The Pharmaceutical Industry at a Glance New drugs approved in Japan in 2019 and their review period

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Tomoyuki Shibuguchi, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute
Shinichiro Awamura, Senior Researcher

The Pharmaceuticals and Industrial Policy Research Institute (PIIPRI) continuously collects and analyzes information on drug approvals and their review periods based on information published by regulatory ^{authorities1)}. This time, we report on our survey on the approval status of new drugs and new regenerative medicine products approved in Japan from January to December 2019, and their review periods.

Research Methods

The survey covered drugs listed in the "List of New Drugs Approved " ²⁾ on the PMDA's website. Items were counted by review report, and those for which multiple companies simultaneously submitted public knowledge applications for the same drug ingredient in response to requests from academic societies, etc., and those for which multiple ingredients were approved for combination drug therapy, etc., were counted as a single drug item. Approval information for each item was extracted from review reports, lists of approved items for new drugs, package inserts, and pharmaceutical bulletins. Standard statistical analysis software Stata/IC 14.0 for Windows (Stata Corp LP, College Station, TX, USA) was used for analysis to calculate the review period (application date to approval date).

Number and Breakdown of New Drugs Approved

Figure 1 shows the annual number of new drugs approved in Japan during the 10-year period from 2010 to 2019. The number of new drug items approved in Japan in 2019 was 130, an increase of 21 items from 2018; this was higher than the average number of items approved in 2010-2018 (116 items). Of these, 39 new medicinal products containing new active ingredients (NMEs) were approved, an increase of 2 NMEs over 2018.

The breakdown of approved items is then shown in Table 1. The breakdown by application category shows that in 2019, in addition to the aforementioned NMEs, there were 45 new indication drugs and 18 new dose drugs; these numbers increased from 2018. In addition, there were 8 biologics follow-on products (biosimilars ⁾³⁾, an increase of 2 compared to 2018 and the highest number to date.

By review category, 92 NMEs were under regular review and 38 were under priority review. Priority review items accounted for 29% of all approved items, a high level (around 30%) since 2014. In addition, 35 of the priority review items were orphan drugs, an increase of 3 items compared to 2018. Orphan drugs have been increasing since 2014, hovering around 20-30 items, but 2019 had the highest number to date with 35 items (Figure 2).

Review period for new drugs

Figure 3 and Table 2 show the annual review periods for new drugs approved from 2010 to 2019.

The median review period for all 130 products approved in 2019 was 9.9 months, similar to 2018, with little variation around 10 months since 2011, when the review period was significantly reduced. By examination category, the median duration was 10.7 months for normal examination items (excluding expedited processing items) and 8.4 months for priority examination items, which was also similar to the duration since 2011. The 80th percentile of the review period for regular review items (excluding expedited processing) was 11.8 months, which was within the target of 12 ^months4). The 80th percentile of the review period for priority items was 8.7 months, which was also within the target period of 9 months4 ⁾.

The median review time for the 39 NMEs approved in 2019 was 10.0 months, 0.3 months shorter than in 2018, while the median review time for non-NMEs was 9.9 months, 0.2 months longer than in 2018. The median review time for some NMEs was similar to that of non-NMEs, although some NMEs took longer to review.

Analysis of New Medicinal Elements (NME)

The study examined the breakdown of NMEs (39 products) approved in 2019: 24 (61%) were NMEs under normal review, 12 (31%) were orphan drugs (including priority review and pioneer review designation), and 2 (5%) were priority review products other than orphan drugs.

The number of NMEs approved in 2019 for biopharmaceuticals was 10, accounting for 26% of all NMEs. The number of biopharmaceutical NMEs approved in 2019 was 10, accounting for 26% of all NMEs. Of these, 5 were antibody drugs and 2 were from domestic firms (including joint ventures). By drug class, anti-malignant tumor drugs were the most common, accounting for 9 items or about 23% of all NMEs. Other metabolic drugs accounted for 8, CNS drugs 5, biological products 4, circulatory drugs 3, and chemotherapeutic agents 3, in that order.

Approval of Products Subject to the Pioneering Review and Designation System

Two items were approved in 2019 for the pioneering review designation system (in order of approval, Vindakel capsules and Rosrytrek capsules: Table 4). The review periods for those items were 4.8 and 5.9 months, respectively, which were very short compared to the median review period for priority review items of 7.7 months. For priority review, which is one of the special measures for items subject to the Priority Review Designation System, the target total review period from application to approval is set at 6 ^months4), and it was confirmed that the two items approved in 2019 were approved within that target period.

Approval of new regenerative medicine products and their review period

The following section presents the approved new regenerative medicine products approved in recent years and their review periods (Table 5).

In 2018, the additional efficacy and indication of autologous cultured epidermal jace and Stemilac Injection, the first regenerative medicine product for spinal cord injury, were approved; in 2019, two products were approved: Kymriah intravenous infusion, the first CAR-T cell therapy in Japan, and Collategene for intramuscular injection, the first product for gene therapy in Japan. The two products were approved in 2019. The approval of Stemilac Injection and Collategene for intramuscular injection was granted for 7 years and 5 years, respectively, with a time limit and conditions. The review periods were 9.3 months, 6.0 months, 11.1 months, and 14.1 months, respectively. The target review period for items subject to the accelerated review designation system is 6 months, while the target review period for regenerative medicine products is 9 months for 50% of all priority items and 12 months for 50% of normal ^items4).

Conclusion

A survey of the review periods for the 130 new drugs approved in Japan in 2019 was conducted by review category, and it was confirmed that the review periods were similar to those after 2011, when the review period was significantly reduced. In addition, for items subject to the Pioneer Review Designation System, both pharmaceuticals and regenerative medicine products were reviewed as expeditiously as in the previous year.

In November 2019, the revised Pharmaceutical Affairs Law was enacted, and the Pioneer Review Designation System and the Conditional Accelerated Approval System, which have been in operation on a trial basis since 2015, became law. With the diversification of treatment modalities, including the emergence of gene therapy drugs, it is hoped that innovative pharmaceuticals and regenerative medicine products will be filed and approved in Japan ahead of the rest of the world and reach patients as quickly as possible.

Supplement

Since some items have significantly longer periods or shorter periods due to special exceptions, the main basic statistic is the median, and the number of samples (N), mean, and standard deviation (SD) are also shown. Figures 3 and 4 are also presented as box-and-whisker plots to show the distribution of the data. The middle line of the box in the box-and-whisker diagram indicates the median (50%), and the bottom and top lines of the box indicate the first quartile (25%) and third quartile (75%), respectively. In other words, if there are 100 samples, the 25th sample value is the first quartile point, the 50th is the median, and the 75th is the third quartile point. Proximity values (whiskers) above and below the box indicate the samples farthest from the median within 1.5 times the height of the box (length of the first quartile to the third quartile). Outliers outside the proximity are shown as points.

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