No Vidaza in the UK?

[Birgitta-A]

Hi all members in the UK,
The National Institute for Health and Clinical Excellence NICE in the UK doesn’t recommend treatment with Vidaza for MDS patients – the experts don’t think it is cost-effective !
http://www.healthhombre.com/articles/20090803

Do tell them that you need possibility to get the drug!
Kind regards
Birgitta-A

[Neil Cuadra]

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.

[Birgitta-A]

Hi Neil,
Yes, you must be right. I can't believe that Vidaza won't be approved in the UK!

As far as I understand we could comment the different sections in the preliminary NICE document. Closing date for comments is 24 August 2009.

I have given them my comments - the document seems to be open for everybody - even persons not living in the UK.
http://www.nice.org.uk/guidance/inde...rticle&o=45032
Kind regards
Birgitta-A

[helen c.]

#1 i cant believe there are so few people in the uk that have mds #2 i cant believe they put a price on a drug that is actually helping people. vidaza really helped my husband last year he became transfusion independant. i am firmly convinced that if treatment had not been stopped b/c of his complete response he would not be where he is now, but that was the treatment then so i dont blame anyone. all i know is that for 6 months last year we got out life back. and he was happy so now we are in the battle again and who knows what new treatment is just around the corner i still have him and i praise GOD for that

[Ray Rogers]

Excerpt from article appearing at:
www.pharmatimes.com/UKNews/article.aspx?id=16353

... "despite agreeing that Vidaza is indeed clinically effective within its licensed indications, NICE’s Committee felt that treatment with the drug is just too costly for use on the health service. Although it was unable to determine a final cost-effectiveness estimate, the Committee concluded that the ICER would be significantly higher than the manufacturer’s base case scenario of £66,000 per QALY (quality-adjusted life year), and so could not endorse its use, even when taking into account new guidance for appraising end-of-life drugs."

"David R Hall, Chairman of the MDS UK Patient Support Group, said patients will be 'devastated' by NICE's preliminary recommendations. 'Celgene’s VIDAZA has brought a ray of hope to MDS sufferers, particularly those with the higher risk conditions' as it 'replaces the uncomfortable and tedious blood transfusion regime' and extends expected survival, he explained. 'Placing an arbitrary value on human life is presumptuous in any situation,' he said, and added: 'When suffering in old age, with premature death are the criteria, our society needs to look critically at the avenues through which public funds are dispensed'."

The article goes on to indicate: "The Committee’s conclusions could still be contested, however, and the closing date for comments is August 24 August in time for a Second Appraisal Committee meeting on September 3."