NICE issues draft “no” on daratumumab combination

NICE has issued a draft "no" on the novel triplet combination of daratumumab (Darzalex®) for the treatment of patients with relapsed or refractory myeloma.

Patient advocacy news // 12th July 2018

The National Institute for Health and Care Excellence (NICE) has issued a draft “no” on the treatment combination of daratumumab (Darzalex®), bortezomib (Velcade®) and dexamethasone for the treatment of patients with relapsed or refractory myeloma.

This draft decision means that, as it stands, NICE will not recommend that this novel triplet combination be made routinely available on the NHS in England and Wales.  However, this decision is now subject to consultation and there is still the possibility of a positive decision being reached.

Myeloma UK Chief Executive Rosemarie Finley said: “This draft negative recommendation is obviously very disappointing for myeloma patients in England and Wales.

“However, we are pleased that at this stage the Committee has recognised evidence from Myeloma UK and leading clinicians that this is a “game-changing” treatment, which delivers significant and clinically important survival benefits for patients.”

“It is therefore vital that NICE and the company explore every possible option to deliver a positive outcome. We will also continue to do everything we can to secure a “yes” for myeloma patients and their families.”

Daratumumab, which is marketed by the pharmaceutical company Janssen, is being appraised by NICE in combination with bortezomib and dexamethasone for patients at their second line of treatment (also known as first relapse).

Daratumumab 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 Horizons Infosheet.


What happens now?

NICE have issued the draft guidance in an Appraisal Consultation Document (ACD) and will now ask questions to gain any information that could help turn the draft “no” into a final approval.

Myeloma UK will continue to work with the company and with NICE and will respond to the negative draft ACD, making the case for daratumumab to be approved.

Once NICE has gathered views from all stakeholders (deadline 2 August), it will meet again to consider its decision. The date for this meeting has not yet been decided.


Why did NICE issue a draft ‘no’ on daratumumab, bortezomib and dexamethasone?

NICE have recognised the benefits which have been demonstrated for daratumumab in clinical trials. The ACD states that it has a “statistically significant and clinical important effect” on progression free survival (that is the time that it keeps myeloma under control). NICE have also agreed that daratumumab is an innovative treatment which “is a step-change” in the treatment of people with multiple myeloma.

However, NICE must consider whether the benefits of treatments can be judged a cost effective use of NHS resources. At the moment, their estimates of daratumumab, bortezomib and dexamethasone’s cost-effectiveness are above the threshold that they use to judge whether a treatment should be approved.

This is based on a range of factors. For example, NICE consider that the company have overestimated survival benefits for this treatment and underestimated the survival benefit of the key comparator treatment.

During the consultation period the company, 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 a revised discount scheme which can make the treatment more cost effective.


What does it mean that the treatment is being considered for use at “second line”?

When NICE consider whether to approve a treatment this involves considering at what point in the treatment pathway it should be used. All approvals for myeloma treatments state which patients are eligible to receive the treatment. For example, carfilzomib is currently only available to patients as a second line of treatment. (This is also described as first relapse.)

The company proposed that daratumumab, bortezomib and dexamethasone should be considered by NICE at second line because clinical trial results show that it is particularly effective in this patient population and because there is an unmet need at this point in the treatment pathway.

If the treatment is approved it would be available only to patients at second line.


Is daratumumab likely to be approved on the NHS?

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 and with the company to make a case for its approval


Who does this decision affect?

The decision affects myeloma patients living in England and Wales.

The Scottish Medicines Consortium (SMC) has not yet assessed daratumumab, bortezomib and dexamethasone. 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.


Further information

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