Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals.

ABSTRACT:

Objectives

To quantify and characterise the usage of expanded access (EA) data in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs). EA offers patients who are ineligible for clinical trials or registered treatment options, access to investigational therapies. Although EA programmes are increasingly used to collect real-world data, it is unknown if and how these date are used in NICE health technology assessments.

Design

Cross-sectional study of NICE appraisals (2010-2020). We automatically downloaded and screened all available appraisal documentation on NICE website (over 8500 documents), searching for EA-related terms. Two reviewers independently labelled the EA usage by disease area, and whether it was used to inform safety, efficacy and

SUBMITTER: Polak TB 

PROVIDER: S-EPMC8739059 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature