Question to the Department of Health and Social Care:

To ask His Majesty's Government what steps they have taken to deliver on the commitment made in the Life Sciences Vision, published in July 2021, to introduce new treatment possibilities such as novel cell and gene therapies for patients with rare genetic diseases such as haemophilia.

The Life Sciences Vision sets out how we will accelerate the adoption and uptake of innovative new treatments and technologies, including for rare diseases. The Accelerated Access Collaborative is central to delivering these commitments and has initiatives aimed at accelerating adoption of innovation in the National Health Service.

The UK Rare Diseases Framework outlines our priorities in tackling rare diseases such as haemophilia. This includes improving access to specialist care, treatment and drugs. England’s first Rare Diseases Action Plan sets out actions to achieve this, including developing a strategic approach for gene therapies and other Advanced Therapy Medical Products. Two gene therapies for adults with haemophilia B are expected to be available within the next few years.