UK must do more on access to rare disease medicines, says ABPI

There is growing evidence that it is becoming harder to bring new medicines, including those for rare and ultra-rare diseases, to NHS patients.

Ahead of Rare Disease Day on 28 February 2025, the ABPI is calling for a greater ambition to make the UK a more attractive place to develop and launch new medicines for rare diseases.

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increase in the proportion of NICE evaluations that are being terminated (the company submission has either been withdrawn during the process or a submission has not been made at all).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 tenth (England) and thirteenth (Scotland) when compared to other European countries. [5]

Dr Richard Torbett, ABPI Chief Executive, said: “Ensuring the NHS is fit for the future will require giving it the tools it needs to treat the 1 in 17 of us who will experience a rare disease. The latest action plan on rare diseases does many good things, but unless we address the UK’s historic and ongoing disinvestment in medicines, we will continue to see NHS patients with rare conditions missing out on the latest innovations.

“This means fixing the current skyrocketing repayment rates under the Voluntary Scheme for branded medicines and making sure that the way NICE evaluates medicines for rare diseases does not prevent companies from bringing them to the UK.”

NICE has recently consulted on proposed changes to its criteria for evaluating a very small number of ultra-rare disease medicines through its Highly Specialised Technology (HST) evaluation programme. [6]

The ABPI has expressed its concerns about the proposals as we do not believe they meet NICE’s stated ambition for the programme and will further decrease the already very limited number of medicines which are routed to it. The impact of this will be felt by patients with ultra-rare lifelong debilitating conditions and no adequate treatment options.

The ABPI does not consider the proposals in the HST consultation to be in line with NICE’s commitment under the Rare Diseases Action Plan to support the rapid adoption of effective new treatments for NHS patients with rare diseases, and has strongly urged NICE to reconsider them.

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, 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.