Rational Rationing The Role of Research Michael Rawlins Chairman, - - PowerPoint PPT Presentation

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Rational Rationing The Role of Research Michael Rawlins Chairman, - - PowerPoint PPT Presentation

Rational Rationing The Role of Research Michael Rawlins Chairman, National Institute for Health and Clinical Excellence, London Emeritus Professor, University of Newcastle upon Tyne Honorary Professor, London School of Hygiene and Tropical


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SLIDE 1

Michael Rawlins

Chairman, National Institute for Health and Clinical Excellence, London Emeritus Professor, University of Newcastle upon Tyne Honorary Professor, London School of Hygiene and Tropical Medicine

Rational Rationing

The Role of Research

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SLIDE 2

Resource Constraints

Healthcare Expenditure (US$ per person)

1000 2000 3000 4000 5000 6000 7000 8000

Cost (US$) per person per year

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SLIDE 3

GDP and Healthcare Expenditure

2007

1,000 2,000 3,000 4,000 5,000 6,000 7,000 10,000 20,000 30,000 40,000 50,000 60,000 70,000 80,000 90,000 Per capita GDP (Int$) Per capita healthcare expenditure (Int$)

R = 0.776 R2 = 0.602

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SLIDE 4

NICE guidance

  • 1. Clinical:

– Technology appraisals – Clinical guidelines – Interventional procedures – Medical technologies – Diagnostics

  • 2. Public health
  • 3. Quality standards and metrics

– Quality & Outcomes Framework – NICE Quality Standards

  • 4. NHS Evidence
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SLIDE 5

NICE guidance

Type Published In development

Technology appraisals 200 130 Clinical guidelines 130 52 Interventional procedures 322 30 Medical technologies 8 Diagnostics 3 Public health 27 33 Total 679 256

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SLIDE 6

Technology appraisals

Health technologies encompass:

– Pharmaceuticals – Devices – Surgical (and other) procedures – Diagnostic methods

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SLIDE 7

Clinical guidelines

“Systematically developed statements to assist practitioner and patient decisions about appropriate health care for specific clinical circumstances”. Institute of Medicine

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SLIDE 8

Principles

  • 1. Robust
  • 2. Inclusive
  • 3. Transparent
  • 4. Independent
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SLIDE 9

Clinical Evaluation

  • 1. Randomised controlled trials
  • 2. Observational studies
  • 3. Systematic reviews
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SLIDE 10

Randomized controlled trials

advantages

  • 1. Minimises bias
  • 2. Minimises confounding
  • 3. Minimises random error
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SLIDE 11

Randomized controlled trials

disadvantages

  • 1. The null hypothesis
  • 2. P-values
  • 3. Generalisability
  • 4. Multiplicity
  • Stopping rules
  • Subgroup analyses
  • Safety analyses
  • 5. Cost
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SLIDE 12

Comparative effectiveness

  • 1. Direct comparisons
  • A versus B
  • 3. Indirect comparisons
  • A versus placebo
  • B versus placebo
  • Impute A versus B
  • 3. Mixed treatment comparisons
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SLIDE 13

Observational Studies

  • 1. Historical controlled trials
  • 2. Concurrent cohort studies
  • 3. Case-control studies
  • 4. Case series (registries)
  • 5. Case reports
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SLIDE 14

Systematic reviews

Efficacy:

– Good at synthesizing RCT evidence – Weak at incorporating observational data

Safety:

– Good at synthesizing RCT evidence – Very weak at synthesizing observational data

Cost effectiveness:

– Very poor

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SLIDE 15

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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SLIDE 16

Cost Utility Analysis

Costs (and savings):

– direct – indirect

Benefits:

– improvement (change) in HRQoL (utility) – time for which it is “enjoyed”

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SLIDE 17

Cost Ineffectiveness

Probability

  • f

Rejection Cost per QALY A US$ 30,000 B ₤45,000

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SLIDE 18

Decision-making

  • 1. Scientific judgements
  • Reliability of the evidence-base
  • Appropriateness of sub-groups
  • Generalisablity
  • Capture of quality of life
  • Handling uncertainty
  • 2. Social value judgements
  • Severity of disease
  • End of life interventions (“rule of rescue”)
  • Age
  • Health inequalities
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SLIDE 19

Social 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

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SLIDE 20

Case-by-Case Decisions

Factors taken into account include:

– severity of the underlying condition – extensions to end of life – stakeholder persuasiveness – significant clinical innovation – children – disadvantaged populations – corporate responsibility

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SLIDE 21

Recommendations >£30,000 per QALY

Product Condition QALY (£)

Severity Significant innovation

Riluzole Amyotrophic lateral sclerosis 40,000 Trastuzumab Early breast cancer 37,500 Imatinib Chronic myeloid leukaemia 36,000 to 65,000 Pemetrexed Mesothelioma 34,500 Sunitini Advanced renal carcinoma 50,000 Lenalidomide Multiple myeloma 43,000

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SLIDE 22

Conclusions

  • 1. Rationing can (and should) be rational
  • 2. Research methodology needs improving
  • less resource-intensive approaches to RCTs
  • creative use of observational data
  • capture the potential of digital technology
  • 3. Earn and retain the trust of all our

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