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

Tobias B. Polak, David Gj Cucchi, Joost van Rosmalen, Carin A. Uyl-de Groot

Research output: Contribution to journalReview articleAcademicpeer-review

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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/or resource use. We qualitatively describe the five appraisals with the most occurrences of EA-related terms. Primary outcome measure Number of TAs that used EA data to inform safety, efficacy and/or resource use analyses. Results In 54.2% (206/380 appraisals), at least one reference to EA was made. 21.1% (80/380) of the TAs used EA data to inform safety (n=43), efficacy (n=47) and/or resource use (n=52). The number of TAs that use EA data remained stable over time, and the extent of EA data utilisation varied by disease area (p=0.001). Conclusion NICE uses EA data in over one in five appraisals. In synthesis with evidence from well-controlled trials, data collected from EA programmes may meaningfully inform cost-effectiveness modelling.
Original languageEnglish
Article numbere052186
JournalBMJ Open
Issue number1
Publication statusPublished - 6 Jan 2022


  • general medicine (see internal medicine)
  • health economics
  • health policy

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