No room for complacency – the UK must do more on access to rare disease medicines

So far this year, by an unscientific count of press notices on the NHS England and NICE websites, at least three new medicines for rare diseases have been approved for patients on the NHS, and that’s just by today, Rare Disease Day. (28 Feb)

To get there, the UK medicines regulator, the MHRA, had to assess the quality, efficacy and safety profiles of the medicines, before issuing a licence for their routine use. The health technology assessment body, NICE, then had to determine that the medicines represented an appropriate use of NHS resources, given the clinical benefits they bring to patients. And finally, the NHS and the company had to agree the final terms around cost and supply.

In theory, this system should lead to patients getting the medicines they need, while the taxpayer gets a good deal on price. However, for many patients with rare conditions, there is a problem – and we can see the evidence for this in three ways.

Terminated NICE appraisals

A NICE appraisal is terminated when a new medicine is in the NICE work programme and has been granted a UK licence, but the manufacturer either does not make a submission to NICE, or withdraws an ongoing submission.

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. That’s the level of increased terminations for all medicines, not just those for rare diseases. The primary reason for these terminations is that companies consider the medicines will be rejected by NICE. The end result is that these medicines, despite having been licenced for use in the UK, will not be available to UK patients.

ABPI and BIA survey of members

In support of the recent Rare Diseases Action Plans, the ABPI and the BIA undertook a survey of our member companies on various issues about rare disease medicines. It covered a wide range of regulatory and commercial issues. The Department of Health published a summary of their response to that survey today and you can see their detailed response to the findings via the UK rare diseases forum online platform.

That survey revealed that since 2018, there were 16 out of 64 medicines indicated for rare diseases and approved by the MHRA which companies decided not to submit to NICE for evaluation.

Companies cited the very strict entry criteria for the NICE Highly Specialised Technologies (HST) evaluation programme, and the low prospect of NHS reimbursement at sustainable values for these medicines as key contributors to these decisions.

The ABPI and the BIA also asked companies to consider their pipeline for medicines indicated for rare diseases to be launched globally over the next five years and assess the approximate proportion of these their company expects to launch in the UK.

Concerningly, out of the 18 companies that responded, 11 reported that they expected to launch less than 75% of their rare disease pipeline in the UK over the next five years.

Reasons for this included the low likelihood of a positive reimbursement decision from NICE and the increasingly unfavourable commercial environment in the UK, including the level of payment rates under the current Voluntary Scheme (the VPAG). These payment rates represent the amount of revenue that companies are required to pay back to the government if NHS use of branded medicines exceeds an agreed level.  At the time of the survey, payment rates were just 15 percent – now they have rocketed to 22.9 per cent of industry UK turnover in 2025.

EFPIA Patients WAIT indicator data

The European equivalent of the ABPI, EFPIA, publishes annual data on how long it takes for patients in different countries to get access to medicines, including those for rare diseases. NHS patients with rare diseases are falling down this waiting list.

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.

Conclusion

The evidence shows little room for complacency. The UK has a problem getting rare disease medicines to UK patients. That problem is growing, and current action plans don’t go far enough to address it. Medicines policy over the past two decades has locked the UK medicines market into managed, long-term decline.  The environment worsens each year in real terms, reducing expenditure on medicines in relation to total NHS spend and in terms of international comparators. Cost effectiveness thresholds in England are amongst the lowest in the world and have declined around 50 percent in real terms over the last twenty years. Even when medicines are approved by NICE, adoption at local NHS level is often slow and highly inequitable. These policies are having a profoundly negative effect on patient access, thereby impacting clinical trial attractiveness, NHS productivity, investment and growth. Unless we fix rocketing VPAG rates and improve recognition of value in NICE’s evaluation methods, UK rare disease patients face an increasing risk of not getting access to new medicines, which often represent their only treatment option.

Companies do not take the decision to delay or not launch new medicines lightly. They spend hundreds of millions in investments and years of research to develop those medicines. Those involved want everyone who can benefit from their hard-won innovations to do so. However, if they are to continue to be able to bring new innovative medicines to UK patients, they need sustainable prices that adequately balance the large investments and high risks taken in the development process, as well as the many ideas that fail and will never reach patients.

The government, the NHS, and regulators are doing many good things for people with rare diseases. Through the Rare Disease Action Plans for England, Scotland, Wales and Northern Ireland, there is a genuine commitment to keeping that momentum going, and some of the actions in today’s Plan, for example, for England on ATMPs and individualised therapies, are very welcome.

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.

We will continue to be around the table with the Department of Health, NHS England, NICE, the MHRA and all of our other health system partners to solve the problems we face and make the UK an attractive place to research, develop and launch new rare disease medicines. People with rare diseases in the UK and their families deserve nothing less.