Europe vs USA: New drug approvals
pharmafile | June 1, 2017 | Feature | Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing | NDA, regulatory affairs
Dr Terese Johansson, Regulatory Affairs Consultant NDA Group AB, examines the drop in US approvals while observing no similar trend occurred in the EU during 2016.
The US had a significant drop in approvals in 2016 compared to 2015 but continues to grant more expedited and non-standard review approvals compared to the EU. The US drop in approvals for 2016 is not observed in the EU but is likely to be more prominent in the EU during 2017. The trend that many companies first seek approval in the US persists. Large pharma continues to dominate the approval statistics, with oncology being the most common therapeutic area.
The US had a significant drop in approvals in 2016 compared to 2015 but continues to grant more expedited and non-standard review approvals compared to the EU. The US drop in approvals for 2016 is not observed in the EU but is likely to be more prominent in the EU during 2017. The trend that many companies first seek approval in the US persists. Large pharma continues to dominate the approval statistics, with oncology being the most common therapeutic area.
Last year there were a total of 74 new drug approvals granted in the US and the EU that meet our selection criteria*. Of these, 19 were approved only in the EU, 19 only in the US, and 36 were granted in both regions during 2016. However, 17 of the EU approvals already had approval in the US 2015 or earlier, while only 6 US approvals were approved in the EU since before – indicating that the trend to first seek approval in the US persists.
Compared to previous years there was an overall drop in approvals for the US, it’s not observed in the EU but is expected to be more prominent during 2017. 35 of the approvals were classified as novel drugs (e.g. NAS, NME or BLA), nine of these were approved only in the EU, nine only in the US and 17 were approved in both regions. For the EMA it’s the fewest approved since 2011, while the FDA has not approved this few since 2010.
Thirty-nine drugs for rare diseases (orphans) were approved in the US and 14 in the EU. The overall trend for more orphan approvals are expected to continue as FDA and EMA during the last ten years have approved orphan drug designations at record rates for each following year. Among the noteworthy orphan approvals are Exondys 51 (for the treatment of Duchenne muscular dystrophy, only approved in the US), Darzalex (for the treatment of multiple myeloma, approved in the US 2015 and in the EU 2016) and Spinraza (for the treatment of spinal muscular atrophy, only approved in the US).
Expedited drug development and non-standard review approval pathways are the new norm in the US but in the EU special approval procedures are not as common. During 2016, special approval procedures like conditional and accelerated approval pathways were issued for seven of the new approvals in the EU, the majority in oncology and for orphan diseases – compared to the US where special approval pathways and designations like Fast Track, Breakthrough, Accelerated Approval and Priority Review were used for 18 of the new approvals.
To support early drug development and speed up the evaluation and approval process, EMA have encouraged the industry to seek early interaction via scientific advice with the the regulatory and HTA bodies, as well as interactions with the committee for advanced therapies (CAT). The intention is to enable early and continued interaction and dialogue between pharma industry and regulators. In the first quarter of 2016, EMA launched a scheme for PRIority MEdicines (PRIME), aiming to optimise development plans and provide accelerated assessment to medicines of major public health interest. Furthermore, in the last quarter of 2016 EMA launched a new pilot for tailored scientific advice for biosimilars.
Looking at the therapeutic areas, by far the busiest was oncology. Noteworthy approvals are Lartruvo (for the treatment of soft tissue sarcoma, approved in the EU and US), Rubraca (for the treatment of ovarian cancer, only approved in the US), Tecentriq (for the treatment of urothelial carcinoma, only approved in the US), and Venclexta (for the treatment of chronic lymphocytic leukemia, approved in the EU and US).
The trend that smaller companies take products all the way to the market persists but large pharma still dominate. Large pharma accounted for 53% of the new approvals vs 47% from small and medium sized pharma. For large pharma it’s a decrease compared to previous years when they contributed with 72% in 2015 and around 64% in the two previous years.
Related Content
FDA accepts NDA for Karuna’s schizophrenia treatment
Karuna Therapeutics has announced that the US Food and Drug Administration (FDA) has accepted its …
FDA accepts Sun Pharma’s NDA for deuruxolitinib
India-based pharma company Sun Pharmaceutical Industries has announced that the US Food and Drug Administration …
Bristol Myers Squibb’s NDA accepted by FDA
Global pharmaceutical company Bristol Myers Squibb (BMS) has announced that its New Drug Application (NDA) …
