Policy Research Institute page Approval Status of Drugs for Unmet Medical Needs

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Based on the results of the "Medical Needs Survey on 60 Diseases " *1 conducted by the Japan Health Sciences Foundation, we analyzed the status of pharmaceutical companies' efforts to address unmet medical needs. The results of the survey showed that most of the new drugs approved for 60 indications by 2022 were anti-cancer drugs, and a mid-term analysis of these anti-cancer drugs by category showed that each category had its own characteristics.

1. Introduction

Based on the results of the "Medical Needs Survey on 60 Diseases" conducted by the Human Science Foundation (HS Foundation), Policy Research Institute compiles data on new drug approvals and development pipelines and continuously analyzes the status of pharmaceutical companies' efforts to address unmet medical needs *2*3.

In this report, we report on our analysis of new drugs approved from January 2019 to the end of December 2021, as reported in the July 2022 Policy Research Institute News, plus new drugs newly approved through December 2022. The latter half of the report focuses on the area of anticancer drugs, where the number of approved new drugs has been increasing in recent years, and analyzes the changes in the number of anticancer drug approvals in three periods from January 2014 to December 2022.

The approved items of new drugs are those approved as "drugs containing new active ingredients," "new efficacy drugs," "new regenerative medicine products," or "additions to the scope of application of regenerative medicine products" in the items discussed by the Pharmaceutical Affairs and Food Sanitation Council of the Ministry of Health, Labour and Welfare or in the items reported.

2. number of new drugs approved by treatment satisfaction and drug contribution

The HS Foundation has continuously surveyed "treatment satisfaction" and "drug contribution" for 60 diseases selected as "serious diseases," "diseases that significantly impair QOL," "diseases with a large number of patients," "diseases with high social impact," etc. in the form of a questionnaire to physicians from 1994 to 2019. The results of the latest 2019 survey are shown in Figures 1-A and B. With advances in treatment methods and drugs, treatment satisfaction and drug contribution have improved significantly for some diseases. On the other hand, these graphs show that there still remain many diseases that are rated as not yet fully effective in terms of treatment and drugs, or "unmet medical needs".

Figure 1-A Satisfaction with treatment
Source: Human Science Foundation FY 2019 Domestic Basic Technology Survey Report "Medical Needs Survey on 60 Diseases (6th)"

Figure 1-B Contribution of drugs (medicines) to treatment
Source: Human Science Foundation FY 2019 Domestic Basic Technology Survey Report "Medical Needs Survey on 60 Diseases (6th)"

Using the results of the HS Foundation 2019 survey*4, the National Institute for Policy Studies investigated how many drug items were newly approved for these 60 diseases in Japan during the four-year period from 2019 to 2022 ( Figure 2 ). The size and value of the circles indicate the number of newly approved drug items (new drugs).

Figure 2 Number of new drug approvals (2019-2022) by treatment satisfaction and drug contribution (2019)
Note: Numbers (including those in parentheses) indicate the number of new drug approvals for the relevant indication; if the same drug is approved for two different diseases among 60 diseases, they are counted separately.
Note: The number of approved drugs for "neuropathic pain" and "CKD/Chronic Kidney Disease" has been changed from the previous survey due to a review of disease definitions.
Source: "Medical Needs Survey on 60 Diseases (6th)," a report on the 2019 Domestic Fundamental Technology Survey by the Human Science Foundation.
https://u-lab.my-pharm.ac.jp/~soc-pharm/achievements/ (Reference date: May 8, 2023), Review Report, New Drugs for Tomorrow, Pharmaceutical and Industrial Policy Institute Policy Research Institute News No. 66 (July 2022), Pharmaceuticals and Medical Devices Agency ( Prepared by the Pharmaceuticals and Industrial Policy Research Institute based on the PMDA's List of Approved Drugs (New Drugs) for Fiscal Years 2022 through 2022.

Over the past four years, the number of new drug items approved for the 60 diseases in the HS Foundation survey was 181 (including items with multiple indications in one approval). Of these, 73 were considered "new molecular entities (NMEs)," accounting for 40.3% of the total. 181 of these were approved by domestic companies (74) and foreign companies (107) *5.

Looking at the number of approved products related to 60 diseases by quadrant, the percentage of new drug approvals for diseases in the first quadrant, where both treatment satisfaction and drug contribution are 50% or higher, was 87.8% (159/181), higher than the 85.7% in the 2022 Policy Research Institute news results and higher than the 85.7% in the 2022 Policy Research Institute news results. This was a result of the concentration of new approvals in disease areas where both drug satisfaction and contribution were high. In particular, many new drugs were approved in the area of anti-cancer drugs, with 25 approved for "malignant lymphoma," 16 for "lung cancer," 14 for "leukemia," 10 for "breast cancer," 8 for "colorectal cancer," 6 for "liver cancer (hepatocellular carcinoma)," 5 for "prostate cancer," 3 for "stomach cancer," and 2 for "cervical cancer. Drugs targeting cancer diseases in the first quadrant accounted for the majority (56.0%, 89/159). As for the number of approved drugs in quadrants 2, 3, and 4, 6 (3.3%), 7 (3.9%), and 9 (5.0%), respectively.

