Cfor you Small portion From us 20,000 jins It can cause disease when disabled – but this piece is responsible for that Thousands of rare disorders. The difficulty is: What can the doctor do to treat them? In a condition as common as type 2 diabetes, the basic biology is similar in millions of patients. Your doctor can prescribe Metformin. But in the case of a genetic disorder, the mutation may only affect a small number of people worldwide. In many cases, doctors won’t even be able to figure out which mutation is responsible, let alone how to fix it.
New breakthroughs are being made in the field of gene editing Headlines. But treatments are expensive and complex to develop. The cost of providing any new drug to patients now costs about $2 billion, in part because of this, as Brian David Smith notes New medicines and fair prices“The success rate, from discovery to market, is minimal” and there are approved treatments for “less than 10% of the 8,000 diseases that affect humans.” He argues that commercial incentives skew innovation toward lucrative cancer drugs and long-term treatments for large populations. Complex gene therapies for very rare conditions are viewed as too expensive to develop and too small to benefit.
Take, for example, the revolutionary gene therapy of 2022 to treat a young patient with relapsed T-cell leukemia at Great Ormond Street Hospital (GOSH) in London. “We will end up with treatments that work, but no one wants to pay for them,” Waseem Qassem, the pediatric immunologist leading the work, warned earlier this year. The sustainability of pioneering work cannot depend on philanthropy alone. Its technological advance – off-the-shelf modified T cells – requires industrial-scale manufacturing to become widely available, and the market alone will not be able to build it. Josh research It hints at the promise – and limits – of the treatment.
Boom-era biotech money moved into silicon chips and artificial intelligence. That’s why a milestone in primary editing – DNA search-and-replace technology – was announced last May with a caveat. Prime Medicine, the company behind this treatment, successfully treated a teenager with a rare immune disorder, but… He said Will stop working in the future. The geopolitics of Big Pharma is also changing. Companies are shifting research and manufacturing toward countries with broad and long-term political support, such as China and the United States. Britain looks exposed. Donald Trump has already forced the UK to accept higher prices for NHS medicines under the threat of tariffs. This means less money for patient support and advanced treatments.
Gene therapy costs per patient can rise to Six or seven numbers. If the treatment exists and the business model does not exist, the latter must change. Britain needs state-backed manufacturing to achieve medical sovereignty – although it should not ignore the fact that China is already able to produce some gene therapies at much lower cost. What is needed now is a renewed social contract under which creativity is shared, not hoarded. Gene therapy can treat rare diseases such as dialysis or organ transplantation: it is a collectively funded NHS service, not a market good.
Many experimental treatments can remain within long-term NHS research programmes, offered at cost while the science matures. Only a publicly funded global health system Fair access and lifelong follow-up – could make gene therapy a medicine, not a commodity. Without the NHS and charities, discoveries remain in the laboratory. In this way they become part of the common good.