2. RBM: Getting Beyond the Promise and Getting To Value
SCOPE Conference, February 2019
Gary Thompson, VP Strategic Consulting, Medidata Solutions

3. RBM: Lots of Promise… Where’s the Value?
May 2013: Current model for RBM laid out in TransCelerate whitepaper
Nov 2016: Reinforced and formalized in regulations with release of ICH E6:R2
July 2018: 32% of industry respondents have yet to develop an approach to KRIs*
Nov 2018: FDA concerns with slow industry adoption of risk-based approaches and realizing speed and cost benefits**
* “Risk-based Monitoring: Widespread Implementation is Underway, but Still Messy”, CenterWatch Weekly, July 9, 2018
** “Harnessing Real World Evidence for Safety and Innovation”, Scott Gottlieb, M.D., Commissioner of Food and Drugs, Remarks to Reagan-Udall Foundation, November 19, 2018

4. Why Haven’t Companies Seen Enough Value to Pursue RBM More Aggressively?
Companies miss value in RBM for one of three reasons
Confusion about what RBM is and how they should be implementing it
Uncertainty over what their RBM value proposition is
Challenges
with the discipline, time and resources required to implement and execute RBM at scale

5. Clarifying RBM: What It Is
Iteration
Risk Assessment
KRIs
QTLs
Ongoing Monitoring
Quality Report
Statistical/Analytical
Approaches
Planning
Execution
Outcomes
Remote Monitoring
Where
Central Monitoring
How
Risk-based Monitoring
Why
ICH E6:R2
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Strategic
KRIs
Risk Assessment
AI/ML
Data Point-Level
Quality/Integrity
Tactical
Data-Point -Level Quality/Integrity
Traditional Approaches
Analytics-Powered Approaches

6. Clarifying RBM: How It Works
Trial-Level Quality/Integrity
Trial-Level
Quality/Integrity
Strategic
View
Risk Assessment
KRIs
AI/ML
Data Point-Level
Quality/Integrity
Tactical
View
Data Point-Level Quality/Integrity
Risk-Based and
Analytics-Powered Approaches
Traditional Approaches
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Strategic
Trial-Level Quality/Integrity
Trial-Level Quality/Integrity
Strategic
Trial-Level Quality/Integrity
Strategic
Trial-Level Quality/Integrity
Strategic
Strategic
KRIs
Risk Assessment
Strategic
KRIs
Risk Assessment
KRIs
Risk Assessment
KRIs
Risk Assessment
KRIs
Risk Assessment
KRIs
Risk Assessment
AI/ML
AI/ML
AI/ML
AI/ML
AI/ML
AI/ML
Data Point-Level
Quality/Integrity
Data Point-Level
Quality/Integrity
Data Point-Level
Quality/Integrity
Tactical
Data Point-Level
Quality/Integrity
Tactical
Data Point-Level
Quality/Integrity
Tactical
Data-Point -Level Quality/Integrity
Data Point-Level
Quality/Integrity
Tactical
Data-Point -Level Quality/Integrity
Tactical
Data-Point -Level Quality/Integrity
Tactical
Data-Point -Level Quality/Integrity
Data-Point -Level Quality/Integrity
Data-Point -Level Quality/Integrity
Traditional Approaches
Analytics-Powered Approaches
Traditional Approaches
Analytics-Powered Approaches
Traditional Approaches
Analytics-Powered Approaches
Traditional Approaches
Analytics-Powered Approaches
Traditional Approaches
Analytics-Powered Approaches
Traditional Approaches
Analytics-Powered Approaches

7. How RBM Generates Value
“Perfect”
Wasted
Effort
Target (KRI)
QTL
Valuable
Effort
Quality
(eg., protocol compliance,
data quality, etc)
Cost ∞
(eg., financial, human resources, time, delays to patients)

8. How RBM Generates Value
Cost
Quality
(eg., protocol compliance,
data quality, etc)
(eg., financial, human resources, time, delays to patients) ∞
“Perfect”
Target
QTL
Wasted
Effort
Valuable
Company A (overinvestment in quality activities)
Consistently meeting safety, quality, and compliance demands, but with excessive burden and time
Opportunity to decrease costs while maintaining high quality
Investment exceeds risk = Wasted efforts
Risk exceeds investment = Quality gap
Optimal risk-based quality investment
Company B (underinvestment in quality activities)
Concerned about / unsure of consistent quality and compliance delivery, but challenged to make sharply higher investments
Opportunity to increase quality with minimum, targeted additional investment
Relative Risk
(Criticality
and Frequency)
Various Clinical Trial Processes / Data

9. Statistical/Analytical
A Complete RBM Capability is Worth it… and also Complex, Expensive, Demanding
Entry Point – Targeted SDV:
Reduce site monitoring costs
Iteration
Targeted SDV
Issues Management
Integrated Workflow
Risk Assessment
KRIs
QTLs
Ongoing Monitoring
Quality Report
Statistical/Analytical
Approaches
RACT
Industry: TBD
Central Monitoring
AI / ML
Planning
Execution
Outcomes
Entry Point - Risk Planning and Issues Management: Improve Control of the Quality Process
Entry Point – Central Monitoring:
Augment quality assurance through analytics
ICH E6:R2
Solutions

10. RBM Value: Achievable with the Right Approach
Getting to RBM Value requires clear, smart decisions
Get clear
on the components of RBM and what’s needed for your goals
Be smart
about which of the RBM value propositions is right for your company
Be decisive
on your best path: All-in or start with a specific entry point

11. Thank you.