Australian access to FDA-approved breakthrough therapy designation medicines: a 10-year review.

ABSTRACT:

Background

Regulatory pathways adopted by the United States Food Drug and Administration (FDA) and Australian Therapeutic Goods Administration (TGA) enable expedited approval of medicines that are thought to offer significant clinical advantage over existing options for severe diseases.

Objectives

To review Australian accessibility to medicines approved through the FDA breakthrough therapy designation (BTD) process including timelines and approvals by the TGA and Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme (PBS).

Methods

Retrospective review of published reports from the FDA, TGA, PBAC and PBS for BTDs from 1 January 2013-31 August 2023. Uniform data about BTD and milestone dates were collected. Analysis included

SUBMITTER: Lingaratnam S 

PROVIDER: S-EPMC11340226 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature