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Improved patient access scheme allows NICE to recommend azacitidine to treat myelodysplastic syndromes

In draft guidance issued today, NICE recommends azacitidine (Vidaza, Celgene) as a treatment option for people with the following conditions who are not eligible for haematopoietic stem cell transplantation: intermediate-2 and high-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia. The recommendation is conditional on the manufacturer providing azacitidine at a discounted cost as part of a patient access scheme.

During the public consultation on the previous draft, Celgene submitted a revision to its original patient access, in which azacitidine is provided with a discount.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE said: "Azacitidine is the first drug that has been developed specifically for treating myelodysplastic syndromes. It is not a cure, but it does have the potential to extend patients' lives by an average of nine months. It is a very expensive drug, but the manufacturers have submitted a patient access scheme where the cost will be reduced. We are therefore very pleased to be able to recommend azacitidine as a cost effective use of NHS resources."

Myelodysplastic syndromes (MDS) are a group of bone marrow disorders, where the marrow doesn't produce enough of one or more types of blood cells. The majority of patients with MDS receive best supportive care in current clinical practice and some patients receive low dose chemotherapy. There are approximately 700 patients with MDS in England and Wales. This is the second Final Appraisal Determination (FAD) for this appraisal and the draft guidance is now with consultees, who have the opportunity to appeal against it. NICE has not yet issued final guidance to the NHS.

Ends.

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Notes to Editors

About the appraisal

1. The draft guidance will be available from 17 February at: http://guidance.nice.org.uk/TA/Wave18/19

2. The Committee agreed that azacitidine met the criteria for being a life-extending, end-of-life treatment and considered that, on balance, the additional weight that would need to be assigned to the QALY benefits in this patient group for the cost effectiveness of azacitidine to fall within the current threshold range was acceptable.

3. The committee agreed that the most plausible ICER for azacitidine in the overall patient population was approximately £47,200 per QALY gained. This included the revised patient access scheme.

4. The manufacturer agreed with the Department of Health that the level of the discount would remain confidential.

About NICE

5. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health

6. NICE produces guidance in three areas of health:

  • public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
  • health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
  • clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

7. NICE produces standards for patient care:

  • quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
  • Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients

8. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.

This page was last updated: 16 February 2011

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Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.