London School of Hygiene & Tropical Medicine

The National Institute for Health and Care Excellence (NICE) has recommended the use of Bosutinib (trade name Bosulif, Pfizer) on the NHS to treat those with chronic myeloid leukaemia. The drug was previously only available through the Cancer Drugs Fund (CDF).

The CDF was set up to help patients access drugs rejected by NICE for widespread use on the NHS. The decision was made earlier this year to reform the service, with NICE beginning a reappraisal of all drugs currently in the CDF.

In 2013 Bosutinib was not granted NICE recommendation but has now become the first drug to be approved under the reappraisal process. The drug is used to treat chronic myeloid leukaemia when other treatments are no longer viable or cause severe, adverse side effects.

So should NICE have reversed their previous decision on Bosutinib? What precedent does this reversal set for future drugs in the CDF reappraisal process? Richard Grieve, Professor of Health Economics Methodology at the School, explains:

"NICE had previously considered Bosutinib poor value for money.  This new decision exposes a major problem with the reformed Cancer Drug Fund process in that it relies on observational (non-randomised) data. For such data to help NICE improve population health, it is essential that outcomes are compared between similar patients who do, and do not take the new drug. However, in the Bosutinib appraisal, the new drug was compared to a small sample of patients who received an alternative drug some years previously. The evidence submitted to NICE on whether Bosutinib is effective, did not allow for differences between patients who took the new versus the old drug.

"The development of innovative drugs which could help cancer sufferers are to be welcomed - but they must be proven to be effective and cost-effective before they are offered to patients. Today's decision highlights that there is a clear danger NICE will make future decisions based on weak, biased effectiveness evidence. Unless the new NICE process directly addresses these profound difficulties, the NHS will waste large amounts of scarce public money on new drugs with high prices but unproven benefit to patients."