The Pharmaceutical Industry at a Glance Comparison of New Drug Approval Status and Examination Periods in Japan, the U.S. and Europe -Focusing on the approval results in 2019-.

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Masao Yoshida, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute
Tomoyuki Shibuguchi, Senior Researcher

The Pharmaceuticals and Medical Devices Agency (PMDA), the U.S. Food and Drug Administration (FDA), and the European Medicines Agency (EMA) publish information on approval and review periods for pharmaceutical products in Japan, the United States, and Europe. ⁽ PMDA), the U.S. Food and Drug Administration (FDA), and the European Medicines Agency (EMA), and continuously collects and analyzes the information published on their respective websites. In Policy Research Institute News No. 58 ⁽²⁾, we compared the approval status and review period of new drugs in Japan, the U.S., and Europe, focusing on the approval results in 2018, and in Policy Research Institute News No. 59 ^{(3), we} reported the results of our analysis of information on drugs approved in Japan in 2019. In this news item, we investigated the number of new drugs approved in Japan, the U.S., and Europe in 2019 with respect to the number of approved items, review periods, and other factors.

Survey Method

Based on the information published on the respective websites of PMDA, FDA, and EMA, standard statistical analysis software Stata/IC 14.0 for Windows (Stata Corp LP, College Station, TX, USA) was used, and the review period was calculated from the date of application for approval to the approval date The period of review was calculated as the period from the date of application for approval to the date of approval.

In Japan, as in Policy Research Institute News No. 59, the target drugs were those listed in the "List of New Drugs Approved " ⁴⁾ on the PMDA website, and the number of items was counted for each review report. New Molecular Entity (NME) was counted only if the application was classified as a new active ingredient.

In the U.S., New Drug Applications (NDA) and New Biologic License Applications (BLA) approved by the FDA Center for Drug Evaluation Research (CDER) and listed in "CDER Drug and Biologic Approvals for the Calendar Year " ⁵⁾ were included. NMEs were counted for drugs listed in the "New Molecular Entity (NME) Drug & Original BLA Calendar Year Approvals. The NME count included drugs listed in the New Molecular Entity (NME) Drug & Original BLA Calendar Year Approvals.

NMEs were included in the table for drugs classified as "New active substance".

The special regulatory measures included Priority Review, Accelerated Review, Orphan, Fast Track, and Breakthrough Therapy in Japan, Priority Review, Accelerated Approval, Orphan, Fast Track, and Breakthrough Therapy in the U.S., and Accelerated Assessment, Orphan, Fast Track, and Breakthrough Therapy in Europe. Accelerated Assessment, Orphan, Conditional Approval, Exceptional Circumstances, and Priority Medicine (PRIME). The details of each special regulatory action are summarized in the Supplement.

Comparison of the Number of New Drugs Approved in Japan, the U.S., and Europe

The number of new drugs approved in Japan, the U.S., and Europe over the past five years (2015-2019) is shown in Figure 1. The number of new drug items approved in Japan in 2019 was 130, an increase of 21 over 2018 and the highest in the past five years. Of these, 39 NMEs were approved, an increase of 2 over 2018. In contrast, the U.S. approved 116 products and 48 NMEs, both fewer than in 2018, when the number of approved products was higher, and the number of NMEs was similar to 2015 and 2017. Europe had 111 approved items and 28 NMEs in 2019, the lowest number of items in the past five years.

Number of items approved under special regulatory measures (NME)

The number and percentage of NMEs approved in Japan, the U.S., and Europe over the past five years (2015-2019) that received special regulatory treatment in each region were examined (Figure 2).

Of the 39 NMEs approved in Japan in 2019, 14 (35.9%) received priority review (including orphan drugs), 1 (2.6%) received expedited review (excluding priority review), 12 (30.8%) received orphan drugs (including those designated under the Pioneer Review Designation System), and The number of items designated under the Predicate Evaluation Designation System was 1 (2.6%). The percentages of priority review and orphan drugs were about 35-45% and 30-35%, respectively, since 2013. This was also the first time since 2012 that an NME was approved under expedited review (excluding priority review).

In the United States, 9 (18.8%) of the 48 NMEs approved in 2019 received Accelerated approval, the highest number since 2011, and the second highest percentage since 2016. Priority review was 28 (58.3%), Orphan designation was 21 (43.8%), Fast Track designation was 17 (35.4%), and Breakthrough Therapy designation was 13 (27.1%). REVIEW had an overall percentage consistently above 55% since 2014.

For Europe, 7 (25.0%) of the 28 NMEs approved in 2019 received Conditional approval, a similarly high percentage as in 2016. In addition, two products (7.1%) designated as PRIME, initiated as a measure to promote new drug development in Europe, continued to be approved in 2018. On the other hand, three (10.7%) were approved for Accelerated Assessment, a similarly low level as in 2018; six (21.4%) were designated Orphan, the lowest percentage in the past five years; and no products were applicable for Exceptional Circumstances There were no items in the list of Exceptional Circumstances.

