A customisable model that estimates the cost of thalassaemia care – an imperative tool for policy planning and delivery
Cost of illness (CoI) is defined as the value of the resources that are expended or forgone as a result of a health problem. It includes health sector costs (direct costs), the value of decreased or lost productivity by the patient (indirect costs), and the cost of pain and suffering (intangible costs).
In 2021, TIF proceeded to the development of a CoI Model for Thalassaemia to quantify the burden of thalassaemia on health systems and allow decision makers to translate the adverse effects of thalassaemia into monetary terms.
TIF anticipates that the estimates generated by the CoI Model for Thalassaemia will be used to:
- define the magnitude of the disease in financial terms in different settings;
- justify the development of disease-specific policies for the prevention, clinical management and cure of thalassaemia;
- assist in the allocation of funding and resources by decision makers and reduce healthcare waste;
- provide a basis for policy and planning with regard to prevention and control; and
- provide an economic framework for the evaluation of existing thalassaemia control and management programmes
Such a model, simple in inception but powerful in execution, may be used to help national / regional competent authorities include thalassaemia in policy planning. It may also be used by health authorities, health partnerships and consortia both nationally and transnationally to identify and address more effectively the huge unmet needs of patients. It is a powerful advocacy tool for the implementation of disease-specific policies that will enable the better allocation of resources (healthcare, human, financial) that will ensure patients live a better quality of life.
Aspiring to inform national level policy, the nuances of healthcare services provided in developed and developing countries were taken into consideration during the development of the model so as ensure its fitness for purpose in usability and adaptability. Thus, the model is flexible to account for country-specific adaptations.
How was the model developed
To ensure the thalassaemia model is robust, defendable, relevant and reflective of the expected drivers of clinical and cost-effectiveness, the methodology outlined below was followed:
- A targeted literature review of published economic models, fully aligned to the aims of the model and formalised in a search strategy and any existing HTA evidence in thalassaemia thus informing the cost-of-illness model conceptualisation. The review furthermore identified relevant input parameter profiles to support model analysis and value demonstration e.g. Model design, Clinical evidence, Treatments, Model parametrisation, Existing models etc.
- The model conceptualisation and specifications were derived by relevant data sources including the aforementioned targeted literature review, insights from international experts in the field of thalassaemia, health economists and other relevant key opinion leaders.
- A robust, user-friendly and transparent cost-of-illness model was developed enabling customisation and adaption to local settings thus ensuring flexibility and functionality.
The developed model has already been tested, to confirm that it has been robustly and accurately implemented, establishing the cost-of-illness model face validity. Model verification has ensured that the programming and physical implementation of the conceptual model has been completed correctly. To ensure appropriate validation the model has been applied to two countries with available data (United Kingdom and Iran).
Read more about how the model was developed and validated in TIF’s Global Thalassaemia Review 2021 (Chapter 9).