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Review
. 2022 Jan 6;12(1):e052186.
doi: 10.1136/bmjopen-2021-052186.

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

Tobias B Polak  1   2   3 , David GJ Cucchi  4 , Joost van Rosmalen  2 , Carin A Uyl-de Groot  5
Affiliations
Review

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

Tobias B Polak et al. BMJ Open. .

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/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.

Keywords: general medicine (see internal medicine); health economics; health policy.

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Conflict of interest statement

Competing interests: CU-dG has received unrestricted grants from Boehringer Ingelheim, Astellas, Celgene, Sanofi, Janssen-Cilag, Bayer, Amgen, Genzyme, Merck, Glycostem Therapeutics, AstraZeneca, Roche, and Merck. TBP works part-time at myTomorrows and holds stock in myTomorrows. JvR and DGJC declare no conflict of interest.

Figures

Figure 1
Figure 1
Screening and selection of technology appraisals from NICE. EA, expanded access; HST, highly specialised technology; MTA, multiple technology appraisals; NICE, National Institute for Health and Care Excellence; STA, single technology appraisal.
Figure 2
Figure 2
Technology appraisals (TAs) using expanded access (EA) data to support safety, efficacy and/or resource use. (A) Venn-diagram displaying the overlap of safety, efficacy, and/or resource use labelling of TAs. (B): Bar chart of TAs published between 1 January 2010 and 1 January 2021 that did (‘yes’) or did not (‘no’) include data EA programmes to support safety, efficacy and/or resource use.
See this image and copyright information in PMC

References

    1. The National Institute for Health and Care . Guide to the methods of technology appraisal. Nice 2018:1–93. - PubMed
    1. Bell H, Wailoo AJ, Hernandez M. The use of real world data for the estimation of treatment effects in NICE decision making 2016;2016:1–61.
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    1. National Institute for Health and Clinical Excellence (NICE) . Guide to the single technology appraisal process, 2009.
    1. National Institute for Health and Clinical Excellence (NICE) . Guide to the multiple technology appraisal process, 2009.

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