Expedited Programs for Serious Conditions: An Update on Breakthrough Therapy Designation

Area of Science:

• Pharmaceutical Sciences
• Regulatory Affairs
• Clinical Development

Background:

• The US Food and Drug Administration (FDA) has established expedited regulatory pathways to accelerate drug development and approval.
• These pathways aim to encourage pharmaceutical innovation and increase the annual number of approved products.

Purpose of the Study:

• To describe FDA's expedited regulatory pathways for drug development.
• To focus on the benefits and limitations of the Breakthrough Therapy Designation (BTD) pathway.

Main Methods:

• A comprehensive literature search was conducted using online databases (Medline, Scopus).
• Relevant materials included journal articles, press releases, government documents, and news articles.
• Searches were supplemented by the FDA website and general internet search engines.

Main Results:

• The FDA introduced four expedited pathways, including the Breakthrough Therapy Designation (BTD) established in July 2012.
• BTD is granted to drugs for serious conditions with preliminary evidence of substantial improvement over standard care, aiming for earlier patient access.
• BTD status increased from 11% in 2013 to 22% in 2014 among approved new molecular entities, indicating its growing use and success.
• BTD fosters collaboration between the pharmaceutical industry and the FDA.
• Concerns include abbreviated tolerability/efficacy data, manufacturing scalability, and resource strain on both industry and the FDA.

Conclusions:

• Breakthrough Therapy Designation (BTD) impacts various stakeholders, including patients, sponsors, and regulatory agencies.
• The program's goal is to identify promising drug candidates early, expedite development and review with FDA guidance, and ensure rapid patient access.
• Ongoing collaboration among the FDA and stakeholders addresses challenges to refine and improve the BTD program.