Potential inhibitors of a target biomolecule, NAD-dependent deacetylase Sirtuin 1, were identified by a contest-based approach, in which participants were asked to propose a prioritized list of 400 compounds from a designated compound library containing 2.5 million compounds usingin silicomethods and scoring. Our aim was to identify target enzyme inhibitors and to benchmark computer-aided drug discovery methods under the same experimental conditions. Collecting compound lists derived from various methods is advantageous for aggregating compounds with structurally diversified properties compared with the use of a single method. The inhibitory action on Sirtuin 1 of approximately half of the proposed compounds was experimentally accessed. Ultimately, seven structurally diverse compounds were identified.

Japan's conditional/time‐limited early approval program, initiated in 2014, aimed to advance regenerative medicine by expediting market access. However, the withdrawal of autologous skeletal myoblast sheets (Heartsheet) due to ineffectiveness raises concerns about the balance between rapid approval and scientific integrity. While the program seeks to boost innovation, it risks endorsing costly, unclear treatments under national health care. This case highlights the need to refine regulatory approaches, ensuring clinical efficacy and fiscal responsibility in regenerative therapies.

Regulatory authorities must balance ensuring evidence of efficacy and safety of new drugs. Various regulatory pathways, such as the accelerated approval program in the United States (US), allow authorities to quickly approve drugs for severely ill patients by granting market authorization based on surrogate end points and pending confirmatory trials. In this cross‐sectional study, we considered 23 indications of cancer drugs that received accelerated approval by the US Food and Drug Administration (FDA) but were subsequently withdrawn as of April 2023. Our investigation extended to assessing the regulatory status of these accelerated approvals in the European Union (EU) and Japan, examining relevant regulatory documents and identifying factors contributing to the withdrawal in the United States. Comparing regions, we found that for 52% (12/23) and 30% (7/23) of withdrawn accelerated approvals in the United States, sponsors had also sought marketing authorization from the European Medicines Agency (EMA) and Japan's Pharmaceuticals and Medical Devices Agency (PMDA), respectively. As of the April 30, 2023 study cutoff date, 83% (10/12) of drug–indication pairs remained approved by the EMA, while the PMDA retained 100% (7/7). For these indications, the time from FDA withdrawal until the study cutoff date ranged from 0.23 years to 11.45 years for EMA approvals (median: 1.28 years) and 1.10 years to 11.45 years for PMDA approvals (median: 3.22 years). These findings highlight substantial regulatory discrepancies concerning cancer drugs with unconfirmed benefits. Addressing these discrepancies may involve requiring pharmaceutical companies to confirm clinical benefits using more robust end points and fostering international harmonization in regulators' assessment.

Background: Nonmedical use of prescription drugs can cause overdose; this represents a serious public health crisis globally. In this digital era, social networking services serve as viable platforms for illegal acquisition of excessive amounts of medications, including prescription medications. In Japan, such illegal drug transactions have been conducted through popular flea market applications, social media, and auction websites, with most of the trades being over-the-counter (OTC) medications. Recently, an emerging unique black market, where individuals trade prescription medications—predominantly nervous system drugs—using a specific keyword (“Okusuri Mogu Mogu”), has emerged on X (formerly Twitter). Hence, these dynamic methods of illicit trading should routinely be monitored to encourage the appropriate use of medications. Objective: This study aimed to specify the characteristics of medications traded on X using the search term “Okusuri Mogu Mogu” and analyze individual behaviors associated with X posts, including the types of medications traded and hashtag usage. Methods: We conducted a cross-sectional study with publicly available posts on X between September 18 and October 1, 2022. Posts that included the term “Okusuri Mogu Mogu” during this period were scrutinized. Posts were categorized on the basis of their contents: buying, selling, self-administration, heads-up, and others. Among posts categorized as buying, selling, and self-administration, medication names were systematically enumerated and categorized using the Anatomical Therapeutic Chemical (ATC) classification. Additionally, hashtags in all the analyzed posts were counted and classified into 6 categories: medication name, mental disorder, self-harm, buying and selling, community formation, and others. Results: Out of 961 identified posts, 549 were included for analysis. Of these posts, 119 (21.7%) referenced self-administration, and 237 (43.2%; buying: n=67, 12.2%; selling: n=170, 31.0%) referenced transactions. Among these 237 posts, 1041 medication names were mentioned, exhibiting a >5-fold increase from the study in March 2021. Categorization based on the ATC classification predominantly revealed nervous system drugs, representing 82.1% (n=855) of the mentioned medications, consistent with the previous survey. Of note, the diversity of medications has expanded to include medications that have not been approved by the Japanese government. Interestingly, OTC medications were frequently mentioned in self-administration posts (odds ratio 23.6, 95% CI 6.93-80.15). Analysis of hashtags (n=866) revealed efforts to foster community connections among users. Conclusions: This study highlighted the escalating complexity of trading of illegal prescription medication facilitated by X posts. Regulatory measures to enhance public awareness should be considered to prevent illegal transactions, which may ultimately lead to misuse or abuse such as overdose. Along with such pharmacovigilance measures, social approaches that could direct individuals to appropriate medical or psychiatric resources would also be beneficial as our hashtag analysis shed light on the formation of a cohesive or closed community among users.