Real-world data from expanded access programmes in health technology assessments
| Autor: | Polak, Tobias, Cucchi, David G.J., van Rosmalen, Joost, Uyl - de Groot, CA (Carin), van de Vathorst, Suzanne |
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| Přispěvatelé: | Epidemiology, Health Technology Assessment (HTA), Public Health |
| Jazyk: | angličtina |
| Rok vydání: | 2022 |
| Předmět: | |
| Zdroj: | BMJ Open, Vol 12, Iss 1 (2022) BMJ Open, 12(1):e052186. BMJ Publishing Group BMJ Open Polak, T B, Cucchi, D G, van Rosmalen, J & Uyl-de Groot, C A 2022, ' Real-world data from expanded access programmes in health technology assessments : A review of NICE technology appraisals ', BMJ Open, vol. 12, no. 1, e052186 . https://doi.org/10.1136/bmjopen-2021-052186 |
| ISSN: | 2044-6055 |
| Popis: | ObjectivesTo 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.DesignCross-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 measureNumber of TAs that used EA data to inform safety, efficacy and/or resource use analyses.ResultsIn 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).ConclusionNICE 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. |
| Databáze: | OpenAIRE |
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