NICE process and methods

4 Topic identification, prioritisation and selection

4 Topic identification, prioritisation and selection

4.1 Topic identification and prioritisation

The NICE Medicines and Prescribing Centre is responsible for managing the process of topic identification, prioritisation and selection for 'Evidence summaries: unlicensed and off-label medicines' (ESUOMs). Topics are selected where a demand for information has been identified.

There is no established horizon scanning service to identify potential topics for this work stream. A new approach to gathering this information is therefore required.

Figure 1 The main steps in the process for topic identification, prioritisation and selection for ESUOMs

Figure 1 The main steps in the process for topic identification, prioritisation and selection for ESUOMs

Stage 1: Identifying potential topics

Partner organisations[3] are approached up to twice a year to suggest potential topics. They are asked to consider the exclusion criteria in box 1 when suggesting topics. These criteria are designed to filter out topics unsuitable for development into an ESUOM and ensure each topic selected will add value. Previous inclusion of an unlicensed or off-label medicine in a NICE clinical guideline is not necessarily an exclusion criterion for its selection for an ESUOM (see section 1.2).

Box 1 Long list: exclusion criteria

  • Use of the unlicensed or off-label medicine instead of licensed alternatives is being proposed solely on economic grounds.

  • The topic relates to the use of a licensed medicine for a licensed indication but by an unlicensed delivery route or in a modification to the licensed formulation.

  • The topic covers population screening, immunisation or HIV treatment (as these are normally covered by other bodies).

  • High-quality, up-to-date reviews are already available from a NICE-accredited source.

  • A marketing authorisation for the medicine in this indication is being sought and is expected to be granted within 2 years.

The suggestions from the partner organisations and any other potential topics identified through NICE networks, are compiled to form a list of potential topics.

Stage 2: Filtration: applying the exclusion criteria

The NICE Medicines and Prescribing Centre check the potential list of topics against the exclusion criteria (see box 1). Topics that do not meet the criteria are removed from the list and a long list for prioritisation is created.

Stage 3: Prioritisation: developing the short list

The Unlicensed and Off-label Medicines (UOM) Prioritisation Panel (see section 3.4) meets twice a year to consider the priority for each topic on the long list. They advise NICE on the priority of each topic using the prioritisation criteria in box 2.

Box 2 Short list: prioritisation criteria

  • Demand for information: what is the volume of requests from the NHS for information on the topic?

  • Clinical impact: what is the potential of the medicine (if effective) to improve significantly the quality of life for the people in whom it might be used?

  • Variation in practice: is there large variation in clinical practice, significant divergence of clinical opinion, or reports of difficulty of access to the unlicensed or off-label medicine?

  • Need for information: how much uncertainty exists on the risk:benefit balance of the unlicensed or off-label medicine?

The advice from the UOM Prioritisation Panel is considered by NICE and used to produce the short list of topics that NICE intend to develop as ESUOMs.

Stage 4: Approval

The Director of the NICE Centre for Clinical Practice reviews the short list of potential ESUOM topics and gives approval of the topics on which NICE should proceed to develop ESUOMs. Once topics are approved, the manufacturers[4] of the medicines are informed of the intention to produce these ESUOMs and the topics are added to the NICE Forward Planner, as well as being added to the ESUOM page of the Medicines and prescribing section of the NICE website

Stage 5: Development

The NICE Medicines and Prescribing Centre develops an ESUOM for each of the approved topics.


[3] Partner organisations are organisations with a particular interest in the unlicensed and off-label use of medicines. They include professional and patient bodies and trade organisations such as the Royal College of Paediatrics and Child Health, Rare Diseases UK, the Chief Pharmacists Network and the Association of the British Pharmaceutical Industry (ABPI). In addition, the NICE Medicines and Prescribing Associates may also suggest topics. A list of partner organisations is available on the NICE website, but is not intended to be exhaustive, as we aim to engage with all who can help identify potentially suitable topics.

[4] For some unlicensed or off-label medicines, more than 1 manufacturer may be involved in the development of the drug. On these occasions, all relevant manufacturers based in the UK are contacted for information.