The Department of Health says it is taking steps to build a better evidence base for when off-label drug treatment is appropriate, with the aim of improving clinical practice and the consistency of care offered to patients with rare conditions.

Ministers say they will commission expert assessments of the evidence on the use of off-label medicines including, but not exclusively, those used to treat rare conditions. The work will be delivered by the National Institute for Health and Clinical Excellence (NICE) through its NHS Evidence arm, beginning next spring, and the assessments will be designed to inform doctors' decision-making and patients' choices, not to provide a "yes" or "no" recommendation, it says.

Explaining the background to the initiative, the Department notes that most drugs are licensed for use in conditions where the manufacturers are able to recruit enough patients into clinical trials and where they will yield sufficient sales to justify running the trial. However, 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.

Similarly, NICE cannot normally appraise drugs outside their licensed indication, meaning that this use cannot be recommended for the NHS, while at the 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, says the Department.

Announcing the initiative, Health Secretary Andrew 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," he added.

The announcement has been welcomed by clinicians and patient groups. Andrew Wilson, chief executive for the Rarer Cancers Foundation (RCF), pointed out that while the objective should always be to develop licensed drugs for rare diseases, this is not always possible, so this initiative is "a common-sense move which will make a big difference." 

"When there are no licensed drugs available for a condition, it can be difficult to know how best to treat it. It is important that doctors are able to use their clinical judgement. In order to support them, they need to have access to advice and information on what works best," he said.

In rare autoimmune diseases, guidance to support the NHS has been "sadly lacking," said David Jayne, director of the vasculitis and lupus clinic at Addenbrookes Hospital. For some conditions, professionals have produced valuable advice, but this has lacked a national profile, he said, and combining the expertise of these professional bodies with the authority of NICE could be "immensely valuable." 

The initiative "will help address variability in the quality of care and will also remove unnecessary duplication," he said, adding: "it is an essential prerequisite to introducing national quality standards of care for rare diseases."

And Professor Anthony Redmond, chair of the Arthritis and Musculoskeletal Alliance (ARMA), noted that many of the more than 200 musculoskeletal conditions are very rare, which means there may be no drugs licensed to treat them.

"NICE could play a valuable role in collating the evidence about what works in these conditions, and this will be an important resource for clinicians in providing the best possible treatment to patients, and for patients in making an informed decision about the most appropriate option for them," said Prof Redmond.

The Association of the British Pharmaceutical Industry (ABPI) said that it welcomes all initiatives which seek to improve appropriate access to medicines and improve the health of UK patients, and that while it supports this initiative in principle, "it will always be preferable for a medicine to be licensed for the purposes for which it is used and has been subject to the rigorous scrutiny of the regulatory authorities."

"The regulatory and licensing system in the UK exists to protect the public, so the circumstances when an unlicensed or off-label medicine is prescribed should continue to be strictly limited to occasions when there is no suitable licensed alternative available and use is in the best interests of the patient concerned," said ABPI chief executive Stephen Whitehead.