TY - JOUR
T1 - Real-world data from expanded access programmes in health technology assessments
T2 - A review of NICE technology appraisals
AU - Polak, Tobias B.
AU - Cucchi, David Gj
AU - van Rosmalen, Joost
AU - Uyl-de Groot, Carin A.
N1 - Funding Information: This research was supported via an unrestricted grant from HealthHolland: EMCLSH20012. HealthHolland is a funding vehicle for the Dutch Ministry of Economic Affairs and Climate Policy that addresses the Dutch Life Sciences & Health sector. Publisher Copyright: © Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
PY - 2022/1/6
Y1 - 2022/1/6
N2 - 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.
AB - 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.
KW - general medicine (see internal medicine)
KW - health economics
KW - health policy
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85122944934&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/34992108
U2 - https://doi.org/10.1136/bmjopen-2021-052186
DO - https://doi.org/10.1136/bmjopen-2021-052186
M3 - Review article
C2 - 34992108
SN - 2044-6055
VL - 12
JO - BMJ open
JF - BMJ open
IS - 1
M1 - e052186
ER -