3. trend of approval of anticancer drugs in 60 diseases

Figure 3 shows the percentage of new drugs approved since April 2009 for 60 diseases, of which 10 cancer diseases (malignant lymphoma, leukemia, lung cancer, stomach cancer, colorectal cancer, liver cancer, pancreatic cancer, prostate cancer, breast cancer, and cervical cancer) were reported in previous Policy Research Institute News, 2009 to 2011, The data are shown for each of the three years from 2014 to 2016 and 2019 to 2021, with the addition of a new three-year period from 2020 to 2022. In addition, it shows changes in the average treatment satisfaction and drug contribution for the 10 cancer diseases in the HS Foundation surveys reported in 2009, 2014, and 2019; the number of approved products associated with 60 diseases increased to 110, 124, 133, and 146, respectively. Of these, the number of new drugs for the 10 cancer diseases increased over time to 28, 45, 62, and 76, respectively, or 25.5%, 36.3%, 46.6%, and 52.1% in percentage terms, indicating that anticancer drugs accounted for more than half of the new drugs in the survey. Along with the increase in the number of anticancer drugs, the average treatment satisfaction and drug contribution for the 10 cancer diseases also increased over time, with a particularly large increase in drug contribution.

Figure 3 Number of Approved Drugs and Drug Contribution in 10 Cancer Diseases
Note: Cancer diseases covered are malignant lymphoma, leukemia, lung cancer, stomach cancer, colon cancer, liver cancer, pancreatic cancer, prostate cancer, breast cancer, and cervical cancer
Note: Treatment satisfaction and drug contribution are averages for 10 malignant neoplastic diseases.
Source: Human Science Foundation Domestic Basic Technology Survey Report "Medical Needs Survey on 60 Diseases" (2009, 2014, 2019)
Prepared by the Pharmaceuticals and Industrial Policy Research Institute based on the PMDA's list of approved items (new drugs, new regenerative medicine products, etc.) for fiscal 2022-2022.

4. trend of anticancer drug approvals by category

To investigate the factors behind the recent increase in the number of approved anticancer drugs for 10 cancer diseases, we analyzed the increase or decrease in the number of new types of anticancer drugs, focusing on molecular-targeted drugs and antibody drugs.Going back to the 2014 HS Foundation survey (the 5th survey) *6, the three-year period from 2014 to 2016 was defined as "2010s The three-year period from 2014 to 2016 was classified as the "mid-2010s" ("mid-2010s"), the three-year period from 2017 to 2019 as the "late-2010s" ("late-2010s"), and the three-year period from 2020 to 2022 as the "early-2020s" ("early-2020s"), and new drugs approved during each three-year period were classified into the "molecular target drugs", "antibody drugs" and "other anticancer drugs" categories*7 . The number of new drugs approved in each of these three years was classified into the categories of "molecular target drugs," "antibody drugs," and "other anti-cancer drugs.

The number of anticancer drugs in the three categories remained unchanged at 45 in both the mid- and late-'10s, but the number of approved drugs increased significantly to 71 in the early '20s ( Figure 4-A ). Looking at the transition by category, the number of "molecular-targeted drugs" increased over time from 10 to 18 to 27, and their percentages were 22.2%, 40.0%, and 38.0%, respectively. Antibody drugs" increased from 10 to 19 to 27, and the percentages were 22.2%, 42.2%, and 38.0%, respectively. The percentages for "other anticancer drugs" were 55.6%, 17.8%, and 23.9%, respectively, for the categories of 25 → 8 → 17.

Figure 4 New Drugs Approved for 10 Cancer Diseases Classified by Approval Category (2014-2022)
Note: Targeted cancer diseases are malignant lymphoma, leukemia, lung cancer, stomach cancer, colorectal cancer, liver cancer, pancreatic cancer, prostate cancer, breast cancer, and cervical cancer
Source: "Medical Needs Survey on 60 Diseases (6th)," a domestic basic technology survey report for fiscal 2019 by the Japan Health Sciences Foundation.
Prepared by the Pharmaceuticals and Industrial Policy Research Institute based on the PMDA's list of approved items (new drugs) for FY2022.

Next, we analyzed the trends by category to determine whether the increase in the number of approved anticancer drugs was due to an increase in the number of NMEs or an increase in the number of indications added to existing drugs. In the early 20s, the number of NMEs in the three categories was 20, while the number of additional indications was 51 . This suggests that the increase in the number of approved anticancer drugs in recent years was largely due to the addition of new indications.

Figure 5 New Drugs Approved for 10 Cancer Diseases Classified by Approval Category (2014-2022)
Note: Cancer diseases covered are malignant lymphoma, leukemia, lung cancer, stomach cancer, colon cancer, liver cancer, pancreatic cancer, prostate cancer, breast cancer, and cervical cancer
Source: "Medical Needs Survey on 60 Diseases (6th)," a report of the 2019 Domestic Basic Technology Survey by the Foundation for the Advancement of Human Sciences
Prepared by the Pharmaceuticals and Industrial Policy Research Institute based on the PMDA's list of approved items (new drugs) for FY2022.

