Since its creation in 1999, the National Institute for Health and Care Excellence (NICE) has been considered a world leader in assessing the clinical value and cost-effectiveness of new medicines. Originally created to end the ‘postcode lottery’ in healthcare, where access to treatments depended on where people lived, NICE’s methods have remained broadly unchanged for the past 20 years despite the increasing sophistication of innovative treatments within the modern medicine pipeline.
The work of NICE has been highly regarded by international healthcare bodies for the perceived robust methodology of its review process, and countries around the world have sought to learn lessons from England as they introduce their own health technology assessment (HTA) systems and guidelines. However, in the years since it was first established, NICE has failed to update its methods to keep pace with changes in the way drugs are developed and the kind of breakthrough medicines that are starting to make it to market to treat ever increasingly complex diseases. In this era of precision medicine, where a medicine might only work in a small percentage of patients, but work exceptionally well, NICE’s methods are falling short.
The Institute of Cancer Research recently carried out a study which showed that innovative new cancer drugs are taking much longer to reach patients in England than conventional drugs. [i] Highly innovative cancer drugs take a mean of 14.3 years to progress from the patent priority date to availability on the NHS, compared with 11.1 years for low innovation drugs. [i] To compare this with our European neighbours, patients in France and Germany are five times more likely to get access to a new medicine than patients in the UK. [ii]
Drug combinations are also very difficult to assess fairly through the current appraisal process, and as drug development becomes more sophisticated, clinical endpoints other than overall survival may provide greater insights into value and measuring quality of life. New innovations such as gene and cell therapies and personalised medicines may also face hurdles when it comes to the appraisal process, because the current review method cannot necessarily appreciate the value and cost effectiveness of treatments of this kind. As a result, the current HTA process is no longer fit for purpose and urgently needs to evolve. The bottom line is that patients across England need to be given faster and earlier access to the best innovative medicines, in line with patients across Western Europe.
What is already being done to future-proof the appraisal process?
NICE itself recognises that the healthcare system in England has evolved and that new technologies are now more complicated to evaluate. After announcing the NICE evaluation and methods review, Sir Andrew Dillon, NICE chief executive, said: “NICE is undertaking this review at a time of unprecedented change in the healthcare system, where developments such as personalised medicine, digitalisation of health, and use of cell and gene therapy, mean products are becoming ever more challenging to evaluate.” [iii] The outcomes of this review are currently anticipated at the end of 2020. [iv]
Quality-adjusted life years (QALYs) – are they still relevant?
One of the major shortcomings of the current assessment system is the reliance on the inflexible and outdated quality-adjusted life year (QALY) measurement for deciding the cost-effectiveness of drugs. The QALY contains two components: how much extra length of life taking a medicine gives a patient, together with an estimate of the quality of that life. This QALY system was originally intended to manage NHS spending on ‘blockbuster’ drugs – usually conventional medicines for long-term illnesses, such as high cholesterol and high blood pressure, affecting large numbers of the population. However, over the past few decades, healthcare has moved away from a reliance on blockbuster drugs and is evolving towards the development of more targeted combination therapies that treat complex diseases with smaller patient populations, such as rarer cancers. Such modern medicines are clearly delivering value beyond what NICE is currently measuring, and hence the QALY should not be the only factor in NICE decision making.
Gaining a broader view
Drug appraisal processes need to reflect the broader value of treatments to patients such as the wider ‘societal’ impact, rather than relying on a crude method of cost-effectiveness analysis.
Under the current NICE appraisal system, wider economic impacts associated with a health intervention, such as carer costs, unemployment, and the burden of disease to society are not taken into account. As a result, the true value of an innovative medicine that either a) alleviates costs in other parts of the health and social care system or b) improves outcomes for patients and those around them, is not accurately assessed and patient access may be jeopardised.
Nobody understands the impact and burden of a disease better than the patient, which is why patient advocacy groups need to be more involved in consultations surrounding the value of treatments to ensure that the wider economic costs and patient-relevant benefits associated with a health intervention are taken into consideration by NICE during appraisals.
Drugs that are ‘too effective’
State-of-the-art treatments are transforming the outlook for patients, particularly for those with cancer. The pace of innovation in oncology medicine is rapid and new technologies such as targeted medicine, immunotherapy and advances in radiotherapy have shown positive results in terms of survival and quality of life for patients.
However, some of the new generation of oncology treatments have been turned down by NICE. This is because, ironically, a drug can often be ‘so effective’ that the long-term survival data required by the NICE HTA process cannot be captured within the limited timeframe of a clinical trial. The data is seen to have too much ‘uncertainty’ because a high number of patients have survived, so mortality data is limited. The parameters through which NICE determines uncertainty therefore need urgent re-evaluation, as clearly having more patients live longer is a positive thing and should not be penalised.
How can NICE address uncertainty when assessing new treatments?
Uncertainty can be addressed in two different ways, or a combination of both: through further data collection (for example through patient registries), or a flexible pricing arrangement, such as an outcomes-based pricing agreement.
NICE should for example build into its decision-making framework a more flexible criterion for evidence that could be considered ‘uncertain’, including unpublished information, international evidence and all real-world evidence gathered during a potential conditional approval period – particularly where evidence from randomised control trials is limited or unavailable. NICE should seek to align its criteria for evidence with that used by regulators, such as the European Medicines Agency (EMA). It is also important to ensure that, alongside companies, patient groups are involved in the designing of conditional approvals of new treatments, to ensure the data being gathered during this period accurately reflect patient-relevant outcomes and address the uncertainty.
Is ‘flexible pricing’ the future?
As healthcare evolves, the current rigid pricing model used by NHS England also needs to evolve. The current ‘one drug, one price’ system does not accurately reflect the multi-indication nature of most modern medicines, particularly those used in cancer. There are instances where some patients can access treatments, while others who would benefit from the same treatment, but for a different condition or indication, are being denied because the treatment has not been recommended by NICE due to a lack of flexible pricing.
NICE and NHS England must now accept a more flexible pricing model where different prices are set for each major indication approved for a medicine, based upon the value demonstrated in clinical trials and real-world settings, which would in turn allow patients to receive access to the latest wave of innovative oncology medicines. The current methods review presents a real opportunity for NICE to implement fundamental reforms that would enable it to keep up with the pace of change in health technology. This will support patients getting access to new medicines, enable the NHS to secure value for money, and ensure the UK remains a world leader in pharmaceutical research and development. Will NICE rise to the challenge?
Jennifer Lee is EMEA Therapy Area Market Access Leader at the Janssen Pharmaceutical Companies of Johnson & Johnson, and Janssen UK representative on the NICE Methods Review.
References
[i] E Sharpe, R Hoey, C Yap and P Workman. From patient to patient: analyzing access to innovative cancer drugs. Drug Discovery Today (2020). Available at: https://doi.org/10.1016/j.drudis.2020.01.004. Last accessed April 2020. [ii] ABPI. Why is it important to improve access to medicine in the UK? Available at: https://www.abpi.org.uk/new-medicines/medicine-pricing-in-the-uk/why-is-…. Last accessed April 2020. [iii] NICE. NICE announces details of health technology evaluation methods review. Available at: https://www.nice.org.uk/news/article/nice-announces-details-of-health-te…. Last accessed April 2020. [iv] NICE. Changes we’re making to health technology evaluation. Available at: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/ni…. Last accessed April 2020.Job code: CP-151528. Date of preparation: April 2020. This article has been sponsored and developed by Janssen-Cilag Limited.
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