OBJECTIVES: Real-World Data (RWD) continues to gain traction across the product lifecycle. Through unsolicited physician requests, Early Access Programs (EAPs) deliver treatments to patients, prior to local approval, when standard and clinical trial options are exhausted.

While their goal is access to treatment, data collected from these heterogeneous patient populations presents unique opportunity for insight. The UK’s Early Access to Medicines Scheme (EAMS) provides access, with the opportunity to generate RWD a core principle. However, most UK EAPs are outside of EAMS, so identifying a low burden model for RWD collection would be useful.

METHODS: An observational study in a rare, hereditary disease was designed to include UK patients accessing EAP treatment via a non-EAMS arrangement.

Utilising Health Research Authority (HRA) feedback, with National Institute for Health and Care Research (NIHR) National Contract Value Review (NCVR) providing a standardised approach to site negotiations, the study was set up. Following collaborative review, Research Ethics Committee (REC) gave their positive opinion.

RESULTS: Study went live in June 2023, completing by October. 100% of EAP patients participated (all were given the opportunity to opt out), this reflected patients’ trust/confidence in their care teams. Baseline/longitudinal data on clinical characteristics and treatment response were collected from medical records, including some patient-reported outcomes. Publications to share with the scientific community are being developed.

CONCLUSIONS: The UK NHS research environment provides features to support and expedite studies working only with data, such as: Proportionate ethical review, NCVR, NIHR and Clinical Research Networks. Care teams are well supported (e.g. Clinical Trial Assistants) to deliver projects working with data, gleaning valuable real-world insights. Particularly in rare disease, haematology and oncology, where EAPs frequently provide early access for patients with substantial unmet need.

Although less conventional, with appropriate/compliant study design, this approach to gathering insights outside the EAMS framework was effective.