NICE introduces promising improvements to the routing criteria for highly specialised technologies designed to treat rare diseases

This article considers the impact of the new criteria that became effective from 1 April 2025.

Background to the HST Programme

The NICE HST programme assesses technologies for very rare, and often severe, diseases that fulfil the specific considerations and flexibilities permitted by the programme. The HST programme takes into consideration very rare conditions that have been identified by the National Institute for Health Research Innovation Observatory. This programme is an example of a tool used to address healthcare inequality as it helps to encourage research and innovation in areas where gathering robust evidence can be challenging. Small patient populations are therefore better catered for as treatments are created to meet unmet clinical needs.

Purpose of the amendments made to the existing criteria

The amendments came into force on 1 April 2025. It has been reported that the purpose of the amendments was to "clearly define the circumstances" when it was appropriate to route medicines through the specialised technologies programme. The NICE board has approved the changes to facilitate more consistent, predictable and transparent decisions. With more than 7,000 recognised rare conditions and only one out of 20 of these benefitting from an approved NHS treatment or medicine, the reform is welcomed across the healthcare industry. Observable changes to the criteria include new qualifying statements to improve clarity and efficiency in the routing process and clearer definitions.

NICE HST Programme routing criteria

The NICE prioritisation board assesses whether all four of the HST routing criteria have been met. The four routing criteria are:

• Criterion 1: The disease is ultra-rare and debilitating.
• Criterion 2: The technology is an innovation for the ultra-rare disease.
• Criterion 3: No more than 300 people in England are eligible for the technology in its licensed indication, and the technology is not an individualised medicine.
• Criterion 4: There are no effective treatment options.

To assist with the assessment of the technologies, NICE collects relevant information during scoping from the applicant, and other research or academic sources. For more information about the routing process please visit the NICE HST Routing Process flowchart here.

Impact on the Pharmaceuticals and Life Sciences sector

The updates made to the NICE routing criteria are likely to impact on the wider pharmaceuticals and life sciences sector, particularly biotechnology, in a variety of ways. There are multiple opportunities that are likely to contribute to positive change to the pharmaceuticals sector:

• Uptick in innovation. Greater consistency, predictability and transparency in the routing criteria may provide greater assurance to pharmaceutical businesses and innovators, thereby encouraging innovation. If the framework used to assess the technology is consistent and predictable, it may encourage pharmaceutical businesses and innovators to make an investment in creating specialist technologies as the assessment criteria is reliable. The reliability of the assessment criteria may also help to make the UK attractive to businesses with capabilities of developing expensive, cutting-edge technologies. Accordingly, the new routing criteria may incentivise businesses across the pharmaceutical industry to commit to contributing to the research and development of technologies for ultra-rare diseases.
• Predictable decision making. The updated criteria are expected to be more transparent. This is favourable as it could make the HST programme more accessible to businesses of varying sizes with the competency of developing innovative technologies, capable of treating rare diseases. The transparency and improved consistency of the routing criteria facilitates predictable decision making and offers greater assurance to businesses that the investment in research and development is worthwhile.
• Pursuance of the Rare Diseases Action Plan. The HST Programme places a focus on the development of technologies for ultra-rare conditions which aligns with the Rare Diseases Action Plan that was published under the 2022 to 2024 Sunak Conservative government. Under that programme, the four key national priorities to facilitate the improvement of the lives with people living with rare diseases in the UK were: a) helping patients get a final diagnosis faster, b) increasing awareness among healthcare professionals, c) better coordination of care and d) improving access to specialist care, treatment and drugs.
• Market access. Improved access to treatments for ultra-rare diseases could be possible as a larger proportion of them are on the market. Practitioners and commissioners are better able to get the best care for patients across the UK, faster and on a more consistent basis, provided that the HST programme encourages innovation among companies.
• Increase investment in UK research and development. The standardised routing criteria may attract greater interest from investors into life science and pharmaceutical businesses with capabilities in the design and manufacture of highly specialised technologies. This may be the case as they may have greater confidence in the return on investment given the predictability and consistency of the routing criteria.

What can the Pharmaceuticals and Life Sciences sector take away from this regulatory update?

The pharmaceuticals and life sciences sector will be encouraged by the changes in the NICE routing criteria, as there are opportunities available for growth and innovation which may be lucrative. Organisations across the spectrum of size and profitability will benefit from the improved standardised set of routing criteria.

Innovative businesses operating within the pharmaceuticals and life sciences sector can prepare to benefit from these new and improved routing criteria by:

• Horizon scanning.
• Reviewing funding options for innovative research.
• Reviewing and preparing research and development agreements.
• Consider collaborating with universities as an industry partner for research and development.

If you would like to discuss this article or would like to discuss how we might be able to assist you, please contact Alarna Bond-Farrell in our Pharmaceuticals and Life Sciences team on 07500 110 580, or complete the form below.