Finalist
Consensus Through Connectivity: National Alignment on the Optimal Treatment Journey in Fabry Disease
Summary of work
Fabry Disease (FD) is a rare, X-linked inherited lysosomal storage disorder that can affect the kidneys, heart, and cerebrovascular system. There are approximately 1,700 people living with Fabry Disease in the UK, representing one in 40,000 people.
Although data supported the link between prompt treatment initiation and lower renal and cardiovascular risks, no clear agreement existed within UK clinical practice for the care of Fabry Disease patients, or best practice for transitioning from paediatric services to adult services, leading to disparity of treatment.
Takeda has focused on Fabry Disease for over 20 years and is committed to improving patient outcomes. Having successfully identified the unmet needs and supported by clinical data, Key Opinion Leaders, and patient advocates, the small team took on the challenge of bringing HCPs from all 11 of the UK’s treatment centres together. The goal was to reach alignment on best practice patient care and transition from paediatric to adult services.
In 2023, a campaign and first-of-its-kind face-to-face event were delivered, overachieving on many of Takeda’s objectives and achieving consensus on treatment across all UK Fabry Disease treatment centres, setting the direction for improvements in FD patients’ lives.
Judges’ comments
This was a strong, engaging entry – a good example of good practice. It was well designed and well delivered, with really clear objectives of the campaign metrics. It had good tactics and was well executed, and the judges appreciated that this was engagement in the rare disease space, which is unusual. They liked the clarity about the reasons behind the approach, with really solid rational, and that the learnings were to be incorporated into future planning.
