Campaigners are right to request delay in rule change

But even so, we are deeply troubled by the case of Maddison Crockford-Reid, which we report today.

Maddi has a rare – in fact an ultra-rare –condition that affects only a handful of people in the country. Sanfilippo syndrome, a condition that means the sufferer’s body cannot process sugar molecules properly, is incurable.

However, research is taking place which, one hopes, will shed light on the syndrome and lead to ways of mitigating it. However, new rules brought in by the National Institute of Clinical Excellence, which issues guidelines on which treatments should be available on the NHS, has revealed it is tightening its criteria for funding drugs for rare diseases. With little chance of funding for any drug on the horizon, the chance of the trial that Maddi is participating in being renewed drops – and so does, therefore, any chance of a cure.

While we understand the pressures on budgets, this is a brutal way indeed to go about it.

The news was revealed by Nice this month, and the change of funding comes into effect tomorrow. While there was a consultation into it, the swift implementation of this change of tack means Maddi’s family – like no, doubt, hundreds across the country – have not had much chance to rally against it.

It seems this has not been sufficiently tested. For example, the driver behind the change is the cost per ‘quality adjusted life year’ – a mechanism that naturally discriminates against those with rare diseases as there are fewer people who may benefit, so costs are naturally higher. It does not seem to take into account where current research is, and whether it is worth encouraging. Calls for a ‘pause’ in the change are well judged – there are hard decisions to be made in health funding at all levels, but these should not equate to pulling the rug from under people’s feet in cost-cutting drives.