The treatments provided by the National Health Service of 2020 would seem alien to the Nye Bevan of 1948, although the key principle of free healthcare to all at point of need remains intact. It is tempting to believe that the latest advances in technology are revolutionising healthcare both in the UK and around the globe, but the truth is far more prosaic. As someone who has worked as a doctor at the rock face of the NHS for 35 years, the view that it is a clunky, unwieldy and highly bureaucratic organisation that is chronically underfunded and often relies on the goodwill of dedicated staff to prevent it from failing can be difficult to argue against — and that’s before adding Covid-19 into the mix.
Expecting the NHS to keep up with life-saving developments in health technology and increasing patient demand for such treatments is a huge ask, and one that the health service has a somewhat patchy track record on. This is partly due to how funding streams are allocated, but also because of the time it can take for the National Institute for Health and Care Excellence (Nice) to both appraise and recommend new technologies.
Nice is an independent body, set up more than two decades ago to decide which drugs and therapies are made available by NHS England. Its decisions have life-changing consequences: rapid access to clinically beneficial and cost-effective health technologies has never been more important to patients and their families, as well as the life sciences industry. Nice has evolved its methods of health technology evaluation over the past decade to adapt to emerging changes in this sector. Leaving the European Union has given a fresh impetus to encourage companies to launch their products first in the UK.
This is one of the reasons for the current Nice methods review consultation, which looks at the scientific, economic and methodological evidence that supports a case for change in how it now views health technology evaluation. It also evaluates the attractiveness of the UK as a first-launch country for promising new health technologies to ensure that patients can have access to them as early as possible. The review has two stages; the first looks at the evidence and case for change in this area, whereas the second considers the wider implications for change, including any financial effects. Nice says that three key areas to be considered in the future are genomes, digital technologies and antimicrobial resistance. It states the importance of clearly showing how its methods can support early patient access and ‘future proof’ reviews in years to come.
Advanced therapy medicinal products (ATMPs) have the potential for treating a range of disease areas in the future, including potentially curative options for serious conditions that currently have limited treatments available. These are medicinal products which are either tissue engineered, cell therapy or gene therapy based. They present a challenge both to manufacturers and the NHS, as their delivery is often multi-disciplinary and their high one-off costs pose problems with reimbursement frameworks.
The manufacturing of ATMPs is not only technically difficult but also very expensive, with a high entry into market cost-of-goods price. Complex supply chains are usually involved, often with crucial time-sensitive and temperature-critical constraints applied to them, and hospital pharmacies play a vital role in preparation, handling and storage ahead of use — expertise which may not be universally available.
The economic barriers to the expanding use of ATMPs are perhaps even more difficult to clear, as the existing reimbursement practices in the NHS do not always allow for the long-term and potentially curative benefits of these treatments to be considered when looking at purchasing practices. (The Nice review acknowledges this problem by accepting that these have high upfront costs which cannot be recovered, and that this can create challenges for affordability and implementation in the NHS.)
The UK is now looking at ways of improving patient access to ATMPs, with this Nice methods review being part of this process. Other system partners involved in this area include the Advanced Therapy Treatment Centre (ATTC) Network, which is a coordinated network of treatment coalitions that aims to accelerate the delivery of novel therapies by collaboration between industry and the NHS; and the Accelerated Access Collaborative (AAC), which brings these two groups together to identify innovations that should be made available to patients in the shortest possible time. Interestingly, the Nice review states there is no further case to be made for changing the approach of Nice to service delivery costs in health technology evaluations beyond the considerations made for apportioning costs.
One key question remains and hangs over the whole of this review — will this make a difference to NHS patients, and is the NHS serious about getting innovative treatments to them as soon as possible? (After all, we must not forget that the fax machine remains one of the key pieces of equipment in many GP surgeries up and down the land, despite a series of secretaries of state for health trumpeting the arrival of a joined-up IT system that will seamlessly link primary and secondary care and which has yet to arrive.)
The short answer is yes. The enormous Covid-19 costs to the Treasury will take a year or two to start shaking out and we do not know the exact impact of this on future NHS budget changes and priorities yet. However, hidden away in the small print of the review is a section on the ‘discount rate’ — an oft-forgotten factor in NHS economics but one which can decide whether a treatment comes to market or not. This is an economic procedure used by Nice to incorporate the future health benefits and costs of a medicine into its present-day value.
The rate that Nice most often uses is 3.5 per cent, but it has the discretion to apply a lower 1.5 per cent rate for therapies that offer longer-term health benefits. Until now, it has only chosen to apply this lower rate on exceptionally rare occasions, and this has not only acted as a brake on some future gene therapy avenues opening up but also discriminated against the value of long-term benefit therapies as it is applied over a longer period of time. The methods review has considered this impact, and acknowledges that the evidence supports reducing the discount rate for costs and health effects to 1.5 per cent as its default level. This is a hugely welcome step and may allow companies who were undecided as to whether their new health technologies were fiscally viable to take the plunge and bring their products to market.
As ever, though, where NHS policy is concerned there is always a caveat. Any such potential changes in policy will not be implemented until October next year, meaning that medicines such as gene therapies going through the appraisal process before then will still be facing the very policy challenges that this Nice review aims to address. Add into the mix the long-term impact of the current pandemic and the development of a still vague post-Brexit regulatory and access environment with the life sciences sector, and the dark tunnel towards the light of access to new technologies may stretch further than we might wish. However, that beacon of light may just have got a little brighter for patients awaiting new life-altering therapies.
This article first appeared in a Spectator supplement: The evolution of gene therapy. Sponsored by bluebird bio.
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