A survey of pediatric hematologists/oncologists' perspectives on single patient Expanded Access and Right to Try.

ABSTRACT:

Introduction

Physicians in the United States play an essential role guiding patients through single patient pre-approval access (PAA) to investigational medical products via either the Food and Drug Administration (FDA)'s Expanded Access (EA) or the federal Right To Try (RTT) pathways. In this study, we sought to better understand pediatric hematologist/oncologists' attitudes about seeking PAA, on behalf of single patients, to investigational drugs outside of clinical trials.

Methods

A cross-sectional survey was developed and sent to pediatric hematologist/oncologists via St. Baldrick's Foundation's email distribution list.

Results

Of 73 respondents (10.1% of those who received the survey), 56 met eligibility criteria and are included in the analysis. Over 80% (n</

SUBMITTER: Chapman CR 

PROVIDER: S-EPMC9413614 | biostudies-literature | 2021 Jan-Dec

REPOSITORIES: biostudies-literature