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Better information on unlicensed drugs for rare disease patients

Article: 8034603 pillsNICE is to take responsibility for providing advice on unlicensed and off-label medicines for use in special circumstances. This will help patients with rare diseases get better access to drugs, under new plans announced by the Department of Health.

Unlicensed and off-label medicines have a valuable role. They meet the special needs of certain patients when licensed medicines cannot. However, there is currently insufficient information for doctors and patients to decide whether these medicines are safe and effective, and when they are most likely to yield good patient outcomes.

Patients with rare diseases, such as certain cancers like lymphoma or autoimmune diseases, may have limited access to medicines as there are often not enough patients to run the clinical trials needed to develop a licensed drug. Some of these patients could benefit from treatment with unlicensed or off-label drugs.

It is estimated that around 1,000 specific requests for off-label drug use are made to NHS commissioners in England every year.

Under the new plans, NICE will commission expert assessments of the evidence on the use of off-label medicines, including in rare conditions, beginning in spring 2012. This will not constitute formal NICE guidance, but will be a summary of the available evidence on selected unlicensed drugs to inform local decision-making.

The work will focus on helping clinicians and patients to access information on the "off-label" or unlicensed use of medicines in cases where there is no appropriate licensed alternative to treat a particular condition. Its focus will include, but not be exclusive to, drugs for rare conditions.

Dr Gillian Leng, Deputy Chief Executive of NICE, said: “This is an exciting move that has the potential to make a big difference for a group of patients who are currently faced with a real lack of treatment options.

“We are delighted to be involved and will use our expertise to commission work examining the evidence-base for the effectiveness of off-label medicines. This will help to inform doctors' decision-making and patients' choices.”

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.”

David Jayne, Director of Vasculitis and Lupus Clinic at Addenbrookes Hospital and NHS consultant said:“In rare autoimmune diseases, guidance to support the NHS has been sadly lacking.

“For some conditions, professionals have produced valuable advice but this has lacked a national profile. Combining the expertise of these professional bodies with the authority of NICE could be immensely valuable.

“This announcement will help address variability in the quality of care and will also remove unnecessary duplication. It is an essential prerequisite to introducing national quality standards of care for rare diseases”

Professor Anthony Redmond, Chair of the Arthritis and Musculoskeletal Alliance (ARMA), said: “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.”

Andrew Wilson, Chief Executive of the Rarer Cancers Foundation, added: “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.”

5 October 2011

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Accessibility | Cymraeg | Freedom of information | Vision Impaired | Contact Us | Glossary | Data protection | Copyright | Disclaimer | Terms and conditions

Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.