The year so far has been particularly busy for the myeloma access pipeline with four new myeloma treatments being appraised by both drug approval bodies, the National Institute for Healthcare Excellence (NICE) and the Scottish Medicines Consortium (SMC).
There is also a new treatment for patients with AL Amyloidosis which has begun the appraisal process with NICE.
The appraisal work with NICE continued over the COVID-19 pandemic as anti-cancer treatment was a therapeutically critical topic. This meant appraisals affecting England, Wales and Northern Ireland have continued to be assessed.
In Scotland, the SMC paused its work programme in March due to several clinicians, pharmacists and other healthcare professionals being redeployed to help with the COVID-19 pandemic. Thankfully, the work of the SMC has restarted and they are prioritising anti-cancer treatments as they work through their backlog.
Isatuximab (Sarclisa®), pomalidomide (Imnovid®) and dexamethasone.
The combination of isatuximab, pomalidomide and dexamethasone is currently being considered for myeloma patients who have received at least three previous lines of treatment including lenalidomide and a proteasome inhibitor (bortezomib (Velcade®), carfilzomib (Kyprolis®) or ixazomib (Ninlaro®)).
Myeloma UK submitted evidence for the appraisal in November 2019 and took part in the NICE committee meeting to discuss and review the clinical and cost-effectiveness in May 2020.
Unfortunately, NICE have issued a draft ‘no’ for this triplet combination. The committee recognised the benefits highlighted in the clinical trial but uncertainty remains over the level of benefit it delivers which affects the cost effectiveness.
Myeloma UK took part in the second committee meeting to discuss the issues surrounding the appraisal in July 2020. We are currently awaiting the final decision from this meeting. The final positioning of this treatment will be determined as part of the appraisal.
The triplet combination has been scheduled for appraisal at the SMC with Myeloma UK invited to submit a Patient Group Evidence Submission in November 2020. The Patient and Clinician Engagement (PACE) meeting and SMC committee meeting are still be scheduled.
Carfilzomib (Kryprolis®), lenalidomide (Revlimid®) and dexamethasone
The combination of carfilzomib, lenalidomide and dexamethasone is being considered for patients who have received at least one previous treatment. This appraisal follows a review by NICE of its earlier decision not to approve the treatment.
Myeloma UK submitted evidence in January 2020 and took part in the NICE committee meeting in July 2020.
Unfortunately, NICE have issued a draft ‘no’ for carfilzomib, lenalidomide and dexamethasone. The Committee agreed that this treatment delivered clinical benefit compared to current standard treatments. However, NICE considered that uncertainty remained about the long-term impact of this benefit, making cost effectiveness estimates uncertain.
The decision is now out for consultation in which stakeholders, including Myeloma UK, can submit further comment and evidence. Myeloma UK is currently working with NICE, NHS England and the pharmaceutical company, AMGEN, to resolve the issues and change this ‘draft no’ into an approval.
This treatment combination has also been through the appraisal process with the SMC. Myeloma UK submitted evidence in July 2020 with the PACE meeting and SMC committee meeting taking place in August 2020. We are currently awaiting the outcome of this appraisal and a decision should be made public on the 12 of October 2020.
Lenalidomide maintenance is currently being assessed for the treatment of newly diagnosed patients following high-dose therapy and stem cell transplantation (HDT-SCT). Many patients in the UK will have taken part in the Myeloma XI clinical trial for lenalidomide maintenance and we have been expecting this HTA for some time.
We submitted evidence for the NICE appraisal of lenalidomide maintenance in February 2020. To help support our evidence submission, Myeloma UK conducted a patient treatment survey asking for patients’ views on lenalidomide maintenance. There has been a fantastic response to this survey, which highlighted the positive benefits of lenalidomide maintenance and helped to inform our evidence submission.
Unfortunately, NICE have issued a draft ‘no’ for lenalidomide maintenance. The committee highlighted the clinical benefits of the maintenance treatment but uncertainty remained around the long-term economic projections for the treatment.
The decision is now out for consultation in which stakeholders, including Myeloma UK, can submit further comment and evidence. We will also seek to take part in the second committee meeting when it is scheduled. Myeloma UK is currently working with NICE, NHS England and the pharmaceutical company, Celgene BMS, to resolve the issues and change this ‘draft no’ into an approval.
This treatment combination has also been through the appraisal process with the SMC. Myeloma UK submitted evidence in July 2020 with the PACE meeting and SMC committee meeting taking place in August 2020. We are currently awaiting the outcome of this appraisal and a decision should be made public on the 12 of October.
Daratumumab (Darzalex®), bortezomib , thalidomide and dexamethasone
Myeloma UK submitted evidence for this appraisal in September 2020 with the NICE committee scheduled to take place in March 2021.
Myeloma UK have also been invited to submit a Patient Group Evidence submission to the SMC in October 2020 with the subsequent PACE and SMC committee meetings to be scheduled.
Daratumumab, bortezomib, cyclophosphamide and dexamethasone
The quadruplet combination of daratumumab, bortezomib, cyclophosphamide and dexamethasone is being considered for newly diagnosed AL Amyloidosis patients. This is the first treatment for AL Amyloidosis to go through the NICE appraisal process.
Myeloma UK have taken part in the early process for this appraisal and we expect it to continue into 2021. We also anticipate that this will be heard by the SMC in 2021.
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.
If you would like some more information on any of new treatments or HTA processes then please do get in touch at firstname.lastname@example.org.