15th March 2022 // Shelagh McKinlay
You will have heard that, as of today, daratumumab monotherapy is available at fourth line on the NHS in England and Wales.
This appraisal has been a game-changer; one that has paved the way for more life-changing treatments to be rolled out earlier in the treatment pathway.
Back in 2018, when daratumumab (Darzalex®) was first being evaluated by the National Institute for Health and Care Excellence (NICE) for use at third relapse (fourth line) the clinical evidence available – early yet very encouraging data from a second-phase trial – was too limited to secure approval from NICE.
Developing innovative drugs and treatments, as we know all too well, takes time. But asking patients, particularly when they had experienced several relapses already, to wait years for larger third phase trials to report, was something no one wanted. We knew the treatment was safe and effective, but for a treatment to be provided on the NHS we also have to know whether it offers value for money compared to existing treatments. The evidence wasn’t there yet. The solution to getting it out into clinics where patients could benefit was the Cancer Drugs Fund (CDF).
The CDF allows NICE to provisionally approve a drug showing promise for a period of two years and then put it in the hands of patients in England and Wales as early as possible, while necessary data is being gathered. The decision is then reviewed and, all being well, it is then made routinely available on the NHS. This is the first treatment in myeloma which has been confirmed for routine access after CDF review and it shows that the CDF can enable early access to the treatments patients need.
We knew there was a real unmet need for patients whose options are becoming more limited, and daratumumab offered a step change, introducing a new and different mechanism of action into the treatment pathway.
We pressed for approval by submitting evidence, sharing patients’ first-hand accounts of the benefits of daratumumab monotherapy, and making sure the voice of the myeloma community was heard loud and clear at committee. Thanks to our efforts those who needed it the most were able to access this pioneering treatment years ahead of schedule.
Subsequent clinical trials showed that daratumumab could live up to its early promise and it is now available in combination with other drugs as induction treatment and at second line as well as having just been confirmed in its original fourth line position. (It is also available in Scotland and Northern Ireland).
Patients living with myeloma need to know that when they relapse there is another effective treatment waiting. We need to use every tool at our disposal to get treatments from the research pipeline into clinics as soon as the data allows. The story of daratumumab shows that we can make this happen.
The Cancer Drugs Fund (CDF) is a source of funding for cancer drugs in England. It aims to
provide cancer patients in England with faster access to the most promising new drugs. The CDF only applies to England. Wales and Scotland have different new treatment fund policies and Northern Ireland follows decisions made in other home nations.
The CDF aims to make promising cancer drugs available to patients before they are fully approved for use on the NHS by the National Institute for Health and Care Excellence (NICE). It has three key objectives:
The CDF can be thought of as part of the NICE process for reviewing cancer drugs, providing NICE with a third option when appraising cancer drugs.
NICE can say:
NICE will use the CDF route if it thinks that a drug shows promise but there isn’t enough evidence for a straight “yes” decision. When taking this ‘maybe’ route through the CDF, NICE will look again at the drug, usually after two years or so, to consider the additional evidence that has been collected before making a final yes or no decision.