NHS: Specialised Services

Lord Walton of Detchant Excerpts

Wednesday 18th July 2012 Lords Chamber

[Lord Walton of Detchant]

My Lords, I too am deeply grateful to the noble Baroness, Lady Jolly, for initiating this important debate. I have been a long-time supporter of the Rare Disease UK consortium, now chaired by the man who recently was the director of the Genetic Interest Group. A recent editorial in the British Medical Journal in June said:

“Three million in the United Kingdom have a rare disease, defined in both Europe and the USA as a disease that affects fewer than one in 2000 people. It is well recognised that those with rare diseases face intrinsic inequalities in healthcare, and in response to a 2010 recommendation by the European Commission, the UK government, like other member states, agreed to produce a strategy for rare diseases by 2013”.

The Government at the moment are consulting on this very important topic.

Before I come to that I want to say a word about AGNSS, which has proved to be remarkably successful. It has been funded by top-slicing of funds—up to about £100 million a year—from primary care trusts across the UK and it has enabled companies such as Shire Pharmaceuticals to develop enzymatic treatments which have in fact been able to reverse diseases like those referred to by the noble Lords who spoke earlier about the various storage disorders. It has been able to control disease in people with Fabry disease, Hunter syndrome, Gaucher’s disease and others. It has been immensely successful and its future is therefore crucial. It is important that the Government recognise in the consultation process they are undertaking that the needs will increase as time goes by, because the developments in genetic medicine and molecular biology are revealing in many of these devastating and rare diseases single genes whose effects can be controlled to an extent by new forms of treatment. As time goes by, more and more orphan and ultra-orphan drugs to control these rare diseases are coming on stream.

One major concern about the proposals in the Government’s consultation document is that, while it contains proposals on diagnosis and services for rare diseases, policy and treatment is deflected to forthcoming proposals on value-based pricing. Value-based pricing is unlikely to be capable of dealing with medicines for orphan and ultra-orphan diseases because, after all, the number of patients affected by these conditions is relatively small. The drugs that are being developed are going to be very expensive and they are not going to be commercially viable unless they are sponsored and subsidised by funds from an organisation like AGNSS. This is a crucial issue which I hope the Government will be able to deal with. At a recent meeting Sir David Nicholson suggested that it was probable that the functions of AGNSS would be taken over by the national Commissioning Board. I know no decision has yet been made but will the Government tell us what the prospects are, whether the responsibilities will be extended and whether funding for AGNSS is likely to be increased?

Finally, in the light of my own private research I want to mention a disease called Duchenne muscular dystrophy—a devastating disease of young boys causing progressive muscular paralysis. For the first time certain drugs are now coming on stream which have been shown by clinical trials to be effective in delaying the actual progression of this condition. These drugs are so-called molecular patches which overcome defects in the actual gene. This is a form of exon skipping. For these drugs to be effective, several different types of molecular patches may have to be developed. Speeding up that process for different mutations will be difficult but the safety issues are all the same. Can the Government give us an assurance as these molecular patches for this devastating disease become increasingly available that not every single patch is going to have to be tested and subject to regulatory control, and that regulation covering all these patches may be acceptable?

We are dealing with a very important group of diseases which cause immense human suffering. AGNSS, in some form or other, must continue and it must have its functions and, I believe, its funding increased.