Choices and voices

The opinions and experiences of people with haemophilia increasingly shape the availability and choice of different treatment options. Scientists and pharmaceutical companies who develop new drugs for haemophilia care want to understand people’s preferences. This happens both early on in development and once a new treatment has been released. Researchers want to understand what features people feel are desirable for haemophilia medicines − things like how often a drug has to be taken, or even how it is taken.

In their recent paper ‘Discrete choice experiments: An overview of experience to date in haemophilia’, John Spoors and his colleagues consider one technique used by researchers for this purpose. Known as a ‘discrete choice experiment’ (DCE), it involves asking people to choose between different options to reveal their preferences and what trade-offs they might be willing to accept. For example, maybe people would be okay with taking their treatment more regularly if it meant better protection from bleeds.

With lots of new treatments under development, it’s more important than ever that we understand the preferences of people with haemophilia. What we, as individuals, want from our treatment matters – and our preferences will impact the uptake of new medicines.

The what and the why

John and his colleagues were interested in how DCEs were being used to understand haemophilia care. They found that although the method is good at measuring the preferences of people with haemophilia, it doesn’t provide much information about why people have these preferences. What leads people with haemophilia to make these choices and how likely it is that their choices might change? It’s important that we understand these questions too, and this is something for researchers to address.

One of the challenges of choice experiments is making sure they ask questions that people with haemophilia can relate to and understand. Perhaps there are opportunities to involve people with haemophilia in designing and testing these kinds of surveys.

Shaping the future of haemophilia care

John and his colleagues conclude that DCEs may be helpful for healthcare providers and government bodies to forecast people’s interest in taking up new products and how that might shape healthcare budgets. They believe that data on patient preferences will be increasingly incorporated in the evidence that pharmaceutical companies need to show to healthcare regulators about new drugs.

This means that there will be increased demand for the voices and opinions of people with haemophilia in the coming years. For me, this is exciting – and something for our community to be pleased about.


Further reading

Spoors J, Miners A, Cairns J. Discrete choice experiments: An overview of experience to date in haemophilia. J Haem Pract 2022; 9(1): 50-63. doi: 10.2478/jhp-2022-0006


About the author

Rich Gorman lives with severe haemophilia A. He works as a researcher at Brighton and Sussex Medical School.

Image: Shutterstock/Sylverarts Vectors