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NICE issues draft ‘no’ for carfilzomib combination

Patient advocacy news // 22nd September 2020

The National Institute for Health and Care Excellence (NICE) has issued a draft ‘no’ for the triplet combination of carfilzomib (Kyprolis®), lenalidomide (Revlimid®) and dexamethasone for patients who have already received one prior treatment for their myeloma.

This draft decision means that, as it currently stands, NICE will not recommend that this triplet combination be made routinely available on the NHS as a treatment at first relapse (second line) in England, Wales and Northern Ireland. This decision is now out for consultation.

NICE assess whether a treatment is clinically and cost effective, compared to the current standard treatments.

The Committee agreed with Myeloma UK that more treatment options were needed and that this treatment delivered clinical benefit compared to current standard treatments. However, although six years of trial data was presented, NICE considered that uncertainty remained about the longterm impact of this benefit, making cost effectiveness estimates uncertain.

Two other issues impacted negatively on the treatment’s cost effectiveness:

  • While most myeloma patients at second line receive the combination of daratumumab (Darzalex®), bortezomib (Velcade®) and dexamethasone, this could not be used as the current standard treatment comparator because it is approved through the Cancer Drugs Fund (CDF) and may not be permanently available on the NHS. This meant that the carfilzomib triplet was compared to lenalidomide and dexamethasone alone
  • When a novel drug like carfilzomib is added to another novel treatment like lenalidomide it makes proving cost effectiveness more difficult

Commenting on the decision Laura Kerby, CEO of Myeloma UK, said:

“We are deeply disappointed in the decision not to approve this treatment, especially on top of the recent draft no issued from NICE for lenalidomide maintenance.

Today’s news is only one part of the appraisal process and during the consultation period we urge NICE, the company AMGEN, and NHS England to do everything they can to turn this into an approval for myeloma patients to have access to this beneficial treatment. Myeloma patients need as many treatment options as possible and the clinical benefit of this triplet is not in doubt.”

FAQs

What happens now?

NICE have issued the draft decision in an Appraisal Consultation Document (ACD) and will now ask questions that could turn the draft ‘no’ into a final approval.

Myeloma UK will continue to work with clinicians, the company and with NICE and will respond to the consultation, making the case for carfilzomib, lenalidomide and dexamethasone to be approved. Once NICE has gathered views from all stakeholders (deadline 13 October), it will meet again to consider its decision. The date for this meeting has not yet been decided.

For more information please see our blog on ‘What happens after a draft ‘no’?

Why did NICE say no?

The committee highlighted that clinical benefits of the triplet combination had been demonstrated and agreed with Myeloma UK for the need for more options at second-line of treatment.

However, although six years of trial data was presented, NICE considered that uncertainty remains about the long-term impact of the benefit which makes the cost effectiveness estimates uncertain.

The price of drugs is a factor in NICE decisions but it is important to bear in mind that they do not look at whether a drug is expensive, rather they look at whether it is cost effective; all NICE decisions are based both on the clinical effectiveness and cost effectiveness of the treatment being appraised compared to the current standard of treatment.

In clinical practice, most myeloma patients at second line receive the triplet combination of daratumumab, bortezomib and dexamethasone. This treatment is approved for use within the NHS through the Cancer Drugs Fund (CDF) which is used for funding novel cancer treatments when there is uncertainty around the clinical and cost effectiveness, and there is a need for more data collection.

Treatments approved through the CDF are not approved for routine commissioning and therefore cannot be used as a comparator in NICE appraisal for new treatments. In this case, it means that the carfilzomib combination is compared to the treatment of lenalidomide and dexamethasone alone which has significantly impacted on the cost effectiveness estimate.

In addition, it is challenging to get approval for triplet combination treatments when two novel drugs are used in the same treatment, particularly if they are not made by the same company and a new discounted price cannot be agreed for the triplet “as a whole”.

Can carfilzomib, lenalidomide and dexamethasone be approved through the Cancer Drugs Fund?

The committee considered this triplet combination for approval through the CDF, but as the data from the ASPIRE clinical trial was comprehensive it did not meet the criteria to be approved through the CDF.

Is it likely to be approved?

During the consultation period the company, AMGEN, clinicians, and Myeloma UK will be able to submit further evidence to address NICE’s concerns. The company is also able to come forward with new information which could address the uncertainties highlighted by the committee.

At the moment, we cannot be certain what the outcome will be. It is positive that NICE recognise the clinical benefits that this triplet combination can deliver, but there is still work to be done to resolve the concerns NICE have about its cost-effectiveness. We will continue to work with clinicians, NICE and with the company to make a case for its approval.

What about patients based in Scotland?

Carfilzomib, lenalidomide and dexamethasone is currently going through the approval process with the Scottish Medicines Consortium. Myeloma UK attended the SMC committee meeting that took place in August 2020 and the decision of the SMC is expected to be made public in October 2020.

Further information

If you have any questions or comments on please email policy@myeloma.org.uk or call the Myeloma Infoline on 0800 980 3332 during office hours, Monday to Friday.