Industry Perspective

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Industry Perspective

• Clare McGrath
• Senior Director HTA Policy
• Pfizer Ltd.

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Topics

• Why do we need improved follow-up on the effects
  gained from medicinal products in a clinical
  setting?
• What follow-up and evaluations are being carried
  out today?
• How can this work be further developed/improved?
• What potential benefits can be derived from
  European collaboration in this area?

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Why we need improved follow-up

• Need to know whether evidence based decisions
  implemented and reasons if not.
• Reduce uncertainty in clinical results
• Reduce variation in utilization and outcomes
• Currently, only crude measures of use not linked
  to patient type or outcome
• Need to know what is appropriate use and
  implement policies to rectify under or over use
• Need to understand why outcomes not achieved and
  take corrective action of outcomes not achieved
• Need to know residual unmet need that can be
  fulfilled with new medicines

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Determinants of effectiveness

• Policy level
• Incentives to maximise outcomes and tools to
  achieve that goal
• System level
• Implementation of evidenced based decisions or
  guidelines
• Budget allocation
• Understanding and engagement with evidence
• Available skills and resource
• Provider level
• Knowledge of outcomes and corrective action
• Right patient, right treatment right reason,
  right time, right duration, right dose?
• Patient level
• Adherence

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Elements of effectiveness management
Data analyses
Data generation
Healthcare Patients
Academia Industry
Rational, safe and cost effective use of drugs
Regulatory assessment National reimbursement
decisions
Authorities
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Elements of effectiveness management
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Elements of effectiveness management
Observation MonitoringBarriers to
EffectivenessCorrective Action
Data analysesHypotheses research questions
Data generation
Healthcare Patients
Academia Industry
Rational, safe and cost effective use of drugs
Regulatory assessment National reimbursement
decisions
Authorities
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Elements of effectiveness management
Data analysesHypotheses research questions
Unmet need that new drugs can fulfil
Observation MonitoringBarriers to
EffectivenessCorrective Action
Data generation
Healthcare Patients
Academia Industry
Rational, safe and cost effective use of drugs
Regulatory assessment National reimbursement
decisions
Authorities
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What types of follow-up are currently being
carried out?

• Phase 111b, phase iv trials
• Head to head trials
• Observational studies
• Large simple trials
• Pharmacovigilance
• pharmaco-epidemiology
• Registries, claims data NHS data sets etc
• Risk sharing schemes. CED etc.
• Medical audit
• Utilisation monitoring etc.

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Example of effectiveness management tool Growth
Hormone Deficiency

• KIGS
• Largest Survey on GH therapy in Children
• Established for 20 years
• Safety and efficacy of GH therapy in 60,000
  children across 50 countries
• gt 100 aetiologies of growth failure represented
• Most patients assessed 3 monthly
• Individualised treatment using predictor models
• 45 publications ( many peer reviewed) since 1991

http//intranet.se.pfizer.com/or/ .
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Individualised treatment using predictor models
Growth chart in endoKIGS for a male patient with
GH deficiency. The upper graph shows a plot of
the patients height against the standard growth
curves. The lower graph shows the GH dose that
the patient is receiving.
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How can this work be further developed or
improved?

• Focus efforts to collect more data on areas where
  it will be of greatest value
• Understand what is being done already and whether
  it is useful
• Provide appropriate information at different
  levels in the effectiveness management system to
  drive specific decisions and actions
• Clarify accountability
• Provide incentives
• Facilitate cross country learning

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How can the work be further developed or improved?

• Develop EU wide principles on the purpose,
  governance and information needs for
  effectiveness management
• For chosen disease areas, define required
  information, collate information on what exists
  nationally. Identify gaps and facilitate country
  learning (build on EHR and EUnetHTA WP6)
• Engage providers, health ministries and industry
  in design of effectiveness management systems
• Develop EU wide governance system for access to
  public data building on existing best practises
• e.g. GPRD in UK, Sweden health records or
  PHARMO in the Netherlands
• Develop consensus on good data management and
  analysis methods. Facilitate sharing amongst data
  base owners

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Thanks you for your attention!