National Health Service cost regulators are leaning towards barring the use of Bristol-Myers Squibb's Sprycel (dasatinib), Novartis' Tasigna (nilotinib) and high-dose Glivec (imatinib) for the treatment of certain patients with chronic myeloid leukaemia (CML), a condition that affects around 2,660 people in England and Wales.

In new draft guidance the National Institute for Health and Clinical Excellence says the drugs should not be funded on the NHS in patients resistant to standard-dose Glivec because current evidence on their effectiveness in this setting is "very weak".

Despite recognising the clinical benefit of these drugs the Institute's Appraisal Committee was not totally convinced by the data submitted to support their use in patients who have developed resistance to Glivec. 

For one, it stressed that it was aware of only one comparative trial in the chronic phase of the disease, which pitted Sprycel with Glivec, but noted that it had a poor study design and interpretation problems. 

Furthermore, the Committee said that all clinical studies available were of short duration (relative to life expectancy) and that median overall survival was not reached, and that surrogate outcomes were used to predict overall survival.

Moreover, the drugs certainly don't come cheap: Sprycel costs around £30,477 per year, assuming a treatment regimen of 100mg once daily; Glivec is £41,960 per year, assuming 400mg twice daily; and Tasigna is £31,711 per year, based on 400mg twice daily.

"When we recommend the use of very expensive treatments, we need to be confident that they bring sufficient additional benefit to justify their cost," said NICE chief executive Andrew Dillon, explaining the Institute's position.

Two-tiered system?

But Professor Professor John Goldman, Emeritus Professor of Haematology at Imperial College, London, argues that the decision will have and adverse effect on people with CML and warns of the potential for a two-tiered system under which patients who fail on standard Glivec after NICE's decision are left with few options compared to those treated beforehand. 

"This may put clinicians in England and Wales in a very difficult position, and clearly puts us behind Scotland and the majority of the Western World where these treatments are reimbursed,” he stressed.

The decision certainly marks another blow to patients with CML - earlier this year, NICE issued draft guidance also turning down their use in patients intolerant (not resistant) to standard Glivec, although the appraisal is still ongoing and a final decision is yet to be made. 

Sprycel, Tasigna and standard dose Glivec are also being considered for the first-line treatment of the disease, the Institute noted.