Better access to drugs for rare disease patients promised by UK Health Secretary

UK Health Secretary Andrew Lansley this week announced help for patients with rare diseases, whose doctors may not be able to provide them with unlicensed or “off label” medication. 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, he said. This presents a consistent problem for patients with rare diseases, whose access to medicines is limited because too few patients are available to run a clinical trial.

Similarly, the drugs watchdog the National Institute for Health and Clinical Excellence (NICE) cannot normally appraise drugs outside their licensed indication, which means they cannot be recommended for use on the National Health Service, he said. At local level, the NHS often does not have the evidence it needs to make an informed decision. Evidence on the scale of the problem is limited, but it is estimated that around 1,000 specific requests for off-label drug use are made to NHS commissioners in England every year.

Many rare conditions can be treated with drugs outside their licensed indication. Examples include rarer cancers and autoimmune conditions. Unlicensed or off-label drugs may also be considered for some patients with more common conditions.