In a four-part series, Bluebirdbio 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. In this fourth and last column, Ross Selby, MRPharmS, Bluebirdbio UK’s Access, Value and Evidence Strategy Lead, looks at how aspects such as NICE’s Methods Review could impact the assessment of gene therapies in the UK.

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Gene therapies are set to revolutionise healthcare by treating diseases at the genetic level. They address the underlying cause of disease and can restore a patient to normal or near normal health. As one-time, personalised treatments, gene therapies have the potential to transform current care pathways by offering eligible patients durable outcomes when successful.

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They offer a one-time intervention, when often the alternative is decades of chronic treatment and monitoring, benefitting both patients and carers. Health and social care systems could also benefit, when complex chronic care regimes can be eliminated or greatly reduced, with significant resources re-deployed across the health and social care system; pertinent in a post-covid-19 world. Of course, gene therapy, as well as the procedure needed to prepare patients to receive it, can have serious side effects and there must be a rigorous assessment of potential risks and benefits to identify the right patients for the treatment.

Additionally, the cost-effectiveness of gene therapies, depends very much on their capacity of delivery health and social savings over a patient’s lifetime. As many patients treated with gene therapies will be children and young adults, the treatment may deliver additional societal gains over decades.


There are currently over 950 companies worldwide developing Advanced Therapy Medicinal Products, with therapies being tested in 1052 clinical trials, as of Q3 2019.1 Many of these are gene therapies that may become available in the UK over the next five years.

Provision needs to be made urgently for aligned regulatory assessment, health technology appraisal and NHS managed introduction, both in terms of infrastructure as well as reallocated budget. Life science companies also have a responsibility to set value-based prices and should consider alternative payment models and risk share agreements in collaboration with the NHS and government to further ensure value and affordability.

Gene therapies are positive and disruptive technologies that require whole system change to ensure that a post-Brexit NHS is at the forefront of provision rather than lagging behind its European neighbours. There is a window of opportunity for the MHRA with renewed responsibilities in 2021, as well as the National Institute for Health and Care Excellence currently conducting its Methods Review to ensure both regulatory and appraisal systems are aligned for optimal assessment of modern medicines, including gene therapies.

NICE’s review of the methods is highly significant as it will set the framework for how England and Wales will provide access to new and breakthrough medicines. Areas of focus should include wider recognition of gene therapies and their benefits, including the one-off treatment offer to patients, gains to the health and social care system, plus pragmatic ways to address inherent lifetime uncertainty.

Crucially, for paediatric and young adult patients, there is an additional challenge the NICE methods review needs to resolve. High economic discount rates used in the health economic assessment process by NICE has a prejudicial impact on the cost-effectiveness of treatments that are intended to offer benefits over decades, such as gene therapies. This issue can be easily addressed if Treasury guidance for utilising lower discount rates is adopted.

A successful NICE Methods Review would ensure that the UK has a fit-for-purpose medicines assessment process. This will help to achieve world-leading status for bringing new medicines such as gene therapy to patients and will sustain UK-based research and development investment. This should be prioritised as the end of the EU exit transition period approaches, to ensure the government’s vision of a vibrant post-Brexit economy, fuelled by science and technology, allows the UK to lead the world in healthcare innovation.

1. Alliance for Regenerative Medicine. Quarterly regenerative medicine sector report Q3. 2019. Available at: [Accessed 11 February 2020].