Medical Research: International Rare Diseases Research Consortium

Lord Walton of Detchant Excerpts

Thursday 28th February 2013 Lords Chamber

[Earl Howe]

My Lords, we have just announced the establishment of a rare diseases stakeholder forum. As my noble friend rightly mentions, it will be established shortly to bring together a wide range of stakeholders, including organisations representing those with rare diseases, to ensure that the patient voice is part of the discussion that we must have leading up the publication of the UK plan for rare diseases. The 100,000 genomes initiative, which my Department is funding, is about pump-priming—the sequencing of the genomes of 100,000 NHS patients—with the purpose of translating genomics into the NHS. This capacity will be allocated specifically to cancer, rare diseases and infectious diseases. The service design work will be completed by June and we aim to put contracts in place by April next year.

[Lord Walton of Detchant]

My Lords, does the Minister accept that recent research in genomic medicine has led to the progressive introduction of orphan and ultra-orphan drugs, some of which are capable of reversing partially or completely the genetic effect of many such rare diseases—not least, for example, muscular dystrophy? However, these drugs are extremely expensive and are therefore likely to be commercially unsuccessful because they help only a relatively small number of patients. Now that the Government have abolished the Advisory Group for National Specialised Services, can the Minister assure the House that, when responsibility for providing those drugs on the NHS falls to the national Commissioning Board on the advice of NICE from April this year, those rare diseases and their drugs will be given appropriate priority?

[Earl Howe]

My Lords, yes I can give that assurance. As the noble Lord will know, we laid regulations specifying those specialised and highly specialised conditions which the NHS Commissioning Board will be responsible for commissioning. I can also reassure him that the focus on research into rare conditions will not be lost. Indeed, I am sure that he will be aware that the National Institute for Health Research has recently specifically invited submission of research proposals into interventions for very rare diseases. The call encouraged multidisciplinary research proposals as well as study designs and approaches to recruitment of patients.