Health economics: what is it and why bother?

It may seem unusual that a team of mainly economists is involved in a clinical trial of a medical treatment. However, health economic methods offer invaluable insights into the cost-effectiveness of interventions for governments, individual governmental departments and individuals to make informed decisions on the value of healthcare. 

The health economists involved in MCDS-Therapy have written this blog to tell you more about what they’re doing and why it is important.

Why do we need to think about health economics?

When we think about introducing new treatments, we need to consider whether any benefits that the new treatment provides are greater than its side-effects. We also need to think about whether or not the new treatment uses health care services in the best way possible. The reason for this is that health service resources are always limited and the decision to use them one way means we give up the opportunity to use them in other desirable ways. In addition, even when health services do not need to be directly paid for by the recipient of care, newer treatments may impose additional costs on recipients if they choose to use them. It is therefore imperative that the relative benefits, harms and costs are carefully considered. The additional costs of accessing health care may also be the added time and travel costs for recipients of new treatments – such as taking time off work to take their children to hospital for treatment.

In order to gain an understanding of the relative balance of benefits, harms and costs of a new treatment compared with current practice we can use health economic methods. Health economic methods provide the tools required to measure these costs in relation to health outcomes such as quality of life, and to ultimately allow us to understand if the new treatment intervention being evaluated is worthwhile.

What are we doing as part of this study?

As part of this study, we are carrying out a health economic analysis to understand the costs, benefits and harms of using a drug called carbamazepine for the treatment of metaphyseal chondrodysplasia, type Schmid (MCDS) and how it compares with currently available treatments. As there is no information currently available on the costs, benefits and harms of carbamazepine in patients with MCDS, a health economic analysis will be very useful for future decision-making about this treatment.

This health economic analysis involves two main parts:

  • We will investigate whether the use of carbamazepine as a treatment for MCDS would be good value for money. To work this out, we will build a mathematical model that describes the potential impact that treatments for MCDS may have over the lifetime of a child. This model will be tailored to reflect the different way care is provided in three illustrative countries: the UK, Germany and Italy. If the use of carbamazepine is found to be value for money, then health services will be advised on what finances they need to put in place so that the treatment can be provided to everyone with MCDS in their respective countries. This will be done by completing what is called a budget impact analysis.
  • We will use surveys to understand the preferences of patients with MCDS and their carers for the different outcomes of treatment (benefits versus harms), including how gains in one outcome might be traded off against reductions in another. For patients with MCDS, we will attempt to understand preferences around carbamazepine in terms of quality of care, effectiveness, harms and costs. For carers, we are interested in their preferences around caregiving situations involving carbamazepine treatment, which may be considered in terms of the intensity of the task, duration or availability of support.

Using established survey methods such as a discrete choice experiment (DCE), and best-worst scaling (BWS) we can find out what is the best way to provide care and how important successful treatment of MCDS is compared with the potential risks of the treatment. Both methods are standard practice in health economics and are incorporated into questionnaires given to patients. The first method, a DCE, involves answering a series of questions which ask you to make a choice between two options for the way care may be provided. The second method, a BWS, requires the reader to choose the best or worst option to a question on the survey. By examining the responses to both these methods, we can better understand the preferences of patients with MCDS and their carers for treatment outcomes.

What have we done so far?

Over the first year of the study, our focus has been on understanding the treatments for MCDS that are currently available and the clinical pathways of care that children with MCDS currently follow. These pathways of care will form the basis for the mathematical model that will be used to estimate the value for money of carbamazepine, and for the budget impact analysis. Given the limited information available on this rare condition, by speaking to the clinicians involved in the study we were able to gain an understanding of how people with MCDS are currently diagnosed and treated. We were also able to consider what happens to someone with MCDS over time, including the different treatments that might be used, and the complications (harms) that may be experienced.

Using the information we have gained from clinicians, we developed what is called a conceptual model of care – this is really just a diagram of how care is provided and what might happen over the lifetime of someone with MCDS. This conceptual model was also presented to German- and Italian-based clinicians to get their advice about whether this conceptual model reflects the way care is provided for people with MCDS in their respective countries. By doing this we can understand how the mathematical model might need to be changed to reflect the way care is provided in each country.

We have also made further progress on our study of the preferences of patients and their carers by collating a large body of literature on DCE and BWS studies. We are using this literature to help us design the survey presented to patients, carers and clinicians to elicit their preferences on the benefits, harms and costs of carbamazepine.

What are we going to do next?

Over the coming year, the focus of the health economics team will be on adapting our conceptual models of care into fully-functioning mathematical models and developing the survey of the study of the preferences of patients and carers.  To do this we will need to get information from a variety of sources, such as expert opinion, the clinical study, and previous research. Although this previous research is very limited, a health economics intern at Newcastle University spent some weeks in the summer of 2018 attempting to identify any papers (clinical or economic) in related disease areas.  We will use these as a source of additional information to inform the economic analysis.  However, a vital part of helping to complete this research will be by speaking to people with MCDS and their carers.

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