Gene Editing and the NHS: A Call for a New Social Contract

The Impending Revolution in Gene Editing

The landscape of medicine is shifting dramatically, and at the heart of this transformation lies gene editing. While disruptive innovations promise cures for previously untreatable conditions, the accompanying question is unavoidable: who can afford them? With per-patient costs soaring into the hundreds of thousands, the pressing need for a new social contract is evident.

Understanding the Challenge

Consider this: a mere fraction of our 20,000 genes can wreak havoc when disrupted, causing thousands of rare disorders. Unlike chronic diseases, where treatment pathways are well established, the intricacies of genetic disorders present a labyrinthine puzzle for healthcare providers. The challenge is not merely about developing new therapies; it's also about making them accessible to all.

The Price of Innovation

The reality is grim. The cost of bringing a new drug to market today hovers around $2 billion. As noted by Brian David Smith in his insightful analysis, the commercial landscape has favored cancer treatments with mass-market appeal, sidelining complex gene therapies for rare diseases that lack profitability. Gene editing promises revolution, yet the financial model often undermines its efficacy for those who need it most.

Real-World Implications: The Case of Great Ormond Street

“We are going to end up with treatments that work, but that nobody wants to pay for.” - Waseem Qasim, paediatric immunologist

Take, for instance, the groundbreaking gene therapy offered at Great Ormond Street Hospital in 2022 to treat relapsed T-cell leukaemia. While such innovations herald hope, they also underscore a stark reality: without a robust infrastructure backed by the state, many breakthroughs might remain relegated to the shadows of underfunded research.

The Global Shift in Biotech

The dynamics of the biotech industry are rapidly changing. As firms pivot toward technology sectors like AI and chip manufacturing, the UK finds itself at a crossroads. Attention is drifting towards nations with greater resources and foresight, such as China and the United States. As political pressures mount, the UK's capacity for medical sovereignty hangs in the balance.

A New Social Contract for Gene Therapy

If we accept that gene therapies can spiral into six or seven-figure costs, then we must reconsider how we fund and structure these life-changing innovations. The UK must initiate state-sponsored manufacturing capabilities, not only to ensure broad access but also to protect the integrity of its healthcare system against exploitative pricing models dictated by multinational corporations.

Rethinking Access and Affordability

• Collective Ownership: Like dialysis or transplants, rare-disease therapies could be seen as services funded collectively.
• Long-term Research Commitment: Many treatments could remain in NHS research programs, gradually maturing into effective solutions.
• Equitable Healthcare: A publicly funded system must guarantee access to all, transforming gene therapy from expensive merchandise to universally available medicine.

In conclusion, our success in navigating the new frontiers of gene therapy hinges on how we adapt our systems. By prioritizing equitable access and public funding, we can ensure that medical breakthroughs serve the public good rather than narrow profit margins. Without the NHS and a robust commitment to collective health, we risk allowing these crucial innovations to wither in the labs, never reaching those who stand to benefit most.