Factors Impacting Managed Access/Compassionate Use of Therapeutic Products in Countries

Abstract

Background: Managed Access/Expanded Access/Compassionate Use offers a mechanism of access to a locally unapproved or unavailable product outside of a clinical trial, subject to specific access criteria and local regulations. There is significant variation in the regulations and requirements for Managed Access across countries, with varying levels of awareness and usage by patients and HCPs. Methods: We analyzed Novartis Managed Access request activity between January 01, 2018 and December 31, 2020. For countries with request activity during this three-year period, we investigated, aggregated, and compared select country factors to identify potential causal links, which might explain the trends observed. The factors evaluated included public availability of local regulations, robustness of local regulations (using an eight-factor regulatory framework), clinical trial activity, and select macroeconomic factors. Findings: During the 36-month observation period, a total of 31,712 Managed Access requests were received from 111 countries. About 73% (23,194) of these requests came from 10 countries. Approximately 94% of the requests (29,871) came from countries that had publicly available Managed Access regulations on the internet. In addition, the presence of the eight-factor regulatory framework we posited was a useful indicator of the robustness of a country’s Managed Access regulations, as observed in countries with the highest request rates. Overall, countries conducting more clinical trials on average had higher request rates, with a correlation observed between a country’s economic development and its Managed Access activity. Interpretation: Regulatory robustness, public availability of regulations, as well as local clinical trial activity, correlated positively with higher levels of Managed Access requests activity. This analysis was limited to a single company’s Managed Access dataset and should be supplemented by access to and analysis of other comparable experience across the industry to confirm our initial observations reported here. Funding: Novartis Pharma AG Declaration of Interest: PA, SS, RR, ES, MS, SPK, and SNR are employees of Novartis and PA, SS, RR, ES, and SNR own company shares. PF is an employee of the GE2P2 Global Foundation and GE2P2 Global Advisory Services pbc, which provides bioethics advisory services to Novartis Pharma AG.