The UK has long been a leader in pharmaceutical innovation. However, its ability to provide patients with timely access to rare disease medicines is now under serious threat. While the NHS has approved three new rare disease treatments in 2024, this progress masks a deeper issue: an increasing number of approved medicines never reach UK patients.

This challenge is not theoretical. It is reflected in the data, industry sentiment, and global comparisons. Without decisive action, the UK risks becoming a secondary market for rare disease medicines, limiting access to life-changing treatments and diminishing its role in global pharmaceutical innovation.

The UK has long been a leader in pharmaceutical innovation. However, its ability to provide patients with timely access to rare disease medicines is now under serious threat. While the NHS has approved three new rare disease treatments in 2024, this progress masks a deeper issue: an increasing number of approved medicines never reach UK patients.

This challenge is not theoretical. It is reflected in the data, industry sentiment, and global comparisons. Without decisive action, the UK risks becoming a secondary market for rare disease medicines, limiting access to life-changing treatments and diminishing its role in global pharmaceutical innovation.

A System Designed to Work, but Failing in Practice

The UK’s regulatory pathway for rare disease medicines follows a structured process. The MHRA assesses safety and efficacy, NICE evaluates cost-effectiveness, and pricing negotiations determine final NHS access. In principle, this system should deliver both patient access and value for the taxpayer. In reality, it is increasingly dysfunctional.

The Evidence: Declining Access and Industry Retreat

The data shows a clear and worsening trend:

• The rate of NICE appraisal terminations—where companies withdraw or decline to submit medicines for evaluation—has doubled from 9.8% (2016-2019) to 20.1% (2020-2023).
• Since 2018, 25% of MHRA-approved rare disease medicines were never submitted for NICE evaluation, primarily due to low expectations of a positive decision.
• A recent industry survey found that 11 out of 18 companies expect to launch less than 75% of their rare disease pipeline in the UK over the next five years.

The primary reasons cited by industry include:

1. Highly restrictive NICE criteria for the Highly Specialised Technologies (HST) programme.
2. An increasingly unfavourable commercial environment, including the rise in VPAG rates (which require companies to return a percentage of revenues to the NHS), now at 22.9% of industry turnover in 2025, up from 15%.
3. Uncertain NHS adoption even when approvals are granted, leading to delays and regional inconsistencies.

A Diminishing Role on the Global Stage

Once ranked second in Europe for access to orphan medicines, the UK has now fallen to tenth place in England and thirteenth in Scotland. Meanwhile, Germany continues to set the benchmark, with faster approvals and higher adoption rates.

This decline has direct consequences. If the UK is seen as an unattractive launch market, companies will increasingly deprioritise it in global regulatory and commercial strategies. The impact will be profound: fewer clinical trials, slower patient access, and a diminishing share of pharmaceutical investment.

The Path Forward: A Strategic Shift is Needed

The UK Rare Disease Framework has played a critical role in driving collaboration between regulators, industry, and healthcare providers. However, it does not go far enough to reverse these trends. A new approach is required.

Key Actions for Policymakers

1. Reform NICE evaluation criteria to reflect the true value of rare disease medicines.
2. Recalibrate VPAG payment rates to ensure the UK remains commercially viable for new medicine launches.
3. Accelerate NHS adoption processes to prevent regional disparities in patient access.
4. Extend the UK Rare Disease Framework beyond 2026 to sustain momentum and drive long-term improvements.

The UK has the scientific expertise, regulatory capability, and healthcare infrastructure to be a global leader in rare disease innovation. But without urgent policy changes, patients will face increasing barriers to life-changing treatments.

The Question for Industry Leaders

Is the UK willing to redefine its approach and reclaim its leadership in rare disease medicine, or will it continue on a path of managed decline?

The answer will shape not only patient outcomes but also the future of the UK as a centre for pharmaceutical innovation.