Honors Theses

Date of Award

Spring 5-8-2026

Document Type

Undergraduate Thesis

Department

Chemistry and Biochemistry

First Advisor

Harrison Lee

Second Advisor

Erin Holmes

Third Advisor

Kristi Reece

Relational Format

Dissertation/Thesis

Abstract

This study examines the ethical and structural limitations of the Expanded Access program in the United States, with particular focus on its application to terminal pediatric illness. Although the program is intended to provide patients with life-threatening conditions access to investigational therapies outside of clinical trials, its effectiveness in serving critically ill children remains uncertain. This program is analyzed through careful consideration of regulatory review and ethical theory. Key policies governing Expanded Access are examined alongside scholarly discussions of pediatric autonomy and parental authority.

A case study of ONC201, a now-approved investigational drug, is used to illustrate how Expanded Access operates in practice under real-world conditions. Findings indicate that the Expanded Access program creates significant structural and ethical barriers that limit access for terminally ill pediatric patients. The requirement for seamless coordination among multiple stakeholders, including physicians and pharmaceutical companies, provides significant opportunity for error. Moreover, the framework prioritizes clinical trial enrollment and regulatory compliance over individualized care, even in cases where prognosis is poor and standard treatment options have been exhausted. The analysis also reveals that parental decision-making, while central in pediatric care, is frequently subordinated to institutional assessments of risk, raising concerns about how best interests are defined in end-of-life contexts.

The study concludes that the current Expanded Access policy is not adequately designed to meet the needs of terminally ill children. A more flexible and ethically grounded approach is necessary, one that preserves essential regulations while allowing greater responsiveness to individual circumstances. Such a model would better account for the unique ethical considerations present in care for terminally ill pediatric patients, particularly in situations where investigational therapies may represent the only remaining option.

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