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W 10: Process and Pitfalls of Preparing Breakthrough Therapy Designation Documents

Poster Presenter

      Robin Whitsell

      • President
      • Whitsell Innovations, Inc.
        United States


We will detail the Food and Drug Administration (FDA) submission process for Breakthrough Therapy Designation (BTD) documentation, highlighting strategies for efficiency and discussing potential pitfalls.


Whitsell Innovations, Inc. (WI) has examined our processes for writing BTD submissions to FDA since the 2012 inception of this designation. This poster will: detail BTD requirements, map best-practice processes, recommend strategies for success, and identify potential pitfalls in team engagement. Authors: Lisa Vadola, PhD, Karry Smith, PhD, MPH, Kelly Kilibarda, PhD, Monique Pond, PhD, Ann Winter-Vann, PhD, Natalie Herr, PhD, and Robin Whitsell, BA, BPh


Thirty years ago, Congress introduced the Orphan Drug Act to provide incentives for the pharmaceutical industry to pursue drug development programs for rare medical conditions. However, this act did not address the long cycle times required for FDA review, prompting the creation of expedited review programs. Introduced in 2012, the BTD provides drug sponsors with opportunities to shorten both their investigational new drug (IND) application review time and their overall development time, potentially leading to significantly faster availability to patients. Submission of a request for BTD can occur concurrently with the IND or afterwards, but ideally no later than the end-of-Phase 2 meeting. Per the FDA’s website, the formal request for BTD must include a cover letter, information about the drug or biologic, the basis for considering the drug to be intended to treat a serious condition, and preliminary clinical evidence that the drug may demonstrate substantial improvement over available therapies. The submission of this request involves careful coordination between the Sponsor, medical writers, and other study personnel. We have created a process map and a strategy sheet to aid the medical writer in performing an information gap analysis at the initiation of authoring, managing the authoring and review of the document, and coordinating handoff to publishing and submission functions.


Using the strategy sheet and guidelines WI has provided will allow for efficient submission of the formal Request for Breakthrough Therapy Designation documentation.

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