NICE at 25: Technology Appraisals – Faster Access to Modern Treatment?

The early years

Initially, NICE was characterised as a national body providing independent guidance to “the new NHS” being created for the 21st Century. NICE’s evidence-based recommendations were meant to be just that: recommendations, and many of the early ones applied to well-established treatments such as wisdom tooth extractions and proton pump inhibitors. It is also remarkable that, in the early years, the cost-effectiveness of treatments was not benchmarked against an explicit cost-effectiveness threshold to determine how good value-for-money the assessed treatment was.

Thresholds, the first risk-sharing agreement, and mandatory funding

As the NICE technology appraisal process was introduced, an obvious question was how to determine that the health gains from a new technology were ‘worth’ the cost.

A key milestone in NICE’s evolution was the acknowledgement of an explicit cost-effectiveness threshold, representing the opportunity cost of introducing a new intervention in the system and providing a benchmark to guide its decision-making. With an explicit threshold, the NHS and manufacturers started to reach commercial arrangements to achieve access within the value threshold. The step was challenging as it changed NICE’s initial remit, creating a perceived link between NICE decisions and prices for medicines. Beta-Interferon, a new treatment for Multiple Sclerosis (a disease that was poorly served by existing treatments), was the driver for taking the step. The evidence for the effectiveness of this new treatment was still immature, and NICE was unable to recommend its use as cost-effective within the £20k-£30k threshold. As political pressure increased to make these treatments available, the solution was to develop a risk-sharing agreement with the manufacturers. This complex and administratively burdensome arrangement did not initially lead to further similar types of agreements. But this solution to achieving access to new medicines in an attractive way for all stakeholders could not be ignored. Two less burdensome agreements were reached in 2007 and 2008, and their use was formalised in the 2009 Pharmaceutical Price Regulation Scheme agreement. Commercial access agreements are now the norm, especially for medicines used in the hospital setting, with 40 agreements in 2023.

That a medicine was deemed cost-effective by NICE did not necessarily result in consistent use across the NHS, mainly because local implementation of NICE recommendations was uneven. The solution was the establishment of the “funding direction” in 2002, making local commissioners and providers legally responsible for making funding available for a NICE-recommended treatment within three months of guidance publication whilst not challenging the autonomy of clinicians. 

“NICE Blight” and process reform   

In the early 2000s, there were a growing number of cases of “NICE blight”, where the NHS would not fund new treatments until NICE had approved their use, a process that could take years after a new treatment was licensed.

This issue was highlighted by trastuzumab, a biologic treatment for breast cancer. This medicine had been initially licensed for two uses in the latter stages of the disease, but it took two and four years after the license was granted for NICE to reach decisions on its use. In 2005, initial trial results showed very promising outcomes for the use of the medicine in the early stages of the disease. The driver to support the adoption of new effective technologies was being felt, especially from increasingly organised cancer patient groups. The assessment for this third indication for trastuzumab was completed within a month after license and informed the development of the “Single Technology Appraisal” process launched in 2005. NICE were now more clearly focused on assessing new medicines as close to license as possible.

End of life and, the Cancer Drugs Fund 

Trastuzumab was an early headline example of what was to become, and still is, a significant period of innovative launches of oncology medicines, extending life to patients with a short life expectancy. These provided challenges not only for NICE’s HTA process but also for the methods and thresholds used.

The launch of four medicines for renal cancer provided a focus for these pressures, raising the question of whether there should be instances where patients should be paying for treatments. In 2008, the Government commissioned Mike Richards, the NHS National Cancer Director, to produce a report on improving access to medicines. Following his recommendations, NICE introduced an “end-of-life” policy. This effectively increased the threshold for medicines that met specific criteria, including extending life expectancy for patients by at least three months. In recognition of innovations in areas outside of cancer, this principle has been extended to other classes of medicines in 2023 with the introduction of the Severity Modifier.

An additional policy to improve access to oncology treatments was put in place in 2011, with the establishment of a £200m dedicated fund for new oncology medicines not recommended by NICE. The rationale for this was twofold: recognising that the use of oncology medicines was below international averages and providing time to collect further evidence on the effectiveness of these treatments. By 2015, the budget for the Cancer Drugs Fund had reached more than £300m, and its management was proving challenging for NHS England. To put this short-term solution onto a longer-term footing, the management of the Cancer Drugs Fund was incorporated into the NICE HTA process, with the introduction of the category of “Recommended for Use within the Cancer Drugs Fund”. Launched in 2016, these decision categories enhanced NICE HTA methodology, formalising the principle of funding with evidence collection.

NICE and NHS England

Further reorganisation of the NHS in 2011 saw the creation of NHS England. This body assumed responsibility for the delivery of NHS services in England and, therefore, implementation of NICE’s recommendations. The last two changes to the NICE processes discussed here can be understood as responses to challenges faced by NHS England.

One of the most significant recent drivers has been the development of medicines that effectively cure chronic diseases. The first of these were medicines for Hepatitis C. Their introduction provided NHS England with significant operational and budgetary challenges. To address these issues, NHS England took steps to manage their introduction through commercial agreements with the manufacturers of these medicines. In recognition of the need to better plan and manage similar cases, this process of negotiation with manufacturers was formalised with the introduction of the Budget Impact Test. Effectively, this is an easing of the funding directive for those medicines that provide affordability challenges for the NHS, facilitating their managed introduction over a longer period.

The budget impact test was subject to a joint consultation by NICE and NHS England, recognising the close working relationship between these two organisations. This was also the case for the Highly Specialised Technologies process. Historically, the evaluation of medicines addressing very rare conditions was not usually undertaken through a NICE appraisal and instead sat with various specialist centres. NHS England assumed a centralised and standardised approach to managing specialist services, so their evaluation naturally moved to NICE. Similar to the drivers that led to the introduction of the end-of-life criteria, a new evaluation route was created to address the specific challenges created by these medicines, including uncertainty and unmet need.

Looking back, NICE has been successful in evolving their technology appraisal processes to address the changing landscape in which they operate. They have been able to reach a positive decision in 84% of cases and continue to support the process of faster access to modern treatments.

Looking forward, there will still be significant pressures on NHS spending and challenges to equitably provide access to new treatments. New treatments for Alzheimer’s disease look likely to be the next test for the technology appraisal processes and anticipated breakthroughs in areas such as digital health technologies and diagnostics will drive further reform to NICE processes and methods.