“The 16th Annual Meeting of the Society for Regulatory Science” Held

The “16th Annual Meeting of the Society for Regulatory Science” was held on September 4–5, 2026, at the National Center of Sciences (Chiyoda Ward, Tokyo), under the theme “Regulatory Science in the Era of Digital Transformation (DX) in Healthcare.”

Introduction

The Regulatory Science Society (RS Society) serves as a platform where professionals from the clinical setting,universities and research institutions, industry, and regulatory authorities to openly discuss research findings and ideas regarding regulatory science for pharmaceuticals and medical devices, thereby promoting the advancement and dissemination of this academic field.Sixteen years after its establishment, the concept of regulatory science is becoming widely accepted.

In 2026, the conference was held over two days, September 4–5, under the theme “Regulatory Science in the Era of Healthcare DX,” featuring lively discussions across various program sessions.
This academic conference consisted of a Chairperson’s Lecture, two Special Lectures, 15 symposia, and general presentations (17 oral presentations and 46 poster presentations).

Symposium 13

The Impact of Measures to Promote Orphan and Pediatric Drug Development

Chair: Mr. Yuji Matsukura, Director, Review Management Department, Pharmaceuticals and Medical Devices Agency (PMDA)
　　　Mr. Yuji Kashitani, Chairperson, Regulatory Affairs Committee, JPMA

In Symposium 10, based on regulatory reforms following the Study Group on the Future of Pharmaceutical Regulations, the current status and challenges in the development of orphan drugs and pediatric medicines were discussed from the perspectives of government, academia, and pharmaceutical companies.

First, Mr. Mamoru Narikawa, Director of the Review Center at the Pharmaceuticals and Medical Devices Agency (PMDA), explained that the 2024 revision of the Orphan Drug Designation System has enabled the definition of medically and pharmacologically reasonable patient populations, designation in cases where existing treatments are insufficient,designation when existing treatments are insufficient, and the ability to seek consultation in the early stages of development—all of which have led to a significant increase in the number of designations. On the other hand, given that some designated drugs may not qualify for priority review even after designation, it was noted that while the predictability of the system must be enhanced, it is also necessary to continuously evaluate whether the policy is achieving its original objectives and to consider the next steps for promoting development.Regarding pediatric medicines, measures such as making the formulation of development plans a non-mandatory obligation and establishing a system that limits the re-review period to a maximum of 12 years have been put in place; however, future challenges include fostering coordination among systems, clarifying development requirements, and continuously improving the infrastructure for conducting pediatric clinical trials.

Next, Mr. Hidefumi Nakamura, Director of the Research and Development Supervision Department at the National Center for Child Health and Development, introduced support systems provided by the academic community, including the Japanese Society of Pediatrics, the Pediatric Drug Development Network, and the Pediatric Clinical Trials Network.In addition to providing medical advice, these networks support companies’ development efforts from a practical standpoint by assessing the feasibility of clinical trials, selecting trial sites, and training clinical trial coordinators with expertise in pediatrics.Furthermore, regarding pediatric cancers and rare, intractable diseases, the approach presented was to first consider participation in international collaborative clinical trials and, if conducting such trials proves difficult, to explore the use of data from sources other than clinical trials.It was pointed out that companies should not merely wait for guidelines or directives from regulatory authorities, but should incorporate research findings into their own decision-making and global development plans, and proactively propose optimal development strategies.

Next, Mr. Takao Abe, a member of the JPMA Regulatory Affairs Committee, reported that while the JPMA’s corporate survey revealed many companies were prompted to seek consultation on orphan drug designation following the revised notification, there were also cases where companies abandoned the development of imported products or the addition of new indications because they were not granted orphan drug designation.Regarding the benefits of designation as perceived by companies, compared to the results of the previous survey, there were many cases where companies cited the “Bonus for Promoting New Drug Creation and Resolving Off-Label Use” (currently: the Innovative New Drug Price Maintenance System) and the “Marketability Bonus” as advantages.Seventy-three percent of products that met the designation requirements were deemed ineligible for priority review, with “medical necessity” cited as the reason in nearly all cases. Although the system revisions are encouraging companies to seek designation consultations, the need was highlighted to further enhance the predictability of the positioning and judgment criteria for priority review from the perspectives of promoting development and ensuring patient access.

Furthermore, Committee Member Chikako Yagi of the JPMA Regulatory Affairs Committee reported that, according to the JPMA’s corporate survey, development is proceeding in Japan for approximately 70 percent of projects undergoing pediatric development in Europe and the United States, and more than 70 percent of projects are considering domestic development at the same time as in Europe and the United States.The report indicated that pediatric drug development in Japan is shifting from merely following the lead of Europe and the U.S. to early participation in international joint clinical trials.

During the panel discussion, topics such as priority review for orphan-designated products, the rationale for treating pediatric development plans as a “best-effort obligation” rather than a “mandatory obligation,” and the international harmonization of pediatric dosage forms and strengths were discussed.It was explained that the “best-efforts obligation” is a system designed to encourage pediatric development while avoiding situations where development in Japan—including that of medications for adults—is abandoned due to increased burdens on companies.Furthermore, while acknowledging the increase in the number of orphan drug designations and constraints on review resources, participants shared the view that it is necessary to continue examining the appropriate approach to priority review in light of the system’s fundamental purpose: to deliver necessary medicines to patients as quickly as possible.

