Making Difficult Decisions

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Making Difficult Decisions

• Michael Rawlins
• Chairman, National Institute for Health and
  Clinical Excellence, London
• Emeritus Professor, University of Newcastle upon
  Tyne

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NHS Act (1977)
UK parliament NHS vote
Secretary of State for Health
Primary Care Trusts
Other bodies (eg HPA)
NICE
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NICEs History

• 1999 Technology appraisals
• Clinical guidelines
• 2002 Interventional procedures
• 2005 Public health
• 2009 Expansion
• Appraisals
• Clinical guidelines
• New programmes
• Quality standards and metrics
• NHS Evidence

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NICEs Purpose

• To provide health professionals with advice on
• securing the highest attainable standards of
  clinical care for National Health Service
  patients
• promoting and sustaining the public health

Effectiveness
Cost effectiveness
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Healthcare expendituretotal per capita OECD
member states
Total per capita healthcare expenditure (US)
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Healthcare expenditure and GDPOECD member states
USA
R 0.855 R2 0.733
Luxembourg
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Published NICE guidance
Type Numbers
Appraisals 170
Clinical guidelines 101
Interventional procedures 294
Public health 19
Total 584
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Application of NICE Guidance
Type England Wales Scotland N. Ireland
Technology appraisals
Interventional procedures
Clinical guidelines
Public health
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Technology appraisals

• Health technologies encompass
• Pharmaceuticals
• Devices
• Surgical (and other) procedures
• Diagnostic methods
• Health promotion techniques

Clinical effectiveness
Cost effectiveness
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Appraisal processmultiple technology appraisals
Independent assessment report
Ministerial referral
Consultees
Appraisal Committee ACD
Appraisal Committee FAD
Consultees
or
Publish
Appeal to NICEs Board
Consultees
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Appraisal processsingle technology appraisals
Expert Review Group
Manufacturers assessment report
Ministerial referral
Consultees
Appraisal Committee ACD
Appraisal Committee FAD
Consultees
or
Publish
Appeal to NICEs Board
Consultees
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Appraisal processappeals

• Grounds
• Failure to apply a fair process
• Perversity of the Appraisal Committee
• The final advice exceeds NICEs legal powers
• Appeal panel
• Two board members
• One NHS representative
• One person with relevant industry experience
• One lay member

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Principles of NICE guidance

1. Robust
2. Inclusive
3. Transparent
4. Independent
5. Legal powers

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Robust

• Clinical/scientific/technical considerations
• Systematic review of the published (and
  unpublished) literature
• 2. Critical examination of the evidence base
• Avoiding the use of hierarchies
• Qualitative as well as quantitative evidence
• 3. Accepting the need for judgements
• Scientific
• Social

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Inclusive

• Consultees comprise all relevant
• manufacturers
• professional organisations (national)
• patient organisations (national)
• two volunteer Primary Care Trusts

All with rights of appeal
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Transparent

• Open access to
• relevant published and unpublished data
• economic models
• systematic reviews
• reasons for decisions
• No access to
• Data submitted in confidence
• Economic models with in confidence data

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Independence

• NICEs advisory bodies include
• NHS doctors
• NHS nurses
• NHS pharmacists
• NHS managers
• Pharmaceutical physicians
• Academic statisticians
• Academic economists
• Patient advocates
• Patients

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Legal obligations

• Statutory Instruments
• as amended
• Directions
• NHS Act (1977)
• Administrative Law
• Fair processes
• Reasonableness
• Limits of legal powers
• European Law

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Clinical Evaluation

• 1. Randomised controlled trials
• Observational studies
• Systematic reviews

Avoiding hierarchies of evidence
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Economic Evaluation

• Overarching principles
• 1. Economic perspective
• NHS and PSS
• 2. Cost effectiveness
• Not affordability or budgetary impact
• 3. Balance between
• Efficiency (utilitarianism)
• Fairness (egalitarianism)

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Cost Utility Analysis

• Costs (and savings)
• direct
• indirect
• Benefits
• improvement (change) in HRQoL (utility)
• time for which it is enjoyed

Incremental cost effectiveness ratio
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Cost Ineffectiveness

• Probability
• of
• Rejection
• ICER (cost per QALY)

100
1. No empirical basis
2. Inflexible
3. Assumes reliability of QALY
4. Implies efficiency has an absolute
priority over equity
0
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Cost Ineffectiveness

• Probability
• of
• Rejection
• Cost per QALY

B
30,000
A
20,000
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Decision-makingScientific judgements

• Including
• Reliability of the evidence-base
• Appropriateness of sub-groups
• Generalisablity
• Capture of quality of life
• Handling uncertainty

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Decision-makingSocial value judgements

• Citizens Council
• 30 members
• Cross-section of England and Wales
• Serve for 3 years (one third retiring annually)
• Meet twice a year for 3 days
• Deliberative process
• Reports directly to the Board

Culturally and context specific
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Decision-makingCase-by-case

• Factors include
• severity of the underlying condition
• extension to end of life
• stakeholder persuasiveness
• significant clinical innovation
• children
• disadvantaged populations

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Recommendations lt30,000 per QALY
Topic QALY (000) Generalisability Stakeholder persuasion
LBC (cervical screening) 2
ECT (depression, psychosis) lt10
The QALY is a tool not a rule
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Recommendations gt30,000 per QALY
Topic QALY (000) Severity End of life Stakeholder persuasion Significant innovation Disadvantaged population Children
Riluzole (MND) 38-42
Trastuzumab (breast cancer) 37.5
Imatinib (CML) 36-65
Pemetrexed (mesothelioma) 34.5
Sunitinib (renal cancer 55
Human growth hormone Uncertain
The QALY is a tool not a rule
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Anti-cancer drugsvalue-for-money
Incremental cost per QALY ()

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Median Monthly Costsnew anti-cancer drugs at
launch
at 2007 prices
Monthly treatment costs US
Bach et al 2009
Year
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Appraisalstreatment condition pairs
Decision Numbers
Full use 98 (29)
Restricted use 188 (55)
Only in research 21 (6)
No use 32 (9)
Total 343 (100)
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Conclusions

• NICEs decisions are grounded on the available
  evidence
• But conditioned by scientific and social value
  judgements
• And tempered by its legal obligations

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Healthcare expenditure total per capita (Europe)
Total per capita healthcare expenditure (US)
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Healthcare expenditure and GDPEurope
R 0.894 R2 0.799
Per capita GDP (US)
Per capita healthcare expenditure (US)
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Appraisal processsubsequent appeals
Institute appeal
High Court
European Court of Justice
House of Lords
Court of Appeal
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IHE Innovation Forum II
Making Difficult Decisions