NHS: Specialised Services

Baroness Thomas of Winchester Excerpts

Wednesday 18th July 2012 Lords Chamber

[Baroness Thomas of Winchester]

My Lords, I am grateful to my noble friend Lady Jolly for this debate. I do not have to declare an interest because, although I have a rare disease, I do not have an ultra-orphan disease. There will be a lot of repetition in what we are saying but perhaps the very fact that we all want the same thing will send a powerful message to the Government. That message has been particularly loud and clear from all the groups that have been lobbying us and are very worried about the future of the commissioning of services for these very small populations of patients with ultra-rare diseases. They all want a version of AGNSS to continue its invaluable work and they do not want its expertise and experience to be lost when the NHS Commissioning Board takes over the responsibility of commissioning specialised services.

It is something of an irony that as medical research finds more and more treatments for these ultra-rare diseases, and as improvements in diagnosis mean that more people will have a correct diagnosis and therefore potentially live longer because their condition can be treated, the actual drugs and therapies they need might be deemed unaffordable. That is why we need AGNSS more than ever at this point where research is at the forefront of the Health and Social Care Act. That Act now gives the Secretary of State, for the first time I believe, the duty to promote research in the NHS. This will inevitably mean that new therapies will be found for rare disorders. This should be great news, but will it be for the very small proportion of the population who have these ultra-rare conditions? Will they be denied access because of the high cost of treatment? The key question is what value-based pricing, due to be introduced in January 2014, will mean for high-cost low-volume drugs and whether these can be adequately assessed within a new value-based pricing framework.

The Department of Health says that the new system will give patients and clinicians greater access to clinically effective and cost-effective medicines. But experts are not convinced that the system will work without the AGNSS framework, particularly given the situation in Scotland where AGNSS does not operate. The last thing we want is to go back to the old days when people diagnosed with Pompe disease, for example—an ultra-rare but treatable neuromuscular condition which affects fewer than 100 people in England—were not always certain that enzyme replacement therapy would be licensed in England because NICE had to be satisfied that it would be cost-effective. Through the leadership of Sir Michael Rawlings, national commissioning of such orphan drugs was transferred to an advisory body which later evolved into AGNSS, and the dreaded threat of a postcode lottery was removed.

In its short life, as we have already heard this evening, AGNSS has garnered high praise for its thorough evaluation process, which is seen to be open and transparent, using the uniform expertise to evaluate funding for service provision and therapies, thereby avoiding inefficient and artificial separation of commissioning for drugs and services. It is likely that this good practice has led to the UK being looked on favourably as a destination for pharmaceutical industry-sponsored clinical trials. Surely we all want this to continue and develop. Can the Minister say what the timescales are for confirming the future work of AGNSS and can he confirm that the Secretary of State for Health has the duty under the new Act to provide specialised services for all who need them, however rare their disease?