Pharmaceutical Medicine最新文献

Background: Poor adherence to glaucoma medication regimens may be associated with subsequent optic nerve damage and irreversible visual loss. Specific barriers to effective patient adherence in low-middle income countries are not fully recognized and new disease-specific instruments to assess adherence have been developed.

Objective: The purpose of this cross-sectional study was to evaluate adherence of primary open-angle glaucoma (POAG) patients to treatment in a middle-income country.

Methods: POAG patients were recruited from the Glaucoma Service - Irmandade da Santa Casa de Misericordia de Sao Paulo, Sao Paulo, Brazil. Clinical and demographic data were retrieved from participants' electronic records. All patients answered the Glaucoma Treatment Compliance Assessment Tool (GTCAT). This 27-item questionnaire was designed to evaluate multiple behavioral factors associated with glaucoma medication adherence.

Results: The sample comprised 96 patients with POAG. The mean age was 63.2 ± 8.9 years; 48 were male and 48 female; 55 (57.3%) were White, 36 (37.5%) African-Brazilian, and five (5.2 %) were of mixed color. Most patients (97.9%) had less than a high school degree and all had a family income < US$10,000. The GTCAT identified 69 (71.8%) patients who "sometimes forget to use drops," 68 (70.8%) patients who "sometimes fall asleep before dosing time," and 60 (62.5%) patients "whose drops aren't with them at the time to take them"; 82 (85.4%) patients admitted to using "reminders to take medications." Eighty-two (85.4%) patients agreed that "doctor answers my questions," and 77 (80.5%) said "they are happy with their eye doctor."

Conclusions: The GTCAT identified a number of mostly unintentional factors associated with adherence in this cohort of Brazilian patients. The data may impact on how to understand and improve adherence to ocular hypotensive treatment in the Brazilian population.

Background: In Australia, facilitated regulatory pathways (FRPs) became available with the introduction of priority review (PR) in 2017 and provisional approval (PA) in 2018, which aim to facilitate expedited review and approval for novel medicines. The pathways were developed in consultation with a wide range of stakeholders and have since been utilised by pharmaceutical companies for various therapeutic products. However, the perceptions of the firsthand users of these pathways have not been evaluated in Australia.

Objectives: We have conducted a survey of Australian regulatory professionals aiming to solicit the perceived benefits, barriers to utilisation, shortcomings and proposed modifications to utilising these pathways. We have also solicited the users' perspective on key aspects of the pathways, including overall satisfaction, regulatory burden, availability and ease of use of guidelines, regulator support, impact on company strategy and recommendations for improvement.

Methods: A survey was developed and distributed to Australian regulatory professionals from the pharmaceutical industry who had submission experience of new medicine applications via either PR, PA or the standard registration pathway to the Therapeutic Goods Administration (TGA). The questionnaire consisted of 44 questions with a skip logic and the option for free text comments.

Results: We received responses from 16/42 companies that had utilised these new pathways. Nine respondents had experience with the PR pathway and ten with the PA pathway. The respondents were generally satisfied with the effectiveness of the PR process in expediting registration approvals, but they were ambivalent towards the PA pathway in terms of overall satisfaction and timelines. Respondents expressed a desire for further improvements in the speed of approval, earlier access for patients across various pathways and introduction of new Health Technology Assessment processes for medicines approved under PA.

Conclusion: While the FRPs have been an important and positive development in the Australian regulatory landscape, there remain opportunities for further improvements, some of which have been highlighted by this study and may help inform future regulatory decisions.

Background: Coroners, who hold inquests to determine the causes of unnatural deaths in England and Wales, having recognised factors that could cause other deaths, are legally obliged to signal concerns by sending 'Reports to Prevent Future Deaths' (PFDs) to interested persons. We aimed to establish whether Coroners' concerns about medications are widely recognised.

Methods: We searched MEDLINE, Embase and Web of Science up to 30 November, 2022 for publications linking PFDs and medications using a combination of search terms "coroner*", "inquest*", "medicine*", "medication*" and "prevent*". We also searched the BMJ, a UK journal that carries news items; and the databases Nexis Advance and News On the Web for reports in national newspapers between 2013 and 2022, using the search terms ("regulation 28" OR "prevent future deaths" OR "prevention of future deaths") AND "coroner". We recorded the number of publications, as well as their citations in Google Scholar at 23 May, 2023.

Results: Only 11 published papers on medicines referenced UK PFDs, nine of which were from our group. The BMJ carried 23 articles mentioning PFDs, five related to medicines. Of 139 PFDs (out of over 4000) mentioned in national newspapers, only nine related to medicines.

Conclusions: The PFDs related to medicines are not widely referred to in medical journals or UK national newspapers. By contrast, the Australian and New Zealand National Coronial Information System has contributed cases to 206 publications cited in PubMed, of which 139 are related to medicines. Our search suggests that information from English and Welsh Coroners' PFDs is under-recognised, even though it should inform public health. The results of inquiries by Coroners and medical examiners worldwide into potentially preventable deaths involving medicines should be used to strengthen the safety of medicines.

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.

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.