Takeda’s Ninlaro rejected by NICE for multiple myeloma

Takeda has vowed to work with the National Institute for Health and Care Excellence to get NHS funding approved for its mutilple myeloma drug Ninlaro following an initial rejection by the cost watchdog.

Ninlaro (ixazomib) plus lenalidomide and dexamethasone is authorised to treat multiple myeloma in people who have had one or more previous therapies, but in clinical practice is likely to be used only for those who have had two or three previous therapies, according to the Institute.

Explaining its initial rejection of the drug, it noted that the main clinical trial for the drug is ongoing and only limited data are available, and it is not yet clear whether Ninlaro prolongs life compared with the current treatment for people who have had two or three therapies.

In addition, NICE argues that Ninlaro’s benefit on the length of time people live without their disease progressing appeared to reduce after longer follow-up.

For people who have had two or three therapies, the minimum estimate of cost effectiveness varied between £138,000 and £176,000 per quality-adjusted life year (QALY) gained compared with current treatment, and thus falls well outside the bounds of what would normally be considered value for money for the NHS. Also, for patients who have had one previous therapy, the drug was less effective than the current treatment (bortezomib plus dexamethasone) and cost more, the Institute concluded.

"This draft negative recommendation is obviously very disappointing for myeloma patients in England and Wales," said Kate Morgan, policy and public affairs manager at Myeloma UK. "Whilst we understand the difficult remit NICE has in assessing new drugs, we hope that over the coming weeks they will fully engage with the company on the uncertainty in this appraisal and help reach a positive outcome for myeloma patients, who not only want treatment options that are effective and tolerable, but also appreciate the convenient option of taking an oral treatment."

Multiple myeloma is an incurable cancer of the plasma cells, which are found in the bone marrow and normally produce antibodies to help fight infection. It is the second most common type of blood cancer and there are around 5,000 new cases and 2,700 deaths related deaths each year in the UK.

According to Takeda, some currently available therapies for multiple myeloma "can be a burden on the patient both due to toxicity and the requirement to visit hospital for multiple infusions or injections", and this "can affect the ability of patients to stay on treatment which may ultimately contribute towards a relapse of their disease."

Ninlaro, the firm stressed, was approved in Europe on the back of data from the pivotal Phase III TOURMALINE-MM1 trial, which showed that the all oral combination of Ninlaro, lenalidomide and dexamethasone boosted progression-free survival by around six months, or 35 percent, when compared with placebo-based regimen. In addition, the triple combination had limited additional toxicity and was well tolerated by patients with no adverse impact on quality of life.

"We’re obviously disappointed with NICE’s draft decision. However, we won’t be walking away from the process and are totally committed to working with NICE and NHS England to find a solution, as achieving access to our medicines for patients in need is our first and foremost concern," commented Adam Zaeske, managing director UK & Ireland at Takeda UK.

"It is very challenging, under current NICE methodologies, for innovative medicines used in combination to demonstrate cost-effectiveness but Takeda will do everything we can to rise to the challenge."