Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to support the use of evidence-based off-label medicines where there is no commercial incentive for a pharmaceutical company to seek a marketing authorisation.

The Medicines and Healthcare products Regulatory Agency (MHRA) is an executive agency of the Department of Health and Social Care and regulates medicine, medical devices and blood components for transfusion in the UK. The MHRA is responsible for ensuring medicines meet appropriate standards of safety, quality, and efficacy.

In the UK, off-label prescribing refers to the use of a licensed medicine outside the terms of its marketing authorisation, such as for a different condition, dose, or age group. The MHRA regulates the licensing, safety and quality of medicines and monitors adverse effects but does not control medical practice. Responsibility for off-label prescribing lies with the prescriber, who must ensure it is in the patient’s best interests, supported by evidence, and discussed with the patient.

The General Medical Council (GMC) provides professional standards, while NHS trusts and the National Institute for Health and Care Excellence (NICE) may set local or national policies to guide safe and appropriate use.

Where there is no commercial incentive for a pharmaceutical company to seek a marketing authorisation, the use of independent, evidence-based guidelines (such as those developed by NICE, professional bodies, or specialist societies) can support clinical decision-making and standardise safe practice. Published research, systematic reviews, and expert consensus also provide a foundation for justifying off-label use in areas such as paediatrics, oncology, and rare diseases.

Thus, the MHRA oversees the medicine itself, while prescribers, the GMC, and the NHS govern how off-label prescribing is carried out in practice, supported by independent clinical evidence and guidelines when licensing is not pursued.

The MHRA encourages Marketing Authorisation Holders to vary their licences when there is sufficient evidence to change the indication approved. The MHRA will reach out to ask companies to assess when appropriate. This does not indicate that the MHRA will approve the licence, but are prepared to assess the evidence provided.