UK Mechanisms to Address Rare Disease Challenges: Does the UK Go Far Enough?

OBJECTIVES: Rare conditions affect fewer than one in every 2,000 people. There are an estimated 7,000 different conditions and, owing to their rarity, people living with rare diseases face challenges with the health and care system, and with wider public services. One in 17 people are affected by a rare condition at some stage in their lifetime: in the UK alone, this amounts to over 3.5 million people.

The UK government and devolved administrations published the UK Rare Diseases Framework in January 2021, setting out a shared vision for addressing health inequalities and improving the lives of people living with rare diseases. This included a priority to “improving access to specialist care, treatments and drugs”. The aim of this poster is to map the current mechanisms available in the UK to address UK rare disease challenges and answer the question ‘does the UK go far enough for rare diseases?’.

METHODS: Previous challenges for rare diseases in HTA were identified as part of an ISPOR international working group. The UK access landscape was evaluated against two topline HTA issues; no tailored HTA method for rare diseases and uncertainties for HTA authorities.

RESULTS: A number of mechanisms are available in the UK to help address rare disease challenges. However, not all challenges are adequately addressed

CONCLUSIONS: Rare diseases face significant challenges within the UK. Current mechanisms, processes and methods to fairly reflect societal preference for rare diseases have so far fallen short. The UK system continues to adapt and look at ways to partner with industry to tackle these issues with the aim of being ‘open to business’. However, politicians at the highest levels need to decide if the UK wishes to attract companies to invest, or to become a fourth launch region behind the US, EU and Japan.