The Pharmaceutical Industry at a Glance Development Status of Pharmaceuticals for Unmet Medical Needs -Trends in 2020-.

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Akira Nakao, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute

Introduction

The Pharmaceutical Industry Research Institute (PIRI) 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 surveys on medical ^needs1) conducted by the Human Science Foundation (hereinafter referred to as "HS Foundation"). ²⁾, 3.

In the previous Policy Research Institute News No. 59 (March 2020), based on the results of the 2014 Treatment Satisfaction Survey, we showed the number of development pipelines as of January 2020 and the number of drug approvals from 2011 to 2019 for 60 diseases, as well as the number of drug approvals for 10 cancer types included in the 60 diseases The relationship between the number of drug approvals for the 10 cancer types included in the 60 diseases and the degree of improvement in 5-year survival rates was analyzed. Based on the "Medical Needs Assessment on 60 Diseases (6th)" published in March 2020, this report shows the number of development pipelines as of the end of August 2020 for the latest 60 diseases, as well as the number of approved drugs for some selected diseases among the 8 diseases newly added to the 60 diseases (however, 2 new diseases are split among the diseases that have been covered previously). We report the results of the survey on approved drugs and products in development for several selected diseases.

New Drug Development Status by Treatment Satisfaction

Figure 1 plots the diseases along the treatment ^{satisfaction4)} (horizontal axis) and drug ^{contribution5) (} vertical axis) in the 2019 HS Foundation survey, and shows the number of developed ^products6) surveyed this time in terms of circle size and numerical value. The same 20 pharmaceutical ^companies7) as in the previous survey were included in the total number of development items (Phase 1 to Filed) in Japan as of the end of August ²⁰²⁰⁸⁾. The number of applicable development items was ²⁸⁴⁹⁾, of which 122 (43%) were new active ingredients (NMEs). As in the previous survey, the ratio of the number of NMEs developed for oncology to the total number of NMEs developed for 60 diseases was very high, at 50% (143/284). The number of NMEs in the 10 cancer disease development items was 44, or 31% (44/143). The low rate of NMEs in oncology compared to the total is considered to be due to the fact that a single drug is often developed for multiple oncology diseases or in a more subdivided manner for the same oncology disease.

One of the major differences between the current survey and the previous survey is that the percentage of products developed in the so-called first quadrant, which is the area where treatment satisfaction is 50% or more and drug contribution to treatment is 50%, increased from 64.4% to 79.6%. This was considered to be due to an increase in treatment satisfaction for "lung cancer," which has the largest number of products developed among the 60 diseases, from 37.3% to 52.4% between the FY2014 and FY2019 surveys. ⁹⁾ In conjunction with this increase, the percentage of treatment satisfaction increased from 50% to 50%. In conjunction with this, the percentage of the number of products developed in the second quadrant, which indicates treatment satisfaction of 50% or less and drug contribution to treatment of 50% or more, decreased from 29.2% to 4.6%.

Compared to the 61.7% (37/60) of the 60 diseases included in the first quadrant, the number of developed products (79.6%) in this area is quite large. However, 10 out of 60 diseases are cancer diseases, and 9 cancer diseases other than pancreatic cancer are included in the first quadrant, and for example, leukemia, a type of blood cancer, can be broadly classified into 4 types: acute/chronic x lymphoid/myeloid, and there are many different types of "00 cancer". It is therefore logical that the ratio of new drug development items in the first quadrant, which includes nine cancer diseases, is high.

Even if the percentages indicating satisfaction with treatment and contribution to drug development both exceed 50, the breakdown of satisfaction and contribution to drug development is that "somewhat satisfied (contribution)" is much higher than "fully satisfied (contribution). Most of the remaining percentages that did not reach 100 were "unsatisfied" and "not contributing much," but some of them included responses such as "treatment is not working" and "there are no drugs that work. This suggests that there is still plenty of room and demand for pharmaceuticals to contribute to the treatment of diseases, even if they are in the first quadrant. For reference, Table 1 summarizes the results of the HS Foundation survey for FY 2014 and FY 2019, as well as the number of products under development and the number of NMEs by disease for 20 pharmaceutical companies as of the end of August 2020.

Eight Newly Added Diseases

In this year's HS Foundation survey of medical needs, some diseases were replaced in light of the significant changes in the healthcare environment. To borrow a phrase from the report by the survey team, the eight diseases that were excluded are: "hyperuricemia/gout," "dyslipidemia," "chronic hepatitis C," "chronic hepatitis B," "HIV/AIDS," and "sinusitis" as diseases with high medical satisfaction and drug contribution; "sleep apnea syndrome," which is not treated primarily with drugs; and A total of 8 diseases, "IBD/Inflammatory Bowel Disease," which is a generic name for "Ulcerative Colitis" and "Crohn's Disease," were excluded, and "ALS/Amyotrophic Lateral Sclerosis," "Non-tuberculous Mycobacterial Infection," "Idiopathic Pulmonary Fibrosis," "Systemic Scleroderma," which were listed as new diseases for which new diagnosis, treatment methods and drugs are desired to be developed, were excluded. In addition to the six new diseases listed above, the following two new diseases have been added: "chronic constipation," for which many people are suffering and a new drug has recently been launched, and "sarcopenia," which has become a major problem due to the increase in the number of elderly people.

