Orphan Drugs Availability in Israel: A Base for Policy Development
Author(s)
Daphna Arbel, M.Sc., MBA1, Omer Ben-Aharon, MBA, PhD2, Moriah Ellen, Professor3, Nadav Davidovitch, Professor1.
1Health Policy & Management, Ben Gurion University of the Negev, Beer Sheva, Israel, 2Ben-Gurion University of the Negev, Beer Sheva, Israel, 3Ben Gurion University of the Negev, Beer Sheva, Israel.
1Health Policy & Management, Ben Gurion University of the Negev, Beer Sheva, Israel, 2Ben-Gurion University of the Negev, Beer Sheva, Israel, 3Ben Gurion University of the Negev, Beer Sheva, Israel.
OBJECTIVES: To map and analyze current global and Israeli approval of orphan drugs (ODs), identify key trends, and assess potential impact on the Israeli healthcare system, which currently lacks a formal definition and policy regarding orphan drugs
METHODS: A comparative analysis was conducted of all approved ODs by the FDA and EMA from January 2000 to December 2024, and all drugs approved in Israel up to February 2025. Identification and matching of drugs across the lists were performed using both trade and generic names, supplemented by manual verification when necessary. Pharmacotherapeutic groups were derived from received EMA file; in cases of missing information, ATC Level II was added. Drugs were categorized according to ATC I
RESULTS: During the study period, 798 ODs were approved by the FDA, 272 by the EMA, and 502 in Israel (matching either FDA or EMA). There were 243 ODs approved by both the FDA and EMA; 191 (79%) are also approved in Israel. Additionally, 361 orphan drugs were solely approved by the FDA, and 185 were approved as ODs by the FDA and as regular drugs by the EMA(42% and 77% approved in IL, respectively). . The predominant therapeutic area among approved ODs by FDA, EMA and IL, respectively, is Antineoplastic and immunomodulating agents (37%, 44%, 44%), followed by Alimentary tract and metabolism (10%, 17%, 11%) and Nervous system (9%,8%, 9%).
CONCLUSIONS: Analysis of OD registration indicates that approximately 60% of drugs approved by either the FDA or EMA are registered in Israel, with a trend toward antineoplastic agents and drugs approved by both the FDA and EMA, either as ODs in both or OD by the FDA and regular approval by EMA.A higher percentage of drugs approved by both agencies are approved in IL compared to the FDA only.
METHODS: A comparative analysis was conducted of all approved ODs by the FDA and EMA from January 2000 to December 2024, and all drugs approved in Israel up to February 2025. Identification and matching of drugs across the lists were performed using both trade and generic names, supplemented by manual verification when necessary. Pharmacotherapeutic groups were derived from received EMA file; in cases of missing information, ATC Level II was added. Drugs were categorized according to ATC I
RESULTS: During the study period, 798 ODs were approved by the FDA, 272 by the EMA, and 502 in Israel (matching either FDA or EMA). There were 243 ODs approved by both the FDA and EMA; 191 (79%) are also approved in Israel. Additionally, 361 orphan drugs were solely approved by the FDA, and 185 were approved as ODs by the FDA and as regular drugs by the EMA(42% and 77% approved in IL, respectively). . The predominant therapeutic area among approved ODs by FDA, EMA and IL, respectively, is Antineoplastic and immunomodulating agents (37%, 44%, 44%), followed by Alimentary tract and metabolism (10%, 17%, 11%) and Nervous system (9%,8%, 9%).
CONCLUSIONS: Analysis of OD registration indicates that approximately 60% of drugs approved by either the FDA or EMA are registered in Israel, with a trend toward antineoplastic agents and drugs approved by both the FDA and EMA, either as ODs in both or OD by the FDA and regular approval by EMA.A higher percentage of drugs approved by both agencies are approved in IL compared to the FDA only.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR153
Topic
Epidemiology & Public Health, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Approval & Labeling, Insurance Systems & National Health Care, Public Spending & National Health Expenditures, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases