The Burden of Haemophilia

The Haemophilia Society worked with the University of Chester to plan, lead and deliver a research study that, for the first time, looked 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 are 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).

Why is the study so important?

As new treatments for haemophilia continued to be licensed we need detailed data on what life is life living with haemophilia to help us make the case that they are clinically and cost effective.

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. The results of the first CHESS Study have been published but further stages of the work are underway.

Future studies will expand the data to children and other bleeding disorders such VWD. We’ll keep our members posted as things progress on our website and social media.