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

Abstract

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

An approach to data collection in compassionate use/managed access

“…Novartis uses an end-to-end online system accessible to all physicians worldwide for the receipt of the initial patient request ( Aliu et al, 2022 ). Once patients are on treatment, physicians can also request product resupply via this system for patients deriving benefit, at which timepoint key follow-up efficacy parameters on the patients are captured for specific compounds and indications, according to the principles outlined in Table 1 .…”

An 8-Factor Regulatory Framework to Facilitate Patient Compassionate Use Access

“…Notably, only a handful of requests were received from countries with no regulations, mostly lower-income and lower-middle income countries. 1 The COVID-19 pandemic highlighted the challenging dynamics with accessing unlicensed therapies and vaccines, especially in countries where there were limited to no regulations in place, as in many instances CU was the only possible access mechanism. 8 Even in instances in which overarching regional CU frameworks exist, eg, in the EU, individual member states develop and implement their own local regulations and procedures, which have substantial differences in terms of requirements, assessment time, and documentation, as well as in collection and use of data from CU programs.…”

“…The 8 factors we posit here are useful indicators of the robustness of local CU regulations, as higher CU request rates were observed in countries where regulations fulfill most of these factors. 1 A definition of the roles of involved stakeholders and the requirements for the overall CU request process including evaluation, documentation, and shipment would provide clarity to patients, treating physicians, and other involved stakeholders, potentially reducing the bureaucracy and burden of CU management. The involvement of HAs, ethic committee (EC)/IRB review, and requirements for informed consent would ensure adequate patient safeguards and holistic local oversight, hence mitigating the risks of potential CU misuse or recklessness.…”

“…For the CU process to be effective and to adequately address patients' needs, it requires comprehensive regulations and active involvement and collaboration of several stakeholders (requesting physicians, their patients, caregivers, and other health care professionals [HCPs], patient advocacy groups, health authorities [HAs], as well as pharmaceutical companies). 1,2 The plurality of CU regulations or nonexistence of such regulations in some countries has resulted in disparity, delay, and lack of patient access to potentially life-saving therapies as well as access inequity across geographies globally. 1,3,4 In some countries, the available regulations, though comprehensive, may be too stringent and hinder access for the potential CU population.…”

“…1,2 The plurality of CU regulations or nonexistence of such regulations in some countries has resulted in disparity, delay, and lack of patient access to potentially life-saving therapies as well as access inequity across geographies globally. 1,3,4 In some countries, the available regulations, though comprehensive, may be too stringent and hinder access for the potential CU population. 5,6 Besides the risk of delayed or lack of access, for patients, there is the risk that the local CU framework may not provide adequate safeguards to ensure appropriate use and monitoring of the therapies.…”