Pharmaceutical Industry at a Glance Approval Status of Drugs for Unmet Medical Needs

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The Office of Pharmaceutical Industry Research Natsuko Watanabe, Principal Investigator
The Office of Pharmaceutical Industry Research Shinji Tsubakihara, Principal Investigator

Summary

1. Introduction

The Office of Pharmaceutical Industry Research The Japan Health Sciences Foundation (hereafter referred to as "HS Foundation") has continuously analyzed the status of pharmaceutical companies' efforts to address unmet medical ^needs2) by compiling data on new drug approvals and development pipelines based on the results of the Medical Needs ^Survey1) conducted by the HS Foundation.

The HS Foundation dissolved at the end of March 2021 after the sixth survey in FY 2019, but after five years, the survey on medical needs was taken over by the Laboratory of Social Pharmacy at Meiji Pharmaceutical University in the same format as the Foundation's survey. The 2024 ^survey3), the seventh survey in total, was conducted between December 12, 2024 and January 31, 2025, mainly targeting general internal medicine specialists working at hospitals.

Satisfaction with treatment" in this survey is an indicator calculated by summing the percentages of physicians who responded "fully satisfied" and "somewhat satisfied" for each disease, and "pharmaceutical contribution" is similarly calculated by summing the percentages of physicians who responded "fully contributing" and "somewhat contributing" for each disease The "drug contribution" is similarly calculated by adding up the percentages of respondents who answered "fully contributing" and "somewhat contributing. Therefore, it should be noted that even if these indicators show high values, it does not mean that sufficient satisfaction or contribution has been established for the treatment or therapeutic agents for the disease in question.

The 60 diseases to be surveyed are those considered important after discussion by the survey team. Specifically, they include serious diseases, diseases that significantly impair QOL, diseases with a large number of patients, and diseases with a large social impact. The purpose of this report is to summarize the status of new drugs approved in Japan for these 60 diseases between 2020 and 2024 based on the results of the most recent treatment satisfaction survey (FY2024), and to clarify the composition of new molecular entities (NMEs) by modality and characteristics by therapeutic area. The purpose of this report is to summarize the status of new drugs approved in Japan during 2020-2024.

In addition, by comparing the results of the surveys in FY2014 and FY2019, we extracted diseases with significant differences in treatment satisfaction and drug contribution, and discussed whether the number of new drug approvals and the impact of innovations such as new modalities, new pharmacological actions, etc. can be inferred.

Survey Methodology

Based on the results of the "Medical Needs Survey for 60 Diseases (Results of FY2024 Survey)" published in "Pharmaceuticals and Medical Devices Regulatory Science" (Vol. 57, No. 1), the number of new drugs approved for 60 diseases from 2020 to 2024 was identified from the "Approved Drug Items" on the website of the Pharmaceuticals and Medical Devices Agency (NIH). The PMDA also compiled data on new drugs approved for 60 diseases between 2020 and 2024 based on the results of the "Medical Needs Survey (FY2024). The PMDA publishes the data for each approved drug category for each fiscal year, but the data were compiled for the calendar year in previous research reports by the National Institute for Policy Studies (NIPR), so the data were compiled according to customary practice. The modality survey of these new drugs was based on the commercial database "New Drugs for Tomorrow" (prepared by Technomic) and the review reports of each drug.

In this paper, new molecular entities (NMEs) are defined as "drugs containing new active ingredients (including combination products)" and "newly approved regenerative medicine products (including those approved with conditions and time limits)" among the new drugs.

Results

(1) Number of new drugs approved by treatment satisfaction and drug contribution

Figure 1 plots diseases along the treatment satisfaction (horizontal axis) and drug contribution (vertical axis) for 60 diseases selected as "serious diseases," "diseases that significantly impair QOL," "diseases with a large number of patients," "diseases with a large social impact," etc. in the 7th Survey reported in 2024, and shows the number of drugs approved in the last 5 years (2020 - The number of new drugs approved in Japan in the last five years (2020-2024) that are indicated for the 60 diseases is indicated by the size of the circle and the numerical value.

New drugs are defined as those approved as "drugs containing new active ingredients (including combination drugs)," "new indication drugs," "new regenerative medicine products," and "additional indications for regenerative medicine products.

The total number of approved drugs during this period was 610, and 237 drugs (including drugs with multiple indications in one approval) and 222 products were approved for 60 diseases in the 7th Survey. Of these, 99 (90 products) were new molecular entities (NMEs), accounting for 41.7% (99/237).

Looking at the 60 diseases by quadrant, the "quadrant 1" diseases, in which both treatment satisfaction and drug contribution were greater than 50%, were the most common, at 40 (66.7%), and included 9 of the 10 malignant neoplastic diseases.

