• Martin Kwok, Theresa Foster, and Michael Steinberg.
• Biogen, Inc., Cambridge, Massachusetts; MCPHS University, Boston, Massachusetts.
• Clin Ther. 2015 Sep 1; 37 (9): 2104-20.

PurposeOur aim was to describe the regulatory pathways made available by the US Food and Drug Administration (FDA) to expedite the drug development and approval process, with a focus on the benefits and limitations of the Breakthrough Therapy Designation (BTD) pathway.MethodsPublished materials consisting of journal articles, press releases, government documents, and news articles from pharmaceutical publishers were identified through online databases (ie, Medline and Scopus), the FDA website, and Internet search engines (eg, Google).FindingsTo encourage pharmaceutical innovation and increase the number of products being approved each year, the FDA has introduced 4 expedited regulatory pathways to accelerate the drug development and approval process. The most recent program, enacted in July 2012, was BTD that is given to drugs that treat a serious or life-threatening disease or condition; and preliminary clinical evidence suggests the potential for these drugs to provide a substantial improvement over the current standard of care. The primary basis for the creation of BTD is to provide patients with serious conditions with earlier access to FDA-approved medications. In 2014, 22% of the new molecular entities approved within the Center for Drug Evaluation and Research had BTD status, as opposed to only 11% in 2013, which indicates both the popularity and success of this expedited pathway. Additionally, the creation of BTD has produced a more collaborative working relationship between the pharmaceutical industry and the FDA because both parties have a vested interest in the drug's success. Some of the more notable concerns surrounding these approved breakthrough therapies have been the abbreviated tolerability and efficacy evidence available from accelerated clinical development programs, ensuring the manufacturing aspects keep pace with these accelerated clinical programs, and finally, managing the strain on resources for both the pharmaceutical companies and the FDA.ImplicationsBTD has already had many positive and negative impacts on various stakeholders, including sponsors, investors, regulatory agencies, third-party payors, and patients. The ultimate goal of the BTD program is to identify promising drug candidates early in the clinical development timeline, expedite the development and review processes via intensive guidance from the FDA, and provide patients access to approved therapies as quickly as possible. With the first few batches of BTD product approvals, the FDA and other stakeholders have been working collaboratively to address the various expected and unexpected challenges that have arisen during the BTD process in order to refine and improve this already successful program.Copyright © 2015 Elsevier HS Journals, Inc. All rights reserved.

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