Pharmaceutical Medicine最新文献

Background: In 2012, the East African Community Medicines Regulatory Harmonization (EAC-MRH) initiative was established to improve access to safe, effective, and high-quality medical products to patients in the East African region. The East African Community (EAC) Partner States, the Republic of Burundi, Democratic Republic of Congo, Republic of Rwanda, United Republic of Tanzania, Republic of Kenya, Republic of South Sudan, and the Republic of Uganda, have a population of 290 million inhabitants. The timely access to medical products for this population was to be achieved through harmonisation of regulatory requirements, joint assessments, joint inspections of manufacturing sites, and the strengthening of regulatory systems. The aims of this study were (1) to investigate ways in which the regional initiative could be a well-coordinated and functioning regional assessment and inspection process on which national registration decisions can rely; (2) to investigate whether a sustainable semi-autonomous regional agency could provide regulatory guidance and coordination for the entire region; and (3) to propose a new and improved model for the EAC-MRH.

Methods: Three established questionnaires were used to collect and analyse data on the EAC national regulatory authorities (NRAs) and EAC-MRH initiative 2020-2023: (1) The Optimising Efficiencies in Regulatory Agencies (OpERA) questionnaire was completed by senior officials in the seven authorities that were leading the medicine registration departments about their own respective NRA. The heads of authorities of these NRAs further validated the completed questionnaire, which documented the general organisation of the authorities in terms of their structure, organisation, resources, review process, and timelines. (2) The Process Effectiveness and Efficiency Rating (PEER) questionnaire was completed by the seven authorities to obtain the views of the individual medicines regulatory authorities of the EAC-MRH initiative to identify the strengths and challenges regarding the performance of the joint assessment of the EAC-MRH initiative. (3) The PEER questionnaire, modified for the pharmaceutical industry, was completed by the heads of regulatory units in the pharmaceutical companies that had used the EAC-MRH process for the review and approval of their applications.

Results: The number of applications received for joint reviews increased from nine applications in 2015 to 44 applications in 2023, and the median review time reduced from 553 calendar days in 2015 to 259 calendar days in 2023. A key benefit for pharmaceutical companies using the work-sharing initiative to apply for marketing authorisation was the reduced burden associated with the need to prepare only one application for submission and eventual access to several markets simultaneously.

Conclusions: The EAC-MRH initiative can only be effective and efficient

This paper reviews the pivotal role of the Medical Advisor (MA) within the medical department of the pharmaceutical industry, highlighting their essential contribution to the development, commercialization, and appropriate use of medicines. In an environment characterised by stringent regulations and increasing demands for transparency, the MA role has become an indispensable position for ensuring optimal integration between scientific innovation and commercial strategy. The work of MAs facilitates cross-functional collaboration between a board spectrum of internal teams and external stakeholders, ensuring that scientific and clinical knowledge is effectively integrated at every stage of the product lifecycle. This includes leveraging emerging new technologies such as artificial intelligence and digital health solutions, as well as using machine learning to enhance predictive analytics. Such integration is critical to addressing unmet medical needs while aligning these initiatives with broader business objectives to drive innovation, market competitiveness, and patient outcomes. We explore the key responsibilities of today's MAs, which include contributing to the generation of data through clinical trials and post-authorization studies, providing continuing medical education, and communicating accurate information to diverse audiences, including healthcare providers, regulators, and patients. Furthermore, MAs promote innovation and therapeutic progress, acting as guardians of medical ethics. This review aims to provide a comprehensive understanding of the strategic role of MAs in bridging the gap between scientific research and clinical practice. Their contributions are critical to addressing current industry challenges, ensuring that companies remain competitive and making a significant contribution to patient wellbeing and medical progress.

The evolving landscape of healthcare demands a transformation in Medical Affairs (MA) to address new challenges and opportunities. This article presents the Strategic Scientific Advisor (SSA) role as a critical innovation within our MA department, aimed at enhancing early engagement with stakeholders and strategically positioning assets within the healthcare ecosystem. The SSA role was developed following a comprehensive external assessment and an internal review of the MA department's structure to integrate the CARABELA methodology, which serves as the foundation for SSA activities. CARABELA is a joint initiative between AstraZeneca and key scientific societies to optimize clinical practices in specific chronic diseases using a holistic strategy, whose implementation is monitored in each hospital by applying specifically designed indicators. The SSA role design, guided by the Strategic Scientific Area Plan Framework, emphasizes early proactive engagement, evidence generation, and a multidisciplinary approach to optimizing clinical practices. By fostering collaboration across internal teams and external partners, the SSA supports activities throughout the entire lifecycle of the product, facilitating early access strategies, and building tailored plans to address unmet medical needs. In contrast to traditional Medical Science Liaisons (MSLs), SSAs play pivotal roles in driving long-term healthcare outcomes, aligning with the Medical Affairs 2030 vision. The SSA is trained and evaluated to catalyze a strategic shift, enabling MA to address the complexities of healthcare ecosystems, improve clinical practices, and contribute to patient-centered innovations. This transformation underscores the critical need for MA professionals to expand beyond traditional responsibilities and lead initiatives that shape the future of healthcare.

