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1. Clinical Data Management and Clinical Data Science
A Practical Approach
Urosh Vilimanovich, PhD
Director Clinical Data

2. Main Tools For Clinical Trial Management
2

3. Clinical Data Science and
Biostatistical Services
3
∞ Clinical Data Management
∞ Pharmacovigilance
∞ Central Monitoring
∞ SDTM and ADaM datasets
and define.xml
∞ Biostatistics
∞ Statistical Report Writing
Conduct Finalize
Plan
∞ Protocol Development
∞ CRF Development
∞ Clinical Advisory and Scoping

4. • Responsible for managing the life cycle of clinical data from collection to their delivery for
statistical analysis in support of regulatory activities.
• Primarily focuses on dataflows and data integrity (i.e., data is managed the right way).
Clinical Data Management
4
Clinical Data Science
• Evolution of Clinical Data Management.
• Encompasses processes, domain expertise, technologies, data analytics and Good Clinical
Data Management Practices essential to prompt decision making throughout the life cycle of
Clinical Research.
• The strategic discipline enabling the execution of complex protocol designs in a patient
centric, data driven and risk-based approach ensuring subject protection as well as the
reliability and credibility of trial results.

5. 5
Trial
Workflow
Clinical Research Informatics
Access
Access
Set-Up
Scale
Improve
Enroll
Collect
Monitor
Close
Analyze
Plan & Design
Decomission
1
2
3
4
Implementation
Conduct
Closure
Lesson
s
Learne
d

6. Plan and Design – Study Protocol
6
Primary Endpoint:
The average change in CAT Score in participants administered BOTOX compared to placebo at Day 28 from Day 1 (baseline).
Primary Endpoint:
The change in CAT Score will be summarized using mean, standard deviation, standard error, 95% confidence interval, minimum, 25th
percentile, median, 75th percentile and maximum. The change in participants’ CAT Scores will be calculated as the CAT Score on Day
28 minus the CAT Score at Day 1 (Baseline). Distribution and average CAT Scores, and change thereof, will be presented graphically
using boxplots and/or error bar diagrams. The significant difference between the mean or median change of CAT Score will be
assessed using a two-sided independent samples t test or Mann-Whitney U test, depending on data distribution. The level of
significance for this analysis will be set to 0.05.
Sample size calculation is based on the primary endpoint using the following assumption:
• CAT score reduction from D1 to D28 is at least 2 units more in BOTOX arm compared to placebo
• standard deviation (SD) of the difference is 2 units
• 80% statistical power
• significance level α=0.05, two-sided testing
A sample size of 17 per arm (34 in total) is sufficient to achieve 80% power to reject the null hypothesis that the mean difference of a
change in CAT Score of 2 ± 2 units using a significance level (alpha) 0.05 using a two-sided independent samples t test. Considering a
15% dropout rate, the final sample size is 20 participants per study arm (40 participants in total).

7. Plan and Design – Study Protocol
7
Screening Baseline V2 V3 V4 V5 V6 V7 V8 V9 V10 V11 V12 V13 V14 V15 EOT
D-14 to 0 D1 D3 D5 D7 D10 D14 D21 D28 D35 D42 D49 D56 D63 D70 D77 D90
+/- 1d +/- 1d +/- 1d +/- 1d +/- 1d +/- 1d +/- 3d +/- 3d +/- 3d +/- 3d +/- 3d +/- 3d +/- 3d +/- 3d
Informed Consent X
Demographics &
Medical History
X
Inclusion/Exclusion
Criteria Evaluation
X X
Physical Exam X X X X X X X X X
Vital Signs X X X X X X X X X
Height and Weight X
mMRC Dyspnea
Scale
X
Pregnancy Test X
Hematology/Serum
Chemistry
X X
Oxygen Saturation X X X X X X X X X X X X X X X X X
Pulmonary Function
Test (Spirometry)
X X X X X X X X X X
Randomization X
COPD Assessment
Test (CAT)
X X X X X X X X
6MWT X X X X X X X
Randomization X
Administer
Treatment
X
Concomitant
Medication
X X X X X X X X X X X X X X X X X
Adverse Events X X X X X X X X X X X X X X X X X

8. Plan and Design – EDC Documents
8

9. Plan and Design – EDC Documents
9

10. Collect – EDC Documents
10

11. Monitor – Real Time Reporting
11

12. Monitor – Real Time Reporting
12

13. Monitor – Real Time Reporting
13

14. 14
Monitor - Real Time Reporting – Site Performance

15. 15
Monitor - Real Time Reporting – Site Performance

16. Finalize – Lock, Analyze, and Report
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Database Lock SDV  Medical Review  Coding  DM Review  PI Sign Off  DB
Lock
Data Export  SDTM Dataset  ADaM Dataset  Define.XMLs
SAS / R Programing  Primary and Secondary Endpoints  Safety
SAR Writing  Clinical Study Report  Regulatory Submission
Statistical Analysis
Report
Statistical Analysis
Dataset
Preparation

17. Thank you.
Wemedoo AG
Sumpfstrasse 24
6312 Steinhausen
Switzerland
info@wemedoo.com
+41 41 740 01 24
www.wemedoo.com