In recent years, the National Institute for Health and Care Excellence (NICE) has increasingly agreed to reimburse innovative products with high levels of uncertainty as part of managed access agreements (MAAs) while new data are collected; namely, this has occurred through the new Cancer Drugs Fund (CDF) and highly specialized technology (HST) appraisal pathway. This research aimed to provide a review of ongoing data collection arrangements as part of MAAs agreed with NICE.

We reviewed all current MAAs entered into between the National Health Service (NHS) England and manufacturers as of 24 November 2017 and extracted relevant information related to the data collection arrangements.

Thirteen MAAs were identified (10 through the CDF; 3 through HST). All MAAs involved an observational data collection agreement. The source of observational data collection was existing NHS databases (11 MAAs: 85 percent), existing independent registries (1 MMA: 8 percent [ataluren]); bespoke MAA registry maintained by manufacturer (1 MAA: 8 percent [asfotase alfa]), and registries developed as a requirement for regulatory approval and maintained by the manufacturer (1 MAA: 8 percent [elosulfase alfa]). Only 4 MAAs (asfotase alfa, ataluren, elosulfase alfa, and venetoclax) had observational data collection as the sole basis of the data collection agreement. The other 9 MAAs (69 percent; all from the CDF) also required on-going data collection from clinical trials as a key component of the data collection agreement.

This research shows that current MAAs have predominantly utilized either ongoing data collection (e.g. from RCTs) or existing registries to date for which limited additional set-up administration and costs would be required. However, NICE plan to increase the use of MAAs, with ongoing NICE consultation for changes in the appraisal process to expand MAAs to include all indications. In future, manufacturers will have more opportunities to explore and leverage innovative and bespoke MAAs to help achieve access.