Sorry, the evidence isn’t strong enough

WHILE I have the most profound sympathy with the agony that these families are enduring, I believe NICE has made the right move in deciding that the new American drug Brineura should not be available on the NHS.

Policies like this are always tough, but it is far too simplistic to characterise such a decision as a triumph for cold- hearted accountancy. In truth, NICE had to look beyond costs and consider two other crucial factors.

The first is the effectiveness of the drug, for which there is, so far, little hard evidence, given how limited the studies have been.

The second is the nature of how the drug is administered, which carries the risk of both greater suffering and patient morbidity.

In practice, justifiable parental emotions have to be weighed against clinical realities.

During a 30-year career as a cancer surgeon, I have seen how so many drugs, introduced with an initial fanfare, have failed to live up to expectations. Brineura could be another such case. Sadly, even based on a tiny sample in its trials, the results of Brineura have not been especially promising.

Even its most enthusiastic advocates do not claim Brineura is any kind of cure for the disease. As far as I understand, the only benefit is a possible slower decline in terms of walking ability.

The challenging mode of delivery also gives cause for caution.

It has to be administered directly into the ventricles (central cavities) of the brain and initially requires major surgery to drill holes in the skull and then to insert the necessary tubes, followed by weekly injections through these devices.

It involves dangers not only from the operation but from infections. If I were a parent of a child with Batten, I would not be pushing for this invasive treatment.