Classification: Official
Publication approval reference: PR00254

The Medicines Repurposing Programme identifies and progresses opportunities to use existing licensed medicines in new ways. Our aim is to improve clinical outcomes, patient experience, and value for money through a tailored support programme.

The programme considers medicines with a potential repurposed use that is not within the terms of an existing marketing authorisation. For example, when the repurposed use:

is for a new condition or patient group or requires a different dose or treatment schedule
requires a reformulation, a different mode of administration or a different strength of the medicinal product.

The programme can support generic, biosimilar, and branded medicines. Candidate medicines need to meet all the following eligibility criteria to be considered for entry into the programme. In exceptional cases, the programme’s steering group may decide to waive one criterion.

Eligibility criteria

Patient/service user benefit and evidence base

1. Some evidence of safety and efficacy in the repurposed use already exists, such as a completed phase 2 trial (note 1).

2. The available evidence suggests that the repurposed medicine provides clinical outcomes and patient benefits that are as good as, or better than, the current NHS standard of care (note 2). The appendix provides guidance on how existing evidence is assessed.

Need for repurposing

3. There is current (or likely future) support or demand for the medicine from clinicians or patient groups, or there is a national policy requirement for its consideration (for example, if the repurposed medicine creates additional resilience in treatment options for a particular indication).

4. NHS patients do not currently have equitable access to the medicine in its repurposed use; that is:

the medicine is not currently used in the NHS for the repurposed indication, or at the repurposed dose, or in the repurposed formulation, or
there is some NHS use of the medicine in the repurposed indication/dose/ formulation, but the variation in use is not explained by different clinical need, individual patient choice or the demographics of an area, and further action is expected to enable increased and equitable access.

Feasibility

5. The medicine is already licensed in Great Britain or the UK.

6. The actions required to repurpose the medicine are within the capability of the programme and the supporting agencies, feasible within the resources and capacity available, and likely to be successful in promoting increased and/or more equitable access within the NHS.

Prioritisation criteria

When a medicine meets the eligibility criteria, the medicines repurposing steering group will use the following criteria to inform decisions about which medicines to adopt into the programme, and their relative priority. The criteria act as a guide to deliberative discussion; there is no scoring system and medicines will be judged against each other for the available resource, which may vary from time to time. Each case will be assessed on its individual merits – not all criteria need to be met.

1. The programme will prioritise those treatments with greater patient benefit compared with current NHS standard of care (note 2). Patient benefit could include:

improved clinical outcomes – which could encompass length of life, quality of life or both
improved experience of care
reduced health inequalities.

When discussing patient benefit, the steering group will consider the number of patients affected and the proportion likely to benefit from the repurposed medicine.

2. The programme will prioritise treatments that have the potential to deliver good value for money for the NHS. Medicines that increase costs may be eligible for the programme, provided they are expected to be cost-effective (note 3). The steering group will also consider the value for money of the repurposing project itself, considering the expected expenditure, degree of risk and potential benefits to patients and the NHS.

For proposals submitted by pharmaceutical companies, the steering group will consider: relevant legislation, the principles referred to in existing policies such as the NHS Commercial Framework for new medicines, who will own intellectual property generated during the project, whether there is a compelling need for support from the programme, and whether the repurposed medicine will be available to the NHS at a price that represents good value for money.

3. The programme will prioritise treatments that have the potential to help manage NHS and social care capacity. For example, the programme may prioritise medicines that reduce the need for emergency admissions, outpatient appointments, or surgery (provided clinical outcomes are expected to be at least as good as the current NHS standard of care).

Over time, the steering group would like to test and demonstrate the success and long-term viability of the programme by building a broad portfolio of medicines. The programme will therefore aim to encompass a range of conditions, care settings, commissioning arrangements and stages of development, and also to test different types of partnership agreement to secure a licence.

Decision options

Having assessed candidate medicines against the eligibility and prioritisation criteria, the steering group will choose from three options.

1. Enter the programme. The programme team will actively support the repurposing project, using programme funds when appropriate, and will coordinate and communicate across agencies. However, being in the programme does not guarantee a specific outcome such as funding a trial or granting a licence variation. This is because each agency involved in the programme acts independently and subject to its own statutory and regulatory governance framework.

2. Place on the monitor list. The programme team will check for developments at a specified date, such as the anticipated completion date of ongoing research. Being on the monitor list does not guarantee future entry to the programme.

3. Stand down. No further action would be taken until a voluntary-sector organisation, healthcare professional or company provides clear evidence to the programme to show that the situation or evidence base has materially changed, and that as a result the medicine may now meet the eligibility and prioritisation criteria.

The criteria will be reviewed in January 2025, or sooner if operationally necessary.

Appendix: Evidence assessment

When assessing the existing evidence base, the programme will consider the following issues when relevant:

The research design and risk of bias, with a preference for research higher up the hierarchy of evidence when feasible.
The population(s) studied and generalisability to NHS patients.
The dose of the repurposed medicine and the rationale for choosing that dose.
The comparator(s) and their relevance to the standard of care in the NHS (see note 2).
The outcomes used, with a preference for measures of health benefits and adverse effects that are important to patients and their carers.
Whether the observed treatment effect is large enough to be viewed as clinically meaningful.
In general, the programme prefers direct evidence gathered using the population, intervention, comparators and outcomes of most relevance to the NHS. Where indirect evidence is used to support a proposal, the programme will consider the generalisability of that evidence. For example, if the research used a surrogate outcome such as a biomarker, the programme will consider whether it is a good predictor of outcomes that matter to patients and their carers.
Large-scale evidence generation may be challenging for rare conditions. The assessment will take this into account and will not disadvantage rare conditions.

Note 1: Phase 2 clinical trials are designed to study a medicine’s efficacy and safety. The trial is conducted in people with the relevant condition or disease, and measures common short-term adverse effects and risks.

Note 2: For some conditions, there may not be published guidelines or an established standard of care. In which case, proposers are asked to seek clinical advice on the usual treatment options for the condition, where possible from more than one NHS provider, and to include this in their proposal form.

Note 3: When assessing financial impact, the programme will consider NHS and personal social services costs for the repurposed medicine compared with current standard of care. Thus, the analysis will include not only the cost of the medicine itself but also wider impacts such as the need for follow-up care. A detailed cost analysis may not be available at the initial prioritisation stage. Instead, when appropriate the programme will consider a summary analysis of the main cost elements in a care pathway.