Making cell and gene therapies a true health system priority will improve the lives of those that might benefit in the years ahead and will maximise the holistic benefit for UK plc, says Nicola Redfern of NJ Redfern Ltd

By Nicola Redfern, NJ Redfern Ltd

What a great way to start 2024! It’s a real honour and privilege to be asked to contribute to articles here, alongside Sheela Upadhyaya and Michelle Conway, and what better than to focus my first one on a topic I’m passionate about and a project I actively contributed to. 

During 2023 we all heard and read about multiple advances in the gene and cell therapy (C&GT) arena with many announcements confirming regulatory approval and/or highlighting the promise of significant improvements to quality of life and life expectancy. 

These “advanced therapies” are often referred to under a singular umbrella term, but actually represent many different and uniquely precise technologies—from gene addition and cell therapy to more recent gene editing techniques. While these differ there is common ground in the underlying science and the intent of treatment, which is fundamentally different to more traditional medicines.

This ongoing progression is cause for celebration and we are now seeing real-world examples of these medicines changing lives—particularly in cancer and rare diseases, where clinicians and those living with the conditions have historically been faced with limited treatment options. The potential for both widespread patient and system benefits is incredibly promising, with advanced therapies offering an opportunity to entirely reconsider how we view health today, across an increasing number of different and more prevalent disease areas.

Despite this there are still barriers to unlocking the full potential of the innovation. Routine access in clinical practice around the world has many hurdles, with payers struggling to value the technologies within existing frameworks, and health systems facing logistical and staffing challenges given competing priorities and their often-complex delivery.

There is also an ever-increasing need for updated education so clinicians feel confident discussing C&GT as a choice when in routine clinical appointments with the individuals and families who might benefit. 

Here in the UK we therefore start 2024 with progress still needing to be made for patients and society to truly benefit from the transformative impact of these treatments. 

The Advanced Therapies Treatment Centres (ATTC) network is trying to ensure this becomes a reality and recently launched its call to action.

Their recommendations follow a collaborative meeting in October, where almost 100 influential, knowledgeable and experienced minds from government agencies, industry, the NHS and people with lived experience of relevant conditions, met at the Royal Society of Medicine in London to discuss barriers to advanced therapies adoption in the UK and explore solutions.

The report was published in December recommending change and investment. The full report can be found here, and we would encourage all to not only read but consider how you add momentum in the coming weeks to this call for action. 

The report offers a list of clear recommendations and detailed solutions to the challenge. In summary it calls us to:

1. Leverage existing world-leading expertise to enable the UK to be the best place internationally to conduct clinical trials and deliver advanced therapies—a discussion linked to Lord James O’Shaughnessy’s report.
2. Realise the full wider value of advanced therapies for society by revisiting the parameters within which value for this type of innovation is defined, including (but not limited to) enabling new payment models to be incorporated into the value discussions—a change which will require treasury engagement! 
3. Invest in supporting workforce and delivery infrastructure to allow the UK to benefit from advanced therapy delivery at scale.
4. Improve the collection of and use of data across all healthcare organisations to harmonise the advanced therapies ecosystem.
5. Perhaps most importantly, in order 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. Getting traction behind this taskforce and ensuring the right individuals sit on it to drive positive momentum is absolutely critical.

Whether you are touched personally by a condition that could benefit from one of the current or future cell or gene therapies, work within the health system and are involved in delivering this amazing step forward in science, are helping drive things forward in an academic institution or biotech/pharmaceutical company or sit in a position to influence the direction of travel, now is the time to make some noise and make a difference. 

Without attention it will become another report that sits on a shelf. The energy, aspirations and commitment in the room at the October event suggests this cannot be the case. 

As Neil Watson, co-director of the Northern Alliance ATTC and lead for the initiative, said, “Fundamentally, no change is not acceptable.”

About Nicola

Nicola set up NJ Redfern Ltd in 2022 to enable her to consult, coach and collaborate with other organisations, following her experience at bluebird bio. She has a long history in rare disease and oncology within the pharmaceutical and biotech industry and has worked closely with the Cell and Gene Therapy Catapult and the ATTC network in the UK. She is a member of the International Society of Cell and Gene therapy (ISCT) ethics sub group, contributes to the UK ATMP Engage community and projects, and speaks at various congresses and webinars about the challenges of bringing cell and gene therapies to market.

Nicola is committed to and motivated by ensuring people living with significant health challenges have a choice and are able to access transformative treatment options quickly once science and innovation reaches a point to positively impact their lives. She also believes the UK will be stronger if these new treatments are supported and embraced holistically across our ecosystem.

She can be contacted at nickiredfern@gmail.com or connect with her on LinkedIn.

Editor’s note: In the next Industry Insights column, Sheela Upadhyaya will take the spotlight to delve into Rare Disease Day, unveiling a diverse array of activities happening throughout the system to mark the day.

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