Patient advocacy news // 26th August 2021
Patient advocacy news // 26th August 2021
Today the National Institute for Health and Care Excellence (NICE) issued a draft ‘no’ on the use of daratumumab (Darzalex®) in combination with bortezomib (Velcade®), thalidomide, and dexamethasone (DVTD) for the treatment of newly-diagnosed myeloma patients receiving high-dose therapy and stem cell transplantation (HDT-SCT) in England and Wales.
This is the second time the quadruplet combination, which was approved in Scotland back in January 2021, has been rejected by NICE.
The treatment will now go through a third appraisal process in November. Access for patients in Northern Ireland will be based on the final decision from NICE.
Chief Executive, Laura Kerby, said:
“We are once again very disappointed by the committee’s decision. We firmly believe that patients across the UK should have equal access to the most effective treatments and it has been close to a year since Myeloma UK submitted evidence for this important treatment. The reality of this delay and the ongoing discussions is more and more newly diagnosed patients are missing out. We will keep fighting to ensure no-one is left behind and remain hopeful that an agreement will soon be reached and the quadruplet combination finally be made available to patients in the rest of the UK.”
Patient expert, Rosie Dill, said:
“I appreciate this is a complex decision for NICE but I am hoping this combination of drugs is approved at the next committee meeting in November. This treatment will make a huge difference to the lives of so many newly-diagnosed myeloma patients and extend remission.”
Scottish patient Dave McGovern was diagnosed in April 2021 and has since received the quadruplet combination. He said:
“I’m very disappointed that NICE have rejected it. The results have been impressive with my paraprotein levels reduced from 32 to 4 after 2 cycles. It really is working. In terms of side effects, of course, there are ups and downs, but out of 100-odd days of chemo, there have been only about 10 or 15 when it’s been quite bad. I definitely was expecting it to be a much tougher experience than it has been.”
Clinical trial results show that adding daratumumab to the standard treatment of bortezomib, thalidomide and dexamethasone improves patient outcomes and extends remission time.
Although NICE agreed once again that the treatment was effective, the appraisal committee said it needed longer-term data and further evidence to demonstrate the overall extent of the treatment benefit, particularly when given with lenalidomide (Revlimid®) maintenance, before it could make a cost-effectiveness assessment.
Director of Research, Sarah McDonald, said:
“This is a huge frustration for patients in England and Wales, many of whom have already had to endure long delays in referrals and early detection due to the ongoing COVID-19 crisis. The crux of the issue here is the length of time and process which supports drug approval. Lenalidomide maintenance was approved earlier this year while the quadruplet combination was going through appraisal, thereby disrupting the process. NICE’s decision was that there wasn’t enough evidence to assess the clinical benefits of using lenalidomide maintenance following DVTD. While this is disappointing, we hope this additional time will result in approval. We will continue to work with NICE and the manufacturer to help secure access to this treatment. Patients shouldn’t miss out on this treatment because of process and delays.”
What about the rest of the UK?
The treatment combination was approved for patients in Scotland by the SMC in January 2021. The department of health in NI will decide on whether to fund this treatment based on the decision from NICE.
What happens now?
NICE have issued the draft decision in an Appraisal Consultation Document (ACD) and will now ask questions that could turn the draft ‘no’ into a final approval. Most myeloma drug approvals result in an initial draft “no” so unfortunately this step in the process is not unusual.
Myeloma UK will continue to work with clinicians, the manufacturer and NICE and will respond to the consultation, making the case for Daratumumab in combination with bortezomib, thalidomide and dexamethasone to be approved. Once NICE has gathered views from all stakeholders, it will meet again in November to consider its decision.
For more information please see our blog from an earlier appraisal on ‘What happens after a draft ‘no’?’
Why did NICE say no?
The committee highlighted that results from the clinical trial (CASSIOPEIA) show that adding daratumumab to the standard induction treatment of bortezomib, thalidomide and dexamethasone improves progression free survival and overall survival.
However NICE considered that uncertainty remains about the long-term impact of the daratumumab treatment effect which makes the cost effectiveness estimates uncertain. Further to this, modelling for long-term overall survival did not consider the recent NICE approvals to the myeloma treatment pathway, which includes the approval of lenalidomide maintenance.
The price of drugs is a factor in NICE decisions, but it is important to bear in mind that they do not look at whether a drug is expensive, rather they look at whether it is cost effective; all NICE decisions are based both on the clinical effectiveness and cost effectiveness of the treatment being appraised compared to the current standard of treatment.
Can the treatment be approved through the Cancer Drugs Fund (CDF)?
The committee considered this quadruplet combination for approval through the CDF and noted that the final data analysis of the CASSIOPEIA trial is due in 2023.
The committee stated that the key clinical uncertainty concerning the duration of the daratumumab treatment effect is unlikely to be resolved within the CDF’s timeframe and therefore unlikely to benefit from approval through the CDF.
Is it likely to be approved?
During the consultation period the company, Janssen, clinicians, and Myeloma UK will be able to submit further evidence to address NICE’s concerns. The company is also able to come forward with new information which could address the uncertainties highlighted by the committee.
At the moment, we cannot be certain what the outcome will be. It is positive that NICE recognise the clinical benefits that this quadruplet combination can deliver, but there is still work to be done to resolve the concerns NICE have about its cost-effectiveness. We will continue to work with clinicians, NICE and with the company to make a case for its approval.
What about patients based in Scotland and Northern Ireland?
Daratumumab in combination with bortezomib, thalidomide and dexamethasone was approved by the Scottish Medicines Consortium as a treatment for newly-diagnosed myeloma patients receiving HDT-SCT in January 2021.
For patients based in Northern Ireland the Department for Health makes decisions on which treatments to fund through the NHS. They can follow guidance issued by NICE and the SMC but normally follow the guidance issued by NICE.