Background　The digitization of clinical trial documents has been promoted in Japan. The Clinical Trial Document Support System (CtDoS2) was widely used for electronic document management but has been discontinued in March 2023. This study aimed to assess the changes in the usage rate of electronic systems, their strengths, and weaknesses before (February 2023) and after (February 2024) the discontinuation of CtDoS2.

Method　We conducted a questionnaire survey to examine the status of the introduction of electronic systems and the status of their contracts with CtDoS2.The survey was conducted among clinical trial office staff at PMDA IRB-registered institutions as of November 30, 2023.

Results　A questionnaire survey was performed among 1244 clinical trial offices, and the response rate was 30.1%. The usage rate of electronic systems increased from 15.2% in February 2023 to 25.3% in February 2024. The usage rate was higher in facilities that had contracts with CtDoS2 compared to those without. Facilities that introduced electronic systems reported advantages such as reduced paper usage and decreased work hours, and their satisfaction levels were high. Both the dissatisfaction among facilities that introduced electronic systems and the reasons for non-introduction among facilities that have not introduced electronic systems included cost issues and the burden of creating regulations and manuals.

Background　Despite the international recognition of task‑based cost estimation using the Fair Market Value, its implementation in Japan remains limited, and its benefits and challenges remain mostly unelucidated. This study aimed to clarify the implementation status, advantages, and challenges of task‑based cost estimation using Fair Market Value in Japanese medical institutions.

Methods　A questionnaire survey was conducted in 154 institutions that had experience with this ap-proach, and a total of 63 institutions (33 clinics and 30 hospitals) were included in the analysis.

Results　The results indicated that task‑based cost estimation based on the Fair Market Value had been introduced primarily as a test case. The most common trigger for its implementation was a request from sponsors. Although improved transparency and greater acceptability were an-ticipated as potential benefits, the most frequent response in the survey was that the effects remained unclear due to the recent introduction of the system. The most commonly cited challenge was the increase in operational burden. Additional issues included the need for cost‑sharing adjustments with Site Management Organizations, concerns regarding revenue reduction, and cost allocation among internal departments. Furthermore, in clinics, difficulties in negotiations with sponsors were reported.

Conclusion　The widespread adoption of task‑based cost estimation using the Fair Market Value would require substantial contributions from sponsors; however, it is crucial that medical institutions take an active, independent role in developing the requisite institutional infrastructure.

Objectives　Designs of phase I oncology trials are generally classified into 3 classes: rule-based, model-assisted, and model-based designs. 3+3 design, Bayesian Optimal Interval design, and Bayesian Logistic Regression Model design are representative designs from each class. In Japan, Pharmaceuticals and Medical Devices Agency has released a “Check List for 30-day-Clinical Trial Notification Review on an Initial Clinical Trial Notification (Oncology Drugs)” for 30-day-CTN Review of oncology drugs and an “Early Consideration” specifying points to consider particularly with a focus on safety when evaluating operating characteristics of a dose-escalation trial based on statistical considerations. The performance of the adopted design should be evaluated by simulation according to these notifications. Corresponding R packages and software such as Trial Design are useful, but study-specific changes, such as application of single patient acceleration (SPA) at low doses, may also be considered. In such cases, tools to apply such modifications more efficiently are desirable considering development timelines and technical complexity. Therefore, we develop an application tool using Shiny to evaluate the operating characteristics of each class of designs incorporating SPA.

Conclusion　The developed tool is capable of calculating evaluation metrics required in the Early Consideration, enabling efficient and effective consideration of designs and negotiations with regulatory authorities. The R script download function is employed, ensuring reproducibility of results. The tool is not intended to have all of the expected functionality. Users are advised to download the R script and make additional modifications (e.g., data generation, discontinuation criteria, and so on) as needed when study-specific changes are to be incorporated.

Background Post-marketing surveillance (PMS) in Japan is conducted under the Good Post-marketing Study Practice (GPSP) ordinance, which does not specify requirements for informed consent (IC) or ethical review. As PMS increasingly involves data collection beyond routine clinical practice and wider dissemination of results, ethical considerations have become more important. This study aimed to compare perceptions of IC, ethical review, survey content, and survey-related cost between hospitals and pharmaceutical companies.

Methods A nationwide anonymous self-administered questionnaire survey was distributed to 1,270 hospitals and 89 pharmaceutical companies.

Results Responses from 188 hospitals and 64 companies were analyzed. IC was considered mandatory by 14.9% of hospitals and 40.6% of companies, with significantly higher recognition among foreign-affiliated companies (p<0.05). Ethical review was conducted or requested for all PMS in 59.6% of hospitals but only 10.9% of companies (p<0.05). Survey content concerns were reported by 56.4% of hospitals, and 60.6% of hospitals and 45.3% of companies experienced survey content beyond routine clinical practice. Regarding costs, 53.2% of hospitals considered payments insufficient, often due to CRC involvement and survey content beyond routine care.

