Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether his Department has made an assessment of the potential impact of suspending the medicines repurposing programme on people affected by rare conditions.

In April 2025, a decision was made to suspend the national Medicines Repurposing Programme. The decision was made with consideration to the following factors:

• The programme team and partners have learned that there are fewer repurposing opportunities in practice than was originally envisaged when the programme was established. Partly this is because many opportunities to use existing medicines in new ways can be delivered without a formal repurposing programme;
• Very few repurposed medicines have a strong enough research evidence base to support a licence variation application; and
• The planned integration of NHS England into the Department.

There are alternative sources of advice and support for clinicians or charities wanting to repurpose a medicine, including for a rare condition, such as the Medical Research Council, National Institute for Health and Care Research, and scientific advice from the Medicines and Healthcare products Regulatory Agency (MHRA).

Medicines repurposing means using an existing medicine in a new way, outside of its current marketing authorisation. Clinicians are very familiar with the option of prescribing medicines outside their licence, known as off-label use, particularly within specialised care. Off-label access is often agreed at a local level, informed by the available evidence and clinical guidelines, and where the clinician feels this best meets the needs of the patient. When appropriate and supported by the available evidence, national guidance from organisations including the National Institute for Health and Care Excellence or the British National Formulary could potentially support equitable patient access to repurposed medicines.

A further potential route to support patient access to a repurposed medicine used in specialised care in England is to develop a national clinical commissioning policy. A policy will confirm whether a specific treatment should be routinely available to eligible patients in the National Health Service. A policy can only be developed if the medicine is used within prescribed specialised services, namely services for which NHS England is the accountable commissioner, and not services where the accountable commissioners are integrated care boards. National clinical policies are based on the available research evidence and may be subject to a process of funding prioritisation, depending on the cost of the treatment concerned. A NHS clinician can propose a new or revised national clinical commissioning policy where there is sufficient supporting evidence. More information on the process is available at the following link:

https://www.youtube.com/watch?v=Ya1A9WTzQXQ

NHS England develops clinical commissioning policies in line with published methods and eligibility criteria, which have been subject to consultation. A clinical commissioning policy can recommend medicines that are used on-label or off-label. More information on the policies is available at the following link:

https://www.england.nhs.uk/publication/methods-national-clinical-policies/

The national medicines repurposing programme included a small number of medicines to treat rare diseases. In some cases, the programme supported academics to obtain MHRA scientific advice and gather evidence to support a licensing application. To date, no licensing applications have been received via the programme for medicines to treat rare disease.