Vision launched in UK to maintain leadership position in cell and gene therapies

Cell and gene therapies are advanced medical treatments for certain patients with debilitating or life-shortening diseases. They work by either altering the content of a person’s genetic code, or by replacing diseased or dysfunctional cells with healthy ones. The UK was the first country in Europe to reach agreements that would see some patients routinely access a form of a cell therapy known as CAR T-cell therapy. This is a complex treatment where patients’ cells are collected, engineered and returned to the body to fight specific cancers.

Kite, a Gilead company, is a leader in developing and manufacturing CAR T treatments, and along with other pharmaceutical companies has worked closely with the National Institute for Health and Care Excellence (NICE) and NHS England in partnership to ensure timely access to these treatments.

‘At Kite, our purpose is to research therapies that have the potential to improve outcomes in cancer and ensure they are delivered without delay. Yet the way we review medicines hasn’t always kept pace and doesn’t always recognise wider social benefits, long-term savings or the challenges we face with treatments with small clinical studies,’ said Veronique Walsh, general manager at research-based biopharmaceutical company Gilead Sciences.

To enable the NHS to plan more effectively for these therapies, of which hundreds are in development, the Cell and Gene Therapy Catapult – an independent organisation established by Innovate UK, a government organisation – has worked alongside the wider community to develop a ‘National Cell and Gene Therapy Vision for the UK’. The Vision document sets out some of the challenges and future considerations for the government and aims to help the UK remain a world leader in this area.

While the health service’s long-term plan has set out NHS England’s intent to establish its position as a leader in the use of cell and gene therapies – with a commitment to offer ‘more personalised therapeutic options’ – the Vision document stresses that this intent now needs to stretch across the regulatory, access and care pathways.

‘The recent NICE methods review provided a significant opportunity for regulators, industry, clinicians and patient groups to work together to co-create solutions to long-standing access challenges in oncology – but we fell short of the ambition set out in the government’s life sciences vision. We need to go further. The patient voice needs to be more prominent in Health Technology Appraisal decisions – and this is particularly important in rare conditions. We also need to see an important technical change to the discount rate and for this to be applied to one-time, high-cost medicines to reflect the potential long-term benefit of these treatments,’ Walsh explained.

According to the Vision statement, the NHS also needs to consider and further develop the infrastructure and workforce capacity to be able to cope as more cell and gene therapies are made available. Adopting a national, strategic approach that takes a holistic view of barriers that could impede the uptake of these treatments will ensure readiness of the NHS for the future.

Walsh concluded: ‘Moving forward, true collaboration across industry, regulators, the government and the cancer community will be critical to ensuring that not only do patients have sustainable access to the latest innovative treatments, but that the UK fulfils its ambition to become a leading life sciences superpower.’

Visit www.gilead.co.uk for more information