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W-13: Influence of Orphan Drug Designation, Breakthrough Therapy Designation, and Advisory Committees on NDA/BLA Review Timelines

Poster Presenter

      Brittany Dustman

      • University of North Carolina
        United States


The objective of this study is to determine how NDA/BLA review timelines are influenced by regulatory designations (Orphan Drug and Breakthrough Therapy) and the occurrence of Advisory Committee Meetings.


Systematic analysis of data from the following FDA reports from 2012 to 2016 was conducted: 'Rare Disease and Orphan Drug Designation Approvals,' 'Breakthrough Therapy Approvals,' and 'NDA and BLA Calendar Year Approvals.' The FDA database 'Drugs@FDA: FDA Approved Drug Products' was also utilized.


One hundred thirteen NDAs/BLAs were approved with Orphan Drug Designation (ODD) between 2012 and 2016. These applications had an average FDA review time of 9.2 months, and nearly all (90.3%) were approved in the first review cycle. Twenty seven (23.9%) of ODD approvals went to Advisory Committee (AC) Meetings prior to approval, which extended the average approval time: 10.6 months for reviews that required an AC vs. 8.9 months for those that did not. Of the ODD approvals, 54.9% also received Priority Review Designation. This subset was reviewed within 7.9 months, or about 1.3 fewer months than the average ODD approval. Only 6.5% of NDAs/BLAs with both ODD and Priority Review were not approved in the first cycle. However, a higher percentage of applications with ODD and Priority Review (29.0%) required an AC, which led to an average review time 3.3 months longer than applications with the same designations that were not evaluated by an AC. Thirty three NDAs/BLAs were approved with Breakthrough Therapy Designation (BTD) between 2013 and 2016, with an average FDA review time of 6.6 months, and all but one application were approved in the first review cycle. All BTD applications also received Priority Review. Only 12.1% of these applications required an AC prior to approval, and this extended the average review time by over 2 months: 8.7 months for applications that went to an AC compared to 6.2 months if no AC was held.


Obtaining ODD and BTD has an influence on the outcome of an NDA/BLA review designation (Priority Review vs. Standard Review) and review timelines. More than half of ODD approvals also received Priority Review, which led to a shorter review time compared to ODD applications without Priority Review. Though Priority Review is not automatically granted to BTD applications, all such applications received Priority Review. This strong correlation likely contributed to BTD approvals enjoying a 2.6 month shorter average review timeline than ODD approvals. Thus, sponsors should seriously consider a separate application for Priority Review if BTD is attained. Though the FDA does not automatically extend PDUFA goal dates if an AC is required, this study shows that sponsors should plan for a longer review timeline if an AC is anticipated. ODD approvals with an AC had a review period 1.3 months longer than those without an AC, while BTD approvals with an AC were 2.5 months longer. This could potentially be due to the fact that, if no AC is required, the FDA may be more likely to expedite these reviews and grant approval ahead of the PDUFA date. However, if an AC is required, approval must be put off until after the meeting. First cycle approval is a goal for all sponsors, as multiple cycles can greatly extend review times. Most applications with either an ODD or BTD were approved in the first review cycle. This is potentially due to the intensive FDA guidance these development programs receive as part of the designation. This benefit should be considered by sponsors during development of clinical and regulatory strategy. As this study shows, receiving BTD, ODD, and/or Priority Review decreases average review times, while multiple review cycles and AC Meetings will likely extend review times. It is essential for regulatory professionals to consider these elements when developing realistic projected dates for drug approval and market launch.

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