Factors distinguishing important identified risks from important potential risks in orphan and nonorphan drugs: An analysis of safety specifications of Japan and European Union risk management plans

Hirota, Saeko; Yamaguchi, Takuhiro

doi:10.1002/pds.4646

Cited by 3 publications

(1 citation statement)

References 19 publications

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“…Moreover, in the context of rare diseases, clinical trials are often uncontrolled or comparative data might have been collected from a historical control group. Therefore, the safety profile remains poorly known at the time of approval [11,12,[29][30][31]. Some new generation of orphan drugs target diseases which were always lethal diseases in the young and create a new population of survivors who have a course of disease never previously seen.…”

Section: Pharmacovigilance Studiesmentioning

confidence: 99%

The challenges of pharmacoepidemiology of orphan drugs in rare diseases

Lapeyre‐Mestre

2020

Therapies

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Section: Pharmacovigilance Studiesmentioning

confidence: 99%

The challenges of pharmacoepidemiology of orphan drugs in rare diseases

Lapeyre‐Mestre

2020

Therapies

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Background Factors Contributing to Safety Warning Discordance in the Initial Labeling of New Drugs in Japan, the United States, and the European Union

Fukuda,

Narukawa

2024

Clin Pharma and Therapeutics

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Drug labels summarize essential safety information for healthcare professionals and patients. However, studies have reported a discordance of safety information in drug labeling among countries/regions. We aimed to identify the characteristics of adverse events associated with discordant safety warnings during the initial labeling of new drugs approved at approximately the same time in Japan, the United States, and the European Union. Safety warning discordance/concordance between two countries/regions and the explanatory variables were assessed using multivariable logistic regression. The safety warning concordance rate was 71.0% (152/214) for the United States and the European Union, 59.5% (135/227) for Japan and the United States, and 64.3% (144/224) for Japan and the European Union. A significant association with discordant safety warnings was revealed for “adverse event rate” and “warning status in a similar drug” between the United States and the European Union; “adverse event rate,” “adverse event included in important medical event list,” and “warning status in a similar drug” between Japan and the United States; and “adverse event included in important medical event list” and “warning status in a similar drug” between Japan and the European Union. Clarifying and publicizing the reasons for safety warnings, along with an awareness of the factors associated with the discordance identified in this study, will help healthcare professionals, patients, marketing authorization holders, and regulatory authorities around the world share the background of country/region‐specific warnings, reducing the possibility of confusion among them due to the discrepancies.

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Factors Influencing Classifications of Safety Specifications in a Risk Management Plan for Antineoplastic Agents Approved in Japan: A Review and Descriptive Analysis

Ezaki

Hirakawa

Hanaoka

et al. 2021

Ther Innov Regul Sci

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Factors distinguishing important identified risks from important potential risks in orphan and nonorphan drugs: An analysis of safety specifications of Japan and European Union risk management plans

Abstract: Factors affecting risk categories were different between Japan and the EU, which might contribute to the difference in safety specifications between these 2 regions.

Cited by 3 publications

References 19 publications

The challenges of pharmacoepidemiology of orphan drugs in rare diseases

The challenges of pharmacoepidemiology of orphan drugs in rare diseases

Background Factors Contributing to Safety Warning Discordance in the Initial Labeling of New Drugs in Japan, the United States, and the European Union

Factors Influencing Classifications of Safety Specifications in a Risk Management Plan for Antineoplastic Agents Approved in Japan: A Review and Descriptive Analysis

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