pISSN 1226-6051
eISSN 2508-786X

Table. 4.

Table. 4.

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


Program Fast Track Breakthrough Therapy PRIME Accelerated Examination
Features/ Benefits • Actions to expedite development and review • All Fast Track designation features • Early appointment of and initial kick-off meeting with a CHMP/CAT Rapporteur • Intensive guidance on the program and data preparation
• Rolling review • Intensive guidance on an efficient drug development, beginning as early as Phase 1 • Assigning an dedicated contact point of EMA • Iterative scientific advice over the entire development pathway
• Organizational commitment involving senior managers • Iterative scientific advice on the overall development plan • Approved based on the data collected up to therapeutic exploratory study
• Dedicated toolbox for quality data package development • Approvable based on a surrogate or an intermediate endpoint

Qualifying criteria • Drug products intended to treat a serious condition AND nonclinical or clinical data demonstrating the potential to address unmet medical need • Drug product intended to treat a serious condition • Medicinal product for target conditions with an unmet medical need • Drug intended to treat a serious condition without effective therapy or to do with improved effectiveness
• OR designated as a qualified infectious disease product • AND preliminary clinical evidence demonstrating substantial improvement on a clinically significant endpoint(s) over available therapies • Demonstrating the potential to address the unmet medical need • Or orphan drug
• Available data supporting the potential for a major therapeutic advantage to patients • Or innovative biotherapeutic for bioterrorism or pandemic infectious disease

Time of request • With IND or after • With IND or after • At gaining compelling non-clinical data in a relevant model with early evidence of promising activity (proof of principle) • At gaining preliminary clinical data that confirm clinically meaningful efficacy from target disease.
• Ideally, no later than the pre-BLA or pre-NDA meeting • Ideally, no later than the end-of-phase 2 meeting • And completing first-in-human studies indicating adequate exposure for the desired pharmacotherapeutic effects and tolerability

Guidelines • Expedited Programs for Serious Conditions - Drugs and Biologics (2014) • EMA Guidance for applicants seeking access to PRIME scheme (2023) • Guideline on the application criteria for the fast track review of pharmaceuticals (2021)
• Expedited Programs for Regenerative Medicine Therapies for Serious Conditions (2019)

BLA, Biologics License Applications; EMA, European Medicines Agency; FDA, Food and Drug Administration; IND, Investigational New Drug; MFDS, Ministry of Food and Drug Safety; PRIME, PRIority MEdicines; NDA, New Drug Application

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