CancerChronic Myeloid Leukaemia (CML) patients with advanced disease in England face an uncertain future due to an inequity restricting access to a treatment compared to patients in Scotland and Wales.  

Ponatinib, a novel new tyrosine kinase inhibitor (TKI), 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. Despite this, it was not selected for NICE review due to the small patient population; therefore those affected in England currently have to apply through NHS England's Cancer Drugs Fund (CDF) for access. However, the CDF has restricted access only to those patients with the rare T315I mutation (2%-20% of the CML patient population), a small part of the label.   

Results of a poll released by the National Cancer Research Institute (NCRI) CML Working Group has revealed that 83% of respondents believe the 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.

Leading CML experts and patient advocates feel that patients are being overlooked and denied access to effective treatments, and that the disparity in access and use of treatments is failing patients and call for a resolution as quickly as possible.

CML is a rare blood cancer with around 700 new cases each year in the UK. It affects economically active people with around 50% of UK cases in people aged under 65 years. Many patients with a new diagnosis of leukemia have a prolonged clinical benefit from targeted therapy with tyrosine kinase inhibitors (TKIs), however, there is a high unmet need and poor prognosis for patients with advanced disease who develop resistance and intolerance to other therapies. Once available treatment options are exhausted, the prognosis is poor. Despite advances in treatment, there remains a need for additional therapies for the management of CML.  

The online poll was conducted amongst the National Cancer Research Institute (NCRI) CML Working Group (a group of the UKs leading CML clinicians), chaired by Professor Mhairi Copland, Professor of Translational Haematology, University of Glasgow.  The poll revealed concern among the leading experts that the current restrictions compromise the ability of doctors to give optimal care to CML patients in England. The poll also revealed that the majority of respondents believe that the CDF's decision to restrict ponatinib puts cost savings above best practice.

Prof Mhairi Copland, Chair of NCRI 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.”

Dr Dragana Milojkovic, Department of Haematology, Imperial College School of Medicine Hammersmith Hospital, London, added: “As physicians, we strive to do the best for our patients and managing their illness with the correct treatment at the appropriate time without delay according to the stage of their disease is essential part of this process.  The restricted use of therapies imposed by the CDF have seriously limited our ability to treat patients effectively, which is a barrier especially when treatments are used according to their full license elsewhere.”