The NHS is blocking access to genomic medicine
Approval processes are so slow in the UK that revolutionary treatments are not made available
Next month, the NHS will become the first health system in the world to offer whole genome sequencing to patients where clinically appropriate. Heralded by NHS leaders as “a new era of genomic health”, the goal is to use these data and new technologies to decode and treat previously intractable diseases, to move away from symptomatic treatments to cures and prevention.
The Prime Minister has said that she wants the UK to lead the world in this new science – to continue a tradition of innovation in this country that will “extend horizons and transform lives”.
Theresa May’s ambition to lead the world in genomics and precision medicines is one that we all want to support. As a scientist, doctor and CEO of Vertex, a biotech company that is pioneering precision medicines, I know the promise these scientific advances hold to change lives, but I also know the challenges that must be overcome to realise their full potential. This is not because the science is lacking, but because a fundamental shift in thinking is still needed by governments, regulators and policymakers in how they assess the value of this innovation.
Cystic fibrosis (CF) is an excellent example of this challenge. In 1989, when the cystic fibrosis gene was first identified, we did not know how mutations in the gene caused the condition. There was nothing to treat the underlying cause of the disease and people could only seek treatment for their symptoms.
After nearly 20 years of research and development by hundreds of Vertex scientists, and the design, synthesis and testing of more than 400,000 unique molecules, we have done what was once thought impossible – discovered and brought to nearly half of all CF patients the first medicines to treat the underlying cause of this devastating disease. Today, we have multiple medicines approved by the EU and the USA, and more coming down the line, with the ultimate goal of curing CF once and for all.
To complete this remarkable cycle of innovation, we now need the Government to do its part, by providing patients with access to these medicines. Unfortunately, three years after approval of these treatments, this has still not happened because our scientific innovation is outpacing the UK medicines evaluation system.
The evaluation criteria and processes used by NHS England and the National Institute for Health and Care Excelence (Nice) are currently preventing them from being made available to patients. Despite universal acceptance of the benefits that these medicines will bring, people in the UK have been waiting for access for more than 1,000 days, while thousands of people with CF in other countries in Europe and the US have been benefiting from them for years.
CF patients do not have time to wait. Half of those with this cruel disease will die before they are 31. Science has delivered the breakthroughs, but the system is blocking access. This is particularly heart-breaking in the UK, which has the second largest number of CF patients in the world.
In 2016, the UK’S own chief medical officer recommended a fundamental shift in how new transformative medicines are developed and appraised for use in healthcare systems. The appraisal system in the UK needs to reflect that the genes and pathways underlying genetic diseases seldom respond to traditional pharmaceutical approaches, and so precision medicine requires risk-taking innovation.
The Life Sciences Industrial Strategy, a report made to the Government just last year, echoes many of these sentiments. It outlines the need for industry to take on bold, far-sighted ambitions in the life sciences with the intention of creating commercial success, underpinned by novel technology and higher-risk science. The strategy singles out a handful of successful biotech companies with highly innovative products, including Vertex. Unfortunately, unlike many other European countries, NHS England and Nice have not yet followed these recommendations and evolved their evaluation criteria for these types of transformative precision medicines.
The Government must act now, not just for more than 10,000 people currently living with CF in the UK, but also for people suffering from many other kinds of genetic diseases.
We are standing on the cusp of genomic medicine becoming an everyday reality. As a company at the cutting edge of scientific advances such as gene therapy and gene editing, we need a system that is already thinking about the innovations of tomorrow
– a system that is ready to work in partnership with, and incentivise, innovators to get medicines into the hands of patients as soon as possible.
We will not give up on our ambition to cure CF and other serious diseases that today seem impossible to tackle. While we continue to deliver on the science, the UK Government must now show its commitment to patients and biomedical innovation. Together, we can build a system that works for everyone – for governments, for innovators, for patients – and truly make this genomic revolution a reality.