Question to the Department of Health and Social Care:

To ask His Majesty's Government what assessment they have made of the UK's fall in ranking from fifth to eleventh between 2020 and 2025 in the EFPIA Waiting to Access Innovative Therapies Indicator for orphan medicine availability; and what steps they are taking to ensure the UK remains a priority 'first-launch' market for global biotechnology firms.

No specific assessment has been made. The Government recognises how important it is that patients with rare diseases are able to benefit from access to effective new medicines. The National Institute for Health and Care Excellence (NICE) has a strong track record in supporting access to new medicines for patients with rare diseases with 89% of the rare disease medicines that it has appraised since March 2024 recommended for some or all of the eligible patient population.

Through the Life Sciences Sector Plan and the 10-Year Health Plan, we are taking steps to make the United Kingdom a faster place to approve and adopt new medicines. This includes wider use of real-world evidence, more flexible commercial deals, faster NICE guidance, and a new joint NICE and Medicines and Healthcare products Regulatory Agency pathway to shorten the time from approval to National Health Service use.

We recognise that there is more to do to improve and accelerate access to new medicines for NHS patients and the steps that we are taking as part of the pharmaceuticals partnership with the United States are already resulting in medicines, including medicines for patients with rare diseases, becoming available to NHS patients that may not otherwise have been recommended.