Chronic Myeloid Leukaemia patients in England denied access to effective treatments

Chronic Myeloid Leukaemia (CML) patientswith advanced disease in England face an uncertain future due to an inequityrestricting access to a treatment compared topatients in Scotland and Wales.  

Ponatinib, a novel new tyrosine kinase inhibitor (TKI), was approved bythe European Medicines Agency (EMA) in 2013 as an orphan drug, as itwas deemed of significant benefit to those affected by thecondition. Despite this, it was not selected for NICEreview due to the small patient population; therefore thoseaffected in England currently have to apply through NHS England’s CancerDrugs Fund (CDF) for access. However, the CDF has restrictedaccess only to those patients with the rare T315Imutation (2%-20% of the CML patient population), a smallpart 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 newcases each year in the UK. It affects economically active people witharound 50% of UK cases in people aged under 65 years. Many patientswith a new diagnosis of leukemia have a prolonged clinical benefitfrom targeted therapy with tyrosine kinase inhibitors (TKIs),however, there is a high unmet need and poor prognosis for patientswith advanced disease who develop resistance and intolerance to othertherapies. Once available treatment options are exhausted,the prognosis is poor. Despite advancesin treatment, there remains a need for additional therapiesfor the management of CML.  

The onlinepoll was conducted amongst the National Cancer Research Institute (NCRI) CML WorkingGroup (a group of the UKs leading CML clinicians), chaired byProfessor Mhairi Copland, Professor of Translational Haematology,University of Glasgow.  The poll revealed concern among theleading experts that the current restrictions compromise theability of doctors to give optimal care to CML patients inEngland. The poll also revealed that the majority of respondents believe that the CDF’sdecision 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 managementof some patients with chronic phase CML remains challenging due toresistance and intolerance to existing treatments. These patients have apoor outlook and new innovative treatment options that can potentiallyextend life are important. These treatments should be available on anequal basis to all people with chronic phase CML across the UK; thedecision 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 managingtheir illness with the correct treatment at the appropriate time withoutdelay according to the stage of their disease is essential part ofthis process.  The restricted use of therapies imposed by theCDF have seriously limited our ability to treat patients effectively,which is a barrier especially when treatments are used according totheir full license elsewhere.”