The UK Department of Health has promised to improve the availability of drugs for patients with rare diseases, whose doctors may not be able to provide them with ‘off label’ medication.
Health Secretary Andrew Lansley said the DoH will commission expert assessments of the evidence on the use of off-label medicines, designed to inform doctors’ decision-making and patients’ choices and not provide a yes or no recommendation.
Patients with rare diseases have limited access to medication as manufacturers are often unable to recruit enough of them to run a clinical trial.
In addition, the National Institute of Clinical Excellence cannot normally appraise drugs outside their licensed indication, which means they cannot be recommended for use on the NHS.
At local level, this means the NHS often does not have the evidence it needs to make an informed decision as to whether to provide off-label drugs.
Lansley said: "The NHS we want to create is one where doctors and patients at local level have the power to take the best clinical decisions together.
"Wherever possible we should apply common sense solutions – especially to longstanding problems such as this – to ensure that patients can access the treatment they and their doctor think best for them," Lansley added.
Rarer Cancers Foundation chief executive Andrew Wilson said: "This announcement is good news for patients with rare cancers and will help ensure all patients get access to good standards of care. No patient should be disadvantaged simply because they are unlucky enough to have a rare disease."