Most drugs are licensed for use in conditions where the drug manufacturers are able to recruit enough patients into clinical trials and where they will yield enough sales to justify running the trial. This presents a consistent problem for patients with rare diseases, whose access to medication is limited because not enough patients are available to run a clinical trial.

Under new plans, the Department of Health will commission expert assessments of the evidence on the use of off-label medicines, including in rare conditions. These assessments will be designed to inform doctors’ decision-making and patients’ choices, not to provide a yes or no recommendation.