An analysis of the approval status of additional indications in each category during the nine-year period from the mid-'10s, late-'10s, and early-'20s shows that 32 (23 products) were newly approved for 10 cancer diseases, 42 (17 products) for "Molecular Targeted Drugs," and 30 (23 products) for "Other Anticancer Drugs," while the number of newly approved for "Antibody Drugs" was 51 (51 products). The number of newly approved anti-cancer drugs by category was 30 (23 products). When these products were sorted by category in order of the number of additional approvals ( Figure 6 ), there was a tendency for antibody drugs to receive more approvals for additional indications per drug. In particular, anticancer drugs called immune checkpoint antibody drugs occupied the top three positions.

Figure 6 Number of additional indications for each drug category in 10 cancer diseases (2014-2022)
Note: Horizontal axis indicates additional products approved (n indicates the number of products). Vertical axis indicates the number of additional indications for each product.
Note: Targeted oncological diseases are malignant lymphoma, leukemia, lung cancer, stomach cancer, colorectal cancer, liver cancer, pancreatic cancer, prostate cancer, breast cancer, and cervical cancer
Source: Created by the Pharmaceuticals and Industrial Policy Research Institute based on the PMDA's list of approved drugs (new drugs) for fiscal years 2014 through 2022.

5. number of anticancer drug approvals by domestic and foreign capital

We analyzed the number of approvals in the mid-, late-, and early-periods of the decade for anticancer drugs for 10 cancer diseases, dividing the companies applying for approval into domestic and foreign-capital companies ( Figure 7 ). For anticancer drugs as a whole, the number of items approved by domestic companies increased in the early 20s, while the number of items approved by foreign companies increased approximately 2.5-fold compared to the mid-'10s. By category, foreign firms held the largest share of the "molecular-targeted drugs" category for the entire period, while the number of approved domestic drugs also increased over time. In the "Antibody drugs" category, both domestic and foreign firms showed growth, with domestic firms holding the lion's share of the market for the full year.

Fig. 7 Ratio of In-House Products in 10 Oncological Diseases (2014-2022)
Note: Oncological diseases covered are malignant lymphoma, leukemia, lung cancer, stomach cancer, colorectal cancer, liver cancer, pancreatic cancer, prostate cancer, breast cancer, and cervical cancer.
Source: Prepared by the Pharmaceuticals and Industrial Policy Research Institute based on the PMDA's list of approved drugs (new drugs) for fiscal years 2014 to 2022.

Summary and Discussion

The number of new drugs approved in Japan during the four-year period from 2019 to 2022 was overlaid on the treatment satisfaction (horizontal axis) and drug contribution (vertical axis) for 60 diseases in the "HS Foundation Survey 2019 (6th)," showing that the percentage of approved drugs for diseases in the first quadrant, where both treatment satisfaction and drug contribution are 50% or higher, is 87.8%. The number of approved drugs for diseases in the first quadrant, where both treatment satisfaction and drug contribution are at least 50%, was high at 87.8%, and was concentrated in the first quadrant. In particular, anticancer drugs accounted for about 60% of the items in quadrant 1, and in the last three years, about half of the new drug approvals for 60 diseases were for anticancer drugs.

An analysis of the background to the recent increase in the number of anticancer drugs shows that the reason is that the number of "molecular-targeted drugs" and "antibody drugs" has increased over time, and that the number of approvals for additional indications has had a greater impact than the number of NMEs approved. In particular, the number of approvals for immune checkpoint antibody drugs, which target a molecule called PD-1 that evades attack by autoimmunity commonly expressed in many cancer tumors, is likely to provide more opportunities to add indications for multiple cancer diseases. In addition, some molecular-targeted drugs were approved for additional indications for multiple cancer diseases that share the same genetic mutation, even though they are found in different organs.

The increase in the number of anticancer drug items over time was accompanied by an increase in treatment satisfaction and drug contribution in the HS Foundation Survey, suggesting that advances in therapeutics and the increase in the number of therapeutic drug items have contributed significantly to the clinical practice of cancer disease. However, as mentioned above, there remain many diseases with low treatment satisfaction and drug contribution and unmet medical needs among the 60 diseases as rated by physicians. In addition, even for diseases in the high quadrant of treatment satisfaction and drug contribution, such as oncology, a closer look reveals that few respondents are "fully satisfied with treatment" or "pharmaceuticals are making a sufficient contribution," and in fact, oncology diseases that have a significant impact on patients' life expectancy still do not fully meet medical needs. In fact, cancer diseases, which have a significant impact on patients' life expectancy, still do not fully meet medical needs.

It is an important social responsibility of pharmaceutical companies and the pharmaceutical industry to develop "breakthrough new drugs" as well as to expand the indications of existing drugs to meet the unmet medical needs of all patients. To ensure that the activities of pharmaceutical companies and the pharmaceutical industry can be objectively evaluated as a contribution to patients, the Pharmaceuticals and Industrial Policy Research Institute (PIIPRI) plans to continue its research and analysis of the approval status and development status of pharmaceutical products in terms of treatment satisfaction and pharmaceutical contribution to disease.

( Shinji Tsubakihara, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute)

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