This cohort study examines the disparity in compassionate use access observed across countries and explores the key driving factors.

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.

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 prospective follow-up information at product resupply. Although this approach has recently been implemented and utilization of this data has been mainly internal to the company so far, the prospective collection of key efficacy parameters in patients receiving therapies via CU could potentially be used as a supportive set of information collected in a real-world setting to be submitted in addition to clinical trial data, if not as a main source of data for regulatory submission.

This Viewpoint assesses patient access to compassionate use treatments for medical care and advocates a framework for helping to facilitate such access.

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