Pharmaceutical Medicine最新文献

The author argues that notwithstanding available guidelines and established practices, the elaboration of a formal ethics framework specific to medical affairs could improve good practice internationally. He further argues that further and better insights into the theory behind the practice of medical affairs are an essential precondition for elaborating any such framework.

Background: An individual's personal values strongly influence their immediate and long-term decisions. Psychological heterogeneity in clinical trial populations contributes to selection bias and may affect treatment outcomes and inevitably trial results.

Objectives: The objective of this study was to characterize for the first time the main interpersonal values of patients who participated in Phase II and III clinical trials.

Methods: This multicenter observational study included 200 participants from 4 different hospitals who participated in a Phase II or III clinical trial. Patients from different therapeutic areas were included in this study. The patients' interpersonal values were studied using the Survey of Interpersonal Values (SIV). The SIV scale is grouped into six subscales that assess specific personal values: (1) support, the need to be treated with kindness and to receive encouragement from other people; (2) conformity, the extent to which one does what is acceptable and considered socially correct; (3) recognition, the need to be highly regarded and admired, to be considered important and recognized by others; (4) independence, the extent to which individuals feel free to make their own decisions; (5) benevolence, the capacity to understand and show generosity towards the less fortunate; and (6) leadership, the value ascribed to coordinating the work of others, being selected for a leadership position, and being in a position to tell others what to do. The results obtained from the patient population were classified using the following categories: "very high" (P95-P99), "high" (P70-90), "medium" (P35-65) low" (P10-30), or "very low" (P1-5), and subsequently compared with those of the Portuguese normative population.

Results: Compared with the normative population, regardless of the patient's underlying disease, the percentile frequency distributions were significantly higher for the independence (p < 0.001) and benevolence (p < 0.001) subscales, and significantly lower for the leadership (p < 0.001) and recognition (p < 0.001) subscales in the patient population. Patient distribution according to underlying disease differed significantly relative differences in distribution relative to the normative population for the majority of subscales. Non-alcoholic steatohepatitis (NASH), heart failure, myocardial infarction, lung cancer, and rheumatoid arthritis patients were those for which the greatest differences were observed across diseases, while stroke, multiple sclerosis, and HIV patients showed the least differences relative to the normative population.

Conclusions: This novel analysis of the interpersonal values of patients that participate in Phase II and III clinical trials revealed that the patients' interpersonal values largely differed from those of the Portuguese normative population. Better understanding the implica

What is patient-centricity? In some contexts, it has been associated with targeting therapies based on biomarkers or enabling healthcare access. There has been a surge in patient-centricity publications, and in many cases for the biopharmaceutical industry, patient engagement is used to endorse pre-held assumptions at a specific moment in time. Rarely is patient engagement used to drive business decisions. Here we describe an innovative partnership between Alexion, AstraZeneca Rare Disease and patients that allowed a deeper understanding of the biopharmaceutical stakeholder ecosystem and an empathic understanding of each patient's and caregiver's lived experience. Alexion's decision to build patient-centricity frameworks resulted in the formation of two unique organisation design platforms: STAR (Solutions To Accelerate Results for patients) and LEAP (Learn, Evolve, Activate and deliver for Patients) Immersive Simulations. These interconnected programmes required cultural, global, and organisational shifts. STAR generates global patient insights that are embedded in drug candidate and product strategies while helping to establish enterprise foundational alignment and external stakeholder engagement plans. LEAP Immersive Simulations produce detailed country-level patient and stakeholder insights that contribute to an empathetic understanding of each patient's lived experience, support country medicine launches and provide ideas to have a positive impact along the patient journey. Combined, they deliver integrated, cross-functional insights, patient-centric decision making, an aligned patient journey, and 360° stakeholder activation. Throughout these processes, the patient is empowered to dictate their needs and validate the proposed solutions. This is not a patient engagement survey. This is a partnership where the patient co-authors strategies and solutions.

Background: West African Economic and Monetary Union (UEMOA) countries are characterised by a high prevalence of informal use of medicinal plants and traditional medicines by their population for health care, requiring the establishment of pharmacovigilance, in order to monitor the associated health risks. However, the state of implementation of pharmacovigilance for traditional medicines in UEMOA countries is not known.

Objective: This study aimed to assess the state of implementation of pharmacovigilance for traditional medicines in the eight UEMOA countries, describing the relevant community provisions, assessing the integration of traditional medicines monitoring into national pharmacovigilance systems and identifying related national challenges.

Methods: This was a cross-sectional study using questionnaires, conducted between 1 May and 31 August 2022. A face-to-face questionnaire was administered to officials responsible for the issue within UEMOA and the West African Health Organisation (WAHO). A second online questionnaire was specifically sent to the pharmacovigilance focal points of the eight UEMOA countries. Questionnaires were designed using the WHO indicators for pharmacovigilance. The face-to-face questionnaire collected two types of data, namely community policies and regulations on pharmacovigilance and technical and financial support from sub-regional organisations to countries. The online questionnaire sent to countries collected four categories of data on the study issue: structural data, process data, impact data and data on national challenges.

Results: As a community provision, WAHO has a harmonised regulatory framework for phytovigilance. The monitoring of traditional medicines is not effectively implemented in the pharmacovigilance systems of UEMOA countries. Only two reports of adverse events due to traditional medicines have so far been recorded in the Union. The countries have neither funding nor sufficient human resources for pharmacovigilance in general. Monitoring of traditional medicines in the unregulated market, training of stakeholders, risk communication, and integration of traditional health practitioners in reporting systems are the main challenges of countries for the development of pharmacovigilance for traditional medicines.