Looking at the approved items related to 60 diseases in 2020-2024 by quadrant, the percentage of new drug approvals for diseases in quadrant 1 was 86.9% (206/237), with new approvals further concentrated in disease areas with high levels of both treatment satisfaction and drug contribution.

The first quadrant is dominated by products approved for malignant tumor diseases, with 35 products approved for "malignant lymphoma," 22 for "lung cancer," 18 for "leukemia," 15 for "breast cancer," 9 for "colorectal cancer," 9 for "uterine cancer," 8 for "stomach cancer," 6 for "prostate cancer," and 5 for "liver cancer (hepatocellular carcinoma)," while the second quadrant is dominated by products approved for "hepatoma," 9 for "uterine cancer," 8 for "prostate cancer," 6 for "hepatocellular carcinoma," and 5 for "hepatocellular carcinoma (HCC). These items accounted for 60% of the total number of approved items for Quadrant 1 diseases (61.7%, 127/206).

In the single year 2024, 31 new drugs were added to the list of treatments for the 10 malignant neoplastic diseases.

As for the number of approved drugs in quadrants 2, 3, and 4, 12 (5.1%), 15 (6.3%), and 4 (1.7%), respectively.

(2) Modality analysis of approved products for 60 diseases

This section summarizes the approval trends by modality for drugs approved for 60 diseases and NMEs among them during 2020-2024, and provides an overview of the characteristics of recent drug development.

First, Figure 2-1 shows the number of approved drugs for the 60 diseases by modality. Small molecule drugs (SM) continued to play a central role, accounting for 50-70% of the total during the entire period. On the other hand, the percentage of approved antibody drugs (Ab) remained at 20-30%, and the number of approved antibody-drug conjugates (ADC) and multispecific antibody drugs (msAb) will increase in 2023 and 2024, respectively. The diversification of antibody drugs was progressing, with an increase in the number of approvals for antibody-drug conjugates (ADCs) in 2023 and multispecific antibody drugs (msAb) in 2024. Nucleic acid drugs (oligonucleotidetherapeutics; OT) were only approved in one case during the period.

Next, Figure 2-2 shows the number of NME approvals by modality for 60 diseases. while the number of NME small molecule drugs remained constant each year except for 2023, the percentage of approvals for these drugs showed a gradual downward trend until 2023, but had recovered by 2024. The percentage of NMEs approved for antibody drugs showed an overall increasing trend, but the number of multi-specific antibodies and ADCs, which are derivatives of NMEs, increased in 2024, indicating that the diversification of modalities in the field of antibody drugs is progressing. The number of approvals for nucleic acid drugs was only one.

By therapeutic area, the NME for malignant neoplastic diseases (Figure 2-3) was characterized by an increase in the number of approvals for small molecule drugs, multi-specific antibodies, and ADCs in 2024.

On the other hand, in NME for diseases other than malignant tumors (Figure 2-4), small molecule drugs and antibody drugs accounted for the majority, and the diversification of modalities was more limited than in the malignant tumor area. The number of approvals showed an increasing trend through 2022, but a decline was observed after 2023.

(3) Analysis of changes in treatment satisfaction with the drug contribution and the level of drug contribution

In this section, we focus on the diseases that showed a significant increase in treatment satisfaction and drug contribution compared to the FY2014 and FY2019 survey results, and summarize the status of new drug approvals as a possible background for this increase. Note that some target diseases were changed in the FY2014, FY2019, and FY2024 surveys (Table 1). Therefore, diseases for which survey data were not available at the three time points were excluded from the analysis.

To analyze whether the increase in drug contribution spills over to treatment satisfaction, an approximate curve was created from the values at three time points (FY 2014, FY 2019, and FY 2024) for diseases with treatment satisfaction and drug contribution of 50% or higher in the FY 2024 survey, and the rates of change in treatment satisfaction and drug contribution were calculated. Analysis of 38 diseases that were in the first quadrant of the FY2024 survey and for which there were three time points of data showed no correlation between the rates of change in treatment satisfaction and drug contribution, although the linearity was positive (Figure 3 left). This may suggest that treatment satisfaction is not only influenced by the drug, but also by multiple factors such as disease characteristics, other treatment modalities, and patient response. Note that this analysis was not intended to directly examine the causal relationship between the two indices, but was positioned as an exploratory analysis aimed at identifying disease groups in which improvement in treatment evaluation was simultaneously observed, by identifying relative trends in change over time.

