while meeting regulatory
requirements
This is one example of many, where patients can collaborate in the medicines
development process

6. Thanks!
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