EUGM 2011 | JEMIAI | An Early Look at East 6 on the Architect Platform

1. Click to edit Master /tle style
10/26/11
An early look at
Confirm Success with East®
Clinical Trial Design Software System
on the Architect™ platform
6
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2. • East today
• Software vision
• Example in hypertension
• East® demo
• Design questions
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agenda
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47 of the Top 50 biopharmaceu4cal companies use Cytel so:ware!
+ all divisions of the FDA
an industry standard
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what East has changed
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Software vision
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7. • Can I create designs in batches?
• How should I choose the timing of the interim analysis?
• How would I specify a first interim analysis strictly for futility
followed by a second interim analysis for both efficacy and
futility?
• Can I customize the information I would like to use in a
report?
• Can I specify my futility boundary in terms of conditional
power or effect size?
• What are the true savings in sample size if you consider
accrual patterns and overruns?
• Given observed data at an interim, can I compute the
probability of success or simulate the rest of the trial?
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Design questions
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8. • Phase 3 clinical trial testing superiority of an angiotensin II
receptor blocker vs. placebo
• Primary endpoint: change from baseline to week 6 in the
24-hour mean systolic blood pressure by ambulatory blood
pressure monitoring
• Test the difference of two independent means using a 1-
sided a = 0.025
• Would like the trial to have 90% power to detect an effect
size of δ = -1.5 mm Hg when compared to placebo in a 1:1
allocation
• Can assume a common standard deviation of σ = 6 mm Hg
• Accrual rate of 40 patients per month
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Example in hypertension
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9. East® Demo
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10. • Ease of use
• Reliability
• Productivity
• Communication
• Advanced functionality using R
• Breadth of designs available
• Powerful simulation capabilities
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benefits of East
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11. Click to edit Master /tle style
Peace of Mind with ACES®
Controlled and Secured Trial Execution System
Business Confidential26 October 2011 11

12. • Are interim decision rules pre-specified?
• Are they carried out as pre-specified?
• Who has access to them?
• Who prepares the interim report?
• Who has access to the interim report and how is
confidentiality maintained?
• Can interim results be reverse engineered from
actions taken?
logistical and operational biases
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no problem

14. “A well-trusted firewall established for trial
conduct beyond those established for
conventional group sequential trials can help
provide assurance that statistical and
operational biases have not been introduced.”
FDA Guidance on Adaptive Design (2010)
what is required?
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15. • Web based technology to control flow of
information and access to confidential
documents
• Prevents operational bias by including the actual
adaptive algorithm only in restricted appendix to
Data Monitoring Committee (DMC) charter and
tracking access to this document
• Establishes trustworthiness through secure
password protected access to documents,
execution of algorithms, and audit trail
ACES: Access Control Execution System
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16. Create
Documents
(Protocol,
SAP, DMC
Charter)
Store/Archive
Documents
Enroll
Subjects &
Collect
Responses
Send
Response
Data to ISC
Perform
Analysis and
Create
Reports
Send
Analysis to
DMC
SponsorISCDMC
Make
Recommendati
on
Send
Recommendati
on to Sponsor/
Steering
Committee
Make
Decision
about Trial
Steering
Committee
After decision…
1. DMC notified
2. Drug Supply
notified
3. IVRS notified
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traditional process
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17. • Inconsistency
• Insecurity
• Process Enforcement
• Firewall Enforcement
• Lack of Audit Trails
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Concerns with Traditional Interim Analysis
Processes
why do we need ACES?

18. 10/26/11 Europe East UGM2011
Create
Documents
(Protocol,
SAP, DMC
Charter)
Store/Archive
Documents
Enroll
Subjects &
Collect
Responses
Send
Response
Data to ISC
Perform
Analysis and
Create
Reports
Send
Analysis to
DMC
SponsorISCDMC
Make
Recommendati
on
Send
Recommendati
on to Sponsor/
Steering
Committee
Make
Decision
about Trial
Steering
Committee
in
ACES
in
ACES
in
ACES
After decision…
1. DMC notified
2. Drug Supply
notified
3. IVRS notified
in
ACES
ACES process
18

19. 10/26/11 Europe East UGM2011 19
• Automate Analyses
• Improve Efficiency
• Reduce Bias
• SAS Integration
• R Integration (planned)
Workflow
Automation
• Centralized Access
• User Security
• Audited Access
• Categorization
Document
Storage
• Protect Data and
Information
• Maintain Blind
• Protect Study Integrity
• Reduce Bias
Secure
Firewalls
• 21 CFR Part 11
Compliant
• System and Study
Level Reporting
• All Access Recorded
• Guarantees “Who Saw
What and When”
Auditing
• Make Better Decisions
• Make Earlier Decisions
• SAS Integration
• R Integration (planned)
• Customizable
Reporting
• Supports All Major
Browsers
• SaaS Hosted Solution
• 128-bit SSL Encryption
Global
Access
how can ACES help?

20. Thank you
Questions about East®?
Call +1.617.661.2011
http://www.cytel.com/software/east.aspx
10/26/11
Q&A
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