Pharmaceutical Industry at a Glance Development Status of Pharmaceuticals for Unmet Medical Needs

Printable PDF

The Office of Pharmaceutical Industry Research Takuro Utsumi, Principal Researcher
Shinji Tsubakihara, Chief Researcher

Summary

1. Introduction

The Office of Pharmaceutical Industry Research Based on the Medical Needs Survey conducted by the Laboratory of Social Pharmacy at Meiji Pharmaceutical University (the first six surveys were conducted by the Public Interest Incorporated Foundation for the Promotion of Human Sciences), we have compiled data on new drug approvals and development pipelines and ^have continuously ^analyzed pharmaceutical companies’ efforts to address unmet medical needs ^{2, 3, 4)}.

The results of the analysis of the development pipeline based on the most recent Medical Needs Survey conducted in fiscal year 2019 revealed that the discovery of novel molecules with high pathogen-specificity, the development of compounds with high targeting precision for these molecules, and the development of new modalities with innovative mechanisms of action are key factors in addressing unmet medical needs.Against the backdrop of modality innovation, drug development has surged, and as many of the 60 diseases surveyed have shifted to the first quadrant, we report that treatment satisfaction and the contribution of drugs have improved, leading to an increase in the degree to which medical needs are being met.On the other hand, in the most recent analysis of development candidates ⁽OPIR Views and Actions, No. ⁷⁰⁵⁾ (hereinafter referred to as the “previous analysis”), it was highlighted that while development is concentrated on first-quadrant diseases, the lag in development for third-quadrant diseases—which can be seen as emblematic of unmet medical needs—remains a challenge.

In this paper, based on the results of the FY2024 Medical Needs Survey, we summarize the status of new drug development and focus our analysis on the latest trends in modalities—which serve as a benchmark for drug discovery innovation, key to meeting unmet medical needs—as well as on drug development for third-quadrant diseases, a policy priority.

2. Analysis Method

The FY2024 Medical Needs Survey was conducted via questionnaire between December 12, 2024, and January 31, 2025, primarily targeting general internal medicine specialists working in hospitals.This survey covered 60 diseases selected as “serious diseases,” “diseases that significantly impair quality of life,” “diseases with a large number of patients,” and “diseases with significant societal impact.”Furthermore, “treatment satisfaction” is an indicator calculated by combining the percentages of physicians who responded that they were “fully satisfied” and “somewhat satisfied” with treatment for each disease; similarly, “drug contribution” is calculated by combining the percentages of physicians who responded that the drug “contributes fully” and “contributes somewhat.”Therefore, it is important to note that even when these indicators show high values, this does not necessarily mean that sufficient satisfaction or contribution has been established regarding the treatment or medications for the disease in question.

As in previous analyses of development candidates, the scope of this analysis covers domestic development candidates (Phase 1 through application pending) from 20 domestic and foreign pharmaceutical companies. The primary source of information was domestic development ^{pipeline data⁶} disclosed on each company’s website as of the end of November 2025.For reference, we used the Japan Registry of Clinical Trials “Clinical Research Submission and Disclosure System” website, the “Tomorrow’s New Drugs” (produced by Technomic) website, and the “Clinical Trial Information Disclosure” section of the Pharmaceuticals and Medical Devices Agency (NIH) website.Regarding the development status of third-quadrant diseases, we compared changes over time from August 2020 to November 2025 based on data aggregated from each company’s website.In addition, we analyzed approved and developmental products in Japan and the United States using “Tomorrow’s New Drugs,” and investigated companies’ founding years, revenue, and headquarters locations using Citeline’s “ ^{Pharmaprojects®} ” website to define the term “startup.”

Results

(1) Trends in Development Candidates by Treatment Satisfaction and Drug Contribution in the FY2024 Medical Needs Survey

Figure 1 plots the 60 diseases selected in the FY2024 Medical Needs Survey along the axes of treatment satisfaction (horizontal axis) and drug contribution (vertical axis), with the number of development candidates as of the end of November 2025 indicated by numerical values and circle size.

