Gene Editing Breakthroughs Demand New Social Contracts for Access

Just a small fraction of our 20,000 genes can cause disease when disrupted, yet that sliver accounts for thousands of rare disorders. The challenge for doctors arises from the fact that many genetic disorders have mutations affecting only a small number of people worldwide.

For common conditions like type 2 diabetes, doctors can prescribe metformin, but for genetic disorders, identifying the specific mutation and treatment can be complex. Novel gene-editing breakthroughs are making headlines, yet therapies are expensive and complex to develop.

The cost of bringing a new drug to market is now around $2 billion, and there are approved treatments for less than 10% of the 8,000 diseases affecting humans. Commercial incentives tend to skew innovation towards lucrative cancer drugs and long-term treatments for larger populations, leaving complex gene therapies for rare conditions underfunded.

Waseem Qasim, a paediatric immunologist at Great Ormond Street Hospital in London, noted that while treatments may exist, they may not be financially viable. His work involving off-the-shelf edited T-cells requires industrial-scale manufacturing to become widely available, which the market alone may not support.

In May 2023, Prime Medicine announced a milestone in prime editing technology but indicated it would halt future work due to financial constraints. The geopolitical landscape of big pharma is shifting, with research and manufacturing moving towards nations like China and the US.

Britain faces challenges, as Donald Trump has pressured the UK to accept higher prices for NHS medicines, limiting available funds. Per-patient costs of gene therapies can reach into six or seven figures, posing questions about the sustainability of current business models.

A new social contract is needed to ensure equitable access to gene therapies, treating them like essential services funded collectively rather than as market goods. Many experimental treatments should remain in long-term NHS research programmes, offered at cost as the science develops.

A universal, publicly funded health system is essential to ensure that gene therapy becomes a part of the common good, rather than remaining confined to laboratories.