Orphan Drug

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Winner

Vidaza

Celgene

Summary of work

Azacitidine (Vidaza), the first licensed treatment for the management of Intermediate-2 and high-risk Myelodysplastic Syndromes (MDS) across Europe, was seen by clinicians as a breakthrough treatment for patients with limited other options. However, prior to the positive NICE appraisal for Vidaza in February 2011, there was limited experience of prescribing of Vidaza outside the MDS centres of Excellence and larger teaching hospitals. Therefore, following the positive NICE recommendation, Celgene initiated a three-pronged customer support programme, focusing on clinicians, nurses and pharmacists based in District General Hospitals (DGH), to ensure all patients across England were able to access the treatment on the NHS. The support programme, based on critical insights from European markets where Vidaza was widely-used and existing prescribers and UK clinical trialists, focused on:
  • Patient identification and diagnosis
  • Initiation and maintaining patients on treatment
  • Administration and side effect management.
By the end of 2011, sales of Vidaza had trebled with 35 per cent of all eligible patients initiated on the treatment. In addition, the number of cycles of treatment per patient increased throughout the year – testament to the support programme Celgene had invested in.     Executive summary as submitted

Judges’ comments

"Celgene’s strategy was to get out of specialist treatment and focus on a broader market. I was struck by how good the company is at changing the beliefs of people."