Just a small fraction of our 20,000 genes can cause disease when disrupted – yet that sliver accounts for thousands of rare disorders. The difficulty is: what can a doctor do to treat them? In a common condition such as type 2 diabetes, the underlying biology is similar for millions of patients. The doctor can prescribe metformin. But with a genetic disorder, the mutation might only affect a small number of people worldwide. In many cases, doctors won’t even know which mutation is responsible, let alone how to fix it.
Novel gene-editing breakthroughs are making headlines. But therapies are expensive and complex to develop. The cost of bringing any new drug to patients is now around $2bn, in part because, as Brian David Smith notes in New Drugs, Fair Prices, the “success rate, from discovery to market, is tiny” and there are approved treatments for “less than 10% of the 8,000 diseases that affect humans”. Commercial incentives, he argues, skew innovation towards lucrative cancer drugs and long-term treatments for large populations. Complex gene therapies for very rare conditions are seen as too costly to develop and too small to profit from.
Take 2022’s revolutionary gene therapy to treat a young patient with relapsed T-cell leukaemia at Great Ormond Street hospital (GOSH) in London. Waseem Qasim, a paediatric immunologist leading that work, warned earlier this year that “we are going to end up with treatments that work, but that nobody wants to pay for”. Sustaining pioneering work cannot rely on philanthropy alone. His technological advance – “off‑the-shelf” edited T-cells – would require industrial-scale manufacturing to become widely available, and the market alone will not build it. GOSH’s research hints at the promise – and limits – of the therapy.
Biotech’s boom-time cash has moved on to silicon chips and artificial intelligence. That is why a milestone in prime editing – a DNA “search and replace” technology – was announced this May with a warning. Prime Medicine, the company behind the therapy, had successfully treated a teenager with a rare immune disorder but said it would halt future work. The geopolitics of big pharma is also changing. Firms are shifting research and manufacturing toward nations with scale and long-term policy support, such as China and the US. Britain looks exposed. Donald Trump has already forced the UK to accept higher prices for NHS medicines under threat of tariffs. This means less cash to support patients and for breakthrough therapies.
Per-patient costs of gene therapies can go up to six or seven figures. If the cure exists and the business model does not, the latter must change. Britain needs state-backed manufacturing for medical sovereignty – though it must not ignore the reality that China can already produce some gene therapies considerably more cheaply. What’s needed is a renewed social contract in which innovation is shared, not hoarded. Rare-disease gene therapy could be treated like dialysis or transplants: an NHS service funded collectively, not a market good.
Many experimental treatments could remain in long-running NHS research programmes, offered at cost while the science matures. Only a universal, publicly funded health system – with equitable access and lifelong follow-up – can make gene therapy medicine, rather than merchandise. Without the NHS and charities, the breakthroughs remain in the lab. With it, they become part of the common good.
