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The granting by EMEA of full marketing authorisation for Celgene’s Vidaza will bring new hope to MDS patients, particularly those with high risk forms of these diseases that historically have been associated with a bleak outlook.
Patients who are ineligible for stem cell transplant therapy are often subjected to the discomfort, inconvenience and complications that can be associated with essential, regular blood transfusions, a life saving procedure which remains the mainstay of MDS treatment.
Recent published studies indicate that Celgene’s Vidaza presently has the unique potential to increase, significantly, survival of many, although not all, patients with high risk MDS, at the same time reducing suffering and improving quality of life.
However, in the UK, EMEA approval is no guarantee of patient access to such a new and potentially highly effective therapy. Until the National Institute of Clinical Excellence (NICE) has, in turn, exercised its lengthy approval procedure, prescription of demonstrably efficacious drugs may only be at the discretion of local Primary Care Trusts (PCT’s).
The experience of our Group is that a combination of procedural inertia, ignorance of the degree of patient suffering and misplaced costing assumptions, make it extremely unlikely, in the majority of the UK regions, that the required levels of funding will be authorised. This, coupled with the long approval cycle from NICE, represent a frustrating, unnecessarily long and painful wait for medication, after EMEA’s imprimatur.
Let’s hope that the introduction of this new, powerful medication from Celgene, with significant, demonstrable benefits to many of the more severe aspects of MDS, will attract an appropriately urgent and sympathetic response from those charged with its availability and distribution to the patient base.