When examining the number of development candidates for the 60 diseases by quadrant, the number of development candidates for diseases in “Quadrant 1”—where both treatment satisfaction and drug contribution are 50% or higher—totaled 40 diseases,302 products (82.1%)—indicating a further improvement in the fulfillment of unmet medical needs compared to the previous survey. Among these, malignant neoplastic diseases accounted for 186 of the 302 products (61.6%), representing more than half of the total.Among diseases in “Quadrant 3,” where both treatment satisfaction and drug contribution are 50% or less, systemic sclerosis andmultiple sclerosis—saw an increase in drug contribution and moved to the second quadrant. Meanwhile, two diseases newly added to this survey—amyloidosis and developmental disorders—each entered the third quadrant, bringing the total to 10 diseases.Among these, pancreatic cancer saw two additional approved products and Alzheimer’s disease saw four, compared to the previous survey; however, the drug contribution for these conditions did not increase significantly. It also became clear that development for sarcopenia, fibromyalgia, and vascular dementia has still not progressed.

(2) Trends in Modalities for Products Under Development

In this section, we analyzed the domestic development pipelines of 20 domestic and foreign pharmaceutical companies across 60 diseases, broken down by modality. In the previous analysis conducted at the end of June 2023, the number of active ingredients in development was 207, of which 154 were new molecular entities (hereinafter referred to as NMEs).In this latest analysis, conducted as of the end of November 2025, the number of active ingredients in development stood at 224, with 164 being NMEs, indicating an increase in the number of development candidates.Furthermore, when classified by modality, as shown in Figure 2, while small-molecule compounds continued to maintain a steady presence, antibody drugs—which are becoming the focus of development—continued to diversify; in addition to conventional antibody drugs, multispecific antibodies and antibody-drug conjugates were gaining prominence.Furthermore, the number of development candidates for nucleic acid therapeutics and protein/peptide therapeutics—which were advancing in the treatment of malignant tumors in the previous analysis—has also increased, indicating further diversification of modalities.

The graph in Figure 3 shows changes in modality composition by development stage for NMEs targeting malignant and non-malignant tumors. As discussed in the previous analysis, in the field of drugs for malignant tumors, new antibody modalities—such as multispecific antibodies and antibody-drug conjugates—were gaining prominence alongside conventional antibody drugs.Furthermore, the composition of modalities was consistent across all phases (1–3), with no significant differences observed between stages. A similar trend to that observed for drugs for malignant tumors was also confirmed for drugs for non-malignant tumors.Although the proportion of cases where the modality could not be identified and was therefore listed as “unknown” was high in Phases 1 and 2, the number of development candidates such as multispecific antibodies, nucleic acid therapeutics, and protein/peptide preparations is increasing, and it is expected that modality diversification will continue to advance in the future.

Figure 4 shows modality trends by the top disease categories in terms of the number of NMEs under development.Among drugs for malignant tumors, the number of NMEs under development was highest for lung cancer, followed by breast cancer, malignant lymphoma, and prostate cancer; of the 60 drugs for malignant tumors, 21 were in development for lung cancer alone, accounting for one-third of the total.While many lung cancer development candidates had progressed to Phase 3, the number of candidates in Phases 1 and 2 was comparable to that of other cancer types. Furthermore, in prostate cancer, new modalities such as radioligand therapies were identified, indicating a trend toward further diversification.In disease areas outside of malignant tumors, diversification of modalities was also progressing in diseases with a large number of development candidates.While it is important to note that CKD is defined to include diseases such as lupus nephritis and IgA nephropathy, there were many Phase 3 candidates across a wide range of modalities. Furthermore, the number of candidates in development for Alzheimer’s disease and non-alcoholic steatohepatitis (hereinafter NASH) continued to rank among the highest.

