Investing in rarity: improving access for patients and the UK economy

In the UK alone, more than 3.5 million people are living with a rare condition. Yet many face challenges in receiving the care they need.

Only a small proportion of rare diseases have a licensed treatment, and even where treatments do exist, access through the NHS is often limited. Together, these factors continue to drive unmet need – delaying care, worsening outcomes and increasing pressure on families and public services.

Our latest research, commissioned by the Association of the British Pharmaceutical Industry (ABPI), examines what could change if the UK addressed these challenges.

The current landscape: limited treatments, limited access

Despite advances in science and medicine, only around 5% of rare diseases currently have an effective treatment. And even when treatments are available, access is not guaranteed. Just over a third of orphan medicines licensed in the EU are fully accessible to patients in the UK.,

This means that many patients face long waits, restricted eligibility, or are unable to access treatment altogether. For conditions that are often progressive and life-limiting, delays can have lasting consequences.

What could change: becoming a European leader in access

The UK has an opportunity to improve this position. The UK Strategy for Rare Diseases sets out an ambition to improve equity of access and promote R&D in treatments for rare diseases. This is supported by the Life Science Sector plan which aims to make the UK one of the top 3 fastest places in Europe for patient access to medicines and MedTech by 2030.

Our analysis explores the potential impact if the UK became Europe’s leading country for access to orphan medicines, while also supporting the development of new treatments.

The results show that improving both availability and access could deliver substantial benefits:

• 158,000 more patients receiving treatment
• £4.4bn in lifetime health benefits
• £19.4bn in productivity gains over ten years
• Reduced pressure on the NHS through avoided hospital admissions and emergency care

These findings highlight that improving access is not only a clinical issue, but an economic one – with effects that extend across the health system and wider economy.

Many patients and carers reduce their working hours or leave employment altogether. Improving access to treatment can help to reverse this trend, supporting workforce participation and reducing productivity losses.

At the same time, better disease management can reduce the need for intensive healthcare services. Avoiding hospital admissions, stabilising conditions and delaying progression all contribute to easing pressure on the NHS.

These wider effects reinforce the importance of viewing rare disease policy through a broader lens – one that considers long-term outcomes across health, economic and social systems.

Click here to read our full report Investing in rarity: the economic and personal value of improving treatment for rare disease