NICE nod for ‘game-changing’ multiple myeloma treatment via CDF

BioMarin's Brineura to be available on NHS

The Janssen Pharmaceutical Companies of Johnson & Johnson has announced that the National Institute for Health and Care Excellence (NICE) has recommended Darzalex® (daratumumab) plus Velcade® (bortezomib) and dexamethasone (DVd) for use within the Cancer Drugs Fund (CDF), as an option for treating relapsed multiple myeloma in people who have had one previous treatment.

Patients at second line will now have access to a treatment combination, which NICE accepts had a ‘clinically important and statistically significant effect on progression-free survival’. However, Janssen said that it was disappointed that despite this compelling evidence, routine NHS access was not granted.

NICE recognised DVd as a ‘game-changing’ treatment, which improves overall survival and reduces the risk of death by 50%.

The triple therapy combination’s 18-month improvement in median progression free survival (PFS) has also been recognised by patients and clinical experts. NICE however said that the overall-survival data was  ‘immature’ because more than half of the patients treated with the daratumumab combination were alive after nearly four years of follow up.

Myeloma is a relapse-remitting disease and after first relapse, patients can have a decreased response to treatment over time. Gordon Cook, Professor of Haematology & Myeloma Studies at St James’s University Hospital, Leeds, said: “For many patients, the first relapse can have a devastating physical and psychological impact, making it essential to use the most effective treatments as early as possible, to delay relapse and maximise overall survival.

“There is currently a huge unmet need for treatments in this set of patients, so the DVd combination is a long-awaited additional treatment option, which we anticipate to be game-changing for the myeloma community.”

Jennifer Lee, Director of Health Economics, Market Access and Reimbursement (HEMAR) and Advocacy at Janssen, said: “While it is positive news that patients will now be able to access DVd, it is disappointing that NICE – despite recognising the life-extending, step-change this combination represents for patients – did not recommend routine access on the NHS.

“The parameters through which NICE determines uncertainty need urgent re-evaluation. We believe the additional data collected via the CDF will only further validate the long-term survival benefit already seen in this innovative combination. The CDF should be utilised how it was originally intended, where clinical evidence is less robust.”