Comparison of review periods for new drugs between Japan, the U.S., and Europe

The review periods (in months) for new drugs approved in 2019 in Japan, the U.S., and Europe are shown in Table 1.The respective median review periods (in months) for 2019 approved items were 9.9 months in Japan, 9.9 months in the U.S., and 11.9 months in Europe, similar for Japan and the U.S. and longer for Europe, a trend similar to 2018.For NMEs, The median review period for 2019 approved items was 10.0 months in Japan, 8.0 months in the U.S., and 13.8 months in Europe. Japan was 2.0 months longer than the U.S. and 3.8 months shorter than Europe, also similar to the 2018 trend.

Review periods for items that received special measures by the pharmaceutical affairs bodies (NME)

Among the products approved in 2019, those that received special regulatory measures to shorten the review period (Japan: Priority review, Pioneer review designation system, expedited review; U.S.: Priority review, Accelerated Approval; Europe: Accelerated Assessment) are shown in Table 2. Table 2 shows the number of NMEs approved in 2019.

Comparison of Japan, the U.S., and Europe based on the top five review periods (NME)

Among the NMEs approved in 2019, we focused on the top five NMEs with the shortest review periods in Japan, the U.S., and Europe, and investigated their relationship with special measures in pharmaceutical affairs (Table 3).

In Japan, all of the top five NMEs with the shortest review periods were subject to special regulatory measures, including those designated for pioneering review. The review periods for the top 5 NMEs ranged from 3.4 to 8.0 months, which was much shorter than the median review period of 10.0 months for the 39 NMEs.

In the U.S., all of the top five review periods were for multiple special regulatory action designations, which ranged from 3.1 to 5.1 months, shorter than the median review period of 8.0 months for 48 NMEs, as well as 5.7 months for the nine NMEs that received accelerated approval. In Europe, on the other hand, the median review period was 8.0 months for the 48 NMEs.

In contrast, only the top three NMEs in Europe had received Accelerated Assessment, a special regulatory measure designed to shorten the review period. While the top three NMEs had a review period shorter than 10 months, the 24 NMEs ranked fifth or higher were found to have a review period of more than one year.

In addition, one new drug from Japan (ENHERTU and Xospata) was included in each of the top 5 NMEs in the U.S. and Europe. Referring to the wording of the special regulatory measures (see Supplementary Information), those items were confirmed to have been approved as breakthrough new drugs that would bring about essential innovations to serious diseases or breakthrough new drugs that would provide significant public health benefits.

Annual median review time (Japan, US, EU)

Table 4 shows the review period (in months) by year of approval (2000-2019) for drugs approved in Japan, the U.S., and Europe, and Figure 3 shows the annual trend in median review period.

The median examination periods for the entire period covered by the survey were 11.2 months in Japan, 10.2 months in the U.S., and 13.6 months in Europe. Looking at annual trends, the examination periods from 2000 to 2010 tended to be longer in the order of the U.S., Europe, and Japan, but Japan significantly shortened its examination period in 2011, and since then, the median examination periods of Japan and the U.S. have been similar (approximately 10 months), followed by Europe, in that order. Europe also allowed a shortening of the examination period from 2015 (approximately 12 months), and since then, the examination period has remained the same until 2019. In addition, looking at the variation (standard deviation) of the examination period over time, it was confirmed that the examination period in Japan was stable with relatively small variation by item compared to the U.S. and Europe.

Discussion and Summary

This report compiles and compares the number of new drugs approved in Japan, the U.S., and Europe in 2019 and their review periods, based on information published by their respective regulatory authorities.

The number of approved products in 2019 was the highest in Japan in the past five years, while the number of approved products in the U.S. and Europe both decreased compared to 2018. This was partly due to the fact that the U.S. and Europe had the highest number of approved items in 2018 on record. The survey on the number of NME approvals and special measures in the NMEs also revealed two characteristics: the first was the record-high levels of Accelerated approval in the U.S. and Conditional approval in the E.U. The second was the record-high number of Special measures in the U.S. and the E.U. The number of Special measures in the U.S. and the E.U. was also the highest in 2018. Second, the number of approvals for orphan drugs in Japan and the U.S. has remained at high rates over the past five years. These two characteristics suggest that companies around the world continue to invest aggressively in the development of drugs with high unmet medical needs, including those for rare diseases.

In terms of review times for new drugs, Japan's median review time was similar to that of the U.S. and shorter than that of Europe, a trend that has persisted since 2011 and will continue in 2019. It was. To understand this difference in detail, a comparison of the percentage of Priority review-designated items in Japan's NMEs and their review periods with those of the U.S. and Europe shows that not only is the percentage of Priority review-designated items in the U.S. NMEs higher than in Japan, but also their review periods are shorter. On the other hand, in Europe, although the review period for Accelerated Assessment-designated NMEs is the same as that in Japan, the percentage of designated NMEs is much lower, and the review period for NME review items other than Accelerated Assessment is longer. The above indicates that there is a difference in the (median) review period for NMEs in each region. By also looking at the top NMEs approved in 2019 in Japan, the U.S., and Europe, we were able to confirm the situation in which the systems for early commercialization of innovative therapies are being utilized in each region, and items with short review periods are generally designated across multiple special measures in the pharmaceutical affairs.

Currently, Japan (PMDA), the U.S. (FDA), and Europe (EMA) all have special measures in place to shorten review times, with the main goal of speeding up access to innovative drugs for patients. The cycle of pharmaceutical research and development is well underway. As pharmaceutical R&D becomes more diverse and sophisticated, we hope that the system will be further developed to meet the needs of the times.

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