Introduction
The author has no prior experience working in a pharmaceutical company nor direct involvement in drug research and development. However, in 1981, I established a community of researchers focused on applying computational technology—or what we now call ICT—to pharmaceutical research and development, which eventually became the CBI Society. I was involved in its operations until 2010. Prior to that, I was engaged in applying ICT to clinical and biomedical research, laying the groundwork for today’s big data and AI applications in medicine. Additionally, I worked in a national research institute responsible for foundational studies related to pharmaceutical regulations. In this role, I led efforts to establish a global regulatory science network concerning chemical safety in collaboration with WHO and other UN-affiliated organizations. Through these experiences, I learned that understanding the realities of drug development is extremely challenging for those outside the pharmaceutical industry.
One significant change in drug development since the 1980s is the inclusion of molecular biologists alongside experts in medicinal chemistry. Today, clinical researchers, particularly physicians, play a significant role, and expectations for ICT-related professionals, such as data scientists, are rising. This suggests that opportunities for collaboration among researchers from diverse specialties will continue to grow in drug development. However, new participants in drug development often find it difficult to grasp its essence. While part of the challenge lies in the differences between research disciplines, the more fundamental issue is that the pharmaceutical industry operates under strict regulations and within the framework of public health services.
At the same time, public funding for basic life sciences research, exemplified by genome decoding projects, is accompanied by promises of breakthroughs in understanding rare diseases and developing innovative new drugs. Unfortunately, the media, which often amplifies such expectations, does not fully understand the realities of drug development. This misunderstanding extends to many researchers benefiting from these funds. To bridge this gap, we translated a book in 20081, which later led to the translation of another book by the same authors examining the present and future of pharmaceutical R&D2. Using these translations as references, we organized a series of research meetings titled “Exploring New R&D Models for Drug Development,” conducting our own information gathering and analysis. While further details can be found on the event website3, this article summarizes the discussions from these meetings, focusing on the changing landscape of drug development from a researcher’s perspective.
The Crisis in the Pharmaceutical Industry and Its Countermeasures
Around 2010, concerns emerged over the so-called “Pharma Crisis,” driven by the successive expiration of blockbuster drug patents held by major pharmaceutical companies. Over the previous 30 to 40 years, big pharma had been a reliable investment target, delivering double-digit growth. The diminishing appeal of these companies to investors represents one aspect of the crisis. To address these challenges, pharmaceutical executives, tasked with meeting investor expectations, adopted strategies such as mergers and acquisitions, restructuring research and development divisions (including workforce reductions), cost-cutting, and using surplus funds to repurchase shares, thereby boosting stock prices. While these strategies are reasonable survival tactics for publicly traded companies, they do not enable the development of high-risk, socially essential drugs.
Medicines are vital tools for protecting public health, yet only private pharmaceutical companies have the capacity to develop them. However, even if society needs certain medications, private companies cannot pursue them if they are unprofitable. While it is possible to import medicines from abroad without engaging in development, or to generate profits by focusing on generic drugs, such an approach might suffice for nations like Israel or Singapore. However, for countries like the United States, Europe, and Japan—leaders in international science and technology—focusing solely on following in the wake of others is untenable.
In response, these nations have implemented measures to create a business environment that enables pharmaceutical companies to undertake long-term, high-risk research and development. Such measures include stimulating interest in addressing rare diseases with small patient populations, increasing funding for academia to conduct foundational research that can identify or suggest new drug targets, reducing barriers to Phase III clinical trials—the final stage before market approval—and fostering collaboration among private pharmaceutical companies, public institutions, and academia. These initiatives collectively fall under the umbrella of Translational Research (TR).
In simple terms, this initiative aims to extend the efforts pioneered in precision medicine for cancer to other diseases. Key implementation tools include rapidly decreasing costs for genome sequencing, electronic health records, portable and user-friendly biosensors (wearables), and advanced data science. Additionally, the plan actively invites participation from a broad range of people, including patients and citizens, emphasizing inclusivity. This ongoing and detailed observational effort, described as “research for everyone,” aspires to gather data representative of America’s diverse population, transcending ethnic differences. As of now, approximately 200,000 participants have registered. Notably, the initiative seeks to regard participants not merely as subjects of research but as “partners in research,” reflecting a new ethos for the modern era.
Differences Between the U.S., Europe, and Japan
The new trends in drug development described above appear to be reaching Japan as well. The rationalization through mergers has already been experienced. In emerging fields such as biopharmaceuticals, there is an increasing trend of partnerships with or acquisitions of biotech ventures. This raises the possibility of a pharmaceutical company evolving to its ultimate form, where only capital and planning departments remain, while all stages of the pipeline are outsourced to contract organizations, including externalized sales operations. The relationship between companies and their employees, as well as the question of “who owns the company,” differs significantly between the U.S., Europe, and Japan. Even so, the actions of Western pharmaceutical companies will likely influence Japan eventually.
