NHS: GENOMIC MEDICINE

THIS MONTH the NHS will become the first health service 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 she wants the UK to lead the world in this new area of 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 should all want to support. Scientists and doctors know that pioneering precision medicines and their advances change lives, but they will also be aware of the challenges that must be overcome to realise its potential. This is not necessarily 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, scientists 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 scientists, and the design, synthesis and testing of more than 400,000 unique molecules, they have now 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, multiple medicines approved by the EU and U.S. now exist, and there are more coming down the line. The ultimate goal is to cure CF once and for all.

For this remarkable cycle of innovation to be completed, Governments must now play their part, by providing patients with access to these medicines. Three years after approval of these medicines, this has still not happened because scientific innovation is outpacing the UK medicines evaluation system.

The evaluation criteria and processes used by the NHS and the National Institute for Health and Care Excellence (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 don’t really have the 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. The UK 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. Yet, unlike in many other European countries, the NHS and NICE have not yet followed these recommendations and evolved their evaluation criteria for these types of transformative precision medicines.

The Government must surely need to act, 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.

Genomic medicine stands on the cusp of becoming an everyday reality. Those institutions at the cutting edge of gene therapy and gene editing need a system that is already thinking about the innovations of tomorrow. Such systems need to incentivise innovators to get medicines into the hands of patients as soon as possible.

Organisations involved in scientific advances will never give up on their ambition to cure serious diseases that today might still seem impossible to tackle. While they continue to deliver on the science, the UK Government must show its commitment to biomedical innovation if the genomic revolution is to be fully realised.