UK must get ready for new cell and gene therapies, says ABPI

The NHS got off to a good start with the introduction of cell and gene therapies but it must do more to get ready for the arrival of the advanced therapies of the future, according to a new report for the Association of the British Pharmaceutical Industry (ABPI).

Advanced therapies, also known as Advanced Therapy Medicinal Products (ATMPs), offer the potential to treat previously untreatable diseases and have already been brought in for some rarer conditions such as haemophilia [1], spinal muscular atrophy [2], and a rare disease causing blindness [3]. In some cases, these therapies can transform people’s lives with just a single treatment.

The ABPI report, ‘Unlocking access to future ATMPs in the UK: Comparing international approaches’ [4] shows that the NHS has made progress in getting advanced therapies to patients with rarer conditions – but must do more to get ready for the future pipeline of advanced medicines with larger patient populations.

ATMP treatment costs typically come ‘upfront’ requiring a single payment - but patients are often expected to benefit for many years afterwards. The cost of treatment is driven by a range of factors, including R&D and high manufacturing costs, due to the personalised nature of the medicines.

Many existing advanced therapies are in rare disease areas with relatively low numbers of patients, making it easier for the NHS to budget for the treatments. However, some therapies now in development are aiming to address conditions with larger patient populations, including certain types of dementia or Parkinson’s disease. As a result, the health system will need to adapt its approach to paying for these medicines.

Over the past five years, the UK medicines regulator has approved an average of two ATMPs per year, but this is projected to increase up to 10 - 15 per year by 2030. The number of people who could be treated with these therapies may grow to as many as 10,000 per year in the UK by 2028, up from 2,500 people in 2021 [5].

A challenge for the NHS in using advanced new therapies is uncertainty about how long the benefits of any treatment might persist. Clinical trials are necessarily conducted over a relatively short time, which creates challenges for bodies like NICE to value the long-term benefits of these treatments when the evidence is still uncertain.

Richard Torbett, Chief Executive of the ABPI said: “Advanced therapies have the potential to transform patients’ lives with just a single treatment, avoiding a lifetime of care. In the process, these treatments can in some cases save the NHS a lot of money over the long term, which is better for the taxpayer and the patient.

“We should think about health spending as an investment. We need to take a long-term view of the value and cost-effectiveness of medicines, looking not just at the upfront costs, but at the lasting outcomes we want over years and decades. Doing so will require innovative new payment models that can respond to real patient outcomes.”

Peter Wickersham, General Manager UK/Ireland and Vice President of Gilead said: “The UK’s success in delivering CAR-T therapies demonstrated the global leadership it can have – it was the first health system in Europe to make CAR-T cell therapy commercially available to eligible patients. 

“However, the way we assess cutting-edge treatments like cell therapies must evolve in future if the UK is to retain this global leadership position.”

“The ABPI report highlights some critical changes which will help to ensure that we are fit for the next five years of CAR-T therapy and beyond, which would be a win for UK patients.”

Eduardo Cabas, General Manager, UK and Ireland, for CSL Behring said:

“Highly innovative treatments such as gene therapy represent a paradigm shift for patients, doctors and healthcare systems.

“Our experience is that NHS England has shown flexibility in negotiating pioneering outcome-based agreements that make these therapies available to patients while offering the NHS the assurance of value and financial protection it requires.

“Much more will need to be done to ensure that an increasing number of patients can benefit fully from these innovations and for the UK to maintain its position as a global leader in life sciences.”

The report draws on international experience to suggest ways for UK regulators to adjust their approach and make sure the NHS is in a better position to adopt cutting-edge therapies sustainably. It makes recommendations for changes to allow the UK to research and manufacture these medicines more easily:

• Allow NICE to use a lower ‘discount rate’ to determine the future ‘value’ of ATMPs. At present, NICE has to reduce the future value of treatments more than would be the case for other long-term government investments, which might result in some new treatments not being viable. In 2022, NICE itself recommended that the discount rate be lowered from 3.5% to 1.5%. With the VPAG agreement, this change could be implemented at no cost to the NHS.
• Have more flexibility in the evidence needed for advanced therapies, including greater use of real-world evidence – NICE and NHS England should support greater consistency in the way data on the effectiveness of therapies is collected, which would allow more use of real-world data in cost-effectiveness calculations.
• Test out more innovative ways of paying for medicines, that spread out payments over time and link payments to treatment outcomes. NHS England has committed to two pilot schemes, and one example has been seen recently in haemophilia, but the report calls for a breadth of schemes.
• NHS England, NICE, the MHRA and industry should work together to establish a single national platform for collecting data on ATMP treatment outcomes that could support innovative access models for patients – one example exists in Spain.
• Establish a coordination group for advanced therapies to share lessons across the UK nations and support capacity planning. The Genome UK Implementation Coordination Group, is one example of an existing group that could be followed.
• Continue to develop the UK’s ability to manufacture advanced therapies. Capitalising on the opportunity afforded by the new £520m multi-year Life Sciences Capital Grants Facility is one way to do this.
• Improve the attractiveness of the UK as a destination for commercial clinical research into advanced therapies.