Number of drug approvals for the 8 newly added diseases

Of the eight newly added diseases, "ulcerative colitis" and "Crohn's disease" had been surveyed as "IBD/Inflammatory Bowel Disease" in terms of the number of approved and developed products, but the remaining six diseases had never been surveyed. Therefore, this time we surveyed the status of new drug approvals for the above 8 diseases for the past 13 years from 2007 to 2019, covering drugs with new active ingredients and new ^{indications11)}.

As shown in Table 2, in descending order of number of approved drugs, the following new drugs were approved: "ulcerative colitis" (new active ingredient 1, new indication 5), "chronic constipation" (new active ingredient 3, new indication 2), "non-tuberculous acidosis" (new active ingredient 1, new indication 4), "Crohn's disease" (new indication 4), "idiopathic pulmonary fibrosis" (new active ingredient 2), "systemic sclerosis" (new indication 2 (new indication 2), and ALS/Amyotrophic Lateral Sclerosis (new indication 1). There was no drug approved for "sarcopenia" in 2007 or earlier.

In the FY2019 medical needs assessment results for "chronic constipation," "Crohn's disease," and "ulcerative colitis," for which four or more drugs were approved, both treatment satisfaction and drug contribution exceeded 60%, indicating the effect that new drugs have on disease treatment, while for "non-tuberculous acidosis," for which five new drugs were approved in the past 13 years treatment satisfaction was low at 40.3% (63.9% for drug contribution). In "idiopathic pulmonary fibrosis," "systemic scleroderma," "ALS/amyotrophic lateral sclerosis," and "sarcopenia," for which less than two drugs were approved, both treatment satisfaction and drug contribution were below 50%. In particular, "ALS/Amyotrophic Lateral Sclerosis" had the lowest values (14.3% for treatment satisfaction and 15.6% for drug contribution) among the 60 diseases.

Number of drug development projects for the 8 newly added diseases

As shown in Table 1, the number of new 8 diseases developed by the 20 pharmaceutical companies as of the end of August 2020 was 2 for "ALS/Amyotrophic Lateral Sclerosis," 0 for "Non-tuberculous Mycobacterial Infection," 1 for "Idiopathic Pulmonary Fibrosis," 1 for "Systemic Scleroderma," 6 for "Ulcerative Colitis," 5 for "Crohn's Disease," 0 for "Chronic Chronic constipation" was 0, and "sarcopenia" was 0. In the case of "chronic constipation," 5 new drugs have been approved in the past 13 years, and in the FY2019 survey, treatment satisfaction and drug contribution were 81.4% and 90.9%, respectively, while Crohn's disease and ulcerative colitis have a relatively large number of products under development. However, the remaining five diseases are expected to see further improvement in medical treatment satisfaction and drug contribution in the future. However, for the remaining five diseases, the number of development cases was very low, even though the level of medical satisfaction and drug contribution were not so high.

We decided to investigate whether these low development numbers were the current status of drug development in Japan, or whether they just happened to be low among the 20 pharmaceutical companies surveyed. Specifically, the survey was not limited to 20 pharmaceutical companies, but was conducted using the domestic search function of "Tomorrow's New Drugs (Technomic Co., Ltd.)" for domestic development items, covering development from Phase I through the filing stage. Sarcopenia" was searched as "Muscle Loss Syndrome" in "Tomorrow's New Drugs". If the development pipeline was listed on the company's website, that information was also considered. As a result, we found that there are 10 products under development for idiopathic pulmonary fibrosis (including 8 NMEs and 9 products under development by companies), 8 products for ALS/amyotrophic lateral sclerosis (including 3 NMEs and 4 products under development by companies), 3 products for systemic scleroderma (including 1 NME and 2 products under development by companies), and 1 product for non-tuberculous The study found that 3 products for "systemic scleroderma" (including 1 NME and 2 company-developed products) and 1 product for "non-tuberculous acidosis" are under development. It is expected that new drugs will be approved and launched from among these developed products, and that treatment satisfaction and drug contribution will increase.

On the other hand, in the field of "sarcopenia," it was found that there were no products under development even though the scope of the survey was expanded. According to the Japanese Society for Sarcopenia and Frailty, the first sarcopenia treatment guidelines were developed at the end of 2017, but there are no treatment or prevention methods with a high level of evidence at this ^stage12). It is hoped that the pathogenic mechanism of the disease will be clarified in the future, and that disease prevention and treatment methods with a high level of evidence will be found.

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