Last year, our Patient Advocacy Team submitted a response to the first consultation of NICE’s review of methods. This is an important opportunity for us to provide feedback to NICE on the challenges of getting blood cancer drugs approved for use in England. This blog explains what the Methods review aims to do and what this means for those affected by blood cancer.
Who is NICE and what do they do?
The National Institute of Health and Care Excellence (NICE) are the body that make recommendations about whether a treatment should be used on the NHS in England. NICE do this by doing a health technology appraisal (HTA); they look at how effective treatments are compared to other treatments, but also how much they cost compared to those other treatments (cost-effectiveness).
If a treatment is recommended by NICE, the NHS has to make this available to patients by a set deadline.
How do NICE work out if a treatment is cost-effective?
How well a treatment works is determined by working out how much extra life a person gains by having the new treatment, compared to the benefit they would get by having whatever is currently available. They also look at the quality of that extra life too; this is important as survival is not the only thing that is important to people who are ill. Their quality of life during that extra time alive is also important, such as whether they can get out of bed or socialise with family and friends. You can combine the extra life gained and the quality-of-life benefits of a treatment into one measure, used by NICE, called the Quality Adjusted Life Year (QALY).
To work out the cost of the benefits of a treatment, NICE then compare the number of QALYs gained by people treated with the new treatment with the number of QALYs gained by people on the old treatment. They also compare the cost of delivering each treatment. These numbers are all plugged into an equation which leaves you with a cost per QALY, i.e., how much you have to spend on the new treatment to get one year of good health.
But how do NICE decide whether the NHS should pay that amount for treatment? The NHS has a limited pot of money and so wishes to fund only those treatments which provide the biggest gain for the least amount of money. Therefore, usually NICE will only recommend a treatment that costs £20,000 or less per QALY gained by the person treated; in certain circumstances, this can increase (see later in the blog). In theory, by deciding not to fund treatments over this cost, money is then saved that can be spent on treatments elsewhere.
Why did NICE need to review the way it makes decisions?
NICE was committed to review how it makes its decisions in an agreement between the government and the Association of the British Pharmaceutical Industry, signed in 2018. This agreement aims to keep drug prices at a level that the NHS is able to afford. Making sure NICE is making accurate decisions with all the available evidence is an important part of this.
NICE is finding it more challenging to make these decisions recently as treatments are changing from what they are used to seeing. For example, the cancer world has moved away from general chemotherapy treatments to personalised medicines that target specific genes. This is only set to continue. NICE are not proposing that they change the cost per QALY in order to do this; treatments will still need to show they are more cost effective than the current treatment. However, they are open to other changes. Leukaemia Care, along with many others, have provided our thoughts to NICE on what these changes could be.
What are the challenges with the current process for blood cancers, and what have you suggested NICE do about this?
There are issues with the way NICE currently make decisions that have impacted upon the number of treatments approved for blood cancers in recent years. Some of the challenges we have experienced as a charity include:
- Treatments being presented to NICE before they have gone through all the phases of clinical trials. Clinical trials are the main source of evidence used to work out whether the treatment is effective or not, so they need to be well designed and as big as possible. If NICE only have small or early trials to look at, they are less certain about the benefits of the treatment and so less confident about approving it. Because NICE’s decision making relies heavily on evidence generated through big clinical trials, treatments for rarer blood cancers are disadvantaged. Some cancers are so rare that you cannot do a large study in them.
- We have called on NICE to make treatments available where they are uncertain, if they are not sure because a big trial can’t be done. This can’t be changed if the treatment is rare and so NICE needs to be more flexible, otherwise rare treatments will always be disadvantaged.
- NICE have indicated they will be more flexible when making decisions on treatments for very severe conditions, which we support. However, we have asked that rare diseases be considered in the most severe group, as a lack of treatments for rare illnesses makes them severe for the patients affected.
- NICE has a special process for treatments for rare drugs, which is much more flexible. However, the criteria to enter this process are too strict and no cancer treatment has yet qualified. We have called on NICE to review these criteria, so their processes do not discriminate against rare cancers.
- There is also a similar problem with treatments that only treat a certain group of people (e.g., if it is a drug that targets a specific genetic mutation that only some patients have); the smaller you make the group, the harder it is to collect information on that group.
- Again, we have asked NICE to be more flexible and accept that there will be some uncertainty as groups get smaller.
- NICE has proposed that their decision makers should be allowed to only approve treatments in certain groups that the treatment is most cost-effective in. For example, if a treatment is cost effective for all relapsed CLL, but is even more cost-effective in relapsed CLL patients with a specific genetic mutation, this proposal would mean it might only be made available in the group with a genetic mutation. We feel it should be made available to all groups where it is cost effective, benefitting as many people as possible.
- Leukaemia Care, as do many other charities, work hard to get involved in the work of NICE to ensure that patient views are represented in this process. The clinical trials often capture the impact of the treatment on quality of life, but the surveys don’t always capture everything that is important to particular groups of patients. Patient groups often work hard to provide some additional information to NICE, but it is not always clear what the impact of the information we provide is.
- NICE has said, in their consultation, that they are looking to take into account a wider variety of evidence, not just clinical trials. However, we are disappointed they did not include how they would use the evidence submitted by patient organisations in this and have asked them to do so.
Where can I read more about this review?
You can read our full response to this consultation here. We have also worked very closely with other groups on this project, including the Blood Cancer Alliance and Cancer52 (a group of charities focusing on rare and less common cancers). This helps us to have a united voice on issues that affect different groups of cancer patients. You can find out more about the review more generally on the NICE website here.
For more information about the NICE review, please contact our Patient Advocacy team on email@example.com