In a four-part series, bluebird bio is exploring how the NHS can embrace the power of gene therapy as a treatment for rare diseases in the UK, including some of the systemic changes that will be needed. Here in the first column, Nicola Redfern, bluebird bio UK’s General Manager, discusses the potential value that gene therapies could bring from an individual, societal and economic perspective, and why it will be important for the NHS, payers and industry to work together to bring these novel treatment options to patients.

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We live in a new era of medicine. Cell and gene therapies are set to revolutionise healthcare as we know it, opening up a new frontier in medical innovation and treatment options. By treating diseases at the genetic level, the aspiration is to address the underlying cause of disease and restore a patient to normal or near normal health with a one-time, personalised treatment.

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The ability to address the cause of serious genetic conditions, that can impact on the daily life of patients and cause premature death, was once the “stuff of dreams.” Though not without risk (gene therapy itself, and the procedure needed to prepare patient to receive it, can have serious side effects), with more experimental therapies gaining marketing authorisation, this could soon be a reality for those patients who can benefit from it the most.

It is important to appreciate that patients, their families and carers are not the only potential beneficiaries of gene therapy. Health and social care systems may also benefit; if complex chronic care regimes can be eliminated, clinical resources can be re-deployed.

Against the backdrop of the new “covid-19” world, the possibility of reducing the systemic burden created by chronic diseases has never been more appealing. Yet how can society embrace the full potential of these innovations which carry a high up-front price tag in a time of impending austerity, fuelled by the impact of the global pandemic?

Government’s vision is a vibrant post-Brexit economy fuelled by science and technology allowing the UK to lead the world in healthcare innovation. This is in line with NHS England’s long-term plan, which suggests a clear appreciation that treatments like gene therapy have a place in the UK’s healthcare landscape.

A question to resolve is how to ensure licensed therapies gain rapid reimbursement enabling timely patient access, especially when gene therapies differ from the conventional medicines that our current systems were designed to accommodate.

They are complex to design and manufacture, and as “one-time” therapies have the potential to offer benefits that accrue over a lifetime. Gene therapy is a disruptive technology, which extends beyond the impact on care delivery to funding approaches, challenging how gene therapies should be valued, reimbursed and paid for.

The UK aspires to be a forerunner in gene therapy innovation. This requires a shift from collaborative dialogue to action planning and defined timelines to ensure initial momentum in the Accelerated Access workstreams (initiated by the NHS Accelerated Access Collaborative) translates, and the system is “recoded”. The aspiration is to focus resources on addressing the underlying cause of chronic genetic conditions rather than just managing them. This could be value worth paying for.