International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions.

ABSTRACT:

Importance

Compassionate use (CU) is a treatment option for patients with serious or life-threatening medical conditions that provides access to locally unlicensed medications (generally free of charge) when all available treatment options have been exhausted and enrollment in a clinical trial is not possible.

Objective

To examine the disparity in CU access observed across countries and explore the key driving factors.

Design settings and participants

This study analyzed all Novartis CU requests (for individual/named patients and cohort programs) received between January 1, 2018, and December 31, 2020, and investigated selected country-specific factors for association with request activity. Data analysis was performed from February 2021 to February 2022.

Main outco

SUBMITTER: Aliu P 

PROVIDER: S-EPMC9012970 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature