pISSN 1226-6051
eISSN 2508-786X
eISSN 2508-786X
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
| FDA | EMA | MFDS | ||
|---|---|---|---|---|
| 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