AUTHOR=Sarp Séverine , Reichenbach Ramona , Aliu Paul 

TITLE=An approach to data collection in compassionate use/managed access

JOURNAL=Frontiers in Pharmacology

VOLUME=Volume 13 - 2022

YEAR=2022

URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2022.1095860

DOI=10.3389/fphar.2022.1095860

ISSN=1663-9812

ABSTRACT=Compassionate Use (CU)/Managed Access programs provide access to locally unapproved medicines. As these programs become more global and involve a broader range of products, determining whether patients derive benefit from treatment could provide insights into therapeutic use in a real-word setting with diverse pools of patients. CU primary purpose is to provide treatment and it is not targeting research. However, it is increasingly considered as a source of real-world data. In the absence of a harmonized framework on CU data collection, Novartis developed a company-wide guidance to collect baseline patient data and prospectively acquire information at product resupply.