Increasing Use of Compassionate Use/Managed Access Channels to Obtain Medicines for Use in COVID‐19

Abstract

The coronavirus disease 2019 (COVID-19) pandemic has triggered an extensive need for new therapeutics and there has been widespread use of unapproved/repurposed medicines. Several countries have regulations for access to unapproved medicines, known as compassionate use, managed/expanded access or emergency use. The Novartis Managed Access activity for COVID-19 delivered unapproved/repurposed medicines to nearly 6,000 patients over a 6-month period. With the rapid growth of such access mechanisms to address COVID-19, a better understanding of these channels is required.

Characteristics of the Compassionate Use Program in Japan: An Analysis of Expanded Access Clinical Trials from 2016 to 2021

“…9 Other terms for "CU" include "Managed Access, " "Expanded Access, " "Named Patient Supply, " "Special Access, " "Early Access, " and "Temporary Authorization for Use, " which are regulations regarding access to investigational drug use for unapproved medicines. 10 CU has been institutionalized in the United States and Europe for a relatively long time. The history of the initial discussion on CU programs led to their initiation, which was the HIV/AIDS crisis in the 1980s.…”

“…In CU, it is also critical that patients’ access to unapproved investigational drugs does not interfere with the standard clinical trial process required to approve the drugs 9 . Other terms for “CU” include “Managed Access,” “Expanded Access,” “Named Patient Supply,” “Special Access,” “Early Access,” and “Temporary Authorization for Use,” which are regulations regarding access to investigational drug use for unapproved medicines 10 …”

Learning with insufficient data: a multi-armed bandit perspective on covid-19 interventions

“…For example, as early as the spring of 2020, the anti-viral remdesivir was administered to patients with severe covid-19 under the U.S. IND compassionate use regulations (Grein et al 2020 ). Different countries leveraged a variety of compassionate use regulations during the covid-19 pandemic, and internationally standardized “managed access programs” have been proposed (Aliu et al 2021 ).…”

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

“…Recently, CU gained attention during the COVID-19 pandemic as the scientific community considered investigational products or repurposing of approved therapies with antiviral or immunomodulating properties. 5 , 14 , 15 These considerations were influenced by in-country regulatory access mechanisms. There are substantial differences in the CU regulatory landscape across countries, with some introducing or updating their regulations as the landscape evolves.…”