UK must alter approval regime so thousands more patients benefit from future cell and gene therapies

By Chris Wheal

August 22, 2024

Detailed report includes a 12-point plan of action

UK pharma firms have issued the NHS with a 12-point plan to help thousands more patients benefit from future advanced therapies 

The Association of the British Pharmaceutical Industry (ABPI) praised the NHS and the National Institute for Health and Care Excellence (NICE) for adopting cell and gene therapies. But it warned that new treatments, known as Advanced Therapy Medicinal Products (ATMPs), need to be adopted faster. 

The 12-point plan is included in the ABPI report ‘Unlocking access to future ATMPs in the UK: Comparing international approaches’.

Thousands more helped

The report says the regulator has approved an average of two ATMPs per year, but this is projected to increase to between 10 and 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.

This is an altered landscape from the current focus on niche conditions suffered by small numbers of patients and it means the NHS needs to change the criteria it uses to judge the value of new treatments.

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.

NICE and easy

The ABPI said: “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.”

Global leadership

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.”

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 12 recommendations 

1. The National Institute for Health and Care Excellence (NICE) should utilise the existing flexibility to apply the 1.5 per cent discount rate for ATMPs where relevant to ensure its appraisal process does not disadvantage future innovative treatments with long-term benefits, including many ATMPs. This would reflect evolving Health Technology Assessment (HTA) academic best-practice developments and the experience of several counties of periodic revisions to discount rates, and ensure that access to pipeline ATMPs is made possible through appropriate recognition of their value.

2. NHSE and NICE should work with industry to accelerate the implementation of a rapid entry to managed access (REMA) process by using ATMPs to pilot new approaches. Currently, the CDF and IMF require a full appraisal both on entry into and exit from managed access, which increases the burden on NICE and other stakeholders involved in technology appraisals, risking delays as the number of future ATMPs increases. A REMA process would help provide a sustainable solution to these challenges. Learnings from the pilots could also help identify options to streamline the appraisal process for transitioning from managed access to routine commissioning.

3. NHSE should ensure timely preparation for the launch of new ATMPs by being open to discussions with companies on the potential role for innovative payment models prior to marketing authorisation. Although NHSE already offers engagement with health technology developers, the specific issue of innovative payment models is not meant to be discussed until after the NICE process. In many cases, this is far too late to avoid delays in patient access if a more innovative contract is negotiated. Therefore, NHSE should align with the company earlier on the challenge facing a particular ATMP and the range of alternative options that might be investigated, rather than assuming a simple commercial discount is the solution, which will not be appropriate for many ATMPs.

4. NHSE should implement its commitment in the 2024 VPAG to undertake two innovative payment model pilots in a timely manner. At least one of the pilots should be for an outcomes-based agreement with payments spread over a longer contract duration than those used to date. The decision in the 2024 VPAG to pilot innovative payment models is welcomed, considering the urgency of acting to support patient access to the ATMP pipeline, and it will be important to realise the opportunities of these pilots to identify more sustainable solutions to patient access. This should include the possibility for contracts of longer durations than those used to date, as uncertainty over treatment outcomes may extend for several years longer than NHSE is currently accustomed to. 

5. Accounting regulations have long been identified as an issue for ATMPs, but action has not been taken to address the problem. NHSE, the Department of Health and Social Care (DHSC) and HM Treasury should explore how accounting rules can be adjusted to enable payments over multiple years. The UK is lagging behind other countries in addressing the uncertain impact of these rules and should instead implement any changes needed to enable the use of spread payments. NHSE’s commitment in the VPAG to undertake two innovative payment model pilots provides an opportunity to explore amendments to accounting regulations that are necessary to implement the pilots. 

6. NICE should increase flexibilities in evidence requirements and adopt a more risk-neutral approach to managing uncertainty for all ATMP appraisals. A clear strategy for health data in the context of ATMPs will become increasingly important as the role of real-world evidence is expected to increase for both managed-access approaches for supporting technology appraisals and for innovative payment models that use patient outcomes. To enable this and address the uncertainty, NICE and NHSE should support greater consistency in data collection to facilitate NICE’s continued use and acceptance of real-world data in appraisals. 

7. NHSE, NICE, the Medicines and Healthcare products Regulatory Agency (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. This would draw on other countries’ learnings on overcoming data-collection challenges, and should also enable secure and appropriate utilisation of patient data. 

8. ATMPs recommended for use by NICE should be included in the NHSE Innovation Scorecard and Estimates Report to understand whether uptake is in line with NICE eligible populations. This data should be scrutinised by an action-orientated cross-sector working group with policy makers, with plans put in place to understand and address instances of lower-than expected uptake. This will help to deliver on the promise of a streamlined end-to-end pathway for ATMPs, which is vital for underpinning the attractiveness of the UK to this sector. 

9. The UK government and devolved governments should establish a coordination group for ATMPs to share learnings across the UK nations and support capacity planning and decision-making across the respective health services. As shown by the example of the Genome UK Implementation Coordination Group, collaboration at UK level is consistent with the devolved nature of healthcare policy and could support maintaining equitable access across the UK. Wider pathway recommendations for the UK 

10. The UK must continue to develop ATMP manufacturing activities, including both early-stage and late-stage production and the effective targeting of research and innovation funding, as well as capitalising on the opportunity afforded by the new £520 million multi-year Life Sciences Capital Grants Facility. This will help to secure future, larger manufacturing operations and maintain 'sticky', high-value ATMP manufacturing jobs that have been created. 

11. The government should prioritise improving the UK ecosystem for delivering commercial clinical research, which will increase the attractiveness of the UK as a preferred destination for ATMP clinical trials. This should include implementation in full and at pace of the recommendations of the O’Shaughnessy Review, and passing outstanding UK clinical trials legislation to enhance the UK’s attractiveness for inward investment. New clinical trials legislation is essential following the UK’s exit from the European Union. However, this faced delays in the previous Parliament, causing uncertainty and enabling the UK’s competitors to gain an edge in attracting R&D investment. 

12. A strengthened and refreshed Innovative Licensing and Access Pathway (ILAP) should offer a streamlined end-to-end pathway for ATMPs that helps align all system partners from development to patient access. The MHRA should ensure it has the right levels of expertise and capacity to support the delivery of timely scientific advice, and clinical trial and drug licensing approvals as the evolving ATMP pipeline matures. The regulatory environment is key to unlocking growth and attracting and retaining inward investment to the UK.