The future of access for patients to life changing advanced therapies lies in the hands of policy-makers and, in order to remain a life-sciences ‘superpower’ the UK needs to act now.

In October almost a hundred influential, knowledgeable and experienced minds from government agencies, industry, the NHS and people with lived experience met at the Royal Society of Medicine in London to discuss barriers and develop solutions to the UK Advanced Therapies Adoption Challenge.

The challenge amounts to an urgent need for rapid change and improvement in the field to maximise patient access to these therapies. Attendees jointly aligned on a need for investment nationally, to tackle disease, increase patient access to potentially life-saving medicines and to boost the UK economy – all achievable with the right resourcing and support.

Instigated by the Advanced Therapy Treatment Centre (ATTC) Network, which is coordinated by the Cell and Gene Therapy Catapult, the event drew from the exceptional expertise of participants to compile the ‘Advanced Therapies Adoption Challenge in the United Kingdom’ report.

From the report comes a list of clear recommendations and detailed solutions to the challenge, with headline recommendations being:

  1. Leverage existing world-leading expertise to enable the UK to be the best place internationally to trial and deliver advanced therapies.
  2. Realise the value of advanced therapies to ensure that UK patients are able to benefit from the latest cutting-edge health technologies.
  3. Invest in supporting workforce and delivery infrastructure to allow the UK to benefit from advanced therapy delivery at scale.
  4. Improve the use of data system-wide to harmonise the advanced therapies ecosystem.

To cement advanced therapies as a strategic, UK-wide health and life sciences policy priority and deliver the clear recommendations and detailed solutions within the report, there is a call to establish a new, multi-stakeholder, cross-departmental advanced therapies taskforce.

Advanced Therapies are often referred to under this umbrella terminology, but actually represent many different and uniquely precise technologies – from gene addition and cell therapy to more recent gene editing techniques. The science and progression of these technologies is cause for celebration and we are now seeing real-world examples of these medicines changing lives – particularly in rare diseases, where patients have historically been challenged with a limited availability of treatment options.

Whilst the latest generation of these innovations offer clear benefits, they present different hurdles when compared to historically ‘traditional’ medicines, such as the complicated manufacturing processes. However, the potential for widespread patient benefits is incredibly promising, with advanced therapies offering an opportunity to entirely reconsider how we view care today.

The UK remains a leader within Europe on advanced therapy clinical trials but Europe itself is now being left behind on the number of new trials being initiated when compared to both North America and Asia. Nationally the UK has exceptional expertise and the advanced therapy community is poised to lead the way in the research, testing, and implementation of these therapies. However, change is needed to deliver these ambitious at scale.

Whilst the UK is continuing to make rapid progress in the space, with the annual investment into advanced therapies increasing year on year and a doubling of employment into the industry (from 3,033 in 2019 to 6,956 in 2021) there is still huge opportunity for advancement.

By embracing an ‘innovation-first’ mindset and acting swiftly, experts at the event suggested investment into reinforcing the UK’s already highly skilled research workforce and expediting trial approvals were outlined as key priorities.

Professor Neil Watson, report author and co-director of the Northern Alliance ATTC, says: “Fundamentally, no change is not an option. We have such a prime opportunity to truly become one of the best places in the world to develop, launch, and receive advanced therapies.

“Policy-makers must be ready to grasp this opportunity to improve both the UK economy and, importantly, patient access by investing in advanced therapies.

“Within the current and very near future science, there is potential and real possibility for conditions such as diabetes, cardiovascular disease and Alzheimer’s to benefit from gene and cell therapies.

“The science suggests this is achievable, and that within a decade many people will be able to sit with their doctor to discuss how a cell or gene therapy might be part of their treatment – and this is not just for rare diseases or advanced cancer as it is now”.

Professor Gillian Leng, Commissioner on the NHS Innovation and Life Sciences Commission and President-Elect of the Royal Society of Medicine said: “The prospect of transforming people’s lives through advanced therapies is hugely exciting. We have begun to see how these innovations can make a significant impact on diseases such as cancer and rare inherited conditions, and we may soon see the same transformative impact on more common, priority public health conditions.

“I have been fortunate enough to observe the evolution of these technologies first-hand, including the system-wide challenges of funding, adoption, and implementation. With innovation often comes disruption – and this is particularly true for advanced therapies.

“To truly maximise the potential of advanced therapies for patients of the future, we must work to get the environment right now. We must overcome barriers in the system and reconfigure patient pathways to ensure people benefit from the best examples of these innovations. Achieving change won’t be simple but it is encouraging to see that progress is already being made.

“The future looks bright. To get there we must use the vast expertise at our disposal and drive forward the most tangible, implementable solutions to establish a flexible platform for advanced therapies.”

Matthew Durdy, Chief Executive at the Cell and Gene Therapy Catapult, supported the call to action saying: “Delivering advanced therapies at scale is essential to realising the true value of cell and gene therapies and the opportunity they provide to transform healthcare. By ensuring that patients have access to these transformative medicines when approved, the health system will meet demand, bring down cost and stimulate investment.”

Read or download the report here.