The Pharmaceutical Industry at a Glance Development and Approval Status of Drugs for Unmet Medical Needs Trends in 2019

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Eriko Hashimoto, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute

The Pharmaceuticals and Industry Policy Institute (PIIPI) continuously analyzes the status of pharmaceutical companies' efforts to address unmet medical needs by compiling data on new drug approvals and development pipelines based on the results of a survey on medical ^needs1) by the Human Science Foundation (hereinafter referred to as the HS Foundation) The 60 diseases covered in the survey are those considered important based on discussions by the survey team, and specifically include serious diseases, diseases that significantly impair QOL, diseases with a large number of patients, and diseases with a large social impact ^. Based on the results of the most recent treatment satisfaction survey (2014), this report presents the number of development pipelines as of January 2020 and the number of drug approvals from 2011 to 2019 for these 60 diseases, and analyzes the relationship between the number of drug approvals for the 10 cancer types included in the 60 diseases and the improvement in 5-year survival rate. The relationship between the number of drug approvals and 5-year survival improvement for the 10 cancer types included in the 60 diseases is analyzed.

New Drug Development Status by Treatment Satisfaction

Figure 1 plots the diseases along the treatment satisfaction (horizontal axis) and drug contribution (vertical axis) in the 2014 HS Foundation survey, and shows the number of developments investigated in this study in terms of circle size and numerical ^value7). The same 20 pharmaceutical ^companies8) as in the previous survey were included in the total number of development items (Phase 1 to Filed) in Japan as of January ²⁰²⁰⁹⁾. The number of development projects in Japan as of January 2020 was ²⁹¹¹⁰⁾, and in descending order of number of projects, lung cancer (43), malignant lymphoma (20), breast cancer (18), leukemia (16), and liver cancer (14), indicating that development is active in oncology, with cancer being the top five of the 60 diseases. The 60 diseases include a total of 10 types of cancer. Looking at cancers other than these five, the number of development cases was prostate cancer (10 cases), colorectal cancer (8 cases), stomach cancer (7 cases), cervical cancer (4 cases), and pancreatic cancer (3 cases). Pancreatic cancer, which has the lowest level of treatment satisfaction and drug contribution among cancers, had the lowest number of development cases among cancers, suggesting the difficulty of development.

Of the 291 cases of development totaled in this survey, 164 were for new active ingredients, accounting for 56.4% (71.9% in the 2017 survey). When divided into oncology and other diseases, the percentage of new active ingredients developed was 46.9% for oncology and 65.7% for other diseases, reflecting the high tendency of oncology drugs to add indications for different cancer types or different patient groups within the same cancer type after launch, which was reflected in the development status.

For reference, Table 1 shows the number of development cases by disease from previous surveys to the current survey.

Number of drug approvals by 60 diseases

The number of new drugs approved or added indications between 2011 and 2019 that can be categorized into the 60 diseases covered by the 2014 HS Foundation survey is summarized (Table 2). ¹¹ Looking at the drugs approved or added indications between 2011 and 2019, in terms of cancer, the most common in order of number of new cases are The top cancer types in terms of the number of drug approvals were also seen in terms of the number of drug development and approval.

Looking at the trend of drugs for lung cancer, which had the highest number of development and approval, drugs and immune checkpoint inhibitors that match the type of gene involved in cancer growth, such as EGFR gene mutation, ALK fusion gene, ROS1 fusion gene, and BRAF gene mutation, have been developed and approved one after another in recent years. In addition, drugs that act on different targets, such as drugs for types of cancer that have become resistant to other drugs, PARP inhibitors, and anti-HER2 antibodies, are also being developed. The optimal approach to lung cancer varies depending on the genotype and other factors. The number of treatment options for each type of cancer is increasing, and it is expected that treatment will become more personalized.

Improvement in 5-year survival rate and number of approved drugs for cancer

We would like to look at the extent to which the prognosis of cancer patients has improved as a result of advances in cancer treatment, including the approval of new cancer drugs and the addition of indications to existing drugs. According to data analyzed by the National Cancer ^Center12), the 5-year survival rate by cancer site showed an improving trend for all of these 10 cancer sites. (Figure 2) The 5-year survival rate for patients diagnosed between 2010 and 2011, the most recent data available at this time, was 66.4% for all cancers, an improvement of 4.3 percentage points from the rate for patients diagnosed between 2006 and 2008. The highest 5-year survival rate for patients diagnosed between 2010 and 2011 was 98.8% for prostate cancer, and the lowest was 9.8% for pancreatic cancer. The lowest improvement was 0.6 points for colon cancer (part of the large intestine).

Since the improvement in 5-year survival rates is largely due to the spread of early diagnosis and treatment and improvements in surgical and other techniques, it is difficult to determine the extent to which drugs contribute to the improvement, but we analyzed the relationship between the improvement in 5-year survival rates and the number of approved drugs for each of these cancer types. The vertical axis shows the improvement in 5-year survival for patients diagnosed in 2003-2005 (a group in which drugs approved after 2011 had no effect on 5-year survival) and for patients diagnosed in 2010-2011 (a group in which drugs approved between 2011 and 2016 may have affected 5-year survival), and the horizontal axis shows the improvement in 5-year survival for each cancer type. The number of drug approvals per cancer type from 2011 to 2016 was plotted for each cancer type on the horizontal axis (Figure 3). (Figure 3) Note that leukemia and malignant lymphoma were not included in the analysis because 5-year survival data for patients diagnosed in 2010-2011 were not available.

The improvement in 5-year survival rates for lung cancer and gastric cancer, for which many drugs have been approved, was also significant. The improvement point for breast cancer was not so high, but this may be due to the fact that the original 5-year survival rate was as high as 89.1%. However, prostate cancer, for which the original 5-year survival rate was 93.8%, showed an even greater increase to 98.8%. For liver cancer, there were no new drugs approved during this period, but the 5-year survival improvement points were significant. The main cause of liver cancer is persistent hepatitis B and C virus infection, but there were a total of 20 drugs approved for hepatitis B and C between 2011 and 2016. According to the 2017 edition of the Japanese Society of Hepatology Guidelines for the Treatment of Liver Cancer, "Antiviral therapy after hepatic resection or puncture local therapy may contribute to the prevention of recurrence and improvement of survival in hepatocellular carcinoma caused by viral hepatitis." and suggests that drugs for viral hepatitis may indirectly contribute to improved survival in liver cancer.

Conclusion

The 2019 development and approval status of drugs for unmet medical needs was surveyed.

As in previous surveys, we found active development and approval status in each disease area, but this time we also focused on oncology, as the top five diseases in terms of development were all oncology. For the 10 cancer types surveyed, the number of drugs approved and the number of drugs in development showed similar trends, with high rates for both hematologic and lung cancers, and low rates for pancreatic and cervical cancers. The 5-year survival rates for all 10 cancer types showed an upward trend, confirming that the development and marketing of drugs are playing a role in this trend. On the other hand, there are some cancers, such as pancreatic cancer, where both treatment satisfaction, 5-year survival rate, and the number of new drugs developed are low, and further development of drugs and improvement of diagnosis and treatment are expected.

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