HCD Economics is setting research priorities in the field of rare disease and blood related disorders. Beta-Thalassaemia is the latest disease to be incorporated into the HCD research portfolio. HCD Economics is focused on understanding the beta-thalassemia patient and with that understanding, undertake socio-economic research to generate evidence to support patient advocacy.

The radical improvements in prevention and management of beta-thalassaemia major that have been taking place in the last three decades, primarily in the West, have led to significant progress in thalassaemia prevention and treatment. Thanks to these improvements, patients with beta-thalassaemia major can now have an increased survival rate and a better quality of life.

Dependant on the severity of disease, patients with beta-thalassaemia may require regular blood transfusions supplemented by appropriate iron chelation therapy (ICT) throughout their life. Such treatment impacts the patient medically and socially. This impact is described as the burden of beta-thalassaemia illness. Improved understanding of the beta-thalassaemia patient’s life with their disease is dependent on objective assessment of quality of life and the improvement that new therapeutic approaches can deliver for the patient’s quality of life.

Quality of life benefits can be quantified in the form of health state utility values (HSUVs). Utilities are defined as the strength of an individual’s preference for being in a particular health state, quantified into an index value, typically scaled between 0 (death) and 1 (full health). In assessing the evidence base currently available to support beta-thalassaemia advocacy for appropriate allocation of health resources, a gap in the evidence was identified for data that examines the utility impacts of differing transfusion burden (TB) and ICT.

In pursuit of commencing the creation of an evidence base to elicit the burden of illness of the beta-thalassaemia patient, a study was designed by HCD Economics and research partners including Celgene UK, Bristol Myers Squibb Inc. The study, recruited 200 individuals in an experiment to determine how the general public in the UK value beta-thalassaemia health states with differing TB and ICT. The results are published and available from the Open Access Library of the European Hematology Association.

For more information on the health state utilities study, and for an overview on the HCD Economics beta-thalassaemia research portfolio, please contact the research lead – Hayley Hubberstey. hayley.hubberstey@hcdeconomics.com