(3) Development Status of Third-Quadrant Diseases

1. Analysis of Development Candidates for Third-Quadrant Diseases

In this section, we conducted a detailed analysis of diseases in the third quadrant—those for which both treatment satisfaction and drug contribution are 50% or less—which represent policy challenges. Figure 5 shows the changes over time from August 2020 to November 2025 for domestically developed products from 20 domestic and foreign pharmaceutical companies.For multiple sclerosis and systemic sclerosis, the drug contribution increased in this medical needs survey, causing them to move into the second quadrant. For both diseases, the number of products in development has been steadily increasing since 2020, and further fulfillment of unmet medical needs is expected in the future.On the other hand, eight diseases remained in the third quadrant, as they had in the previous medical needs survey. Among these, Alzheimer’s disease and pancreatic cancer showed a consistent trend of having a higher number of products in development compared to other diseases. Additionally, a steady number of products in development was observed for amyotrophic lateral sclerosis (ALS).The number of products in development for idiopathic pulmonary fibrosis has increased in recent years, and future approval trends warrant close attention. It became clear that development is not progressing for sarcopenia, fibromyalgia, and vascular dementia.

Furthermore, although there is no longitudinal data available for amyloidosis and developmental disorders—which were added to the list of target diseases in this medical needs survey—the number of products in development as of the end of November 2025 stood at one for amyloidosis and two for developmental disorders.

2. Analysis of Two Diseases Where the Contribution of Drugs Has Increased

We analyzed two diseases—multiple sclerosis and systemic sclerosis—which moved into the second quadrant as their drug contribution increased.

Multiple sclerosis is an immune-mediated inflammatory demyelinating disease of the central nervous system and is classified into subtypes such as relapsing-remitting multiple sclerosis (RRMS), primary progressive multiple sclerosis (PPMS), and secondary progressive multiple sclerosis (SPMS). Although no curative treatment has been established, development is progressing, primarily focused on disease-modifying therapies.Since the previous medical needs survey conducted in 2019, siponimod fumarate has been newly approved for SPMS, and ofatumumab has been newly approved for both RRMS and SPMS.In ^{the 2023} Clinical Practice ^Guidelines for Multiple Sclerosis and Neuromyelitis Optica Spectrum Disorders ^⁷), siponimod fumarate is strongly recommended (Grade 1A) for SPMS.Additionally, ofatumumab is weakly recommended (Class 2C) for early treatment of highly active RRMS (assessed based on relapse frequency, MRI activity, the Kurtzke Disability Scale, and brain atrophy), strongly recommended (Class 1B) for SPMS,and is weakly recommended (Class 2D) for SPMS with relapses and MRI activity.*In clinical practice guidelines, the strength of a recommendation is defined as “1: Strong (recommended or not recommended),” “2: Weak (suggested or not suggested),” and the certainty of evidence is defined as “A: Strong,” “B: Moderate,” “C: Weak,” and “D: Very weak.”

Systemic sclerosis is a disease characterized by hardening of the skin and internal organs. It presents with various clinical manifestations, including Raynaud’s phenomenon, skin sclerosis, other skin symptoms, pulmonary fibrosis, scleroderma renal crisis, and reflux esophagitis; drug selection is made based on each specific clinical presentation.Since the previous medical needs survey conducted in 2019, two new drugs—rituximab and mycophenolate mofetil (MMF)—have been approved.In ^the Clinical Practice ^Guidelines for Systemic ^{Sclerosis⁸)}, rituximab is strongly recommended (Grade 1A) and MMF is weakly recommended (Grade 2B) for skin sclerosis, while both agents are strongly recommended (Grade 1A) for interstitial lung disease associated with systemic sclerosis.

It was suggested that the market launch of drugs classified as standard treatments in clinical practice guidelines for both multiple sclerosis and systemic sclerosis may have contributed to the increase in the drug contribution index.

