New treatments for myeloma continue to come through the Health Technology Appraisal (HTA) pipeline.  At the moment, there are four active appraisals at the National Institute for Healthcare Excellence (NICE). 

Daratumumab (Darzalex®), bortezomib (Velcade®), thalidomide and dexamethasone induction treatment 

The quadruplet combination of daratumumabbortezomibthalidomide and dexamethasone is being appraised by NICE as an induction treatment for newly diagnosed myeloma patients before high-dose therapy and stem cell transplantation.  

Earlier this year, NICE issued a draft ‘no’ recommendation for this quadruplet combination. The committee recognised the benefits highlighted in the clinical trial but there were uncertainties in the longer-term data required to make the cost-effectiveness assessment as it doesn’t yet fully demonstrate the length of remission the treatment delivers, or how long it extends life overall. 

In July 2021, Myeloma UK took part in the second committee meeting to discuss the issues surrounding the appraisal. We are currently awaiting the final decision from this meeting.  

This treatment was approved for use in Scotland by the SMC in January 2021. This is the first quadruplet combination treatment for myeloma patients approved anywhere in the UK. 

Ixazomib (Ninlaro®), lenalidomide (Revlimid®) and dexamethasone 

The combination of ixazomib, lenalidomide and dexamethasone is being re-appraised. 

The combination was recommended by NICE through the Cancer Drugs Fund (CDF) in 2018 for patients who had received two or three lines of treatment.  

To support the appraisal Myeloma UK conducted a Patient Treatment Survey to capture the insight and experience of patients who have received this triplet combination. The results from this survey helped to inform our evidence submission which was submitted to NICE in April 2021.  

We are currently awaiting the technical engagement step of the NICE process and the committee meeting will take place later in 2021.  

Ciltacabtagene autoleucel (Cilta-Cel) CAR-T cell treatment 

This CAR-T cell treatment is being considered by NICE for patients who have received three or more lines of treatment or are double-refractory to an immunomodulatory drug and a proteasome inhibitor. 

Myeloma UK submitted evidence for this appraisal in May 2021 with the NICE committee meeting scheduled in November 2021. The final positioning for this treatment will be determined through the HTA process.  

Daratumumab (Darzalex®) in combination with bortezomib (Velcade®), cyclophosphamide and dexamethasone for AL amyloidosis 

The combination of daratumumab, bortezomib, cyclophosphamide and dexamethasone (Dara-CyBord), is being assessed by NICE for newly diagnosed AL amyloidosis patients.  

This is the first licenced treatment for AL amyloidosis to be appraised by NICE for use through the NHS in England and Wales.  

Myeloma UK submitted evidence for this appraisal in June 2021 and the committee meeting is scheduled to take place in December 2021.  

We are also expecting this treatment to be scheduled with the Scottish Medicines Consortium for AL amyloidosis patients based in Scotland towards the end of the year.   

The active HTA schedule is a welcome sign of the continuing advances in treatment in myeloma. We will continue to work with patients, their families and carers, and with clinicians to make the case for the approval of effective new myeloma treatments. We will also continue to update patients and healthcare professionals on the progress of these HTAs. 

More treatments (e.g.  selinexor, melflufen and belantamab mafodotin) should be coming through the HTA pipeline soon but as yet their appraisals have not been scheduled by NICE. 

 If you would like more information on HTA processes, any of the new treatments or expanded access programmes get in touch with the Patient Advocacy Team