This new treatment combination is being made available via the CDF on the NHS for the first time following a recommendation by the National Institute of Health and Care Excellence (NICE). This decision follows an earlier draft negative decision by NICE in July last year.
Daratumumab, which is produced by the pharmaceutical company Janssen, belongs to a group of drugs known as monoclonal antibodies, which work in an innovative way by harnessing the body’s own immune system. You can read more about daratumumab in our Treatment Guide.
Under the CDF, treatments are initially approved for around two years and more data on their effectiveness are collected during this period. At the end of this time, NICE look again at the treatment and make a final decision on whether to approve it for routine use on the NHS.
Myeloma UK Head of Patient Advocacy, Shelagh McKinlay said: “This is really positive news and is a major step-change in the treatment available to patients at this stage in their myeloma.
“For too long there has been real unmet need at first relapse in myeloma. The limited treatment options, particularly for patients treated with bortezomib when newly diagnosed, has meant that patients were missing out at a critical time in their disease pathway.
“We are pleased to have contributed to this approval. We made sure the patient voice was heard by submitting evidence to NICE and encouraging decision makers to understand what access to this treatment means for patients and their families. We commend NICE, NHS England and Janssen for working together to deliver a positive outcome.”
Jeanette Ashworth, 59, is a former specialist nurse who was diagnosed with myeloma in 2015. Following a stem cell transplant Jeanette has been in remission for three years but recently learned that her myeloma had returned. Jeanette said; “This news is extremely important and gives patients at first relapse a chance to approach their myeloma with a treatment that works in several different ways.
“At last we have a new treatment that can be given early in the myeloma journey.”
What does the approval mean for patients?
Only patients at first relapse (also known as second line) will be eligible to receive daratumumab, bortezomib and dexamethasone.
When NICE consider whether to approve a treatment, they must consider the point in the treatment pathway it will be used. All approvals for myeloma treatments include conditions on which patients are eligible to receive the treatment. For example, carfilzomib (Kyprolis®) is currently only available to patients at first relapse.
Janssen applied for daratumumab, bortezomib and dexamethasone to be approved at first relapse only because clinical trial results show that it is particularly effective in this patient population and because there was significant unmet need at this point in the treatment pathway.
If you are a patient at first relapse, there may be reasons why you may not be eligible to receive daratumumab, bortezomib and dexamethasone, similar to the restrictions placed on entry into clinical trials. Your doctors will discuss this with you if applicable.
Does this decision apply to all patients in the UK?
The decision affects myeloma patients living in England and Wales.
The Scottish Medicines Consortium (SMC) will shortly consider a submission for the approval of this treatment combination. Myeloma UK will contribute to this process as it has done with NICE and will do all we can to help deliver a positive decision. In Northern Ireland, the Department of Health, Social Services and Public Safety, recently announced that it would apply its usual decision making process to drugs approved by NICE via the CDF. The Department decides whether to fund a new treatment following publication of the final NICE decision.
What is the Cancer Drugs Fund (CDF)?
The Cancer Drugs Fund is a pot of money which enables NICE to conditionally approve promising treatments while more data are collected about the treatment’s clinical effectiveness and value. This period of data collection is usually around two years. At the end of this period NICE reconsider the treatment and make a final decision.
The Fund is for patients in England but Wales and Northern Ireland have processes in place to enable CDF decisions to be implemented. The system is different in Scotland and there is no direct equivalent of the CDF. However, Scotland does have the New Medicines Fund which covers myeloma.
The CDF is an increasingly common route for funding new cancer treatments. This is now the third CDF approval for myeloma. Previous approvals were for ixazomib (Ninlaro®), lenalidomide (Revlimid®) and dexamethasone in December 2017 and daratumumab monotherapy in January 2018.
Why is daratumumab, bortezomib and dexamethasone being funded via the CDF?
Based on the clinical trial data, NICE agreed that daratumumab, bortezomib and dexamethasone was a “step-change” in treatment, because of the additional remission it delivered in clinical trials (a median of 27 months compared with a median of 8 months delivered by bortezomib and dexamethasone alone). NICE have also previously recognised that there was a real unmet need at first relapse in the myeloma treatment pathway.
However, NICE concluded that the data on how much longer patients lived in total as a result of the treatment (known as “overall survival”) was unclear because the data had not been collected over a long enough period of time. For this reason, they recommended that the treatment be made available through the CDF so that a final decision could be made based on more mature data.