Principles of cost-effectiveness studies and their use in haematology

Elsevier

Available online 2 February 2023, 101441

Best Practice & Research Clinical HaematologyAuthor links open overlay panelAbstract

Health economics is about providing the population with the maximum health possible under budget constraint. The most common method to present the result of an economic evaluation is the calculation of the incremental cost-effectiveness ratio (ICER). It is defined by the difference in cost between two possible technologies, divided by the difference in their effect. It represents the amount of money required to gain one additional unit of health for the population. Economic evaluations are based upon 1) medical evidence of the health benefits of technologies and 2) the value of resources used to achieve these health benefits. An economic evaluation is one type of information that can be used by policy makers, in combination with data on organisation, financing, and incentives to decide on the adoption of innovative technologies.

Section snippetsEconomic evaluation and policy decisions

Why is economic evaluation an important component of health technology assessment and of its current sequel, comparative effectiveness research? Economic evaluations, in addition to effectiveness measures, are necessary to ensure that resources are allocated where they produce the greatest health benefit. Indeed, in a context of limited available financial resources, there is a need for collective arbitration in the resource's allocation.

There are common misconceptions about economic

What are the costs?

The costs that we consider for health economic studies are the value of resources committed to a medical intervention (and therefore lost for anything else). They are estimated by measuring the healthcare resource use associated with each intervention studied during the study period. These can include: (i) hospital inpatient stays and procedures; (ii) hospital outpatient and Accident and Emergency (A&E) visits; (iii) medicines; and (iv) healthcare professionals. When necessary, resources

What are the health benefits, why QALYs and how?

QALYs combine duration of life and quality of life. The use of a single metric for health outcomes is necessary if decisions are to be made for resource allocation across specialities: it is necessary to compare the amount of health produced by, for example an intervention in cardiology and another in ophthalmology or oncology.

The QALY is calculated by simply multiplying duration of survival in a given health state (pre progression, post progression) by a coefficient (or weight) that represent

The results of an economic evaluation

The endpoint for an economic evaluation is typically a ratio, the mean difference in costs divided by the mean difference in health (QALYs). It can be presented on a plane (Fig. 1) the point estimate of the result has for coordinates the difference in costs (vertical axis) and the difference in QALYs (horizontal axis). The incremental cost effectiveness ratio can be represented visually as the slope of the line connecting this point to the origin.

In order to test the robustness of the results,

Sources of evidence

Clinical trial data traditionally constitute the gold standard for determining the safety and efficacy of a new drug. The increasing use of the randomised controlled trial as a vehicle for economic evaluation presents the opportunity to collect prospectively economic as well as clinical data and offers the potential for uncertainty to be quantified through conventional statistical techniques. However, they are usually insufficient to help decision-makers understand how effective or

What are the results of economic evaluations in haematology?

Recent health economic studies in haematology-oncology have focussed on the more expensive therapies, such as chimeric antigen receptor (CAR) T-cell therapy. The list price of these therapies varies between 300 and 350,000€ in European countries, but this figure is not the result of an economic evaluation, only one input for the analysis [16]. The economic evaluation of axicabtagene ciloleucel by the French national authority provides a simple example [17]. The effectiveness results are based

Perspective

Any text on health economics would be incomplete if it left the reader with the impression that policy decisions result directly from the calculations described above. A framework for the analysis of how policymakers use the information is provided by M Goddard et al. in their article on priority setting in health [18]. The world of healthcare delivery is not limited to clinicians and economists, other stakeholders such as politicians, bureaucrats, taxpayers, interest groups and the industry

Practice points

Economic evaluations in haematology are very often relevant, given the high costs and changes in practice in involved.

It is best to plan the economic evaluation as an ancillary study to the clinical trial, this allow prospective collection of data on resource use and quality of life.

The preferred tool for measuring health related quality of life in an economic evaluation is the EQ5D, 3L or 5L

Research agenda

The research agenda concerns both the methods and their political acceptability.

The QALYs are insensitive to the type of diseases considered and compliantly methods such as discrete choice experiments are proposed to reflect societal preferences, for rare diseases for example.

The place of real world data as complement or substitute to data generated by trials is being investigated.

The inclusion of patient-reported outcome measures (in addition to health related quality of life measures) is

Declaration of competing interest

Dr Durand-Zaleski reports fees for speaking and taking part in advisory boards from Amgen, BMS, MSD, Pfizer, Takeda.

References (18)

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