The value conundrum

The precedent set by COVID-19

At the onset of the pandemic, the UK research community came together to develop multi-agency systems to accelerate the set-up and delivery of high priority research. This required government bodies, clinical academic research experts, health authorities, regulators and the life sciences industry to work together in new and extraordinary ways.

A prime example of this collaborative approach was the NHS Health Research Authority’s (HRA) implementation of a fast-track approval process for COVID-19 studies. This process meant that its Research Ethics Committee and approval specialists could complete a full review of a study into specific study details, research methodologies and trial designs in less than 72 hours, down from a typical 40 days.

The Medicines and Healthcare products Regulatory Agency (MHRA) also adopted a more flexible and pragmatic approach, supporting the set-up of clinical studies and the execution of approvals in a matter of weeks rather than the usual months, whilst upholding all essential regulatory and ethical standards.

The way these bodies responded to COVID-19 clearly demonstrated their ability to accelerate research delivery in response to an urgent public health challenge. The question now is whether we can adopt some of these extraordinary ways of working in a more sustainable way, so that they become part of our ‘business as usual’ approach to medical innovation.

If we can maintain and build on even a fraction of what has been achieved over the last year, then the UK has the potential to be the go-to country for clinical research for years to come.

Keeping pace with scientific advances

While COVID-19 has placed the importance of medical innovation under the spotlight, it is not the only disease area where the UK healthcare system has shown flexibility to enable rapid access to the latest medical technology.

The advances in genetics as a result of the Human Genome Project have transformed the way that patients are treated across a vast array of disease areas, including HIV and cancer. Individualised and targeted therapies, along with new manufacturing and quality control processes, have made it increasingly possible to treat conditions that were considered almost untreatable not long ago. And for many patients this has meant significant improvements to their quality of life.

One example of this is CAR-T therapy – a pioneering individualised medicine that re-engineers a patient’s own immune cells at a genetic level to identify and attack cancer. Recognising the potential of this treatment, the NHS, the National Institute for Health and Care Excellence (NICE) and industry worked together to ensure that UK patients were among the first in Europe to access CAR-T in 2018.

Now, the pandemic has demonstrated how much can be achieved when everyone works in a flexible and agile way, in the face of a health emergency. We should harness this new found sense of collaboration to look at the other urgent health crises our society faces – the fight against cancer is one such example. As with COVID patients, cancer patients who would benefit from latest innovations have conditions which, if left untreated, will only worsen with time. As such, we must continue the nimbler ways of working, to ensure we continue to prolong and improve as many lives as possible.

‘We must use this opportunity to reform our health technology assessment methods so that we can properly value innovative therapies, particularly those targeted at smaller patient populations’

What could change look like?

It is important that we continue to incentivise investment in innovation in the UK if we wish to remain leaders in this field, particularly in a post-Brexit landscape. But we must also look to collaborate on how we determine the value of medicines; continuing to think in terms of the wider benefit to patients and society, rather than focusing on a purely cost-driven approach. This will help us sustainably balance the ongoing patient demand on the NHS, against the budgetary issues that we will no doubt face as a country.

Gilead’s belief is simple: that all patients should have access to the treatment that is most appropriate for them. Access to treatments depends on a broad range of factors.

The current NICE appraisal process, which assesses the clinical and cost-effectiveness of treatments for use in the NHS, was created two decades ago at a time when mass-produced treatments for large patient populations were the norm. But as medical technology has improved, so has our ability to target increasingly specific groups of patients and produce treatments for them. CAR-T therapy outlined above is just one example of this.

NICE is aware of these developments and has reflected this in its methodology over the years. It has, for example, allowed more flexible criteria for end-of-life cancer drugs. But its overall approach has not seen much change since its conception.

As the latest generation of therapies is increasingly tailored to the individual patient, the value they generate cannot be measured in the same way. To gauge the true significance of these treatments to the system, we need to understand their value to the patient – including the impact on a patient’s quality of life and ongoing health. With the NICE methods review well under way, the opportunity to make this change is now upon us.

The way in which NICE can support innovation of new treatments and reach those who need them most, is fourfold:
Firstly, at present the discount rate for health benefits is set at 3.5%, meaning the government currently puts a lower value on the long-term effects of health technology that is being developed today. Reducing this to 1.5% would ensure that transformative treatments and potential cures in areas of high unmet need, such as cancer and vascular dementia, are correctly valued.

Secondly, the introduction of a severity modifier to assess whether or not a treatment provides value for money could pave the way for UK patients to have access to the latest healthcare technology.

Thirdly, we must accept uncertainty. Innovative new treatments will inevitably come with a degree of uncertainty and the NICE consultation proposal addresses this fact by suggesting there should be more flexible approaches to interpreting uncertainty in defined circumstances. There should be no balancing areas where uncertainty is used as a negative modifier.

Finally, it is crucial to strengthen the patient and clinician voice in this evaluation process. Their views are invaluable if we want to fully capture what it means to have treatment options where there is none, to live independently and have a better quality of life. Patient groups can provide valuable insights and data but this requires a more structured and inclusive approach to achieve a more rounded evaluation.

We must use this opportunity to reform our health technology assessment methods so that we can properly value innovative therapies, particularly those targeted at smaller patient populations. We must ensure the process clearly identifies their full value, facilitating decisions that lead to the appropriate use of innovative medicines. We are delighted that this need has already been recognised by NICE, political stakeholders and regulatory bodies, but there is still work to be done.

We also need to ensure that the process aligns with the government’s ambition for the life sciences sector in the UK. In the wake of Brexit, the eyes of the world are upon us, and the decisions taken now will certainly shape our life sciences landscape in the years and even decades to come.

Getting this right will not only benefit patients and the NHS, it will also be a major global statement of the UK’s ambition to remain a leader in life sciences, in a world that has already changed – and continues to do so.