Written by Marco Crisci, Senior Analyst
The Association of the British Pharmaceutical Industry (ABPI) recently published a report outlining the need for new payment models, to manage an increased number of advanced therapy medicinal products (ATMPs), expected to become available under the National Health Service (NHS) [1,2]. However, England’s National Institute for Health and Care Excellence (NICE) disagree that changes are necessary [3].
Paying for ATMPs in the UK
Since 2018, the NHS has integrated many ATMPs successfully (e.g., Kymriah, Yescarta, Luxturna, Zolgensma) [2-3]. However, considering the current development pipeline, ATMP regulatory approvals are also expected to increase to 10-15 annually, in contrast to the current annual average of 2 approvals over the last 5 years. Future ATMPs are also expected in prevalent diseases (e.g., dementia, Parkinson’s disease and diabetes), whereas others to date have targeted smaller patient populations. This will impose a greater annual budget impact for the NHS, illustrating a potential need for payment models to cope with these changes [2].
Unlike in other European markets, innovative payment models have not been historically agreed in the UK, as NICE typically favour simple discounts. However, the NHS are currently conducting pilots to explore the practicalities of outcome-based arrangements (OBAs) for ATMPs, under the voluntary scheme for branded medicines pricing, access and growth (VPAG) [4]. Within these OBAs, payments are linked to clinical outcomes achieved by patients. The ABPI report has highlighted that the uptake of such models will be crucial for enabling access to future ATMPs through the NHS.
In June 2024, CSL Behring’s Hemgenix became the first therapy to be recommended for managed access under the Innovative Medicines Fund (IMF) by NICE. Casgevy (Vertex-CRISPR Therapeutics) has since followed suit. The agreements allow for conditional access to treatment on the NHS while more data is collected to support routine reimbursement [3,5]. While these managed entry agreements help to secure faster access, they may not present a long-term solution to manage routine reimbursement for an increasing number of ATMPs.
Potential barriers to innovative payment model implementation in the UK
The ABPI also suggests that staggered payments should be considered to help manage the high upfront cost of single-dose ATMPs over several years (e.g., reducing cost density) [2]. However, as the UK is a single-payer system with fixed annual budgets, staggered payments alone may not be sustainable for the NHS in the long-term, as the level of debit accrued year-on-year will increase in-line with the expected number of ATMP recipients. This financial risk may be partially mitigated if payments are linked to measurable outcomes that reflect the cost-effectiveness of reimbursed ATMPs.
The uptake of OBAs in the UK also demands a willingness to invest in the NHS, which is not currently equipped with the infrastructure and resources to track outcomes in a growing number of individuals, issue payments by results, and manage manufacturer disputes. Ring-fenced budgets (e.g., IMF and Cancer Drugs Fund; CDF) may alleviate the impact of ATMPs on annual NHS budgets, but more investment will also be required to finance these funding pools with the projected influx of available ATMPs.
Other recommendations in the ABPI report [1,2]:
- Payment model discussions should be available with the NHS before an ATMP receives marketing authorisation, to prevent delays in patient access due to price negotiations.
- The new rapid entry to managed access (REMA) process (currently under development by the NHS and NICE) should be piloted with ATMPs, to streamline access through the IMF and CDF (addressing current inefficiencies).
- Reduce the preferred NICE discount rate of 3.5% per year to 1.5%, to better reflect the therapeutic value of ATMPs. This amendment was previously considered by NICE during its 2022 methods review but was not included in the final set of changes.
- Increase the acceptance of real-world evidence (RWE) in cost-effectiveness evaluations; building on NICE’s recently published RWE evaluation framework [6].
Perception of England’s NICE on the ABPI report
In response to the ABPI critiques and recommendations, a spokesperson from NICE has defended their system, emphasising that they “have a strong track record of getting the most cost-effective treatments to patients fast, while ensuring good value for money for the taxpayer, [including] recommending 15 gene, cell or tissue therapies since 2016″. The assessment body suggested that large-scale changes may not be necessary, with NICE already having processes in place to deal with the complexities of ATMPs, including uncertain long-term benefits (evidencing Hemgenix and Casgevy managed entry). They also implied that willingness to accept “appropriate and fair” pricing will be required from industry to share the financial risks taken by the NHS [3].
Key takeaways
As observed in the US and some EU markets (e.g., Italy and Spain), OBAs may be utilised to reassure UK payers on ATMPs with uncertain long-term durability, and to manage high-upfront costs. These alternative agreements may enable sustainable reimbursement of future ATMPs. However, NICE do not agree that the changes proposed by ABPI are necessary. Although NICE have recently agreed managed entry for 2 gene therapies, more ATMPs are expected to become available, which will require investment and adaptation to address associated implementation barriers. To promote continued access to ATMPs in the UK, it will be important that NICE demonstrate the recognition of ATMP value to manufacturers, and a willingness to engage in new, sustainable payment models.
Partners4Access are constantly monitoring payment models negotiated for ATMPs in the UK and across global markets, to best inform our clients’ global PRMA strategies. For more information, do not hesitate to get in touch!
Sources:
1. https://www.abpi.org.uk/publications/unlocking-access-to-future-atmps-in-the-uk/
5. https://www.apmhealtheurope.com/story.php?uid=20095&objet=89264 6. https://www.evidencebaseonline.com/launch-of-nice-real-world-evidence-framework-heralds-a-new-era-in-health-technology-assessment-in-the-uk/#:~:text=Following%20a%20consultation%20process%20in%202021%2C%20in%20June,data%20%28RWD%29%20to%20inform%20health%20technology%20assessment%20%28HTA%29