Symposium 13

Prospects and Challenges for Improving Review Reports

Chair: Yuji Kashitani, Chairperson of the JPMA Regulatory Affairs Committee

In Symposium 13, discussions focused on the future direction of review reports and challenges for their improvement, from the perspectives of the PMDA—which prepares the reports—and academia and pharmaceutical companies, which utilize them.

To begin, Mr. Yuji Matsukura, Director of the PMDA’s Review Management Department, delivered a presentation titled “The Role and Expectations of Review Reports: From the Perspective of a Regulatory Authority.” He explained that review reports are important documents that record the scientific evaluation and review process regarding the quality, efficacy, and safety of pharmaceuticals. He noted that, in addition to ensuring administrative accountability and transparency, these reports are widely utilized in pharmaceutical development,clinical settings, and overseas regulatory authorities. He also explained the future direction of review reports and review operations—including the use of artificial intelligence (AI)—amid the need to balance review quality and efficiency against the backdrop of increasing application and approval volumes.

Next, Mr. Ryuta Asada of Nagoya City University Hospital gave a presentation titled“Utilization of Review Reports and Related Documents for New Drugs in Academia and Associated Challenges.” He explained that review reports serve as a useful source of information for the development of drugs for rare diseases and the design of clinical trials. As one example of how to utilize review reports, he presented the findings of an investigation into an approved drug for which a clinical trial had yielded negative results.He further noted that review reports are crucial documents for understanding the rationale behind approval decisions and key considerations in drug development, and highlighted their potential for use in drug development within academia.

Next, Mr. Yasutsugu Nakano, Chair of the Regulatory Affairs Applications Subcommittee of the JPMA Regulatory Affairs Committee, delivered a presentation titled “A Collaborative Approach to Improving Review Reports and Practical Issues.” He explained that review reports serve as a vital information foundation for communicating the process and rationale behind approval decisions to the public, while also highlighting challenges such as clarity,referencability, and the effective communication of the background and clinical significance of review decisions. He also highlighted the importance of balancing user needs with the practical requirements of drafting and publishing these reports, and proposed the need for a co-creative approach in which stakeholders—including the PMDA, pharmaceutical companies, academia, and the healthcare sector—work together through dialogue to implement improvements in a phased manner.

Furthermore, Mr. Toru Suzuki, a member of the JPMA Regulatory Affairs Committee’s Application and Regulatory Affairs Subcommittee, presented on “Prospects and Challenges for Better Review Reports: Based on the Results of the FY2026 Survey.” Drawing on the results of a survey of JPMA member companies, he outlined expectations for improving the searchability and usability of review reports.While many positive opinions were expressed regarding the use of PDF bookmark functions, the addition of summaries, and the enhancement of benefit-risk assessment items, the need to consider the burden of preparation and operational aspects was also pointed out.Furthermore, the importance of continuing discussions—while balancing user needs with practical constraints—was emphasized in order to create review reports that convey the background and rationale for approval decisions in a more understandable manner.

During the panel discussion, numerous questions were raised from the audience, including the appropriate level of applicant involvement in the preparation of review reports and the possibility of disclosing review reports for rejected products.Regarding the industry’s efforts to enhance the description of benefit-risk assessments and innovation, Mr. Matsukura explained that such descriptions are already being provided appropriately to the extent possible under current conditions. He emphasized the importance of deepening the discussion by first sharing a common understanding of the challenges—based on specific examples—that the industry identifies as issues.In addition, the idea of holding joint study sessions between the PMDA and the industry to exchange views on the ideal format of review reports at the practitioner level was proposed, and a lively exchange of opinions took place regarding how to proceed with future discussions aimed at producing better review reports.

General Presentations <Posters>

During the poster session, the JPMA Regulatory Affairs Committee presented on the following three topics.

P-12　Kazumi Kawabata, Yuki Nishioka (Regulatory Affairs Subcommittee for Drug Applications, Regulatory Affairs Committee, Japan Pharmaceutical Manufacturers Association)

Current Status of Development Projects at Member Companies of the Japan Pharmaceutical Manufacturers Association Regulatory Affairs Committee: Insights from Global Development Activities

P-16 Zenichi Yamamoto (Application and Regulatory Affairs Subcommittee, Regulatory Affairs Committee, Japan Pharmaceutical Manufacturers Association)

2026 Survey on the Review Status of New Drugs and New Regenerative Medicine Products

P-33 Takayoshi Yamaguchi (Application Affairs Subcommittee, Regulatory Affairs Committee, Japan Pharmaceutical Manufacturers Association)

“Pre-disease” from the Perspective of Pharmaceutical Companies—Possibilities for a New Approach Aimed at Extending Healthy Life Expectancy—

The next “17th Annual Meeting of the Society for Regulatory Science” is scheduled to be held on September 3–4, 2027.

(Regulatory Affairs Committee: Tadashi Sugihara, Yuji Ishihara, Takayoshi Yamaguchi, Zenichi Yamamoto, Sayaka Iiyama, Yutaka Takeuchi)

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