Conclusion: The effective compliance of WAHO's harmonised phytovigilance regulatory framework by UEMOA countries and addressing the challenges identified by the countries constitute the basis for the development of pharmacovigilance for traditional medicines within UEMOA.

Background: The Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) is a new validated 14-item patient-reported outcome (PRO) instrument for evaluating daytime functioning in people with insomnia. It comprises three domains: Alert/Cognition, Mood, and Sleepiness.

Objective: The aim of this analysis was to estimate the minimum within-patient change for IDSIQ scores that an adult patient with insomnia would consider meaningful.

Methods: Data were from a randomized, double-blind, placebo-controlled, phase III clinical trial of daridorexant in adults with insomnia. Subjects completed the IDSIQ daily in the evening, with a recall period of 'today', throughout the 3-month double-blind treatment period. Scores were calculated as a weekly average. Each IDSIQ item was scored on an 11-point numeric rating scale ranging from 0 (not at all/none at all) to 10 (very/a lot), with a higher score indicating a greater severity or impact. PRO measures with correlation coefficients ≥0.30 were included in a subsequent anchor-based analysis. For the IDSIQ total score and each IDSIQ domain, meaningful within-patient change was estimated as the minimum score change patients would consider meaningful in an anchor-based analysis using data from PRO instruments capturing daytime and night-time insomnia symptoms (the Insomnia Severity Index [four items, each scored 0-4, with a higher score indicating greater symptom severity; assessed at screening, baseline, month 1 and month 3], Patient Global Assessment of Disease Severity [6-point scale from 'none' to 'very severe'; assessed weekly], Patient Global Impression of Severity [4-point scale from 'none' to 'severe'; assessed weekly], and Patient Global Impression of Change [7-point scale from 'very much better' to 'very much worse'; assessed weekly for night-time and daytime symptoms separately]). A supplemental distribution-based analysis was also conducted to support the anchor-based analysis.

Results: The analysis included 930 subjects aged 18-88 years. Spearman correlation coefficients for the relationships between score changes/ratings for anchors and the IDSIQ (0.36-0.44 at month 1, 0.45-0.57 at month 3) were all above the prespecified threshold of 0.30. Mean IDSIQ score changes at months 1 and 3 based on the different anchors supported meaningful within-patient change estimates starting at 17 points for the IDSIQ total score, 9 points for the Alert/Cognition domain, and 4 points for the Mood and Sleepiness domains.

Conclusion: This analysis demonstrates the meaningful within-patient change for the IDSIQ total score and domain scores, that the instrument is sensitive to changes in the patient experience of insomnia, and that it can be used in clinical trials to evaluate changes in daytime functioning.

Clinical trials registration: NCT03545191 (4 June 2018).

Drugs that have been manufactured or packaged fraudulently are referred to as counterfeit/fake/spurious/falsified drugs because they either lack active ingredients or have the incorrect dosages. Counterfeiting of drugs has become a global issue with which the whole world is grappling. The World Health Organization states the frightening figure in which almost 10.5% of the medications worldwide are either subpar or fake. Although developing and low-income countries are the targets of the large-scale drug counterfeiting activities, fake/substandard drugs are also making their way into developed nations including the USA, Canada, and European countries. Counterfeiting of drugs is leading to not only economic loss but is also playing its part in the morbidity and mortality of patients. The recent COVID-19 pandemic fuelled the demand for certain categories of medicines such as antipyretics, remdesivir, corticosteroids, vaccines, etc., thus increasing the demand and manufacture of subpar/fake medicines. This review articulates the current trends and global impact of drug counterfeiting, current and potential measures for its prevention and the role of different stakeholders in tackling the menace of drug counterfeiting.

Antiseizure medications can cause serious adverse reactions and have deleterious drug interactions that often complicate the clinical management of patients. When the US Food and Drug Administration (FDA) wants to alert healthcare providers and patients about the risk of potentially serious or fatal drug reactions, the FDA requires the manufacturers of these medications to format these warnings within a "black-box" border, and prominently display this box on the first section of the package insert; such warnings are called "black-box warnings (BBWs)". The BBW is a way for the FDA to urge physicians to evaluate patients more rigorously and carefully weigh the risks and benefits, before prescribing medication that has the potential to cause serious adverse reactions, and to formulate a plan for close monitoring during therapy. The FDA BBW provides the extra layer of safety but many healthcare providers fail to comply with these warnings. Currently, there are 26 FDA-approved antiseizure medications in the US market, 38% of which have received BBWs, and most of the antiseizure medications with BBWs are older-generation drugs. Some antiseizure medications have multiple BBWs; for example, valproic acid has three BBWs including hepatotoxicity, fetal risk, and pancreatitis, carbamazepine has BBWs of serious skin and hematological reactions, and felbamate also has two BBWs including hepatic failure and aplastic anemia. The purpose of this review is to provide insight into each BBW received by antiseizure medications and discuss the FDA recommendations for evaluating the drug benefit/risk, and for monitoring parameters before the initiation of and during treatment.

Monoclonal antibodies are an effective and growing class of pharmaceuticals for the treatment and prevention of a broad range of non-communicable and infectious diseases; however, most low- and middle-income countries have limited access to these innovative products. Many factors contribute to the global inequity of access to these products; however, in this report, we focus on clinical and regulatory complexities as further highlighted by the coronavirus disease 2019 pandemic. Despite a higher prevalence of many diseases in low- and middle-income countries, only 12% of clinical trials for monoclonal antibodies are conducted in these countries. Additionally, only a fraction of the available monoclonal antibodies in the USA and European Union are authorized for use in low- and middle-income countries. Through learnings from desk research and global symposia with international partners, we present recommendations to harmonize processes and facilitate regional and international collaborations for more rapid approval of fit-for-purpose innovative monoclonal antibodies and biosimilars in low- and middle-income countries.