The news item about azacitidine at the HealthHombre website says "Appraisal Committee Says Drug Not Recommended as Treatment Option for People with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukaemia or Acute Myeloid Leukaemia." That description may lead readers to the wrong conclusion.
The subject is a recent report by the U.K.
National Institute for Health and Clinical Excellence.
These are the relevant documents, in particular the
Evidence Review Group’s Report (PDF, 268K) from April 2009 and
briefing (PDF, 280K) from June 2009, which evaluate the cost-benefit tradeoff of using azacitidine (Vidaza), not azacitidine's medical efficacy.
In studying the benefits of azacitidine, the NICE review focused on a
randomized trial from 2004 to 2006. The conclusion of that trial was that treatment with azacitidine increased overall survival in patients with intermediate-II and high-risk MDS, CML, and AML compared to conventional care, with statistics as follows:
- Median overall survival: 24.5 months for azacitidine, 15.0 months for conventional care.
- Complete remission: 17% for azacitidine, 8% for conventional care.
- Time to transformation to AML: 17.8 months for azacitidine, 11.5 months for conventional care.
- Transfusion independence (for transfusion dependent patients): 45% for azacitidine, 11.8% for conventional care.
The NICE cost-benefit evaluation used a measure called
QALY, which is the incremental cost per
quality-adjusted life year. In other words, it's a financial question about the cost of the better treatment provided by azacitidine. NICE explains QALY
here.
Data on the cost of azacitidine vs. conventional treatment was provided by Celgene Europe. Depending on which conventional treatment was compared to treatment with azacitidine, results showed that the "cost per QALY gained" for azacitidine was in the £30,000 to £60,000 range. NICE reported these results while raising questions about Celgene's economic model.
Despite the description at HealthHombre, the NICE report appears to stop short of recommending against the use of azacitidine, and recommends further evaluation.