New treatment appraisals continue to come through the Health Technology Appraisal (HTA) pipeline.
Myeloma UK is involved in five active appraisals and one appeal at the National Institute for Healthcare Excellence (NICE).

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

Ixazomib plus lenalidomide and dexamethasone has been routinely approved in England and Wales for myeloma patients who have received two or three lines of treatment.
The combination was originally approved through the Cancer Drugs Fund (CDF) in 2018.

Daratumumab (Darzalex®), bortezomib (Velcade®) and dexamethasone

The combination of daratumumab plus bortezomib and dexamethasone is being reappraised for myeloma patients who have received one previous line of treatment.
Myeloma UK is involved in this reappraisal and submitted evidence on behalf of patients last year. The NICE committee discussed the appraisal at a committee meeting in February 2023, and the draft guidance is expected in March 2023.

Isatuximab (Sarclisa®), pomalidomide (Imnovid®) and dexamethasone.

The combination of isatuximab, pomalidomide and dexamethasone is being reappraised for myeloma patients who have received at least two previous lines of treatment, including lenalidomide and a proteasome inhibitor.
Myeloma UK is involved in this reappraisal and will submit evidence on behalf of patients in April 2023. The NICE committee meeting is scheduled for July 2023.

Daratumumab (Darzalex®), lenalidomide (Revlimid®) and dexamethasone

The combination of daratumumab plus lenalidomide and dexamethasone is being appraised for newly diagnosed myeloma patients for whom high-dose therapy and stem cell transplantation (HDT-SCT) is unsuitable.
Following the first committee meeting in February 2023, NICE published draft guidance not recommending the combination. Although NICE acknowledged that DRD was effective, they could not recommend the treatment because the cost-effectiveness estimates were uncertain and above what they consider to be an acceptable use of NHS resources.
Myeloma UK will continue to be involved in this appraisal and will respond to the draft guidance on behalf of patients in March 2023. The next committee meeting is scheduled for June 2023.

Belantamab Mafodotin (Blenrep®)

Belantamab mafodotin monotherapy is being appraised for myeloma patients who have received at least four treatments and are refractory to at least one proteasome inhibitor, one immunomodulatory drug, and an anti-CD38 monoclonal antibody.
Myeloma UK is involved in this appraisal and submitted evidence on behalf of patients in October 2022. The committee meeting is scheduled for April 2023.

Ciltacabtagene autoleucel (Cilta-Cel) CAR-T treatment

NICE is currently considering this CAR-T cell treatment for patients who have received three or more lines of treatment or are double-refractory to an immunomodulatory drug and a proteasome inhibitor.
A NICE committee meeting is scheduled to take place in March 2023. 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

In December 2022, NICE did not recommend daratumumab plus bortezomib, cyclophosphamide and dexamethasone (Dara-CyBorD) for newly diagnosed AL amyloidosis patients in England and Wales.
This was a blow to patients in England and Wales because the highly effective combination treatment, the first bespoke treatment for AL amyloidosis, had been approved for use in Scotland.
Myeloma UK is appealing this decision and will present our case alongside the pharmaceutical company, Janssen, at the appeal hearing in March 2023.

We will continue to work with patients, their families and carers, and clinicians to advocate for the approval of effective, new treatments. We will also continue to update patients and healthcare professionals on the progress of these HTAs.

If you would like more information on HTA processes, upcoming treatment appraisals, or Myeloma UK’s access work, contact the Policy Team.