International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea

Shin, Eun Young; Hong, Young Jun; Lim, Kyung Min; Kim, Tae Hyang; Lee, Jong Hyuk

doi:10.1080/20523211.2024.2354299

Journal of Pharmaceutical Policy and Practice

2024

DOI: 10.1080/20523211.2024.2354299

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International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea

Eun Young Shin,

Young Jun Hong,

Kyung Min Lim

et al.

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Assessing Post‐Marketing Requirements for Orphan Drugs: A Cross‐Sectional Analysis of FDA and EMA Oversight

Yu,

Lee,

Kim

et al. 2024

Clin Pharma and Therapeutics

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The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) oversee pharmaceutical regulations, including orphan drugs targeting rare diseases with limited patient populations. Post‐marketing studies are crucial for monitoring safety and efficacy, with post‐marketing requirements (PMRs) mandated by the regulatory agencies to ensure compliance. This study aims to compare PMR statuses, objectives, and pivotal trial characteristics of orphan drugs approved by the FDA (n = 154) and EMA (n = 79) from 2008 to 2018, shedding light on regulatory differences and their impact on drug development. Contrary to expectations, our analysis found no significant disparity in the proportion of orphan drugs with and without PMRs approved by both the FDA (48.1%) and EMA (55.7%). Safety concerns surrounding orphan drugs post‐approval, attributed partly to pivotal trial design, underscore the need for robust post‐marketing surveillance. While the FDA primarily focuses on post‐marketing safety (36.1%), the EMA places a higher emphasis on both efficacy and safety (47.1%), reflecting distinct approaches to PMR management between the two regulatory bodies. The observed trend of delayed PMRs at the EMA (47.1%) highlights the importance of effective cooperation between regulators and pharmaceutical companies to ensure the timely completion of PMRs and enhance drug safety.

show abstract

Assessing Post‐Marketing Requirements for Orphan Drugs: A Cross‐Sectional Analysis of FDA and EMA Oversight

Yu,

Lee,

Kim

et al. 2024

Clin Pharma and Therapeutics

View full text Add to dashboard Cite

show abstract

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International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea

Cited by 1 publication

References 14 publications

Assessing Post‐Marketing Requirements for Orphan Drugs: A Cross‐Sectional Analysis of FDA and EMA Oversight

Assessing Post‐Marketing Requirements for Orphan Drugs: A Cross‐Sectional Analysis of FDA and EMA Oversight

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