However, when it comes to government policy, while buzzwords may appear similar, the actual substance often differs significantly. For instance, government support for TR (Translational Research) in terms of connecting academic seeds (from universities, etc.) to pharmaceutical companies appears similar. Still, there has been no substantial strengthening of national research institutions responsible for TR or regulatory science. Instead, the focus has been limited to enhancing administrative or advisory functions. Unlike the U.S.’s NCATS or Europe’s IMI, Japan lacks a centralized research organization that coordinates TR efforts. The much-publicized AMED, touted as the “Japanese NIH,” is not yet comparable to NIH in scale or function.
The essence of promoting TR lies in fostering partnerships among stakeholders with differing roles in drug development. However, in Japan, Public-Private Partnerships or Academia-Industry Partnerships often lack openness. Furthermore, in the U.S. and Europe, patient participation—including involvement from families and advocacy groups—has become a defining characteristic of the new wave of drug development. In Japan, however, the importance of such participation is rarely discussed.
Open Society Drug Development and New Entrants in Healthcare
The changes and trends discussed above are likely already being recognized by stakeholders and researchers in Japan’s pharmaceutical industry. Let us now look further ahead. One relevant example is the legislative initiative in the United States known as the 21st Century Cures Act4. This initiative originated from bipartisan committee activities in the House of Representatives aimed at accelerating translational research (TR) in the U.S. Prominent figures from the FDA and NIH were invited to participate in open discussions held over a year. The bill has been approved by the House with a majority vote. Its key provisions include increasing the FDA’s budget by $550 million over five years ($110 million annually), increasing the NIH’s budget by $8.75 billion ($1.75 billion annually), strengthening TR research, expediting the approval of new drugs and medical devices, and focusing on the recruitment and development of young researchers. While many of the proposed actions have been widely supported, the provisions aimed at accelerating the approval of drugs and medical devices, such as incorporating adaptive clinical trials into testing, have faced significant concerns and dissent. The progress of this bill warrants close attention.
In the U.S., the implementation of universal healthcare under the Obama administration has become a major policy challenge. Simultaneously, there has been a surge in new entrants into the healthcare services sector, ranging from fitness and wellness businesses to those offering insurance-covered medical services. These entrants include major players from retail, telecommunications, automotive, electronics, home appliances, ICT, and other industries. Currently, these companies are primarily focused on meeting consumer needs with convenient services that surround the core healthcare areas of medical diagnosis and treatment. However, capital- and action-driven companies have shown interest in innovating healthcare services and even venturing into drug development5.
From the perspective of drug development, the most notable entrants are global leaders in ICT, such as IBM, Apple, Microsoft, and Google. The transition of the information technology (IT) sector to the information and communication technology (ICT) sector around 1993–94 was driven by the opening of the internet to the general public. The subsequent spread of the internet was closely tied to the advancement of the World Wide Web (WWW) technology introduced around the same time. Over the past two decades, companies offering new internet-based services have emerged, rapidly growing into global giants. These companies stand out for their ability to provide revolutionary services that transform daily life, hone unique management capabilities, amass substantial capital, and boldly venture into new fields. The current reliance of ICT on the internet is analogous to the reliance of biomedical science on genome sequencing technologies (NGS). Such trends are likely to dramatically transform the drug development ecosystem within the next 10 to 20 years.
Changes in the Drug Development Environment and Big Data
As Big Pharma continues to outsource research and manufacturing while maintaining sales, management capabilities, and capital, the potential for dynamic ICT companies to succeed in entering the healthcare and pharmaceutical industries has significantly increased. These companies appear to believe that partnerships with specialized enterprises, universities, or medical institutions can fulfill their needs. Several noteworthy initiatives highlight this trend.
23andMe, which ceased direct-to-consumer services at the FDA’s recommendation, announced this year a collaboration with Genentech to study patient stratification for Parkinson’s disease, focusing on groups valuable for drug development. Similarly, it has partnered with Pfizer on inflammatory bowel disease (IBD) patient stratification.
Earlier, in 2013, Optum, a data analytics service company, and Mayo Clinic established OptumLabs, a research data analysis organization. OptumLabs aims to provide optimal solutions tailored to individual patients at the lowest possible cost by leveraging vast datasets related to medical care and reimbursement.
A typical example of such research involves high-priced cancer drugs, which continue to receive approval. The American Society of Clinical Oncology (ASCO) is undertaking a data analysis project to provide effective treatments at lower costs. The concept of “Learning Healthcare,” which aims to provide the best treatments at the lowest cost, is becoming a zeitgeist in the field of oncology (referred to as Precision Oncology). It symbolizes the end of the traditional “one-size-fits-all” drug sales model.
This shift highlights the growing importance of research into the proper use of drugs, alongside new drug development (Figure 1). Such research includes patient stratification, patient-centered evaluations of drug efficacy, studies on polypharmacy, and optimizing treatment for minimal cost. However, generalizing these concepts across other disease areas requires collecting and organizing clinical data for research purposes.

Figure 1. Future R&D in Drug Development Emphasizing Proper Use and Evaluation.
