Presentation (CTI)

OBJECTIVES: 85% of the global population resides in low- and middle-income countries (LMICs). Manufacturers prioritise drug launches in high-income countries due to greater revenue potential, leading to delays or limited access to medicines in LMICs. A 2024 analysis found voluntary licensing practices in LMICs have stalled, with manufacturers 5 times more likely to launch new products in upper-middle-income countries. This research examines the pathways for accessing unregistered medicines in LMICs, assessing the key barriers encountered by patients and physicians attempting to utilise them, the real-world extent of their use, and the disparities between lower-income and middle-income countries.
METHODS: Secondary research reviewed the regulations governing access and reimbursement for unregistered medicines in 10 LMICs across 4 regions: Egypt, South Africa, Ethiopia (Africa); China, India (APAC); Argentina, Mexico (LATAM); and Turkey, Jordan, Syria (Middle East). The findings were validated through primary research with 20 healthcare professionals experienced in accessing unregistered medicines. Insights were gathered on the regulatory, logistical, and financial barriers to accessing these pathways, and the extent of their use in clinical practice.
RESULTS: Named-patient and compassionate use were identified as key pathways to accessing unregistered drugs. Lower- and lower-middle-income countries reported greater funding barriers than upper-middle income countries, with limited national reimbursement and many patients relying on charitable funding or out-of-pocket payments. Access to unregistered medicines was typically restricted to severe or life-threatening conditions lacking an approved treatment, and national funding required detailed justification. Stakeholders identified logistical challenges, particularly the administrative burden and bureaucratic delays to importation.
CONCLUSIONS: While regulations to support access to life-saving unregistered medicines in LMICs exist, their practical implementation is limited by financial and logistical barriers. Enabling access to medicines through official pathways and compassionate use is essential for patients in LMICs with limited treatment options.