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Table. 5.

Table. 5.

Expedite programs that provide the conditional approval for innovative drug development and approval in the United States, the European Union, and the Republic of Korea


Program Accelerated Approval MAEC CMA Conditional Permission
Features/ Benefits • Approval based on an effect on a surrogate endpoint or an intermediate clinical endpoint that is reasonably likely to predict a drug’s clinical benefit • MA based on less comprehensive clinical data than normally required, • MA without comprehensive clinical data to be collected in a decent timeframe. • Approved based on the data collected up to therapeutic exploratory study
• Under certain specific obligations • Valid for 1 year, renewable annually • Approval on the condition of submitting clinical trial data within a specified period to corroborate the safety and efficacy, subject to opinion of the Central Pharmaceutical Affairs Council
- To reassess the benefit/risk within a time period specified • Convertible to a standard MA after fulfilling the imposed obligations with complete data confirming the benefits continue to outweigh the risks - Data proving efficacy, using surrogate endpoints in terms of pharmacoepidemiology, pharmacotherapy, pathophysiology, and the like
- Product supplied on prescription only in certain cases and only under strict medical supervision - Data proving efficacy on clinical endpoints
- Package leaflet and medical information to state the particulars are as yet inadequate

Qualifying criteria • Drug product treating a serious condition • Medicinal product with inability to provide comprehensive efficacy and safety data due to rarity of the indication • Medicinal product intended for a public health emergency, or for seriously debilitating or life-threatening diseases, including orphan medicines. • Drug used to treat serious major diseases or rare diseases
• AND generally providing a meaningful advantage over available therapies • Inability to provide comprehensive information due to the present state of scientific knowledge • Positive benefit-risk balance • Or innovative biotherapeutic for bioterrorism, pandemic infectious disease
• AND demonstrating an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit or on a clinical endpoint that can be measured earlier than IMM that is reasonably likely to predict an effect on IMM or other clinical benefit • Inability to collect such information because of medical ethics • Potential for submitting comprehensive data post MA
• Fulfilling unmet medical need
• Benefit of immediate availability of the product to patients greater than the risk due to insufficient data

Time of request • To discuss the possibility with the review division during development, supporting the use of the planned endpoint as a basis for approval, and discussing the confirmatory trials to usually be already underway at the time of approval • In advance of MAA • At the time of the application for MA • At gaining preliminary clinical data that confirm clinically meaningful efficacy from target disease.
• Recommending to notify the intention 6-7 months before submission

Guideline • Expedited Programs for Serious Conditions - Drugs and Biologics (2014) • Guideline on procedures for the granting of a MAEC, pursuant to article 14 (8) of regulation (EC) No 726/2004 (2005) • Guideline on the scientific application and the practical arrangements necessary to implement Commission Regulation (EC) No 507/2006 on the CMA for medicinal products for human use falling within the scope of Regulation (EC) No 726/2004 (2016) • Complainant Guide: Instructions for the Conditional Permission of Drug Products (2022)
• Expedited Programs for Regenerative Medicine Therapies for Serious Conditions (2019)

CMA, Conditional Marketing Authorisation; EMA, European Medicines Agency; FDA, Food and Drug Administration; IMM, irreversible morbidity or mortality; MAEC, Marketing Authorisation under Exceptional Circumstances; MFDS, Ministry of Food and Drug Safety

Korean J Clin Pharm 2024;34:39-61 https://doi.org/10.24304/kjcp.2024.34.1.39
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