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Regulatory News: UK plans for wider use of ‘off-label’ and unlicensed medicines

29th November 2011

Last month, the UK’s health secretary, Andrew Lansley proposed plans to initiate the wider use of “off-label” and unlicensed medicines, principally for rare disease patients. 

As part of these plans, NICE (The National Institute for Health and Clinical Excellence) will take responsibility for providing advice to clinicians and patients on “off-label” and unlicensed medicines for use in special circumstances where there is no appropriate licensed alternative available.

The aim is to provide doctors, patients and local level NHS with more information on the use of specific medicines for unlicensed indications by commissioning expert assessments on the evidence available. These reports will NOT be considered recommendations for use on the NHS but aim to inform doctors’ decision making and patient’s choices.

“Off label” medicines refer to the use of a medicine outside its licence indication whereas unlicensed medicines do not have a licence in the UK (but may have a licence in other countries e.g. the US).  It is estimated that around 1,000 specific requests for off-label drug use are made to NHS commissioners in England every year.

Large scale clinical trials can be difficult to conduct for rare diseases due to the limited number of potential patients available to test, which consequently limits the variety of licensed medicines available for treatment. 

This move could potentially be very positive for the thousands of sufferers of rare conditions who currently have little choice and options for treatment, NICE’s involvement will ensure knowledge is nationally shared with regards to the safety and efficacy of these type of products.

The announcement has raised questions within the industry about how useful the reports will really be - if there is sufficient evidence to give a good enough picture of the safety and efficacy of a product for a specific indication, could this be used to support a licence application. Will it set a precedent for the off label use of other products for more common conditions that may have more safety data available?

More will be known once the first of the expert assessments are released, work on which will be started by the NHS Evidence arm of NICE in spring 2012.

Reviewed by Sophie Hobson, Senior Regulatory Affairs Executive