3. Comparison of Approved and Developmental Products in Third-Quadrant Diseases Between Japan and the U.S.

Since the previous analysis, three drugs for Alzheimer’s disease, one for amyotrophic lateral sclerosis, and one for amyloidosis have been approved in Japan for third-quadrant diseases.To investigate the detailed development status of third-quadrant diseases, we utilized search results from “Tomorrow’s New Drugs” as of March 2025 and compared approval and development trends with those in the United States, including companies other than the 20 domestic and foreign pharmaceutical firms.Although the scope initially covered all third-quadrant diseases, diabetic neuropathy was excluded because it is subsumed within the indication for neuropathic pain and was therefore deemed impossible to separate. Furthermore, to account for the impact of cases where Japanese-approved drugs are included in other countries’ development pipelines—which would arise if the comparison were expanded to include other countries—the analysis was limited to a comparison with the United States only.Startups were defined as companies with verifiable annual sales of less than $5 billion in the most recent year and established within the past 30 years; products developed in collaboration with pharmaceutical companies were counted as originating from startups. Companies confirmed to have merged by March 2026 were counted as part of the acquiring company.

As shown in Table 1, while there was no significant difference in approval status between Japan and the U.S. for diseases in the third quadrant, there was a marked difference in the number of products under development. While more than half of the products under development in the U.S. originated from startups, in Japan, products developed by startups were extremely few in number, which significantly influenced the difference in the number of products under development between the two countries.This suggests that challenges remain in implementing measures to promote pharmaceutical development by startups in Japan.Furthermore, regarding Phase 3 and later development candidates in third-quadrant diseases—including those currently under review—in Japan and the U.S., with the exception of Alzheimer’s disease and amyotrophic lateral sclerosis (ALS), there was still no significant difference between Phase 3 and later candidates and the overall number of development candidates.It is important to note that the number of products under development in Japan includes those developed exclusively in Japan by Japanese companies, as well as products based on U.S.-approved drugs due to the “drug lag.”

Here, we analyzed the development status by modality, focusing on the four diseases within the third quadrant that had the highest number of development candidates: Alzheimer’s disease, pancreatic cancer, amyotrophic lateral sclerosis (ALS), and idiopathic pulmonary fibrosis.

Figure 6 shows a comparison of approval and development status by modality for Alzheimer’s disease. In both Japan and the U.S., lecanemab and donanemab—the first antibody drugs targeting amyloid-β—have been approved, expanding treatment options.Alzheimer’s disease treatments approved in the U.S. but not yet approved in Japan consisted solely of combination therapies with existing prodrugs, offering no innovation; therefore, there was no substantial “drug lag” or “loss.” Compared to the analysis limited to the 20 pharmaceutical companies mentioned earlier, the diversification of modalities was progressing both domestically and internationally among products under development.In Japan, development was progressing for small-molecule compounds, antibodies, multispecific antibodies, nucleic acid therapeutics, peptides, and vaccines; however, in the United States, in addition to these, development efforts were underway for cell therapy, gene therapy, and protein-based therapeutics, and there were significant differences in the number of development projects for each modality.

Figure 7 shows a comparison of approval and development statuses by modality for pancreatic cancer.Notably, Zenocutuzumab—the first multispecific antibody for pancreatic cancer, currently under development in Japan—has been approved in the United States. While the pancreatic cancer treatments currently approved in both countries are primarily small-molecule compounds, the modalities of products in development are becoming increasingly diverse, a trend that was particularly pronounced in the United States.In Japan, development is underway for small-molecule compounds, antibodies, multispecific antibodies, antibody-drug conjugates, and nucleic acid therapeutics; however, in the United States, in addition to these, development is progressing for a wide range of modalities, including CAR/TCR-engineered cells, proteins, peptides,oncolytic viruses, vaccines, and cell therapies are also being developed; this resulted in a significant difference in the diversity of modalities between Japan and the U.S.

