A revolutionary wave of genetic medicine is offering hope for thousands of rare, inherited disorders, but a profound crisis in funding and delivery threatens to leave these cures locked in the laboratory. With per-patient costs for advanced gene therapies soaring into six or even seven figures, medical pioneers and health economists are issuing a stark warning: if a cure exists but the business model does not, the model must be radically transformed.
The Promise and Peril of Precision Medicine
While only a small fraction of humanity's 20,000 genes are responsible for disease, their malfunction causes thousands of devastating rare conditions. The challenge has always been one of scale and economics. For common illnesses like type 2 diabetes, standardised treatments like metformin can be developed for millions. In contrast, a genetic mutation might affect only a handful of people globally, making traditional drug development commercially unviable.
Novel gene-editing techniques, such as prime editing – a precise 'search and replace' tool for DNA – are making scientific headlines. However, the pathway from discovery to patient is astronomically expensive and fraught with failure. The average cost of bringing a new drug to market is now estimated at around $2 billion. As noted in the analysis New Drugs, Fair Prices, the success rate from discovery to market is 'tiny', with approved treatments available for less than 10% of the 8,000 known human diseases.
This financial reality skews innovation towards lucrative, long-term treatments for large patient populations, such as cancer drugs, while leaving rare genetic conditions behind. Complex gene therapies are often deemed too costly to develop and the potential market too small to profit from.
Breakthroughs in the Clinic, Barriers in the Boardroom
The tangible success of these therapies only heightens the urgency of the funding dilemma. In 2022, the world watched as clinicians at London's Great Ormond Street Hospital (GOSH) used a bespoke gene therapy to treat a young patient with relapsed T-cell leukaemia. The lead paediatric immunologist on that groundbreaking work, Professor Waseem Qasim, has since cautioned that the sector risks creating 'treatments that work, but that nobody wants to pay for'.
His team's advance – creating 'off-the-shelf' edited T-cells – represents a leap forward. Yet, moving from a research setting to widespread availability requires industrial-scale manufacturing capacity that the current market has no incentive to build. Relying on philanthropy alone is not a sustainable strategy for pioneering medicine.
The economic landscape is shifting further. Investment capital that flooded into biotech has now moved towards sectors like silicon chips and artificial intelligence. This retreat was starkly illustrated in May when Prime Medicine, the company behind a major prime editing milestone, announced it would halt future work despite successfully treating a teenager with a rare immune disorder.
Forging a New Social Contract for Health
The geopolitical context adds another layer of pressure. Major pharmaceutical firms are increasingly shifting research and manufacturing to nations with scale and robust long-term policy support, such as the United States and China. The UK's position looks exposed, a vulnerability highlighted when former US President Donald Trump forced the UK to accept higher drug prices for the NHS under threat of tariffs.
The solution, argue experts, lies in reimagining the social contract for medical innovation. To ensure medical sovereignty and equitable access, Britain must invest in state-backed manufacturing capacity. This is not to ignore global realities—China, for instance, can already produce some gene therapies more cheaply—but to ensure the NHS is not held hostage by market failures.
The vision is to treat rare-disease gene therapy as a core NHS service, funded collectively like dialysis or organ transplants, rather than as a luxury market good. Many experimental treatments could be integrated into long-running NHS research programmes, offered at cost while the science matures.
Only a universal, publicly funded health system like the NHS, with its commitment to equitable access and capacity for lifelong patient follow-up, can transform gene therapy from a high-priced merchandise into mainstream medicine. Without this infrastructure, the breakthroughs remain confined to the lab. With it, they can become part of the common good.
