Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, if he will take steps to strengthen the Orphan drug regulations to encourage research into rare diseases.

The Human Medicines Regulations include specific incentives to support orphan medicines, designed to encourage the development of medicines for rare diseases. The regulations enable the Medicines and Healthcare products Regulatory Agency (MHRA) to grant orphan designation to a medicine intended for the prevention, diagnosis, or treatment of a rare condition. This orphan designation provides the product with a period of market exclusivity for the specified condition, preventing similar competitor medicines from entering the market during this time.

The MHRA further supports developers of orphan medicines by offering a full or partial refund of the marketing authorisation fee following a positive orphan designation. There is evidence that the regulations for orphan medicines do support development, with approximately 150 medicines having been successfully developed and authorised for the diagnosis, prevention, and treatment of rare diseases and given orphan designation in the United Kingdom.

The MHRA will continue to review its approach to orphan medicines and, where necessary, will propose updates to the criteria to further encourage research into rare diseases, to improve patient outcomes.