Penalization and shrinkage methods produced unreliable clinical prediction models especially when sample size was small.

Penalization and shrinkage methods produced unreliable clinical prediction models especially when sample size was small.
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DOI:
10.1016/j.jclinepi.2020.12.005
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发表时间:
2021-04
影响因子:
7.2
通讯作者:
Collins GS
Collins GS
中科院分区:
医学2区
文献类型:
--
作者:
Riley RD;Snell KIE;Martin GP;Whittle R;Archer L;Sperrin M;Collins GS

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When developing a clinical prediction model, penalization techniques are recommended to address overfitting, as they shrink predictor effect estimates toward the null and reduce mean-square prediction error in new individuals. However, shrinkage and penalty terms (‘tuning parameters’) are estimated with uncertainty from the development data set. We examined the magnitude of this uncertainty and the subsequent impact on prediction model performance. This study comprises applied examples and a simulation study of the following methods: uniform shrinkage (estimated via a closed-form solution or bootstrapping), ridge regression, the lasso, and elastic net. In a particular model development data set, penalization methods can be unreliable because tuning parameters are estimated with large uncertainty. This is of most concern when development data sets have a small effective sample size and the model's Cox-Snell is low. The problem can lead to considerable miscalibration of model predictions in new individuals. Penalization methods are not a ‘carte blanche’; they do not guarantee a reliable prediction model is developed. They are more unreliable when needed most (i.e., when overfitting may be large). We recommend they are best applied with large effective sample sizes, as identified from recent sample size calculations that aim to minimize the potential for model overfitting and precisely estimate key parameters. When developing a clinical prediction model, penalization and shrinkage techniques are recommended to address overfitting. Some methodology articles suggest penalization methods are a ‘carte blanche’ and resolve any issues to do with overfitting. We show that penalization methods can be unreliable, as their unknown shrinkage and tuning parameter estimates are often estimated with large uncertainty. Although penalization methods will, on average, improve on standard estimation methods, in a particular data set, they are often unreliable. The most problematic data sets are those with small effective sample sizes and where the developed model has a Cox-Snell far from 1, which is common for prediction models of binary and time-to-event outcomes. Penalization methods are best used in situations when a sufficiently large development data set is available, as identified from sample size calculations to minimize the potential for model overfitting and precisely estimate key parameters. When the sample size is adequately large, any of the studied penalization or shrinkage methods can be used, as they should perform similarly and better than unpenalized regression unless sample size is extremely large and is large.
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