There is growing evidence that it is becoming harder to bring new medicines, including those for rare and ultra-rare diseases, to NHS patients.
While there are more than 7,000 recognised rare conditions, only one out of 20 of these have an approved NHS treatment or medicine to help.[1]
A survey by the ABPI and the Bioindustry Association (BIA) of pharmaceutical companies developing medicines to treat rare diseases found that the majority of companies did not expect their full future pipeline of rare disease medicines to reach UK patients.[2]
Reasons for this included the low likelihood of a positive reimbursement decision from NICE and the increasingly unfavourable commercial environment in the UK, characterised by the rocketing and unpredictable medicines rebate rates under the Voluntary Scheme – now at 22.9 per cent of industry UK turnover in 2025. At the time of the survey, rebate rates were at 15 per cent, well above the 6.8 per cent average between 2014-2022.
This feedback correlates with a concerning increase in the proportion of NICE evaluations that are being terminated (the company submission has either been withdrawn during the process or a submission has not been made at all).[3]
The same ABPI/BIA survey revealed that since 2018, 16 out of 64 medicines indicated for rare diseases and approved by the MHRA, were not submitted to NICE for evaluation.[4]
Despite companies working hard to make medicines available to UK patients, many cited the very strict entry criteria for NICE’s Highly Specialised Technologies (HST) evaluation programme and the low prospect of NHS reimbursement for rare disease medicines as key drivers of these decisions.
Further, NHS patients with rare diseases are falling down the waiting list for access to new treatments. In the last six years, the UK has dropped from being ranked second for availability of orphan medicines (behind Germany), to eleventh (England) and fifteenth (Scotland) when compared to other European countries[5]
The ABPI wants to see the work of the UK Rare Disease Framework continue beyond the end of its current remit in January 2026. Since it was introduced in 2021, the five-year Framework has played a crucial role in driving action, accountability and coordination on rare diseases across the four nations and between stakeholders.
However, it is clear that there is more work to be done to improve the lives of people affected by rare conditions, and the renewal of Framework provides an opportunity to set new ambitions for beyond 2026.
The ABPI will continue to work with the government and all partners to give people with rare diseases better access to medicines.
References
[1] Genetic Alliance factsheet, ‘Access to rare disease medicines in the UK’, accessed Feb 2024, available at https://geneticalliance.org.uk/wp-content/uploads/2024/02/Access-to-medicines-factsheet.pdf
[2] In a 2024 survey to support Action 25 of the Rare Disease Action Plan, the ABPI and the BIA asked companies to consider their pipeline for medicines indicated for rare diseases to be launched globally over the next 5 years and assess the approximate proportion of these their company expects to launch in the UK.
Out of the 18 companies that responded, 11 reported that less than 75% of their rare disease pipeline is expected to be launched in the UK over the next 5 years.
The government has published a summary of its response to the survey.
[3] Over the last decade, there has been over a 100% increase in the rate of terminated NICE appraisals, from an average of 9.8% between 2016-2019 to 20.1% between 2020-2023, according to Office for Health Economics analysis of official NICE data, available on request.
[4] See reference [2] above, survey part 2. The survey did not break down the names of individual medicines.
[5] EFPIA Patients WAIT Indicator Reports 2018 (published 2019) and 2024 (published 2025). 2018 survey available at https://www.efpia.eu/media/412747/efpia-patient-wait-indicator-study-2018-results-030419.pdf and 2024 survey available here efpia-patients-wait-indicator-2024-final-110425.pdf Data in the 2018 report is captured for the UK and not broken down. Data in the 2024 report is split by England and Scotland.
[6] NICE, public consultation launched on changes to NICE’s Highly Specialised Technologies routing criteria, Dec 2024, available at https://www.nice.org.uk/news/articles/public-consultation-launched-on-changes-to-nices-highly-specialised-technologies-routing-criteria
