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Class notes Economic Assessment of Healthcare

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Notes of all the lectures are in the document, which I used to obtain a 7.0 for the subject.

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  • 18 januari 2023
  • 7
  • 2022/2023
  • College aantekeningen
  • J.m. van dongen
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Economic Assessment of Healthcare

Lecture 1: Introduction HTA & Research designs
 Health technology assessment is a scientific study of a health technology, assessing
its health impacts and other aspects, with the aim to inform policy decisions.
 Health effects: efficacy and effectiveness
1. Efficacy: Can it work?
2. Effectiveness: Does it work?
 Aspects to be investigated in an HTA: health effects (efficacy & effectiveness),
technical properties, safety, economic impacts, and social, ethical, legal, or political
impacts.
 Timing of the HTA: Safety -> efficacy -> effectiveness -> economic impacts -> social,
ethical, legal, political impacts.
 In the Netherlands ‘Zorginstituut Nederland’ is the HTA-agency, in the UK it is the
National Institute for Health and Care Excellence (NICE).

 Efficacy research is oftentimes conducted with a placebo, to see whether the drug
has more effect than a pill with no substance. A study with a placebo cannot be
effectiveness research, as in effectiveness research you want to portray the real-life
situation, while in real life a doctor does not prescribe a placebo.
 Observational study easy to conduct, good reflection of usual practice, sometimes
very large populations are required, and confounding by indication is possible
(selection bias).
 RCT is sometimes expensive to conduct, may take a lot of time, and there is selective
patient population (- generalizability).
 Reliability: the degree of stability exhibited when a measurement is repeated under
identical conditions -> it refers to the measuring procedure rather than to the
attribute being measured.
 Validity: the extent to which the study measures what it is intended to measure.
1. Internal validity: the probability that the study design and conduct does not lead
to biased results. In other words, results are valid for the research population.
2. External validity: the probability that results are valid for other populations as
well. In other words, results are generalizable.
 Threats to internal validity:
1. Selection bias: systematic differences in participant characteristics at the start of
a trial.
2. Performance bias: systematic differences, other than the intervention under
study, in the performance of care.
3. Detection bias: systematic differences in obtaining (measuring) outcomes.
4. Selective outcome reporting bias: selection of a subset of the original variables
recorded, on the basis of the results, for inclusion in publication of trials
 Solution to performance bias -> blinding

Lecture 2: Introduction Economic evaluation
 Why do we perform economic evaluations:
1. Scarcity: available resources are never sufficient to allow all available health
interventions to be provided.

, 2. Choices: how can scarce health resources best be used in order to maximize the
health gain obtained from them?
 The basic question of an economic evaluation: is the service or program worth doing
compared with other things we could do with the same resources OR do the extra
costs weigh up to the extra benefits.
 An economic evaluation compares two or more alternatives and examines both
outcomes and costs.
 Incremental Cost-Effectiveness Ratio (ICER) = (Costs1 – Costs2) / (Effects1 – Effects2)
 Types of economic evaluations:
1. Cost-minimization analysis: effects considered equal
2. Cost-effectiveness analysis: disease-specific effects
3. Cost-utility analysis: Quality Adjusted Life Years
4. Cost-benefit analysis: Effects expressed in monetary value
 Decision rule: accept if ICER < predefined threshold, but which threshold.
 Thresholds:
1. UK: 20.000-30.000 per QALY gained
2. US: $20.000 per life year gained, $50.000 per QALY gained
3. NL: €10.000 - €80.000 per QALY gained.
 ICER is a poor predictor of decisions, policymakers may accept an intervention with a
non-cost effective ICER, when:
1. There is lack of an adequate alternative
2. Seriousness of condition (burden of disease)
3. Affordability from the patient perspective
4. Predefined ethical objectives
 Piggyback study -> adding collection of economic data to a clinical trial.
1. Advantages: experimental design, prospective -> patient level data, efficient use
of resources available for scientific studies
2. Aim of clinical trials: demonstrate safety and efficacy in a highly controlled
environment -> internal validity
3. Aim of economic evaluations: informing resource allocation decisions, outcomes
and costs in actual clinical practice -> external validity.
 Clinical trial vs economic evaluation -> pragmatic/naturalistic trial design to improve
the generalizability of the results.
 Study design issues in a pragmatic trial:
1. Comparator: choice of comparator treatment -> usual care, standard care, most
commonly used treatment.
2. Protocol-driven care: resources consumed for trial purposes that would not
typically be consumed in standard clinical practice. Increased resource
consumption, ‘case finding’ -> discovery of a previously undetected condition
during a protocol-mandated visit or diagnostic test, patient compliance.
3. Blinding: patients and providers do not have knowledge of the treatment group
to which patients have been assigned -> all study subjects receive the same tests
and services. Is this actual clinical practice?
4. Study population: in clinical trials patients are carefully selected to minimize
biological variation and highlight the treatment effect
5. Study sites: institutions and physicians should be representative of clinical
practice as a whole.

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