This document discusses the challenges and opportunities of real-world evidence (RWE) in the pharmaceutical industry. It notes that while RWE from real-world data can help improve drug development, operations, and decision-making, there are also challenges to overcome. These include issues with data accessibility, analysis capacity, and balancing limited resources. However, RWE also presents opportunities to increase value, understand effectiveness beyond clinical trials, and gain business insights. Overall, RWE brings significant opportunities if pharmaceutical companies can address solvable challenges through leadership, governance, and transformation.

1. Challenges
&
Opportunities
Real World Evidence in Pharmaceutical Industry
April-2-18
Peter (Quynh) Le

2. April-2-18
DISCLOSURE: NONE

3. OUTLINE
April-2-18
• Introduction
• Challenges
• Opportunities
• Conclusion
• Q&A

4. April-2-18
INTRODUCTION
 Real-World Data (RWD)
 Patient health & healthcare data from a variety of
sources other than RCTs
 Vs – Volume, Velocity, Value, Variety, & Veracity
 Real-World Evidence (RWE)
 Evidence regarding the use, benefits, or risks of a
drug derived from RWD analysis
 Aggregation & analysis of RWD elements
 Pharmaceutical industry
 $18 billion Sales (Source: trade.gov, 2016)
 3.2% Annual growth (Source: IBISWorld, 2017)
 20,309 Employees
 366 Businesses
 Challenges & Opportunities
 R&D
 Manufacturing
 Commercial Operation & Services
 Overall & Specific

5. April-2-18
CHALLENGES - overall
 Environment
o Regulatory & public perceptions
o Health care providers & payers’ acceptance
o Internal preferences/cultural barriers
 Accessibility
o Where and What quality of RWD
o Privacy
 Capacity
o Analysis & interpretation of RWD
o Opinion leaders vs. RWD analytics
 Balance
o Limited resources & Unlimited expectations

6. April-2-18
CHALLENGES - specific
 Data
o Internal vs. External data
o Big in Vs
o Security & governance
o Integration & preparation
o Data exploration
 Organizational
o Talent scarcity
o Transdisciplinary engagement
o Standards & knowledge are behind desire regarding RWE
o Dependency - not only industry but also regulators & 3Ps

7. April-2-18
CHALLENGES - specific
 Modelling complexity
o Justification of Machine learning, Artificial intelligence
o Model interpretability
 Operational & production
o Operational efficiencies through RWE
 Coordination, job, & manufacturing
o AI/ML Model development
 Personal laptop vs. Cloud server
o Model & decision management
 Monitor & manage ML models
 Model predictions + business rules = decisions
o Transform toward outcome-based RWE model
o RWE model itself

8. April-2-18
OPPORTUNITIES - overall
 Improvement
o Development & Standardization
 Systems, data, designs, & analytical methods
 Hypotheses for continued clinical development
o Operation & Services
 Product’s benefit-risk profile, post-market safety
 Approval, pricing, & reimbursement
 Value
o Business critical insights
o ROI – increase value and decrease price
o Gift of abundance/Corporate Social Responsibility
 Expectation
o Quality/Efficacy/Safety vs.
Outcomes/Effectiveness/Economics
o Better treatment earlier
o Better way to analyze dirty/real-word/complex/big data
$

9. April-2-18
OPPORTUNITIES - specific
 Operation
o Jobs – capitalist’s dream vs. workers’ nightmare
o Boom vs. Stall – necessary (RCT) vs. sufficient (RCT/RWE)
o Marketing & Sales
 Physician-Reps relationship
 Preferred prescriptions, Sentiment analysis
o Predict
 Patient behaviour/Patient segmentation/Targeted therapy
 Disease progression, cost
 Decision
o Value in Vs – rapid from RWD/RWE to business decisions
o Rapid accurate individual/patient level predictions
 Treatment response, progression regarding a drug
 Monetization (new oil)
o Control over data generating sources
o Gap in use of RWD/RWE for R&D
$
%

10. April-2-18
OPPORTUNITIES - specific
 RCT pitfalls
 RCT
o Cost, time, representativeness, & long term safety
 RWD/RWE
o Effectiveness with real world patient treatment environment
o Comparative effectiveness & payer's willingness to pay
 Combined RCT/RWE for R&D
o Continuous stream of truly differentiated products
o Empirical business insights:
• How products are & aren't being used?
• Customers’ frustration points, & areas of improvement
 Promising environment
 Strong interested in better treatments earlier (Patients)
 Aspiration regarding cost-effective care (Payers)
 Striving for innovative (Academic/healthcare)

11. April-2-18
CONCLUSION
• RWE brings huge opportunities & solvable
challenges to the pharmaceutical industry
to enhance its competencies, inform
decisions better, & optimize operations
…Governance considerations &
…Leadership determination …
…Transformation …
(Adapted: Strategy & Survey)

12. Thank you!
Q & A
April-2-18
Peter Le