Catapult’s Cell and Gene Therapies Vision for UK as a Global Delivery Leader
Cell and Gene Therapy Catapult has published the ‘National Cell and Gene Therapy Vision for the UK’, a document opening industry eyes to recommendations and strategic directives required to grow the UKs leading position in cell and gene therapies.
In recent years the UK has positioned itself as a leader in everything cell and gene therapy. It has the third largest cluster for cell and gene therapy production worldwide, with 168 ongoing trials taking place in the UK in 2021, making up around 9% of all global trials.
The NHS was the first national health system in Europe to make CAR-T cell therapy available back in 2018. As of 2021, 16 new cell and gene therapies have been approved by the EMA, with 12 of these granted marketing authorisation by the MHRA.
Commissioned by Catapult, for educational and sharing purposes, and funded equally by Innovate UK, Astellas Gene Therapies, Bristol-Myers Squibb, Gilead and Novartis, this forward looking document was built from the insights of Catapult’s Industry Advisory Group and organised roundtable of experts.
“The expert recommendations outlined today, along with continued investment in the sector, will ensure that the NHS is prepared to deliver the next generation of ATMPs, and the UK retains its status as a world leader in the delivery of these therapies,” says Mathew Durdy, CEO of the Cell and Gene Therapy Catapult.
The document provides suggestions to spark conversations considering:
- How can the UK capitalize on the innovative movement in cell and gene therapies right now?
- How can they maintain and build further prominence in this niche market?
- And ultimately, how can they get these novel treatment options to patients in the UK?
Supporting the NHS
The NHS’s Long Term Plan sets out an agenda to reaffirm their position as a clear leader in the use of cell and gene therapies. Reportedly over 300 cell and gene therapies are under development and predicated to move towards approval. We can therefore expect a significant influx of ATMPs into the UK market in upcoming years. However, the expanse of these cell and gene therapies into the NHS will stretch their already strained resources.
Document recommendations for preparations to integrate these therapies and increase patient access and uptake include:
- Developing robust data infrastructure to improve clinical practice and provide informed evidence of these life-saving treatments
- Prioritising the patient experience and being receptive to patient feedback
- Ensuring a national implementation for widespread consistent uptake
As the health services gain scope to on-board cell and gene therapies into mainstream patient use, more NHS sites will need to gain accreditation to administer ATMPs, and be supported with the necessary access to sufficient training, expertise and workforce capacity. Preparation for which is already underway in the form of the Advanced Therapies NHS Rediness Toolkit.
Improving Manufacturing Facilities
The unique nature of advanced cell and gene therapies means its manufacturing is dictated by a confined set of parameters specific to the patient treatment window. This presents manufacturing challenges suggested in the document to be tackled through plans to build domestic manufacturing facilities and specialist treatment centres to support supply and reduce delays. This should also help to address the need for regulated manufacturing solutions to develop and administer point-of-care ATMPs to the patients who need them.
On top of this, concerns exist surrounding the effect the UK’s departure from the EU will have upon its position. Emphasis is placed, in the document, upon the Government to ensure supply chains are uninterrupted and safe product and batch testing certifications are accelerated, to maintain the UK as a future proofed and attractive location for manufacturing.
Gaining Investment and Focusing on Clinical Trials
It is inescapable that the Covid-19 pandemic has impacted the world of clinical trials, ATMP being no exception. Re-establishing itself at the forefront of cell and gene therapy clinical trials is essential to the UKs vision of industry dominance and delivery.
Document suggestions highlight the importance of gaining global investment to continue building and progressing these trials. The document also refers to a new process of submitting to the MHRA and Ethics Committees launched in January 2022, to improve the efficiency and speed of clinical trial assessment.
Finding Finance Solutions
In terms of the complex and unconventional pricing system of these advanced therapies, the document touches on some key suggestions NICE could consider moving forward to better support the adoption of ATMPs, including a 1.5% discount rate in ATMP appraisals.
Recommendations were also included for future reimbursement models, such as annuity payment methods and out-come based agreements to protect the best interest of the NHS, from both an accessibility and value for money point-of-view.
A Final Note
The premise of these recommendations all feed into supporting the UK, and in effect, the NHS, as becoming the world leader in cell and gene therapy delivery. The implications of these recommendations rely upon the collaboration of stakeholders at all levels. Working within the rapidly evolving innovative world of ATMPs, requires a similar collective attitude of evolution and innovation for its successful and sustainable implementation in the UK.