Lenalidomide (Revlimid®) maintenance for myeloma patients who have undergone high dose therapy and stem cell transplantation (HDT-SCT) will be considered for use on the NHS by The National Institute for Health and Care Excellence (NICE) later this year.
This announcement comes after updated advice from the manufacturer Celgene and follows publication of data from the Myeloma XI clinical trial. The appraisal is expected to begin in early December 2019 after “scoping” (clarification of the key issues to examine) is complete. Currently the estimated timescale for a decision is October 2020.
A positive decision from NICE means that lenalidomide maintenance could be offered to over 1300 myeloma patients who undergo HDT-SCT every year. Clinical data shows lenalidomide maintenance after HDT-SCT can improve both progression-free survival (the length of time following treatment before myeloma returns or further treatment is required) and overall survival. Therefore access to this treatment through the NHS has the potential to improve myeloma patient survivability.
Head of Patient Advocacy, Shelagh McKinlay said:
“We recently met with Celgene as part of our ongoing efforts to move forward with this appraisal and we are pleased that a public commitment to submit has now been made.
“However, the system has not been moving fast enough. Every month of delay means patients are missing out.
“Patients in the UK want and deserve access to this treatment. We will keep doing all we can to make sure that the patient voice is heard and to press for swift progress.”
What is maintenance treatment?
Maintenance treatment is the technical term for myeloma treatment which is administered long-term and intended to sustain control of the myeloma and reduce the risk of disease progression. Maintenance treatment is similar to “treat to progression”, and sometimes the terms are used interchangeably.
In this instance maintenance treatment refers to a low dose of long-term treatment given after HDT-SCT.
What is the likely timescale for NICE reaching a decision?
NICE cannot set absolute timelines for all stages of its appraisal process since this depends on each particular appraisal. However, the recent NICE update states that the first committee meeting to consider the appraisal will be on 13 August 2020. NICE currently estimate that a decision would be published in October 2020. However, these deadlines can be subject to change, depending on a range of factors including whether more than one committee meeting is needed.
The timelime for carrying out an appraisal like this at NICE can be found here. It sets out the different stages in the process and includes times for consultation with stakeholders, including organisations like Myeloma UK.
Why is NICE considering lenalidomide maintenance only following a stem cell transplant?
Before a treatment can be used in the UK it must receive a licence, or marketing authorisation. Most treatments are granted a marketing authorisation by the European Medicines Agency (EMA). The only EMA licenced use of lenalidomide maintenance is post HDT-SCT.
In addition, it is worth noting that clinical trial data, including from the Myeloma XI trial, showed that the treatment delivered greater benefit to patients who had undergone a stem cell transplant. Specifically, that there was a significant improvement in overall survival, which was not present for patients who had not had a stem cell transplant.
Why has the timescale been slow?
The use of lenalidomide maintenance for the treatment of myeloma patients following HDT-SCT has been licenced in Europe since 2017 but Celgene advised NICE that they did not have the data needed to make a submission. In addition, discussions on a new commercial deal for lenalidomide, which began in early 2018, were concluded only recently and it was not possible to make progress on a submission for maintenance treatment until this was resolved.
Seeking approval for any treatment is a complex process and maintenance treatment is particularly challenging. It is also important to make sure that the NHS gets value for money and that treatments really deliver what patients need.
NICE has an ambition for appraisals to start before a product’s licence is granted and for the first committee meeting to take place as soon as possible after that. However, this isn’t always possible and depends on a range of factors including whether the company is ready to make a submission to NICE.
What are the next steps for Myeloma UK?
Myeloma UK will keep pressing for faster progress, encouraging Celgene, NICE and NHS England to keep patients at the forefront of their mind and move as quickly as possible.
Over the next couple of months we will also reach out to patients and families to gather evidence and insight into the benefits of lenalidomide maintenance following a stem cell transplant so we are ready to make the best possible case when the appraisal begins.