Figure 8 compares the approval and development status by modality for amyotrophic lateral sclerosis (ALS). Approved products for this disease are primarily small-molecule compounds, and there was no significant difference between Japan and the United States.There were no products approved in the U.S. but not yet approved in Japan; conversely, in Japan, mecobalamin—for which a company submitted an application based on the results of a physician-led clinical trial and received approval last year—was added to the therapeutic lineup. This represents a successful example of collaboration among industry, government, and academia in Japan.Regarding products under development, while diversification of treatment modalities is progressing in both Japan and the U.S., the U.S. held a significant lead in the number of products in development.Currently, in preparation for simultaneous submissions in Japan, the U.S., and Europe, a research team at the Japan Agency for Medical Research and Development (hereinafter AMED) is developing “Clinical Evaluation Guidelines for Amyotrophic Lateral Sclerosis (ALS) Therapeutics” to facilitate international collaboration on clinical trials and accommodate cutting-edge evaluation methods, similar to those in Europe and the U.S.

Figure 9 compares the approval and development status by modality for idiopathic pulmonary fibrosis. In both Japan and the United States, approved products consisted solely of small-molecule compounds, and there was no significant difference in the number of approvals. As with the diseases mentioned earlier, the pipeline in both Japan and the United States is becoming more diverse in terms of modalities, but the United States significantly outnumbers Japan in the number of products in development.Some products in development are unique to Japan; these include protein therapeutics from Japanese startups and cell therapies being developed through physician-initiated clinical trials.

4. Summary and Discussion

(1) Trends in Development Candidates by Treatment Satisfaction and Drug Contribution in the FY2024 Medical Needs Survey

Among the products under development for 60 diseases, 40 diseases—accounting for 302 products (82.1%)—are concentrated in the first quadrant, where both treatment satisfaction and drug contribution are high; it is expected that the degree to which unmet medical needs are met will continue to improve in the future.Furthermore, for systemic sclerosis and multiple sclerosis—which have shifted from the third quadrant to the second quadrant—the number of products in development continues to increase; therefore, further improvements in treatment satisfaction and drug contribution are expected in the next survey.On the other hand, 10 diseases—including amyloidosis and developmental disorders, which were added in this medical needs survey—are included in the third quadrant, where both treatment satisfaction and drug contribution are low; it is necessary to continue closely monitoring trends in development candidates and shifts in modalities for these target diseases.Furthermore, while multiple pipeline products were identified for NASH in the previous survey, no products have yet been approved, and the condition has shifted to a position straddling the third quadrant.Overseas, selective thyroid hormone receptor β agonists and GLP-1 receptor agonists have been approved as highly anticipated new drugs, and we intend to closely monitor future approval trends in Japan as well.

(2) Trends in Modalities for Products Under Development

Regarding modality trends in domestically developed drugs, diversification is progressing for both anticancer and non-anticancer drugs, and this trend is expected to continue.Among anticancer drugs, the number of NME development projects is high for four diseases—lung cancer, breast cancer, malignant lymphoma, and prostate cancer—and new modalities, such as radioligand therapies, have also been identified.In particular, for lung cancer—which accounted for one-third of all anticancer drugs—development candidates are concentrated in Phase 3, and it is expected that new approved drugs will further improve the satisfaction of unmet medical needs in the near future.Among non-malignant tumor drugs, there are many Phase 3 candidates across a wide range of modalities for chronic kidney disease (CKD), and we will be watching closely for future approval trends.Furthermore, for Alzheimer’s disease and NASH, although the high development difficulty has resulted in fewer approved drugs compared to other diseases, the number of drugs in development remains high, and diversification of modalities is progressing; therefore, we can expect an increase in drug contribution with the launch of approved drugs that become standard treatments.

