NICE rejects AbbVie/Roche CLL drug Venclyxto

NICE has rejected AbbVie/Roche's Venclyxto  for chronic lymphocytic leukaemia in its first draft guidance, saying there are too many uncertainties in its clinical evidence base.

Venclyxto (venetoclax) is forecast to be a global blockbuster for the companies, but its £4,800 a month cost has proven to be an obstacle for NICE.

Patients with a 17P deletion or TP53 mutation have alternative treatments available, as NICE has already recommended Gilead’s Zydelig (idelalisib) in first line, followed by AbbVie/Janssen’s Imbruvica (ibrutinib) in second line for this type of cancer.

The first draft guidance from NICE finds Venclyxto to be not a cost-effective use of NHS resources, even though the drug was given extra leeway as an end of life treatment.

The UK's regulator also made the drug available to patients ahead of its marketing authorisation under the Early Access to Medicines scheme.

NICE also decided the drug shouldn't be reimbursed via the revamped Cancer Drugs Fund, which can make the drug available to patients in England for a limited period to allow for more conclusive clinical evidence to be accumulated.

Venclyxto was approved conditionally in Europe on the basis of phase 2 data, which means its marketing authorisation will be reviewed every year until the company can provide late-stage data.

According to the first draft guidance, NICE’s appraisal committee was unable to make a recommendation on Venclyxto because of the small study size.

NICE was also unable to provide overall survival data as the company considered this information to be confidential.

The ruling comes as the UK government works with the pharma industry on a new life sciences industrial strategy. The UK industry continues to call for reform of NICE, which it says needs a root-and-branch review. This includes NICE's use of the Quality Adjusted Life Year (QALY) system to assess cost-effectiveness of drugs, which pharma says is applied to strictly.