Technology Appraisal of Medical Devices at NICE

1
Technology Appraisal of Medical Devices at NICE
Methods and Practice

• Mark Sculpher
• Professor of Health Economics
• Centre for Health Economics
• University of York, UK

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Outline

• Policy context of NICE
• NICE methods
• Are devices different?
• The role of randomised trials
• When do we have sufficient evidence?
• Impact of NICE guidance

3
Background

• Brief overview of NICE
• NICEs decisions on devices
• NICEs methods requirement for decision making
• Are devices different?
• The impact of NICE guidance

4
The National Institute for Health and Clinical
Excellence (NICE)

• Following election of Labour government 1997
• Prolonged controversy about post code
  prescribing in the UK National Health Service
• Wish to de-politicize decisions about which
  technologies to cover in NHS
• Desire to use best available methods to address
  difficult questions
• Focus on drugs but devices also included

5
The NICE process
Selection
Assessment
Appraisal

• Specific technologies
• Lacking in transparency
• Subject to some criteria

• Independent group
• Review plus model
• Good methods
• supported
• Assess company
• submissions
• 6 months or more
• Companies can also
• provide unpublished
• data

• Multi-disciplinary
• committees
• Take information from
• range of sources
• Range of decisions
• possible

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NICE decisions overall
Source Raftery, BMJ 2006.
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NICE and medical devices (1)
Source Raftery, BMJ 2006.
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NICE and medical devices (2)
Source Raftery, BMJ 2006.
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NICE and methods

• 2004 guidance more prescriptive than most
  international guidelines
• Based on a clear view about the use of economic
  evaluation for decision making
• Included the concept of the Reference Case

National Institute for Clinical Excellence
(NICE). Guide to the Methods of Technology
Appraisal. London NICE, 2004.
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The requirements of economic evaluation for
NICE-decision making
Generic measures of health QALYs
Objective function
Decision problem
Clarity about population full specification of
options
Appropriate time horizon Evidence
base Context
Time over which options might differ
Inclusion of all relevant evidence
Relevant to specific decision maker(s)
Uncertainty
Quantify decision uncertainty feed in research
prioritisation
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NICEs preferred methodology the Reference Case
Source National Institute for Clinical
Excellence (NICE). Guide to the Methods of
Technology Appraisal. London NICE, 2004.
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What is the appropriate framework for economic
evaluation?

• Systematic review
• Meta-analysis
• Mixed treatment comparisons
• Differing endpoints and follow-up
• Patient-level and summary data

Evidence synthesis

• Structure reflecting disease
• Incorporation of evidence on range
• of parameters
• Facilitates extrapolation and
• separation of baseline and treatment
• effects
• Probabilistic methods

Decision analysis
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Are devices different? Decision problem

• Need to include all relevant alternatives to the
  technology of interest
• May include pharmaceuticals
• May include sequences and other strategies (e.g.
  diagnostic)
• Need to define relevant populations and
  sub-populations
• May differ between jurisdictions

No clear differences between devices and
pharmaceuticals
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Are devices different? Evidence base

• Less likely to need trials for regulatory
  purposes
• Does not mean should not be used for
  reimbursement
• Typical regulatory trials have limitations for
  economic evaluation
• The evolution of devices over time
• Not unique to devices
• Has implications for evidence gathering
• Need larger longitudinal studies, sub-groups on
  device types
• Comparators may also be changing over time

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Limitations of trials as a vehicle for decision
making
Trial limitations Inappropriate or partial
comparisons More than one trial Partial
measurement Unrepresentative practice Intermedia
te outcomes Limited follow-up No trials
NICE Examples Temozolomide (recurrent malignant
glioma) Drugs for Alzheimers Riluzole
(resource use) Glycoproteins Beta interferon
(MS) Implantable cardioverter defibrillators
Liquid-based cytology
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Distinguishing types of parameters in models
Cost-effectiveness models invariably draw
evidence from multiple sources
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Making trials more naturalistic The design
continuum
Comparators
Placebo controlled
All relevant comparators
Measurement
Few efficacy and safety endpoints
Resource use, QoL
Follow-up
Shortest acceptable for registration
Long-term follow-up
Patients
Tightly defined
Reflective of full range of likely patients
Practice
Highly protocolised
Reflective of routine practice
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When is there sufficient evidence to reimburse?
Implications of getting it wrong
Value of perfect information
X
Decision uncertainty

• What is the probability
• of the wrong decision?
• Joint effect of uncertainty
• in all inputs

What are the implications of a wrong decision in
terms of resources and health?

• Sets an upper bound on the
• value of further research
• Can be calculated overall
• and for individual parameters
• Calculated per patient and
• across a population of patients

Sufficient evidence exists if it is not
cost-effective to undertake further research
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Evidence on impact of NICE decision on the NHS
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Evidence on Orlistat for obesity
Source Sheldon et al. BMJ 2004329999.
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Evidence on ICDs for arrhythmias
Source Sheldon et al. BMJ 2004329999.
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What influences uptake?
Source Sheldon et al. BMJ 2004329999.
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Conclusions

• NICE part of an international trend towards
  greater use of economics in decision making
• NICE is prescriptive about methods
• Few differences between drugs and devices which
  affect appropriate methods
• Impact of NICE guidance has been variable