Conclusions Although IC and ethical review are partially implemented in PMS at certain institutions, the lack of uniform regulatory requirements leads to considerable variability and may result in insufficient ethical oversight. When surveillance includes procedures beyond routine care or research-oriented use of data, ethical frameworks comparable to clinical research and alignment with global standards are warranted. Multistakeholder collaboration is needed to establish standardized rules for IC and ethical review.

The incorporation of patient-reported outcomes (PROs) into clinical research has gained increasing attention in parallel with the global shift toward patient-focused drug development. PROs are essential for evaluating outcomes that are subjective in nature and not adequately captured by clinician-reported assessments or objective clinical indicators, such as symptoms, treatment-related adverse events, health-related quality of life, and patient experience. When collected using well-validated patient-reported outcome measures (PROMs) within appropriately designed studies, PRO data may inform benefit-risk assessment and support regulatory decision-making.

The scientific and ethical use of PROs requires methodological rigor across all stages of clinical research, including study planning, outcome selection, data collection and management, statistical analysis, interpretation, and reporting. Over the past three decades, international regulatory authorities, academic societies, and expert consortia have developed a comprehensive set of PRO-related guidelines. These include guidance for protocol development (SPIRIT-PRO), conceptual frameworks for outcome selection (ISPOR Clinical Outcome Assessment framework), standards for evaluating the measurement properties of PROMs (COSMIN guidelines), ethical guidance for PRO use, recommendations for electronic PRO implementation and data management, statistical analysis recommendation (SISAQOL), and reporting guidance (CONSORT-PRO).

This short report summarizes the current landscape of PRO-related guidelines and highlights recent developments in Japan. The dissemination and practical implementation of these guidelines in the Japanese clinical research context are critical to ensuring high-quality, ethically sound, and patient-centered use of PROs in both clinical trials.

Background In recent years, efforts to incorporate patient perspectives into clinical trial design have advanced, aiming to enhance the overall trial experience. Patient experience data (PED), which captures patients’ daily health conditions, their treatments and care, and the impact of these factors on their lives, has gained increasing attention. Effective use of PED requires continuous patient engagement; however, challenges such as compliance, confidentiality, and ensuring psychological safety remain significant barriers. The use of metaverse technology has been considered a potential approach to address these issues.

Methods To collect patient perspectives on clinical trial participation, a focus group (patient advisory board) was conducted on a metaverse platform, guided by a semi-structured interview. Participants engaged in open discussions using anonymous avatars, and their verbal interactions were transcribed and analyzed.

Results The focus group was held in December 2024 and included six participants with prior clinical trial experience. The metaverse platform was positively evaluated for its immersive experience, anonymity, psychological safety, and freedom of expression. Conversely, participants pointed out some challenges.

Discussion As the importance of patient involvement in drug development continues to grow globally, there is increasing interest in exploring digital technologies as new methods for collecting and using PED. Areas for improvement in the use of metaverse platforms include enhancing user comprehension, improving psychological safety, facilitating emotional expression, and reducing visual strain. Future research should also focus on quantitatively evaluating the benefit of metaverse to establish more robust mechanisms for patient engagement.

Background With constantly ongoing cancer therapy development, clinical trial participation has emerged as an important treatment option, but geographical barriers continue to create disparities in access to participation. Decentralized clinical trials (DCTs) offer the potential to improve clinical trial access, despite remaining challenges in their implementation.

Objectives We used anonymous surveys to evaluate patient and healthcare professional awareness of DCTs and their willingness to use them.

Methods Three surveys were conducted: (1) a questionnaire survey provided to cancer patients in non-urban areas regarding telemedicine and DCTs (IR051015), (2) a survey provided to patients in urban areas and healthcare professionals belonging to the Japanese Society of Medical Oncology (IR051015), and (3) a web-based survey provided to physicians and trial coordinators involved in cancer genomic medicine.

Results Survey 1 revealed low overall awareness: telemedicine (15%), DCTs (10%), eConsent (4%), and D to P with D (4%) and high willingness to participate: telemedicine (73%), DCTs (64%), and eConsent (61%). In Survey 2, 84% and 71% of healthcare professionals considered online follow-up visits and D-to-P with D feasible, respectively. The respective corresponding proportions among patients were 44% and 56%. Survey 3 revealed a travel time of ≥2 hours to be the most common barrier to referral to clinical trials (56%).

Conclusions Despite patients’ willingness to participate in DCTs, preferences for face-to-face care persist. Although DCTs may improve trial access, patient-centered implementation remains warranted.