(3) Development Status of Third-Quadrant Diseases

Among the 10 diseases in the third quadrant, Alzheimer’s disease and pancreatic cancer saw multiple approvals since the previous survey, but the contribution of these drugs did not increase significantly.In Alzheimer’s disease, a drug targeting amyloid-β—considered one of the causes of the disease—was approved; however, since it was launched after December 2023, it was deemed that this survey could not fully reflect its impact. Fundamental treatment for Alzheimer’s disease has only just begun, and surveys on the medical needs related to dementia—a global social issue—should continue in the future.The number of drugs in development for idiopathic pulmonary fibrosis has been increasing rapidly in recent years.Although not reflected in the results of this analysis, nelandomirast—a long-awaited phosphodiesterase 4B inhibitor with a novel mechanism of action that was approved in the United States in October 2025—had been in the application stage in Japan but ^received approval this past ^May⁹).We will be watching closely to see how it is positioned in future clinical practice guidelines. It has become clear that development for sarcopenia, fibromyalgia, and vascular dementia—which were identified as challenges in the previous analysis—has still not progressed. Although limited, development by startup companies has been confirmed overseas; therefore, we believe it is necessary to closely monitor the development status, not limited to the 20 domestic and foreign pharmaceutical companies.Furthermore, regarding amyloidosis and developmental disorders—which were added to this medical needs survey—the number of products in development as of the end of November 2025 stood at one for amyloidosis and two for developmental disorders; we intend to follow up on future development trends.

For systemic sclerosis and multiple sclerosis, which moved from Quadrant 3 to Quadrant 2, drugs launched after the previous unmet medical needs survey have been designated as standard treatments in clinical practice guidelines and are considered to be contributing to the fulfillment of unmet medical needs.In the case of systemic sclerosis in particular, examples of successful public support aimed at addressing unmet medical needs include public approval granted by the Unapproved Drug Review Committee in response to requests from academic societies, as well as approvals based on the results of physician-initiated clinical trials funded by AMED. Moving forward, we look forward to the promotion of development through collaboration among industry, government, and academia to further address these unmet medical needs.

We compared the approval and development status of pharmaceuticals for the 10 diseases in the third quadrant with that of the United States. While there was no difference in approval status between the two countries, significant differences emerged in the number of drugs under development and the diversity of modalities.While pharmaceutical companies have played the leading role in development in Japan, in the U.S., development by startups—in addition to pharmaceutical companies—is thriving. In particular, for diseases with few products in development, such as sarcopenia and fibromyalgia, there was a greater reliance on development by startups.Furthermore, a comparison of products that had progressed to Phase 3 or later revealed that differences had already emerged in Alzheimer’s disease and amyotrophic lateral sclerosis (ALS); it was therefore considered necessary to introduce promising assets and attract overseas startups in the future.While there were no significant differences for diseases other than these two, this was largely because there were no major disparities between Japan and the U.S. in the pipeline products led by pharmaceutical companies, and because many of the products being developed by startups were still in Phase 1 or 2.To curb the drug lag and loss anticipated in the future, it is necessary to build a sustainable domestic drug discovery ecosystem; in addition to fostering startups, support from pharmaceutical companies for drug discovery and the introduction of promising assets is considered crucial.It is hoped that the industry-government-academia collaboration through AMED IND ENGINE (AND-E) ^¹⁰, launched this year in cooperation between AMED and the Japan Pharmaceutical Manufacturers Association ^, will help spur the creation of innovative new drugs by startups and pharmaceutical companies.

5. Conclusion

While progress is being made in addressing unmet medical needs for 40 of the 60 diseases surveyed—representing two-thirds of the total—significant challenges remain for many diseases.This analysis suggests that promoting development through industry-government-academia collaboration, fostering domestic startups, and providing support from Japanese pharmaceutical companies for drug discovery and the acquisition of promising assets are also crucial. To curb future “drug lag” and “loss” and swiftly deliver necessary medical care to society, the establishment of a domestic drug discovery ecosystem is urgently needed.

Back to News List

TOP