01 December 2016
By Joseph Clift, Senior Policy Officer at Asthma UK
For the first time, people in England with one type of severe asthma will have a new treatment targeted at reducing their risk of a potentially life-threatening attack.
Mepolizumab, also known as Nucala, has today been recommended by the National Institute for Health and Care Excellence (NICE) as an add-on therapy for adults with severe eosinophilic asthma.
Highlighting the unmet need
It’s certainly not been a simple process. Just over a year ago, Asthma UK submitted written evidence to NICE making the case that people with severe asthma were in urgent need of new, targeted therapies. In March one of our volunteers, Lehanne, spoke at the first appraisal committee meeting, highlighting the stark challenges of living with severe asthma.
While NICE accepted early on that there was a need for new treatments, it would not accept the cost of making this available – based on the price agreed with the company, and how the overall cost was modelled for the target population. Two further rounds of consultation and committee hearings were needed before NICE and the company reached agreement.
What this means for people with severe asthma
Of the 5.4 million people with asthma in UK, an estimated 3.6% to 5.0% - roughly 250,000 adults and children - have severe asthma. Until now, omalizumab (Xolair) has been the only specialist treatment available across the UK to people with severe asthma, but this isn’t effective for all types of the condition.
Mepolizumab will target severe eosinophilic asthma - where the inflammation of the airways is linked to a particular type of white blood cell (eosinophils). We believe that around 40% of people with severe asthma will have an eosinophilic phenotype – meaning that they may be able to benefit from the new treatment.
As the results of our new pilot survey from earlier this week highlighted, severe asthma can have a devastating effect. The addition of mepolizumab means that some will now have access to a treatment that could lower their risk of having an asthma attack, and reduce the need to be treated with oral corticosteroids.
Why has it taken so long?
We know that mepolizumab is a treatment many of our supporters have been waiting patiently throughout the year to see approved. Many have already been told by their clinicians that they could be eligible to receive it, and for those people today’s news will no doubt come as a huge relief.
Mepolizumab is one of a number of new therapies that have been developed targeted at severe eosinophilic asthma, and a number of issues have delayed its path. NICE has a clear set of criteria against which they assess cost-effectiveness for a new treatment, and whether the NHS can afford to fund it. These are expensive therapies, and if a person responds well to the treatment they could be on it for a long period of time. This has been back and forth as those involved have debated how to best identify the people that would receive the treatment, how long they might be on it, what price the company is charging, and what measures would be in place to withdraw it if it’s not proving to be effective for an individual.
The numbers have been crunched repeatedly through this process, considering the benefits resulting from reducing exacerbations and need for oral corticosteroids against the cost of the drug, and this has at last reached a level where NICE can recommend. The treatment should become available within three months of today’s decision.
What have we learnt?
Research takes time, and a lot of resources, to take a new treatment from the lab to the patient. The road can also be a bit bumpy. Mepolizumab was first registered as a new drug in 1997, with trials in patients with moderate asthma two years later. While the trials demonstrated that the drug could lower blood eosinophils, it didn’t result in overall clinical benefit. This meant mepolizumab was almost abandoned as a potential asthma treatment. As overall understanding of asthma has increased, it was then trialled again in a more targeted population (severe eosinophilic asthma), where it was shown to reduce exacerbations – which led to it ultimately being approved today.
Targeting the right population for mepolizumab is as essential now for clinicians as it was to the researchers trialling it. The treatment, like omalizumab, will not be effective for everyone. Establishing exactly who might respond to these treatments can be tricky, and confounded by a number of different factors. This is why these will be accessible through specialised asthma services – where diagnostic tests and multi-disciplinary clinical teams can help establish whether an individual is likely to benefit.
We hope that mepolizumab is just the first of a new line of treatments available targeted at severe eosinophilic asthma – another treatment, reslizumab, is currently being assessed by NICE. We also need to ensure that today’s decision is followed up with mepolizumab becoming available in Wales and Northern Ireland. But there is still much that needs to be done.
Diagnosing severe asthma is complicated and the process can be extremely stressful for people with severe asthma. Better diagnostic tests are needed to help ensure that people can have a confirmed diagnosis quickly, so that appropriate treatments can be offered.
We also need more investment in research. While today is great news for some people with severe asthma, the majority still do not have effective treatments – and the drug pipeline for this group is far less developed.
Mepolizumab’s availability also highlights the importance of ensuring that services for people with severe asthma are fit for purpose, so that high quality specialist services can be accessed by those that need them wherever they live.
Read about what this decision means for those living with severe asthma in a blog post by Kay Boycott, Asthma + Lung UK CEO.