This data includes controlled data from rigorously designed clinical trials and cohort studies, as well as electronic medical records (EMRs) from everyday clinical settings, often referred to as Real World Data. Recently, there has been growing interest in leveraging personal health records (PHRs), such as health data inputted via smartphones or collected from wearable devices. These data, too, qualify as Real World Data, and more rigorously designed and controlled PHR studies are likely to emerge in the future (Figure 2).
This heightened interest in big data resonates with the zeitgeist emphasizing research on the appropriate use of drugs. It also benefits ICT companies entering the pharmaceutical industry. Regarding big data, the NIH has been driving the BD2K initiative to generate knowledge from big data and supporting platforms like ClinGen, developed by the NCBI, to analyze genome sequence variations in clinical settings. In Japan, there is a rising interest in big data within the context of translational research (TR). However, there are structural challenges, including a shortage of young talent and limited opportunities for full-time employment for these individuals.
These professionals are expected not only to handle computational data processing but also to possess biomedical knowledge and communication skills to engage deeply with experimentalists, clinicians, and other stakeholders. Relying solely on external talent may not be sufficient to address these needs.

Figure 2. Increasing Use of Real World Data in Drug Development Research. Bridging the second and third quadrants involves tertiary prevention research for chronic diseases.
The Day to Reassess the Pipeline Model
As part of our investigation into “new R&D models for drug development,” it appears that the traditional pipeline, a hallmark of the pharmaceutical industry’s work environment, has yet to undergo substantial transformation even as we enter the era of partnerships. While it is evident that parts of the pipeline are increasingly being implemented externally or through collaborations, researchers working within the framework of the pipeline may find it difficult to perceive these changes. However, in the future of drug development, the focus on studying the appropriate use of drugs after their market release will likely increase significantly. The development of new drugs will inevitably align more closely with research into their proper use (Figure 2).
Additionally, trends such as the surge of new entrants into healthcare from ICT and other industries, the rise in business partnerships, and a growing interest in upstream treatments for chronic conditions associated with aging (e.g., Calico, a company established by Google), highlight the emerging realities of the future. The day when these shifts become clearer may not be far off. The necessary perspective involves not viewing drug development from within the pharmaceutical industry but rather analyzing it through the lens of a society transformed by the second revolution of the internet (Figure 3). Within this evolving landscape, the pipeline model may increasingly come under scrutiny. Given the societal changes driven by advancements in internet technologies, it is not far-fetched to imagine that the environment in which drugs developed today will be used in 10 to 20 years could be entirely different.

Figure 3. Rethinking drug development from the perspective of future society rather than traditional R&D pipelines.
Conclusion
Whether or not we are in a crisis is debatable, but it is clear that drug development is at a major turning point. While change offers opportunities, drastic upheavals in the research environment that prevent researchers from focusing on their work can also represent a crisis for them. Without an appreciation for researchers’ fundamental ethos—their desire to bring good medicines to the world—research and development will not succeed. Unfortunately, this critical understanding often seems to be overlooked.
If this article provides “food for thought” about the near future to researchers and stakeholders in pharmaceutical companies and their solution enterprises, it will have fulfilled its purpose.
References and Information
- Nobumasa Kaminuma, supervised by Yukio Tada and Tadashi Horiuchi, “The Truth About Drug Development: From Clinical Practice to Investment” (New Edition to be released July 18th by Nikkei BP): Bartfai T and Lees GV (2006) Drug Discovery: from Bedside to Wall Street. Elsevier/Academic Press: Amsterdam.
- Nobumasa Kaminuma, supervised by Yukio Tada and Tadashi Horiuchi, “The Future of Drug Discovery: Overcoming the R&D Crisis,” Nikkei BP, 2014: Bartfai T and Lees GV (2013) The Future of Drug Discovery: who decides which diseases to treat? Elsevier/Academic Press: Amsterdam.
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ICA Seminar Series: “Exploring New R&D Models for Drug Development”
http://join-ica.org/ws/14rdseminar.html -
21st Century Cures
https://energycommerce.house.gov/ -
21st Century Pharmaceutical Collaboration: The Value Convergence
https://www.pwc.com/us - K. D. Mandl et al., “Driving Innovation in Health Systems through an Apps-Based Information Economy,” Cell Systems 1:8-13, July 29, 2015.

Tsuguchika Kaminuma
Born in Kanagawa Prefecture, Japan, in 1940. Educated at International Christian University, Yale University, and the University of Hawaii. Received a Ph.D. in Physics. Since 1971, has worked at Hitachi Information Systems Research Institute, the Tokyo Metropolitan Institute of Medical Science, and the National Institute of Health Sciences. Conducted research in pattern recognition, medical artificial intelligence, medical information systems, bioinformatics, and chemical safety. In 1981, founded an industry-government-academia research exchange organization (now CBI Society) aimed at theoretical drug design. Later engaged in interdisciplinary human resource development at Hiroshima University and Tokyo Medical and Dental University. Established the NPO Cyber Bond Research Institute in 2011.