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T-25: Impact of US Breakthrough Therapy Designation on Approval Timelines of Drugs and Biologics in the EU

Poster Presenter

      Masooma Razvi

      • Regulatory Affairs Postdoctoral Fellow, GlaxoSmithKline
      • University of North Carolina
        United States


The objective of this study is to compare approval timelines of original US BTD drugs and the respective EU programs to assess implications of regional acceleration mechanisms on earlier access of medicines to treat serious or life threatening conditions with limited treatment options.


For original drugs granted BTD by the FDA CDER (2012-2016), FDA approval packages, EPARs and company press releases were used to identify regional acceleration mechanisms (e.g. accelerated approval, conditional approval, rolling submission, priority review, and scientific advice) that were applied.


The FDA introduced breakthrough therapy designation (BTD) in 2012 to add to their arsenal of expedited programs for serious conditions (i.e. fast track, accelerated approval, and priority review) and expand early access to promising drugs for patients with limited or no available therapies. Understanding if receipt of BTD and other designations in the US aligns with similar acceleration mechanisms in the EU (e.g. conditional authorisation, accelerated assessment, scientific advice, and protocol assistance) is key in today’s global drug development landscape. We hypothesized that regional acceleration in the US following BTD may delay approval timelines for these drugs and biologics in the EU. A total of 59 BTD drugs were approved by CDER (Center for Drug Evaluation and Research) between 2012 and 2016 with 32 of these under an original NDA/BLA. Twenty-seven of these were approved for a similar indication by the EMA. The average approval time for these drugs and biologics was 189.8 days (6.3 months) in the US compared to 269 days (9.0 months) in the EU. Acceleration mechanisms were more widely used by the FDA – 31 (97.0%) priority reviews, 14 (43.8%) accelerated approvals, and 11 (34.4%) rolling NDA/BLA submissions. Additionally, 22 (70%) of these programs were granted BTD during Phase I or II of development. In contrast, of the 27 EU approvals, 18 (66.7%) had accelerated assessment, and 6 (22.2%) were conditionally approved. Furthermore, 20 (74.1%) and 7 (25.9%) of these programs received scientific advice and protocol assistance, respectively. Nineteen (59.4%) of the 32 drugs in the US and 13 (48.1%) of the 27 drugs in the EU received orphan designation. Of note, 19 (70.4%) of the EU market authorisations (MA) were filed within 3 months of the US NDA/BLA submission while on the other hand, 3 (11.1%) of the MAs filed greater than 3 months from NDA/BLA filing in the US, were filed in the EU prior to the US.


Much evidence has been published demonstrating support for FDA’s BTD in accelerating access to new medicines for patients with serious conditions with limited or no available therapies based on preliminary evidence. In addition to BTD, FDA is utilizing other expedited mechanisms (e.g. accelerated approval, and priority review) to further enhance access, but the impact of FDA’s BTD for EU approval timelines remains elusive for many R&D teams. No similar pathway existed in Europe until PRIority Medicines (PRIME) in 2016 which is aimed at increasing early advice from EMA and payers to better plan clinical trial programs and incorporation of real world evidence and is anticipated to lead to a similar improvement in early access for designated programs in the EU. Our analysis indicates that NDA/BLA approval timelines for original submissions in the US are shorter by approximately 3 months compared to MAAs in the EU. Both the US and EU have an arsenal of regulatory acceleration pathways that are used in combination to expedite and streamline drug development in these key markets. On average, the use of acceleration mechanisms was slightly greater by the FDA’s CDER compared to the EMA. Contrary to our hypothesis, NDA/BLA versus MAA filing timelines in the US and EU, respectively, for majority of these drugs/biologics did not differ by greater than 3-4 months. Potential reasons for this may be the large number of programs seeking scientific advice, utilization of additional, well-established acceleration pathways to engage regulatory authorities in early dialogue, and flexibility in acceptance of submission packages in the EU. It will be crucial to note the impact of BTD in the US along with additional designations and how it aligns with similar designations under the PRIME scheme in bringing key medicines to market in the future.