The National Institute for Health and Care Excellence (NICE), the drug approval body for England and Wales, has published draft negative guidance for daratumumab (Darzalex®) monotherapy (for use on its own and not in combination with other drugs).
This means that, as it stands, it is unlikely to be made available in this setting on the NHS for relapsed and refractory myeloma.
Myeloma UK Policy and Public Affairs Manager, Kate Morgan said, “This is a very disappointing decision for myeloma patients in England and Wales, but it was always going to be a challenging appraisal.”
“We understand why NICE has not been able to say yes at this point, given there are major limitations associated with the early nature of the trial data and the lack of direct comparison with the current standard of care. However, since daratumumab was given an accelerated licence by the European licensing body, this negative decision demonstrates the need for NICE and other stakeholders to be better prepared to deal with situations like this. ”
“The myeloma community is extremely hopeful and positive about the potential of immunotherapies. Daratumumab is a highly innovative drug and it is very important that these types of exciting new treatments are made widely available for patients.
“We will therefore work with NICE and the pharmaceutical company, Janssen, to seek solutions to the complex issues that arise in appraisals based on early data.”
NICE has issued a draft decision as it needs more information on daratumumab’s value to the NHS. The main issue is that daratumumab monotherapy was licensed earlier, using data from an earlier phase clinical trial than is normally submitted. NICE concluded that whilst they understood the need for treatments in the relapsed and refractory setting in myeloma, there is just too much uncertainty relating to value of daratumumab monotherapy to be able to say yes at this stage.
This first draft will now be issued to stakeholders for their comments and feedback. During this phase, the submitting pharmaceutical company (Janssen) and patient and clinician groups can submit further information to support approving the treatment. Myeloma UK will be writing to NICE with our feedback and will be working with NICE, myeloma doctors and Janssen over the coming weeks to try overcome the major challenges in this appraisal.
The level of uncertainty outlined in the NICE draft guidance over cost-effectiveness means it will be difficult to get daratumumab monotherapy approved on the NHS for relapsed and refractory patients.
In addition, there are not many other trials in progress looking at daratumumab in this setting. This means there is not likely to be any additional forthcoming data to support the use of the drug as a monotherapy in the relapsed and refractory setting.
However, as this is draft guidance there is the opportunity for more discussion. Myeloma UK will also play a key role in wider policy discussions about how UK drug approval bodies prepare for this type of situation, ensuring that the patient perspective is heard by NICE and other stakeholders.
This is not the only appraisal in the pipeline for daratumumab. NICE is also currently considering a separate appraisal looking at daratumumab in combination with established myeloma drugs – lenalidomide (Revlimid®) or bortezomib (Velcade®) for relapsed and/or refractory myeloma.
Daratumumab belongs to a group of immunotherapy treatments known as “monoclonal antibodies.” Monoclonal antibodies recognise and attach to specific proteins on the surface of myeloma cells, allowing the immune system to attack and remove them. Find out more by downloading our daratumumab Infosheet.
The NICE application was based on two Phase II trials which looked at daratumumab monotherapy in multiply relapsed myeloma patients. The trial results found that, when used on its own, daratumumab had a very beneficial effect on both the level of response and length of remission (i.e. the length of time patients have without their myeloma returning) in relapsed and/or refractory myeloma patients.
NICE guidance only applies in England and Wales. The Scottish Medicines Consortium (SMC), the drug approval body in Scotland, assessed daratumumab monotherapy at the end of 2016 and issued a negative recommendation. We are currently working with the SMC and Janssen to assess whether a resubmission will be made to the SMC.
Northern Ireland usually implement final NICE guidance, following an assessment of the Department for Health, Social Security and Public Safety (DHSSPS) for local applicability.
NICE and the SMC will be looking at daratumumab in combination with other myeloma drugs during the course of 2017.
For more information on myeloma and available treatments, contact the Myeloma Infoline on 0800 980 3332 or email AskTheNurse@myeloma.org.uk.