With this in mind, we focused on diseases that showed relatively large improvements in both treatment satisfaction and drug contribution, and extracted disease groups in which improvements in both indicators were observed simultaneously. Based on the rate of change obtained from the proximity curves, we extracted the diseases that ranked within the top 10 in both treatment satisfaction and drug contribution: (1) atopic dermatitis (11.9%/20.8%), (2) psoriasis (6.9%/12.7%), (3) SLE/systemic lupus erythematosus (6.5%/10.6%), (4) malignant lymphoma (10 These were the four diseases that were mainly or concomitantly associated with immune abnormalities or skin lesions in which immune cells become tumors (Table 2). No clear correlation was observed between the rate of change in treatment satisfaction and the rate of change in drug contribution in these four diseases (Figure 3, right).

Summary and Discussion

In this paper, we examined the relationship between recent trends in drug development and treatment evaluation by combining the results of treatment satisfaction and drug contribution surveys from the 7th Medical Needs Assessment by the Laboratory of Social Pharmacy, Meiji Pharmaceutical University, and the analysis of new drugs and NMEs approved from 2020 to 2024.

(1) Number of new drugs approved by treatment satisfaction and drug contribution

The number of new drugs approved for 60 diseases reached 237 in the last five years, of which NMEs accounted for about 40%. Approvals were concentrated in the first quadrant, where both treatment satisfaction and drug contribution were high, with new drugs for malignant neoplastic diseases in particular accounting for the majority of approvals. Although many malignant neoplastic diseases have recently moved into Quadrant 1, they remain one of the leading causes of death worldwide, and the high level of unmet medical need means that drug development is focused on these areas.

(2) Modality analysis of approved products for 60 diseases

Looking at the modality composition of all approved products for the 60 diseases, small molecule drugs played a central role throughout the entire period, confirming that they continue to be the foundation of drug development. On the other hand, the presence of antibody drugs was maintained throughout the year, and the introduction of derivative modalities of antibody drugs, such as ADCs and multispecific antibodies, has been progressing in recent years, indicating that therapeutic options are expanding.

Especially in the area of malignant diseases, in addition to antibody drugs, ADCs, and multispecific antibodies, drugs with various modalities such as cellular medicine and protein/peptide preparations have been continuously introduced, suggesting that therapeutic options are expanding and therapeutic technologies are becoming more sophisticated. On the other hand, in disease areas other than malignant tumors, no significant changes were observed in the diversification of modalities or the number of NME approvals. In particular, diseases classified in quadrants 3 and 4, where the drug contribution is less than 50%, do not include malignant neoplastic diseases except pancreatic cancer, and as shown in Figure 1, the number of approved products tends to be relatively small compared to diseases classified in quadrant 1, suggesting that the approval trends differ depending on disease characteristics. This suggests that there may be differences in approval trends depending on the characteristics of the disease.

Given this situation, the study indicates the importance for pharmaceutical companies to accurately identify unmet medical needs and continue their efforts to expand treatment options, especially for diseases classified into quadrants 3 and 4, where the drug contribution is relatively low. On the other hand, in many of these diseases, understanding of the pathogenesis and identification of effective drug targets have not progressed sufficiently in many cases, and there are limits to the breakthroughs that can be created solely through the drug discovery efforts of companies alone. Therefore, it is essential for the government to provide continuous support for research infrastructure to elucidate disease mechanisms and deepen basic research, which are prerequisites for drug discovery, and for academia and public research institutions to collaborate with pharmaceutical companies to continue and deepen R&D to address unmet medical needs.

(3) Analysis of changes in treatment satisfaction with the drug contribution and the level of drug contribution

As a result of comparing treatment satisfaction and drug contribution at three points in time (FY2014, FY2019, and FY2024) and calculating the rate of change, four diseases were identified as being within the top 10 in both indices: atopic dermatitis, psoriasis, SLE/systemic lupus erythematosus, and malignant lymphoma. These diseases are caused by immune abnormalities or tumorigenesis of immune cells, and have in common that the introduction of new modalities and drugs with new pharmacological actions in recent years has progressed in parallel with changes in the therapeutic environment for each disease. The new ingredients approved for these diseases are listed in Table 3 by modality.

For atopic dermatitis, small molecule drugs such as Janus kinase (JAK) inhibitors, phosphodiesterase 4 (PDE4) inhibitors, and drugs with multiple dosage forms ranging from topical to oral were approved during this period, expanding treatment options for molecular targets with different inflammatory and immune pathways. Meanwhile, in addition to agents targeting the interleukin (IL)-4/IL-13 pathway, IL-13-specific antibodies and agents that inhibit the IL-31 pathway have been introduced in biologic agents, enabling more sophisticated treatment based on the pathophysiology of atopic dermatitis. These drugs with novel mechanisms of action were effective for patients who had not been sufficiently improved by existing treatments, and the progress of stratified treatment according to severity and symptom characteristics was assumed to have contributed to the increase in treatment satisfaction and drug contribution.

