Gene Editing Breakthroughs Demand New Social Contract for Access

Gene editing technologies are advancing rapidly, prompting discussions about the need for a new social contract to ensure equitable access to these groundbreaking therapies. Currently, a small fraction of the approximately 20,000 human genes can lead to diseases when disrupted, resulting in thousands of rare disorders. The challenge lies in how healthcare providers can effectively treat these conditions, as many genetic mutations affect only a limited number of individuals worldwide.

In contrast to common illnesses like type 2 diabetes, where treatments such as metformin can be widely prescribed, genetic disorders often leave doctors without clear guidance on treatment options. The complexity of developing effective therapies is underscored by the significant cost involved in bringing new drugs to market, which now averages around $2 billion. According to Brian David Smith, author of *New Drugs, Fair Prices*, the success rate from discovery to market remains low, with approved treatments available for less than 10% of the 8,000 diseases that impact humans.

As pharmaceutical companies prioritize lucrative cancer therapies and treatments for larger populations, the development of gene therapies for rare conditions is often seen as financially unviable. A notable example is the revolutionary gene therapy administered at Great Ormond Street Hospital (GOSH) in 2022 to a young patient with relapsed T-cell leukaemia. Waseem Qasim, a leading pediatric immunologist at GOSH, expressed concerns that innovative treatments may become available without corresponding financial backing, stating, “We are going to end up with treatments that work, but that nobody wants to pay for.”

The sustainability of these pioneering therapies cannot rely solely on donations and philanthropy. For widespread accessibility, advancements like “off-the-shelf” edited T-cells necessitate industrial-scale manufacturing, which the market alone is unlikely to support. The recent developments in prime editing technology further illustrate the challenges facing this field. In May 2023, Prime Medicine announced successful treatment of a teenager with a rare immune disorder but indicated potential halts in future work due to financial constraints.

The global landscape for pharmaceutical research is shifting, with companies increasingly relocating their operations to nations like China and the United States, which offer scale and long-term policy support. The United Kingdom faces vulnerabilities in this new environment, especially following actions by former President Donald Trump that pressured the UK into accepting higher prices for medications under the threat of tariffs. This pressure diminishes funds available for patient care and the development of breakthrough therapies.

Cost remains a considerable barrier, with per-patient expenses for gene therapies reaching into the six or seven figures. If viable cures exist but the business model is inadequate, significant changes are necessary. The UK requires state-backed manufacturing capabilities to ensure medical sovereignty, yet it must also confront the reality that gene therapies are being produced more economically in other countries.

A renewed social contract is essential, one in which innovation is shared rather than hoarded. Access to rare disease gene therapies could be structured similarly to services like dialysis or organ transplants—offered as a publicly funded service rather than a market commodity. Many experimental treatments could remain within long-term NHS research programs, provided at cost while scientific understanding evolves.

The vision for effective gene therapy must align with the principles of a universal, publicly funded health system. Such a system would promote equitable access and long-term patient follow-up, transforming gene therapy from a luxury into a common good. Without the support of the NHS and charitable organizations, many breakthroughs may remain confined to laboratories, failing to benefit the broader population. With appropriate frameworks in place, these innovations can become integral to public health rather than mere commercial products.