Andrew Lansley, perhaps learning that he often spends too much time on policy at the expense of the politics, kept his speech to the Conservative Party conference light on technocratic details, as my colleague, Mike Birtwistle, advised last week. Yet one particularly technical announcement quietly remained: news of major progress in the treatment of patients with rare diseases came quietly, via a single unapplauded line in Lansley’s speech to a half empty auditorium.
Under the plans announced, the Department of Health will commission NICE to develop evidence guides on the use of off-label medicines for rare diseases to help inform doctors’ decision-making and patients’ choices. For very rare conditions, there may well be no licensed treatments (it is very difficult to conduct trials given the paucity of patient numbers). In these circumstances, doctors have no choice but to prescribe drugs for other conditions which are biologically similar. However, they do so with little information, which can make it difficult to know whether the treatment is the right thing to do, either from a clinical or NHS perspective.
This paucity of information has meant that there have been unavoidable variations in the care provided to patients with very rare conditions and that commissioners have often been at a loss as to whether to provide funding. For these patients, the progress made by the NHS in tackling the postcode lottery of care has been a distant reality, as they have been left to the hands of local-decision making, often taken by commissioners who had little understanding about the complexities of their very rare condition. This will be welcome news to thousands of patients and clinicians alike.
Whilst the announcement is likely to have come as a surprise to some rare disease charities, others have been plugging away on the issue for some time. The announcement is quintessential Lansley – technical and hardly tabloid fodder. Yet the timing of the announcement is no mystery and it is intended to illustrate a broader theme.
Lansley’s conference speech was always likely to be a platform for campaigners against the Health and Social Care Bill as it came under renewed parliamentary scrutiny; its Second Reading in the House of Lords starts next week. Indeed, the day had begun for the Health Secretary with the latest onslaught, this time in an open letter to the Times from doctors calling for the Bill to be scrapped. However, as with much of the rest of the speech, this announcement was an attempt to move the debate forward from sticky areas of debate on the Bill, to demonstrating why clinical freedom matters. Moreover, the notable lack of any associated spending commitment makes it an affordable win in difficult times. Whilst the programme will go unrecognised in almost all mainstream media, it is a small but significant victory to a long under-prioritised disease area for the NHS.
Download the report from the Expert Group on Rare Autoimmune Diseases, Improving NHS Services for Rare Autoimmune Disease.