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W-45: The Impact of US FDA Breakthrough Designation (BTD) on Global Access to Innovative Medicines

Poster Presenter

Magda Bujar

Senior Manager, Regulatory Programme and Strategic Partnerships
Centre for Innovation in Regulatory Science (CIRS)
United Kingdom

Objectives

To evaluate whether BTD has impact on accelerating not only development and approval in US, but also if this translates into faster approval across other regulators, as well as quicker decision from health technology assessment (HTA) bodies and ultimately greater availability of medicines worldwide.

Method

CIRS databases were used to identify BTD NASs approved by FDA in 2013-2015 compared to non-BTD NAS. NASs were analysed in terms of development (IND) and approval in US; as well as regulatory approval and HTA outcome by major agencies and regulatory approval by emerging countries by the end of 2017.

Results

Twenty-two BTD NASs were approved by FDA in 2013-2015. FDA median approval time was 191 days, compared to 305 for non-BTD NASs (n=103). The IND time was 1538 median days for BTD NASs compared to 2307 for non-BTD NASs. Twenty-one out of 22 (95%) of the BTD NASs were approved by the end of 2017 by EMA, Health Canada, Swissmedic and TGA as well as 17/21 (77%) by PMDA. In comparison, out of the 103 non-BTD NASs, 72% were approved by EMA, 62% by Health Canada and Swissmedic, 59% by TGA and 46% by PMDA. The approval time for BTD vs non-BTD NASs was 357 vs. 431 median days for EMA, 278 vs. 311 for PMDA, 284 vs. 357 for Health Canada, 294 vs. 509 for Swissmedic and 350 vs. 381 for TGA. Time from national regulatory approval to HTA decision was assessed for Australia, Canada, England, France and Germany. This median time was generally faster for NASs with BTD compared to those without, with 15 days for BTD vs. 76 days for non-BTD for Australia, 166 vs. 272 for Canada, 104 vs. 146 days for Germany, 206 vs. 249 days for France and 389 vs. 406 days for England. Regarding the decision outcome (positive; positive with restrictions or negative) this was similar across the agencies regardless of the BTD. For 10 of BTD NASs, data was collected on emerging market approval by the end of 2017 in BRICK-TM (Brazil, Russia, India, China, South Korea, Turkey and Mexico) countries. Eight out of 10 BTD NASs were approved by the majority (4 or more) of the BRICK-TM countries by the end of 2017, but only 2 BTDs were approved by all 7 countries. China had the smallest number of BTD NASs approved with 3 of 10, followed by Turkey with 6; Brazil, India and Mexico with 7; and Russia and South Korea with 8. The median approval time for NASs with BTD were comparable to those without the BTD, except for China, Brazil and India were the median timelines were quicker by 300, 274 and 206 days respectively for BTD NASs compared to non-BTD in those countries.

Conclusion

BTD can be used to expedite the development and review of promising new drugs in US. Nevertheless, this analysis also suggests some ‘halo effect’ as a result of the BTD on the global registration of medicines. BTD was linked to a higher proportion of approved NASs in five other major regulatory agencies, which may due to company strategy to expedite global roll out medicines. Nevertheless, the approval timelines within the five mature agencies (EMA, PMDA, HC, SW, TGA) were also quicker, which is likely related to the use of facilitated regulatory pathways within those agencies to prioritise the marketing authorization process. Time from regulatory approval to reimbursement was also quicker for products with BTD. Nevertheless, the proportion of positive HTA recommendations was not affected, which may be due to the fact that HTA agencies use other criteria when making the reimbursement recommendation, as well as the lack of alignment between facilitated regulatory and access pathways. Finally, the study also suggests a positive effect of the BTD on the availability of NASs in emerging (BRICK-TM) markets, where possible drivers may be modified submission requirements for those products and company strategy supported by the local capabilities of the sponsor. On the other hand, the approval timelines were similar for the majority of emerging agencies regardless of BTD status, which may be due to the lower use or availability of priority pathways within those agencies. Nevertheless, measuring internationalization and global access to medicines enables company-agency and cross-agency discussions to identify possible enablers for improvement to facilitate development, registration and access to medicines globally. This could be through the optimal use of facilitated regulatory and access pathways, alignment of criteria for unmet medical need and submission requirements to ultimately speed up time to market.