A child with muscular dystrophy, using a walking frame, in a park

Our highly specialised technologies (HST) programme is designed specifically to evaluate technologies for ultra-rare, debilitating conditions. It helps encourage research and innovation in areas where gathering robust evidence can be challenging, while ensuring fair access to treatments for very small patient populations.

Ultra-rare conditions are defined as those that affect 1 in 50,000 people or fewer.

The improvements to the HST routing criteria approved by the Board demonstrate our commitment under the Rare Diseases Action Plan to encourage innovation and support the rapid adoption of effective new treatments for NHS patients with rare diseases.

Today’s announcement follows public consultation on proposed changes to the criteria.

“We would like to thank everyone who has contributed to this work and for their help in shaping the final criteria and accompanying qualifying statements,” added Professor Benger. “Our aim throughout this initiative was not to change the number of medicines routed through the HST programme, but to clearly define the circumstances when it is appropriate to do so and improve the efficiency of the routing process.”

The HST programme gives greater value to the benefits from these treatments by having a much higher cost-effectiveness threshold.

Routing a topic to the HST Programme means the NHS must allocate resources if the technology is recommended, diverting them from treatments that could offer greater overall health benefits.

We do not put any limit on the number of treatments that are routed via the HST programme. However, the routing criteria are crucial in helping to ensure that the focus remains on improving access to treatments for ultra-rare conditions, encouraging research and managing the wider impact to the NHS.

Over the past 5 years we have approved 19 new treatments for ultra-rare diseases.

The refined HST criteria will be implemented from 1st April 2025.

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