About the research
The Haemophilia Society is working with the University of Chester to plan, lead and deliver a research study that will, for the first time, look at the full social and economic costs of living with severe haemophilia A or B, across five EU countries – not just to individuals and families, but to national healthcare services, workplaces, communities and society as a whole. Similar research, known as ‘burden of illness’ studies have been conducted for other conditions, or in other countries, but this will be the first of its kind taking place across national borders and aims to include over 1000 people.
We know there are many ‘hidden’ costs involved in having a bleeding disorder. They include things like patients and their carers taking time off work or school, and the impact that has financially and educationally. We know that rates of unemployment and depression are higher among the haemophilia community, so these costs are likely to be significant part of the overall picture.
Also, very few studies have considered the burden that carers - such as parents and siblings of a child - take on in looking after people with haemophilia, and the extra support a family may need over time.
By looking as broadly as possible at the ‘true’ costs, the study aims to fill a major gap in what we know about the ‘burden’, building evidence through a comprehensive, ‘bottom up’, burden of illness study in Europe’s five largest countries; France, Germany, Italy, Spain and the UK.
A range of measures will be used to gather comprehensive evidence that can be properly identified and quantified, by region, country and Europe-wide. They include:
- outcomes reported by patients themselves
- the amount of resources used by each national health service in caring for people with haemophilia
- the costs incurred by people and families themselves over time (medical and non-medical).
At the moment, the steering committee for the study is being recruited, with the aim to recruit study participants in the autumn and with results being published late spring of 2015.
Why is the study so important?
A new generation of longer lasting, or longer acting treatments are in development by pharmaceutical and bioscience companies. Yet to be licensed for use in Europe, these new drugs would reduce the frequency of treatment, which is hoped will improve people’s adherence to their treatment plans. The potential for better long-term clinical, health and economic outcomes and all-round improved quality of life is significant, and similar effects have been seen with improved treatment for other medical conditions.
To fully understand and evaluate the potential impact of these new treatments, alongside existing ones, we need robust data on the ‘real-life’ burden and costs of haemophilia.
Under current commissioning and funding arrangements, assessment still tends to focus on comparing direct costs to the direct / short-term medical benefits, which are most tangible and easiest to measure. This means that, if the price per unit of the new treatments is too high compared to the existing treatments, they may not be taken up by the NHS for mainstream UK use.
Thankfully, the ways in which health technology assessors - such as NICE in England - evaluate and make recommendations are becoming more sophisticated, with the aim of taking the wider, and more long-term costs and benefits into account when assessing whether a new drug offers value for money.
Unless we measure and quantify the wider impacts that a bleeding disorder has on individuals, families, communities and workplaces – such as carers’ ability to work, and long-term costs to healthcare trusts over time – the full, true picture of costs and benefits won’t be available to those who will ultimately decide whether or not to provide the new treatments for patients in each nation of the UK, and Europe-wide.
That’s where this ‘burden of illness’ study comes in – and why we believe this research is so critical to the future lives of the UK bleeding disorder community.
We’ll keep our members posted as things progress on our website and social media.