Transcript NIHRRWDRickLonesFeb2012approved

The Vision For Real World Data – Harnessing
the Opportunities for the UK
Dr Rick Lones BM FFPM MRCGP
Executive Medical Director, UK and Ireland, Bristol-Myers Squibb
and
Co-Chair, ABPI Real World Data Campaign Team
13th February 2012
Objectives
What do we mean by Real World Data?
Where does RWD fit in the new NHS?
Importance of RWD to Industry
The UK context
Page 
“RCTs, long regarded at the ‘gold standard' of
evidence, have been put on an undeserved
pedestal. Their appearance at the top of ‘hierarchies’
of evidence is inappropriate; and hierarchies,
themselves, are illusory tools for assessing evidence.
They should be replaced by a diversity of approaches
that involve analysing the totality of the evidencebase.”
Prof. Sir Michael Rawlins
Harverian Oration to Royal College of Practitioners, 2008
ABPI strategic vision
Page 
ABPI committed to supporting and driving
the UK to be a centre of excellence in
RWD
RWD Campaign team
 White Paper Sept 2011
 Practical Guide May 2011
 Training and Master Classes
A useful definition
“Data collected outside the controlled
constraints of conventional randomized
clinical trials to evaluate what is
happening in normal clinical practice”
Complexity and co-morbidities
Real world data complements and augments RCT data
Post approval
‘market’ population
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Disease specificity
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RCT data vs. RW data
RCTs
Type of Trial
Real World Studies
Experimental / interventional Observational / noninterventional
Primary focus
Efficacy, safety and quality
Effectiveness
Patient population
Narrow and restricted
Wide and unrestricted
Monitoring
Intense (ICH-GCP compliant)
Not required
Comparators
Gold standard / placebo
None / standard clinical practice
Randomisation & Blinding
Yes
No
Cost
££££
£
What do you measure with RWD?
Page 
Outcomes
 Clinical
 Patient reported
Resource use
 NHS
 Patient
 Societal
Treatment pathways
Service models
Patient preference, experience, compliance
How do you get Real World Data?
Page 
Existing electronic health records
New datasets
 Prospectively
 Retrospectively
Collected using a variety of methodologies
• Non interventional research
• Audits
• Service evaluation
• Surveys
The UK regulatory framework
Page 
The UK NHS environment
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Decision makers at national and local level increasingly require
broader and more sophisticated evidence on which to base
informed choices
White Paper: Equity and Excellence: liberating the NHS (July
2010)
Focus on quality and outcomes and NOT targets
Patient perspective is increasingly important
Patient experience and satisfaction
PROMs
Introduction of Value–Based Pricing (2014) – need to be
underpinned by robust evidence of value
Why does the NHS need RWD?
Page 
Meet QIPP
agenda
Patient
expectations
and demand
Localised
Commissioning
UK data
Structural
redesign/
transformation
Deliver
more for
less
VBP
Quality
Outcomes
Cost
effectiveness
and affordability
Importance of RWD to the pharmaceutical industry
Gg
The UK share of
clinical trial activity is
declining…
…could a growth
in RWD research
fill the gap?
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Supporting Industry Objectives
Demonstrating Value
 Resource Use: Individual Episodes
of Care and Total NHS Resources
 Outcomes: Clinical outcomes,
PROs
 Service Delivery: Patient or Clinical
Satisfaction, Capacity, Patient
Journeys
Especially important if RCT data
collected outside the UK
Ggg
Ggg
Supporting Industry Objectives
Getting HTA Ready
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Ensure a robust economic and
budget impact argument
Avoids reliance on RCT data or
‘expert’ opinion
Option to collect in specific UK
countries (AWMSG, SMC)
Ggg
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Supporting Industry Objectives
Capturing Early Clinical Experience
 Suitable alternative to Phase IV
study (esp. high costs
diseases/orphan drugs)
 Local requirements for new
medicines
Evaluating Joint Working
 ABPI Framework advises that
outcomes should be measured
 Baselines need to be established
and tracked
Ggg
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Supporting Industry Objectives
Informing Internal Decision Making
 Clarity on market segments and
size of market
 Competitor products in practice
Documenting Product Safety
 May be regulatory requirement for
approval (RMP, PSUR, PAES)
 Support for labelling changes
Ggg
Ggg
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Alignment with Clinical Development Plan
Discovery
Phase 2
Pre-Clinical
Phase 3
Phase 1
LCM
RCTs focus on EFFICACY, SAFETY and QUALITY; they are SLOW and cost
£MILLIONS
RW Studies focus on EFFECTIVENESS, a more realistic picture of what can be
achieved in normal clinical practice; they are FASTER and typically cost
£THOUSANDS
Product Licensing: From “One-Off” ………
Level of
understanding
of benefit-risk
Withdrawal,
Backlash
Warning,
Dear Dr
MA
Pharmacovigilance
Drug
Development
Phase
Time
Product Licensing: ……….to “Live Licence”?
Level of
understanding
of benefit-risk
Amended
MA/SmpC
Amended
MA/SmpC
Outcomes Research
Earlier
conditional MA
MA
Pharmacovigilance,
Post Marketing
Studies
Drug
Development
Phase
Time
The UK as a Global Player in RWD
The UK has a significant and disproportionate
influence on other markets
 Impact of NICE globally
Unique “cradle to grave” healthcare system
 Wealth of electronic databases
developed over last 20 years
Streamlining of regulatory & governance
frameworks ongoing
Strong links with academia
The Future of RWD in the UK
The UK could become a World Class Leader in RWD, but we must:
 Strengthen the case for the UK to develop its capabilities
 Encourage initiatives to overcome existing challenges
The Introduction of Value Based Pricing offers a
significant opportunity for industry