University of Twente Student Theses
Incorporating background mortality into survival extrapolations: determining the accuracy using a simulation study
Hordijk, D.D. (2023) Incorporating background mortality into survival extrapolations: determining the accuracy using a simulation study.
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| Abstract: | Objective: Clinical trial data is often limited in terms of follow-up time while HTA submissions require assessment of long-term outcomes, thus survival extrapolations are required. Recent guidance from NICE has suggested employing a relative survival approach to incorporate general population background mortality (GPM) into survival extrapolations to better account for increasing GPM as patients age. However, alternative methodologies exist to incorporate GPM, and knowledge on the appropriateness of these methods across situations is lacking. This research uses a simulation study to compare the performance of different approaches for incorporating GPM. Methods: 288 scenarios with different patient characteristics and trial characteristics were assessed with the aim to determine patterns in accuracy, with 2,500 replications per scenario. The performance of the relative survival approach and two other GPM incorporating methods were compared to non-GPM extrapolations. Results: Significant differences in accuracy were found between the methods. The methods that incorporate GPM outperformed non-GPM extrapolations in 226 scenarios (78%) on average. The distribution used to simulate the datasets was not always identified as the best performing distribution. Conclusions: Adjusting for GPM results in more accurate survival extrapolations. Our results provide guidance for selecting between the methods to incorporate GPM within survival extrapolation, given patient and trial characteristics. |
| Item Type: | Essay (Master) |
| Clients: | OPEN Health |
| Faculty: | BMS: Behavioural, Management and Social Sciences |
| Subject: | 31 mathematics, 44 medicine |
| Programme: | Industrial Engineering and Management MSc (60029) |
| Link to this item: | https://purl.utwente.nl/essays/96800 |
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