Pharmaceutical Medicine最新文献

Digital psychiatry tools are increasingly woven into routine care, not only to support pharmacotherapy but also to deliver psychotherapy, sustain engagement and redesign clinical workflows. This narrative review synthesises synchronous (e.g. telepsychiatry and live chat) and asynchronous (e.g. apps, remote monitoring and digital therapeutics/software as a medical device ["SaMD"]) approaches that initiate, monitor or augment medication treatment and commonly paired psychosocial interventions. We outline how these tools can improve adherence, side-effect surveillance, psychoeducation and therapy access while highlighting persistent challenges around evidence strength, workflow integration, equity, reimbursement and regulation. Drawing on emerging hybrid models, such as digital navigators and the digital clinic model, we present pragmatic pathways for safe adoption, data triage and sustained use in real-world settings. We also discuss opportunities for artificial intelligence (AI)-driven personalisation and responsive monitoring, alongside the ethical and implementation considerations they raise. By broadening the lens beyond pharmacotherapy alone, this review provides clinicians and health systems with actionable guidance on selecting, integrating and evaluating digital tools to deliver patient-centred, scalable psychiatric care.

Background: Duchenne muscular dystrophy (DMD) is a rare hereditary neuromuscular disorder caused by mutations in the dystrophin gene, leading to progressive muscle deterioration, loss of ambulation, and reduced life expectancy. The US Food and Drug Administration (FDA) has approved several novel drugs for DMD; however, their utilization, reimbursement, and cost patterns within US Medicaid programs remain unexamined.

Objectives: The purpose of this study was to evaluate the utilization, reimbursement, and cost trends of targeted therapies for DMD in the US Medicaid population from 2017 to 2022.

Methods: A retrospective descriptive drug utilization study was conducted using the national Medicaid drug utilization files that were extracted from the Centers for Medicare and Medicaid Services (CMS) from 2017 to 2022. The study drugs comprised the following four FDA-approved drugs: eteplirsen (Exondys 51^®), golodirsen (Vyondys 53^®), viltolarsen (Viltepso^®), and casimersen (Amondys 45^®). The annual number of prescriptions represented drug utilization and the annual amount of reimbursement represented the total spending, which were calculated for time-series secular trend analyses. The average cost per prescription was also computed as an estimate of drug cost trends.

Results: Over the 6-year period, the total count of DMD prescriptions increased significantly by 2988%, from 643 prescriptions in 2017, when only eteplirsen (Exondys 51^®) was available, to a peak of 19,855 prescriptions in 2022, including all four novel DMD drugs. Additionally, the overall reimbursement grew by nearly 2809%, increasing from US$22,027,999 in 2017 to US$640,890,515 in 2022. However, the average cost per prescription decreased by about 6%, from US$34,258 in 2017 to US$32,279 in 2022.

Conclusion: The considerable rise in the utilization and spending of novel DMD drugs has imposed a significant burden on the Medicaid budget, underlining the need for policy measures to manage rising costs and maintain equal access to treatment.

BACKGROUND AND OBJECTIVES: Additional risk minimization measures (aRMMs), such as direct healthcare professional communication (DHPC) and risk management plan (RMP) educational materials, are required for certain medicinal products to mitigate the risks associated with their use. This research aimed to assess healthcare professionals' (HCPs) awareness and experiences of DHPC and RMP educational materials, identify the safety information most valued by HCPs, and determine ways to improve the processes and content of DHPCs and RMP educational materials.

Methods: An open, anonymous online questionnaire was created and made available to HCPs through their respective professional associations. The Chi-squared test for independence was the primary statistical analysis method used. Content analysis of the open-ended answers was conducted by two independent coders.

Results: A sample size of 185 HCPs (32 physicians, 50 nurses, 26 MSc pharmacists, and 77 BSc pharmacists) was achieved. HCPs showed limited familiarity with aRMMs, with 45% unsure whether they had received DHPCs, 41% uncertain whether they had used RMP educational materials, and 42% unaware where to check whether a product had RMP educational material. Overall, MSc pharmacists demonstrated the highest awareness of RMP educational materials and DHPCs. In addition, 54% of HCPs felt they received insufficient safety information, and 57% desired further training in pharmacovigilance (PhV).

Conclusions: This research reinforces and complements our previous findings on the need to enhance PhV awareness and expertise among HCPs, particularly in areas such as adverse drug reaction (ADR) reporting, additional monitoring, DHPCs, and RMP educational material. Future DHPCs and RMP educational materials should be made more distinguishable to effectively capture HCPs' attention.

Tissue-agnostic therapies, which target molecular alterations across cancer types, have been approved by the US Food and Drug Administration (FDA) since 2017. These approvals were based on clinical trials enrolling patients across diverse tumor types, regardless of the tumor's site of origin. This analysis evaluated the efficacy and safety of FDA-approved tissue-agnostic therapies in patients with lung cancer, the leading cause of cancer-related deaths worldwide, harboring targetable genetic alterations. Current evidence suggests that these therapies show promise in treating NTRK gene fusions (larotrectinib, entrectinib, repotrectinib), BRAF V600E mutation (dabrafenib and trametinib), and RET fusions (selpercatinib) in lung cancer. Reported rates of grade 3 or higher treatment-related or treatment-emergent adverse events ranged from 10 to 59.7%. However, available data are limited by small sample sizes (ranging from 4 to 247 participants per trial-cohort) and the absence of control groups. Larger, controlled studies and real-world evaluations are needed to confirm these findings and inform broader clinical use.