In psoriasis, the approval of oral kinase inhibitors such as topical PDE4 inhibitors, JAK inhibitors, and tyrosine kinase 2 (TYK2) inhibitors, which suppress skin inflammation as small molecule drugs, expanded treatment options, and the use of biologic agents such as tumor necrosis factor (TNF) inhibitors followed, In addition, biologics continued to be approved that target various inflammatory cytokines, including IL-17, which affects skin inflammation and hyperkeratosis, IL-23, which affects joint symptoms, and IL-36, the main etiologic cytokine in pustular psoriasis, a designated incurable disease. These agents are superior to conventional therapies in terms of speed of improvement of skin rash, persistence, and side effect profile, and they have significantly changed the treatment system for refractory psoriasis by targeting cytokines that are associated with each of the various pathological states of psoriasis, enabling treatment according to the pathological state. The development of a modality that enables specific inhibition of the inflammatory pathway by identifying the target molecule that causes the disease was considered to be a factor in improving physician satisfaction with the realization of "personalized treatment" according to the symptoms, pathological conditions, and comorbidities of patients.

In malignant lymphoma, in addition to small molecule drugs, drugs of various modalities such as antibody drugs, multispecific antibodies, ADCs, protein peptides, and even cellular medicine were approved. According to the "Cancer Information Service" of the National Cancer Center, there are more than 100 known types of malignant ^lymphoma4), suggesting that drugs with different actions have contributed to the expansion of treatment options for malignant lymphoma, which shows extremely diverse pathologies. It is particularly important to note that the approval of several types of Chimeric antigen receptor T (CAR-T) cells as regenerative medicine products has made it possible for patients with refractory or relapsed disease who cannot receive stem cell transplants to have access to treatment opportunities. In addition, the approval of several oral small molecule drugs with novel pharmacological actions for various disease types, such as Bruton-type tyrosine kinase (BTK) inhibitors, histone deacetylase (HDAC) inhibitors, and histone methyltransferase (EZH) inhibitors, has expanded treatment options for patients with various types of refractory disease, including those who are able to receive outpatient treatment. The approval of several oral small-molecule drugs with novel pharmacological actions for various disease types, such as inhibitors, has expanded treatment options and provided treatment convenience. The overall effect of these factors may have had a certain impact on physicians' satisfaction with treatment.

For SLE/systemic lupus erythematosus, only one antibody drug, anifrolumab, was approved in the period under review. Although it is a designated incurable disease, it is still mainly treated with glucocorticoids and classical immunosuppressive agents (azathioprine, cyclophosphamide, cyclosporine, tacrolimus), ⁵⁾ and there are only two antibody drugs with new pharmacological actions, including anifrolumab. However, two new drugs (rituximab and voclosporin) were approved for lupus nephritis, a severe complication of SLE, during this period, and it is assumed that this has led to an increased range of treatment strategies for the various symptoms of SLE patients, which has led to improved physician evaluation.

The direction of drug development in recent years has not only seen an increase in the number of new drugs, but also an increase in the sophistication of treatment systems that enable personalized therapy through the expansion of new modalities that elaborately reflect molecular targets according to the pathological conditions. Especially in immunology-related diseases and malignant neoplastic diseases, the introduction of drugs with diverse mechanisms of action has directly led to improvements in treatment satisfaction and drug contribution, and the diversification of modalities will continue to contribute to the improvement of therapeutic value in the medical field. In the future, there is a need for a mechanism to understand the magnitude of needs for each disease and changes in treatment satisfaction over time in parallel with trends in drug development, and to encourage R&D in areas that are truly in need.

5. conclusion

This paper reviews changes in treatment satisfaction and drug contribution based on medical needs surveys, as well as trends in new drugs and new compounds approved in recent years. The analysis confirmed that the introduction of drugs with new modalities and new mechanisms of action, especially in immunology-related and malignant neoplastic diseases, has contributed to the improvement of therapeutic value. On the other hand, treatment options remain limited in some areas and unmet medical needs remain.

In addition, there are some diseases other than malignant neoplastic diseases for which there has been no significant progress in the diversification of modalities and approval trends of NMEs, suggesting that there are still disease areas where therapeutic drug development is inadequate. In the future, it will be important to continuously monitor the magnitude of medical needs for each disease and changes in treatment satisfaction and drug contribution over time, and to examine the relationship with drug development trends from various perspectives.

We hope that the suggestions made in this paper will help us to consider the direction of drug development and lead to ongoing discussions on how to resolve unmet medical needs.

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