NICE set to reject Janssen’s Darzalex for multiple myeloma

Janssen said it is extremely concerned that the National Institute for Health and Care Excellence (NICE) has not recommended Darzalex for treatment of relapsed and refractory multiple myeloma in adults in preliminary guidelines.

Should the Institute’s position become final, patients who have failed nearly all existing treatment options will remain unable to access an innovative and much needed treatment for this incurable form of blood cancer on the NHS - whilst it is already reimbursed in several European countries, the firm stressed.

Darzalex (daratumumab) holds a conditional license in Europe to treat adults with relapsed and refractory multiple myeloma who have been treated before with proteasome inhibitor and an immunomodulatory agent and whose disease as progressed on their last therapy.

However, the cost watchdog has turned down NHS funding for the drug in an appraisal consultation document.

Whilst the committee recognised the need for effective, well-tolerated treatment for people with the disease who have had previous therapies, the quality of  evidence presented meant that it could not fully interpret how clinically effective the drug is, NICE said.

This high degree of uncertainty for clinical effectiveness meant that Darzalex’ cost effectiveness results were unreliable and a most plausible ICER (incremental cost effectiveness ratio) could not be identified.

Janssen says findings from a combined efficacy analysis of two mid-stage trials showed that after a mean follow-up of 20.7 months, the estimated median overall survival (OS) for Darzalex in these heavily pre-treated patients was 20.1 months, with 65 percent surviving up to 12 months whereas current treatment options have shown around an average survival of five to nine months. The overall response rate (ORR) for the combined analysis was 31 percent, and 83 percent of patients achieved stable disease or better.

"This decision is extremely disappointing and highlights that little has changed in the way that medicines are assessed in the UK," said Jennifer Lee, director of Health Economics, Market Access and Reimbursement (HEMAR) and Advocacy at Janssen UK.

"We recognise that NICE is struggling to evaluate breakthrough treatments, such as daratumumab, which receive early approval. NICE must work within defined parameters set by Government, which present real challenges in assessing innovative treatments like daratumumab. It is clear that key reforms are needed to the way NICE reviews such treatments if patients are to be given faster access to the medicines they need."

The UK continues to lag behind in access and uptake of new cancer treatments and has some of the worst survival rates in Europe, the firm argued, noting: "Daratumumab monotherapy is currently reimbursed in 12 European countries. In contrast, since the launch of the ‘new’ Cancer Drugs Fund in July 2016, only one drug has been successfully added to the fund".

"Our primary concern is for UK patients to receive the same, timely access to our medicines as patients across Western Europe," said Lee. "We firmly believe that the clinical effectiveness which has been demonstrated with daratumumab in Phase II studies means that this breakthrough medicine should be made routinely available to NHS patients that need it, and we will continue to work with NICE to make this happen."