Out-Law News 2 min. read
08 Apr 2022, 11:30 am
Major life sciences companies and funding bodies have called for the development of a national vision on cell and gene therapies (CGTs) in the UK.
The joint call, outlined in a paper commissioned by the Cell and Gene Therapy (CGT) Catapult and funded equally by Innovate UK, Astellas Gene Therapies, Bristol-Myers Squibb, Gilead and Novartis, anticipates a significant increase in the number of CGTs coming to market over the next few years.
Agility should characterise the regulatory framework so that it can adapt to scientific and technological developments and address challenges around affordability and patient access
According to the paper, while just 12 CGTs have been granted marketing authorisation by the UK’s Medicines and Healthcare Regulatory Authority (MHRA) since the beginning of 2021, nearly 300 new CGTs are currently in development.
However, the paper highlighted potential barriers to the growth in use of CGTs in the UK in future which, left unaddressed, could threaten the UK’s existing position as a world leader in the provision of CGTs.
The paper said: “The UK has already seized the potential offered by cell and gene therapies, which is highly commendable. However, the UK cannot afford to rest on its laurels and must continue to strive forwards to remain a leading global destination for advanced therapies. A national cell and gene therapy vision will be important to chart the UK’s course over the next decade to truly capitalise on the wave of innovation expected.”
CGTs are treatments based on human cells or genes and a form of advanced therapy medicinal product (ATMPs). Compared to small molecules and protein-based treatments, such as monoclonal antibodies, which require repeated administration to treat the underlying conditions, CGT treatments offer a solution in which a single dose can provide a durable therapeutic effect.
However, the paper, among other things, said that the UK needs to scale-up its manufacturing capacity to meet demand for CGTs in future.
It highlighted the fact that the manufacturing of CGTs is highly individualised and that specialist manufacturing centres are needed so that treatments can be produced at scale. CGTs often have a very short shelf life and the paper highlighted how this can cause a compliance challenge in relation to where the treatments are manufactured and administered. The paper identified forthcoming changes to the regulation of ‘point of care’ products which could help but said the UK needs “to build a domestic manufacturing sector to help secure supply and reduce delays in treatment initiation”.
“To prepare for the expected increase in cell and gene therapies in the UK, a national cell and gene therapy vision should make recommendations on how to increase capacity within existing centres, and how to equip further centres to provide this type of therapy in the future,” the paper said, highlighting possible efficiencies in “estate management” and the establishment of “dedicated cell and gene therapy units with specialist teams” as possible solutions.
Other actions called for in the paper include consideration of new mechanisms for assessing the value of CGTs and appropriately reimbursing manufacturers for use of their treatments on the NHS.
“More flexible pricing arrangements, whereby risk can be shared between manufacturers and payers, such as annuity payment models, which spread the costs of therapies over multiple years, and outcomes-based agreements, which review the effectiveness of new therapies over time to adjust the price paid, could de successfully deployed to address concerns over uncertainty and affordability,” the paper said.
The paper also said there is scope for better collaboration to anticipate pressures that could otherwise fall on the NHS in relation to increased growth of CGTs. Industry has been urged to share information early about the CGTs they are working on to help ensure there is sufficient treatment centre capacity and enable NHS service reconfiguration as required.
Better understanding of the effectiveness of CGTs is also needed, according to the paper, which promoted the concept of real world evidence and improvements to data infrastructure to support capacity planning and fair reimbursement models.
Life sciences expert Helen Cline of Pinsent Masons said: “The UK has the opportunity to build a unique UK ecosystem in which businesses developing these innovative new medical treatments can thrive. Agility should characterise the regulatory framework so that it can adapt to scientific and technological developments and address challenges around affordability and patient access.”
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