cancer cells attackingPatients in England living with advanced chronic myeloid leukaemia (CML) England are missing out on access to a vital drug, which is available in Scotland and Wales.

Ponatinib (Iclusig) is a novel new tyrosine kinase inhibitor (TKI) and was deemed by EMA to be of significant benefit to those affected by CML. 

Despite this, ponatinib was not selected for NICE review, and NHS England's CDF restricts access only to those patients with the rare T315I mutation – a small part of the label. In Scotland and Wales, ponatinib is deemed clinically and cost-effective and accepted for use in line with its full EU licensed indication.  An appraisal of ponatinib by the CDF has been repeatedly delayed, according to the drug manufacturer ARIAD Pharma UK.

A recent poll of the National Cancer Research Institute (NCRI) CML Working Group revealed that 83% of respondents believe the Cancer Drugs Fund (CDF) should change its policy and make ponatinib available to all appropriate patients in England in line with the drug’s full licence indication, as in Scotland and Wales.

CML is a rare blood cancer with around 700 new cases each year in the UK. Approximately 50% of new cases involve people under 65 years of age. Many patients with a new diagnosis of leukaemia benefit from targeted therapy with tyrosine kinase inhibitors (TKIs). Yet there is a high unmet need and poor prognosis for patients with advanced disease who develop resistance and intolerance to other therapies. Once these available treatments are exhausted, the prognosis is poor. Despite advances in treatment, there remains a need for additional therapies for the management of CML. ARIAD has said ponatinib fulfils this important need in the treatment pathway for these CML patients.   

About ponatinib

Ponatinib is a novel new TKI and was approved by the European Medicines Agency (EMA) in 2013 as an orphan drug, as it was deemed of significant benefit to those affected by the condition.  

However, due to the small patient population it was not selected for NICE review due. This means patients in England currently have to apply through NHS England's Cancer Drugs Fund (CDF) for access. 

Currently, ponatinib is only available to patients with the rare T315I mutation, which stands at between 2% and 20% of the CML patient population. 

Commenting on the current situation, Professor Mhairi Copland, Chair of CML Working Group and Professor of Translational Haematology, University of Glasgow, said: “Despite major medical advances, the management of some patients with chronic phase CML remains challenging due to resistance and intolerance to existing treatments. These patients have a poor outlook and new innovative treatment options that can potentially extend life are important. These treatments should be available on an equal basis to all people with chronic phase CML across the UK; the decision should be medical, not geographical.”CML infographic