Exosomes, small extracellular vesicles (sEVs) that range in Size from 30 to 150 nm in diameter, have emerged as crucial mediators of intercellular communication within the central nervous system (CNS). They play significant roles in the pathogenesis and progression of various neurological and psychiatric disorders, including Alzheimer's disease, Parkinson's disease, ischemic stroke, depression, bipolar disorder, and autism spectrum disorder. Exosomes carry a diverse cargo of proteins, nucleic acids, lipids, and other bioactive molecules that can influence neuronal function and synaptic plasticity. In disease states, exosomes derived from stressed neurons or glial cells can propagate neuroinflammation, synaptic dysfunction, and cognitive decline. They may also mediate the spread of abnormal proteins or microRNAs, disrupting neuronal connectivity and neurotransmitter signaling and contributing to the development of proteinopathies and neurotoxicity. Owing to their presence in bodily fluids such as blood plasma, cerebrospinal fluid, and saliva, exosomes hold promise as biomarkers for these disorders. Moreover, their regulatory roles present new opportunities for developing novel diagnostic biomarkers and therapeutic interventions. This review provides an overview of the multifaceted roles of exosomes in neurological and psychiatric disorders. We delve into their contributions to disease pathogenesis, their potential as diagnostic biomarkers, and the innovative therapeutic strategies leveraging exosome-based delivery systems. By exploring the current state of research, we aim to highlight the translational potential of exosomes in revolutionizing the diagnosis and treatment of these disorders.

Background and objective: Regulatory agencies and policy makers increasingly recognize real-world evidence (RWE) as a valuable complement to randomized controlled trials (RCTs) in oncology, yet data on how US physicians who treat cancer use and perceive RWE remain limited. The study aimed to assess attitudes toward RWE among US physicians who treat cancer, including their confidence in interpreting it and reliance across clinical decision contexts.

Methods: A cross-sectional national survey was administered in November 2024 to licensed US physicians who treat cancer, recruited from the American Society of Clinical Oncology (ASCO) member directory, using a random sample stratified by state population. Inclusion criteria were active US medical licensure and current involvement in oncology patient care. The survey instrument included sections on demographics and practice characteristics; RWE familiarity and usage frequency; comparative reliance on RWE versus RCTs in treatment selection, dosing, and outcome prediction (scales ranging from 0 to 10: 0 = complete reliance on RCT data, 10 = complete reliance on RWE); perceived barriers to adoption (4-point scale); and potential facilitators (4-point scale). Categorical data were summarized as counts and percentages, and continuous variables were summarized as means and standard deviations (SD). Chi-squared tests were used to compare categorical variables across groups, paired t tests were used to assess differences in mean reliance scores, and Spearman's rho was used to evaluate correlations. Statistical significance was set at p < 0.05.

Results: In total, 128 completed surveys were received. Overall, 94% of respondents (n = 120) were at least "somewhat familiar" with RWE, 14% (n = 18) used it "often," and 3% (n = 4) reported daily use. 49% (n = 63) felt confident interpreting RWE studies, with late-career physicians (> 20 years of experience) less confident than their early and mid-career peers. Reliance on RWE was lower for treatment selection (mean 3.0, SD 1.7) than for dosing (mean 3.7, SD 2.0) or outcome prediction (mean 3.8, SD 2.0) (p < 0.001). Top barriers included reconciling conflicting RWE versus RCT data, data completeness, and bias. Key facilitators included improved analytical standards, guideline integration, and additional training.

Conclusions: While awareness of RWE is high among US physicians who treat cancer, they apply it selectively on the basis of clinical context, showing notably lower reliance for treatment selection. Addressing concerns about methodological rigor, data quality, and interpretive skills may strengthen RWE's integration into oncology care.

Background and objective: Since the approval of the first biosimilar monoclonal antibody (mAb) in 2014, 18 biosimilar mAbs have been approved in Japan as of December 2023. These biosimilar mAbs are typically developed using data that exhibit comparability with their reference products (RPs) in terms of quality, pharmacokinetics, safety, and efficacy. Although comparative efficacy studies are not necessarily required under Japanese biosimilar Guidelines, such studies have been conducted for all 18 approved biosimilar mAbs. Meanwhile, there is growing global interest in reevaluating the absolute necessity of these studies. The objective of this original research article was to analyze the data packages of biosimilar mAbs using publicly available review reports from 2014 to 2023 to assess the role of comparative efficacy studies.

Methods: First, we identified quality attributes (QAs) that differed between each biosimilar mAb and its RP on the basis of publicly available review reports from the Pharmaceuticals and Medical Devices Agency (PMDA) website. Subsequently, we examined the evidence used to justify these differences.

Results: The results showed that 64% of QA differences were justified using data from other QAs. Conversely, only 6% of QA differences were justified through findings from comparative efficacy studies.

Conclusions: These findings indicate that comparative efficacy studies play a limited role in establishing comparability between biosimilars and their RPs. Therefore, sponsors may consider whether a comparative efficacy study is necessary to determine if differences in comparative quality studies are clinically meaningful, rather than automatically conducting such studies.