Presentation (CTI)

OBJECTIVES: Early or expanded access (EA), also known as compassionate use programmes are increasingly used to provide patient access to investigational medicines (consistent with applicable laws and regulations), while facilitating real-world data collection in a diverse range of patient representation. This investigation aimed to examine the recent usage of data from EA programmes such as the Early Access to Medicines Scheme (EAMS), in National Institute for Health and Care Excellence (NICE) highly specialised technologies (HSTs), and to understand the role EA data plays in NICE decision-making in rare disease.
METHODS: NICE HSTs published between January 2020 and June 2025 were collected and reviewed. Final draft guidance, committee slides and papers were obtained and searched for the keywords ‘early access’, ‘expanded access’, ‘pre-approval access’, ‘named patient access’ or ‘compassionate use’. HSTs in which EA data was incorporated into clinical, economic and/or supportive evidence were included. Relevant EA data was extracted.
RESULTS: NICE have published recommendations for 21 HSTs since 2020 and at least one reference to EA was made in 81%. EA data was used to inform clinical efficacy or safety data in 29% (6/21 HSTs), it served as supportive evidence to economic modelling, budget impact assumptions, or real-world evidence in 5 HSTs, while EA data was not included in 48%. All the technologies which included EA clinical data were recommended for use. HSTs which mentioned but did not integrate EA data cited insufficient results, out of scope comparators, or lack of mandate for further data collection.
CONCLUSIONS: Although EA programmes have the capacity to provide substantial clinical benefits to patients, support real-world evidence generation, and inform payer healthcare decision-making, there remain substantial challenges in integrating retrieved evidence to support health technology assessment submissions in rare disease.