The National Institute for Health and Care Excellence (NICE) has decided against the recommendation of Janssen’s Darzalex (daratumumab) in multiple myeloma. 

In initial draft guidance, NICE determined Darzalex’s supporting data as unclear meaning it could not determine the drug’s cost-effectiveness ratio.

The data was from a trial investigating the drug’s use as a third-line therapy in relapsed and refractory multiple myeloma for patients whose disease had progressed following treatment with a proteasome inhibitor and an immunomodulatory agent.

Janssen’s director of health economics, market access and reimbursement and advocacy, Jennifer Lee, expressed her disappointment with the decision, saying it highlighted “that little has changed in the way that medicines are assessed in the UK.”

“We recognise that NICE is struggling to evaluate breakthrough treatments, such as daratumumab, which receive early approval. Our primary concern is for UK patients to receive the same, timely access to our medicines as patients across Western Europe.”

As Lee alludes to, NICE had previously conditionally-accepted Darzalex based on early data, giving Janssen the time to gather additional data to support the drug’s routine funding through the NHS. Only AstraZeneca’s Tagrisso (osimertinib) has so far been approved in this manner.

There is still hope for Darzalex as NICE will consult on its decision, providing a second draft later this year. To improve its chances, Janssen could offer a discount for Darzalex in conjunction with further supporting data during now and April 7th.

The scenario is a likely one considering the high expectations for Darzalex – J&J projected sales of $5 billion by 2022.

So far, Darzalex has experienced a fairly checkered lifetime. In the US, it had only been approved as a fourth-line therapy for multiple myeloma until the end of last year where the FDA approved its use in combination with dexamethasone and either Celgene’s Revlimid or Takeda’s Velcade as a second-line therapy. The decision greatly broadened its patient population and therefore revenue streams.