Grace periods in comparative effectiveness studies of sustained treatments

Fuente: arXiv
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Autori principali: Wanis, Kerollos Nashat, Sarvet, Aaron L., Wen, Lan, Block, Jason P., Rifas-Shiman, Sheryl L., Robins, James M., Young, Jessica G.
Natura: Preprint
Pubblicazione: 2022
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author Wanis, Kerollos Nashat
Sarvet, Aaron L.
Wen, Lan
Block, Jason P.
Rifas-Shiman, Sheryl L.
Robins, James M.
Young, Jessica G.
author_facet Wanis, Kerollos Nashat
Sarvet, Aaron L.
Wen, Lan
Block, Jason P.
Rifas-Shiman, Sheryl L.
Robins, James M.
Young, Jessica G.
contents Researchers are often interested in estimating the effect of sustained use of a treatment on a health outcome. However, adherence to strict treatment protocols can be challenging for individuals in practice and, when non-adherence is expected, estimates of the effect of sustained use may not be useful for decision making. As an alternative, more relaxed treatment protocols which allow for periods of time off treatment (i.e. grace periods) have been considered in pragmatic randomized trials and observational studies. In this article, we consider the interpretation, identification, and estimation of treatment strategies which include grace periods. We contrast natural grace period strategies which allow individuals the flexibility to take treatment as they would naturally do, with stochastic grace period strategies in which the investigator specifies the distribution of treatment utilization. We estimate the effect of initiation of a thiazide diuretic or an angiotensin-converting enzyme inhibitor in hypertensive individuals under various strategies which include grace periods.
format Preprint
id arxiv_https___arxiv_org_abs_2212_11398
institution arXiv
publishDate 2022
record_format arxiv
spellingShingle Grace periods in comparative effectiveness studies of sustained treatments
Wanis, Kerollos Nashat
Sarvet, Aaron L.
Wen, Lan
Block, Jason P.
Rifas-Shiman, Sheryl L.
Robins, James M.
Young, Jessica G.
Methodology
Applications
Researchers are often interested in estimating the effect of sustained use of a treatment on a health outcome. However, adherence to strict treatment protocols can be challenging for individuals in practice and, when non-adherence is expected, estimates of the effect of sustained use may not be useful for decision making. As an alternative, more relaxed treatment protocols which allow for periods of time off treatment (i.e. grace periods) have been considered in pragmatic randomized trials and observational studies. In this article, we consider the interpretation, identification, and estimation of treatment strategies which include grace periods. We contrast natural grace period strategies which allow individuals the flexibility to take treatment as they would naturally do, with stochastic grace period strategies in which the investigator specifies the distribution of treatment utilization. We estimate the effect of initiation of a thiazide diuretic or an angiotensin-converting enzyme inhibitor in hypertensive individuals under various strategies which include grace periods.
title Grace periods in comparative effectiveness studies of sustained treatments
topic Methodology
Applications
url https://arxiv.org/abs/2212.11398