NICE publishes first Evidence summary: unlicensed or off-label medicine
Tranexamic acid (Cyklokapron, Pfizer) for significant haemorrhage (severe bleeding) following trauma is the first topic to be covered by NICE as part of its new service to provide high quality information to the NHS and patients in England about the use of unlicensed and off-label medicines.
Produced by NICE's Medicines and Prescribing Centre (which was formed following the transfer into NICE of the National Prescribing Centre in April 2011), these outputs provide a summary of the published evidence for selected unlicensed or off-label medicines that are considered to be of significance to the NHS, where there are no clinically appropriate licensed alternatives. They will provide information for clinicians and patients to inform their decision-making and support the construction and updating of local formularies. Although the strengths and weaknesses of the relevant evidence of a drug's efficacy, safety and cost are critically reviewed within the NICE evidence summaries, the summaries do not constitute formal NICE guidance.
Significant haemorrhage and its complications are a considerable cause of death following severe trauma and are responsible for 80% of deaths in the operating theatre and up to 50% of deaths in the first 24 hours after injury. Tranexamic acid is a drug used to stop or reduce unwanted bleeding. It does this by stopping blood clots from breaking down, allowing the clots to stop the bleeding. It is licensed in Europe and the UK for use in certain conditions, for example after surgery, or in women with heavy menstrual bleeding. However, it does not currently have UK marketing authorisation for use after trauma, so use in this way is described as ‘off-label'. Evidence from a large high quality study carried out in 40 countries has shown that a short course of tranexamic acid given within 8 hours of injury to adult trauma patients with, or at risk of, significant bleeding increases the proportion of people who survive their injuries. A further analysis found that giving tranexamic acid within 3 hours of injury was optimal. A health economic analysis has found that tranexamic acid for the prevention and treatment of significant haemorrhage in trauma patients has an incremental cost of $64 international dollars per life saved.
Professor Mark Baker, Director of the Centre for Clinical Practice at NICE, said: “While a licensed medicine meets acceptable standards of efficacy, safety, and quality, clinical situations will arise where the use of unlicensed medicines or the use of medicines to treat conditions for which they are not licensed may be judged by the prescriber to be in the best interest of the patient. This is particularly the case for certain rare diseases, where there are often not enough patients to enlist on the clinical trials that are needed to develop a drug.
“In these situations it is extremely important that any decision to use an unlicensed or off-label medicine is made on the basis of the best available evidence of safety and efficacy and whether the medicine is more or less likely to improve outcomes for individual patients. Up until now there has been a lack of nationally available, good quality information about using unlicensed and off-label medicines. This new resource from NICE, while not formal guidance, will help to ensure that the decisions of clinicians and the choices made by patients are properly informed and made on the basis of the best available evidence. It will also help avoid potential duplication of effort where, as happens currently, different NHS organisations undertake their own analyses of the evidence - NICE will save them the trouble by doing it once.”
NICE has appointed healthcare research company Bazian to produce the evidence summaries which will form the basis of the final NICE information.
Dr Vivek Muthu, Bazian CEO, said: “Bazian's sole purpose and focus of expertise is the independent evaluation of evidence to improve the clinical value of healthcare. We're delighted, therefore, to be working closely with NICE on such an important project, which does just that. This evolving programme will provide easy-to-use summaries of the evidence to inform patients and prescribers, to help with shared decision making, and ultimately to make off-label and unlicensed medicines use safer and more effective. It is a big objective and involves some challenging tasks, and we're excited to be developing this programme in collaboration with our colleagues at NICE.”
NICE evidence summaries for unlicensed or off-label medicines are part of a comprehensive suite of advice and support for delivering quality, safety and efficiency in the use of medicines produced by the Medicines and Prescribing Centre at NICE. Further information about the work of the Medicines and Prescribing Centre at NICE, including details about the evidence summaries currently being developed on unlicensed or off-label medicines and the evidence summaries: new medicines, is available on the newly-launched MPC section of the NICE website.
Notes to Editors
About the Evidence summaries - unlicensed/off-label medicines
1. Although they do not constitute formal NICE guidance, the principles of openness and transparency which are fundamental to NICE's approach to guidance development will be applied during the development of the NICE evidence summaries for unlicensed or off-label medicines. NICE will ensure that stakeholders, including manufacturers, regulators, healthcare professionals and patients and carers are engaged effectively during the development of the Evidence summaries, including topic scoping and consultation on the draft summary.
2. An interim process statement providing an overview of the key process principles and describing all stages of the development of the NICE evidence summaries for unlicensed or off-label medicines is available on the NICE website.
3. The interim process statement will be superseded when the final process guide is issued following engagement and discussions with key groups.
4. The NICE evidence summary for tranexamic acid, together with a version for patients, is available on the NICE website.
5. The following topics will form the first tranche of NICE evidence summaries for unlicensed or off-label medicines:
- Melatonin - sleep disorders in children
- Diltiazem cream 2% - anal fissure
- Glyceryl trinitrate 0.2% ointment - anal fissure
- Midodrine - postural hypotension
- Metformin tablets - Polycystic ovary disease
- Modafinil tablets - decreasing fatigue in MS
- Magnesium glycerophosphate oral - hypomagnesaemia
- Clonidine - ADHD as sole treatment and adjunct
- Rituximab in mantle cell lymphoma
About the Medicines and Prescribing Centre at NICE
1. Further information about the Medicines and Prescribing Centre at NICE, including the Evidence summaries: unlicensed and off-label medicines and Evidence Summaries: new medicines, is available on the NICE website.
1. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health.
2. NICE produces guidance and advice in several areas of health:
- public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
- health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
- medicines and prescribing advice - advice on the use of selected unlicensed and off label medicines where there are no clinically appropriate licensed alternatives, and advice on the use of selected new medicines or new indications for existing medicines that are not being considered by NICE as part of its technology appraisals programme
- clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.
3. NICE produces standards for patient care:
- quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
- Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients
4. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.
Bazian is dedicated to optimising the value that healthcare delivers. It does this by independently appraising and analysing research evidence and data on the safety, effectiveness and cost of medicines, devices, surgery, diagnostics, health services and public health measures. Its clients include NICE, NHS commissioners, NHS Choices Behind the Headlines, health insurers and international agencies.
This page was last updated: 15 October 2012