Trends in compassionate use of medicinal products: Israel 2020-2024.

Abstract

Background: Compassionate use programs allow patients with life-threating and serious conditions to access investigational therapies when standard treatments are inadequate. This study aims to analyze the trends and outcomes of the compassionate use of medicinal products in Israel.

Methods: Data from the Israeli Ministry of Health's compassionate use database (2020-2024) were anonymized and analyzed. Duplicates were removed, and a pivot table was used to assess factors such as active ingredients, indications, treatment counts, and patient demographics. Results were presented as counts and percentages, with treatments over 0.5% of the total classified as common. Statistical analyses included Student's t-test and chi-squared test for subgroup differences (p < 0.05 significant). Commonly used medicinal products were cross-referenced with the MOH drug database for registration and reimbursement status.

Results: A total of 3,284 compassionate treatments were administered, employing 596 distinct medicinal products to address 1,361 conditions in 2020-2024. Temporal analysis identified a peak in 2020, which accounted for 24% of total treatments, followed by a decrease thereafter. Patient age stratification indicated that those aged 65 to 80 received the highest treatment proportion (26%), while the 45 to 65 age group accounted for 19%. Treatments were mainly concentrated in large central hospitals (77%) and the central district (49%), with the southern district showing the least usage. The authorization process was primarily for the continuation of study drug in 63% of cases. Additionally, Belantamab mafodotin and Trametinib were the most frequently utilized medicinal products, accounting for 9% and 8.6% of treatments, respectively. Disease category analysis revealed that relapsed refractory multiple myeloma, central nervous system tumors, and inflammatory bowel disease were among the top conditions treated, varying by age group. Notably, 60% of the most common technologies (13 out of 22) were subsequently included into the national health basket, typically following an extended period of compassionate use that exceeded two years.

Conclusion: The study suggests that Israel's compassionate use programs have accelerated early access to novel therapies for complex conditions and provided a bridge to the inclusion of novel medicinal products in the national health basket. Nonetheless, the study identifies a concerning downward trend in utilization alongside potential access disparities, thereby underscoring the necessity for further targeted investigations.