We thank Drs. Garg, Kaul, and Choudhary for their thoughtful comments. We agree that intention-to-treat (ITT) analysis is best for randomized randomised controlled trials of unselected patients and that per protocol (PP) analysis is best for precision medicine studies wherein biomarker-based patient selection occurs. The latter includes only patients with the relevant biomarker in the analysis [
- Sicklick J.K.
- Kato S.
- Okamura R.
- Kurzrock R.
Precision oncology: the intention-to-treat analysis fallacy.
Eur J Canc. 2020; 133: 25-28
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- Precision oncology: the intention-to-treat analysis fallacy.Eur J Canc. 2020; 133: 25-28
Published online: August 21, 2020
Accepted: July 15, 2020
Received: July 2, 2020
© 2020 Elsevier Ltd. All rights reserved.
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- Precision oncology: the intention-to-treat analysis fallacyEuropean Journal of CancerVol. 133
- PreviewIt has recently been suggested that precision oncology studies should be reanalysed using the intention-to-treat (ITT) methodology developed for randomized controlled clinical trials. This reanalysis dramatically decreases response rates in precision medicine studies. We contend that the ITT analysis of precision oncology trials is invalid. The ITT methodology was developed three decades ago to mitigate the problems of randomized trials, which try to ensure that both arms have an unselected patient population free from confounders.
- Intention-to-treat analysis in precision oncology: A cautious interpretationEuropean Journal of CancerVol. 138
- PreviewRandomised clinical trials (RCTs) are the best available study designs to confirm the effectiveness of a new intervention. The process of randomisation produces two prognostically equal groups which are similar in all aspects except that one group receives the new intervention whereas the other does not. However, these two groups may not remain similar in due course after randomisation if the participants leave their assigned groups because of varied reasons – high cost, more side effects, inconvenience, alternative options, etc.