NICE issues draft ‘no’ for lenalidomide maintenance

Myeloma UK news, Patient advocacy news // 8th September 2020

The National Institute for Health and Care Excellence (NICE) has issued a draft ‘no’ on the use of lenalidomide (Revlimid®) maintenance for the treatment of newly diagnosed myeloma patients following high-dose therapy and stem cell transplantation (HDT-SCT).

This draft decision means that, as the submission currently stands, NICE will not recommend that this treatment be made routinely available on the NHS in England, Wales and Northern Ireland. The decision is now out for consultation and available for review and comment by stakeholders.

The appraisal committee agreed that lenalidomide maintenance extends life and remission time compared with monitoring alone. The committee also agreed that the side effect profile of the treatment was acceptable, referring to the survey evidence presented from the Myeloma UK Patient Treatment survey conducted in February of this year.

Part of the NICE assessment also involves the consideration of NHS resources, and the committee reported that the assessment undertaken by NICE had raised some uncertainties around the long-term economic projections for the treatment.

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

“While this decision is not final and there is still the possibility of approval, this is hugely disappointing news.

It is three years since lenalidomide maintenance was approved for use throughout Europe and it is standard practice internationally. The results from Myeloma XI, the biggest myeloma clinical trial ever conducted in the UK, were published in December 2018 – and yet patients and their families and friends are still waiting. They have been waiting too long and must not be left behind.

Every year around 1400 myeloma patients in the UK undergo HDT-SCT. As long as this treatment remains unavailable on the NHS more than 100 patients every month are missing out on the opportunity to live longer with a better quality of life.

There is no doubt about the clinical benefit that this treatment delivers. It would be appalling if patients were denied time with their loved ones because agreement cannot be reached.

We call on the company, Celgene/Bristol-Myers Squibb (BMS), NICE and NHS England to do everything in their power to resolve the issues raised and deliver a yes for myeloma patients.

We will keep doing all we can to help make this happen.”

Steve Billcliffe, a patient expert, also presented narrative evidence to the committee. He said:

“Whilst I am very disappointed by the overall decision I was pleased to see that NICE have recognised that the results of the clinical trial that I have been taking part in show that lenalidomide increases how long people with myeloma live and extends the time before their condition gets worse.”

I hope that the technical and economic questions raised by NICE can be answered swiftly by the manufacturers of the drug so that this disappointing appraisal decision can be amended and my successful treatment can be approved for newly diagnosed myeloma patients in England, as is the case in other countries in Europe and around the world.”


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 positive final approval.

Myeloma UK will continue to work with clinicians, the manufacturer, and with NICE and will respond to the consultation, making the case for lenalidomide maintenance to be approved. Once NICE has gathered views from all stakeholders (the deadline is 29 September), 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?

NICE have recognised the clinical benefits which have been demonstrated for lenalidomide maintenance in its clinical trials, including Myeloma XI. It highlighted that this would address the unmet need for maintenance treatment following HDT-SCT.

Part of the NICE assessment for all treatments includes consideration of economic data as well as clinical data. This means that the price agreed for a treatment is also an important factor in whether NICE finds a treatment to be an effective use of NHS resources.

Is it likely to be approved?

During the consultation period the manufacturer, clinicians and Myeloma UK will be able to submit further evidence to address NICE’s concerns. The manufacturer 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 maintenance treatment 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 manufacturer to make a case for its approval.

Who does this decision affect?

Around 1400 patients receive HDT-SCT in the UK every year. If the treatment is not approved then over 100 people every month will miss out on the opportunity to live longer with a better quality of life.

Analysis from our Patient Treatment Survey conducted in February of this year shows that patients feel lenalidomide maintenance is well tolerated and provides exceptional clinical benefit. For more information of our survey results please see our blog post ‘Patients are positive about the benefits of lenalidomide maintenance’.

What about patients based in Scotland?

Lenalidomide Maintenance 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 or call the Myeloma Infoline on 0800 980 3332 during office hours, Monday to Friday.