Carfilzomib (Kyprolis®) has been approved for use on the NHS in England and Wales in combination with dexamethasone by drug approval body, the National Institute for Health and Care Excellence (NICE).
The approval applies only to myeloma patients who have had one prior treatment, as long as that treatment did not include bortezomib, which is also known as Velcade®.
It means that the combination of carfilzomib and dexamethasone will be available immediately to eligible patients via interim funding arrangements through the Cancer Drugs Fund in England. NHS bodies in Wales must also make the combination of carfilzomib and dexamethasone available to eligible patients in the next three months.
The submitting pharmaceutical company, Amgen, had originally requested that NICE fully approve carfilzomib for all myeloma patients at first relapse, however it has been approved in a restricted setting. The approval overturns the draft negative guidance NICE issued earlier this year. You can find out more about the decision in the NICE Final Appraisal Document (FAD).
Alongside this positive guidance, NICE also turned down the triple combination of carfilzomib, lenalidomide (Revlimid®) and dexamethasone for relapsed patients.
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Carfilzomib, also known as Kyprolis, belongs to a group of treatments known as proteasome inhibitors and works in a way very similar to bortezomib. For more information, you can read our Kyprolis Infosheet.
Carfilzomib has a European licence for both indications that were assessed by NICE. Both the licence and NICE applications were made on the basis of two large Phase III trials which found that carfilzomib extended the average length of progression free survival (i.e. the length of time patients were in remission) in relapsed myeloma patients compared to the current standard of care.
The reason behind this restriction is complicated. Whilst the company aimed for full approval of carfilzomib and dexamethasone, due to the current myeloma pathway in England, this was not possible.
NICE consider how clinically and cost effective a treatment is compared to the other standard treatments that are available on the NHS in that setting. In this instance, the treatment used as a comparison is bortezomib. However, there is currently a restriction on the use of bortezomib on NHS which means those who have previously had this treatment at first relapse cannot have it again. This means that carfilzomib must be compared under the same restriction, and focus only on first relapse patients who have also not received bortezomib, in order to prove that the treatment is cost-effective.
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We understand why NICE need to approach the appraisal in this way given their remit. However, it is not a sustainable or acceptable situation that patients are unable to access effective new treatments because of limitations in the current myeloma treatment pathway. Myeloma UK will work with all the key players, including NHS England, to deliver a more strategic and “common sense” approach to designing the myeloma treatment pathway.
While NICE did accept that the combination is proven to keep patients’ myeloma under control for longer than the comparator treatments, it did not find the combination to be a cost-effective use of NHS resources. One of the main issues is that carfilzomib was being appraised with two other treatments, including another “novel” treatment, lenalidomide, which makes the combination less cost-effective.
Myeloma UK will continue to seek solutions to the challenges of securing approval for triplet combination treatments.
This decision affects patients in England and Wales. The Scottish Medicines Consortium previously issued a “no” to carfilzomib, lenalidomide and dexamethasone. It is currently considering a submission for the approval of carfilzomib and dexamethasone alone. In Northern Ireland, the Department of Health, Social Services and Public Safety usually make a decision on whether to fund a new treatment following publication of the final NICE decision.