Abstract
Precision medicine presents various methodological challenges whose assessment requires the
consideration of multiple factors. In particular, the data multitude in the Electronic Health Records
poses interoperability issues and requires novel inference strategies. A problem, though apparently a
paradox, is that highly specific treatments and a variety of outcomes may hardly match with consistent
observations (i.e., large samples). Why is it the case? Owing to the heterogeneity of Electronic Health
Records, models for the evaluation of treatment effects need to be selected, and in some cases, the use of
instrumental variables might be necessary. We studied the recently defined person-centered treatment
effects in cancer and C-section contexts from Electronic Health Record sources and identified as an
instrument the distance of patients from hospitals. We present first the rationale for using such
instrument and then its model implementation. While for cancer patients consideration of distance
turns out to be a penalty, implying a negative effect on the probability of receiving surgery, a positive
effect is instead found in C-section due to higher propensity of scheduling delivery. Overall, the estimated
person-centered treatment effects reveal a high degree of heterogeneity, whose interpretation remains
context-dependent. With regard to the use of instruments in light of our two case studies, our suggestion
is that this process requires ad hoc variable selection for both covariates and instruments and additional
testing to ensure validity
| Lingua originale | Inglese |
|---|---|
| pagine (da-a) | 608-621 |
| Numero di pagine | 14 |
| Rivista | Statistical Methods in Medical Research |
| Volume | 27 |
| DOI | |
| Stato di pubblicazione | Pubblicato - 2016 |
OSS delle Nazioni Unite
Questo processo contribuisce al raggiungimento dei seguenti obiettivi di sviluppo sostenibile
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SDG 3 Salute e benessere
Keywords
- C-section
- Precision medicine
- cancer
- electronic health record
- local instrumental variables
- person-centered treatment
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