Introduction: The rapidly evolving healthcare landscape has prompted Medical Affairs (MA) departments within pharmaceutical companies to transition from their traditional role as information providers to becoming strategic partners in the healthcare ecosystem. Responding to the increasing complexity of patient needs and stakeholder dynamics within Spain's national health system, this shift emphasizes the importance of aligning MA functions with broader healthcare goals. Effective transformation requires in-depth assessments of stakeholder trends and expectations. The objectives of this study were to identify key stakeholders in the Spanish healthcare ecosystem, analyze their trends in detail, and evaluate ways in which our MA department should adapt to address them.

Methods: To support this strategic transformation, we conducted a dual assessment focusing on the Spanish healthcare ecosystem. Using a combination of desk analysis and stakeholder research, we examined external and internal dynamics affecting the MA department's role. This approach identified the perspectives and needs of key stakeholders across three main communities: patients, healthcare professionals, and institutional bodies, offering insights into stakeholder-specific expectations and broader industry macrotrends.

Results: The identification of 16 pivotal stakeholders in Spain's healthcare ecosystem underscores the complex array of needs and expectations that shape MA's evolving role. For the patient community, key trends include the demand for accessible information, emotional support resources, and tools to enhance treatment adherence and chronic disease management. Among clinicians and key external experts, there is an increasing need for current medical resources, digital integration in care processes, and collaborative research opportunities. Institutional stakeholders emphasize sustainable integration of therapeutic innovations, budget predictability, and public-private collaboration to enhance healthcare quality and access. Beyond these specific needs, transversal trends affecting all stakeholders were identified, including the acceleration of medical innovation, demand for value-added services, and the expansion of digital tools and data-driven decision making. Macrotrends within the pharmaceutical industry - such as patient-centric approaches, the growth of digital health, and data-focused business models - further shape the industry's response to evolving healthcare challenges. A unified organizational vision, reflected in shared objectives and priorities, is crucial for cohesive strategy implementation.

Conclusion: This research underscores the essential role of MA departments in redefining their influence within healthcare systems. By aligning MA activities with stakeholder trends, pharmaceutical companies can reposition MA as a central pillar within the healthcare ecosystem. Our

Background and objectives: Drug development in schizophrenia is limited by the differential scaling of the active treatment and placebo arms of a study, such that, as the number of sites increases, the magnitude of placebo response disproportionately increases. The objective of this article was to identify factors conducive to efficient recruitment as a step towards trial designs allowing recruitment of more participants per site, leading to reduced variability, and potentially a smaller placebo effect.

Patients and methods: Using the information of 554 individuals, we calculated the percentage of individuals who were screened, consented, and retained in our research, along with rationale for nonconsent. Independent t tests and Chi-squared tests were performed to compare participant characteristics.

Results: Out of the 273 individuals who were fully screened, 84 did not progress to the consented stage owing to various reasons, leading to a total of 189 individuals who were screened and consented and a total of 365 individuals who were either incompletely screened or not consented into a study. Individuals with an externally validated medical history showed the highest yield in being consented and retained in research as new participants. In particular, chart reviews from clinics were highly efficient (25.8%) in facilitating new participants' enrollment, even when these individuals were not actively/prospectively seeking research. The most common reason for nonconsent was difficulty in telephone or email contact. Consenting and nonconsenting participants were similar in demographics, and recruitment sources only differed in their reported substance use.

Conclusions: Referrals and chart reviews from known clinics were the most efficient method in retaining new participants, highlighting the importance of external validation and communication between research and clinical staff within a system. Consenting participants showed no significant differences from the database as a whole, demonstrating that the study samples were representative. Future studies should aim to confirm the present findings at other academic and commercial research centers.

Enrollment of women of childbearing potential in clinical trials from early stages of drug development allows the gathering of relevant safety and pharmacokinetics data in the representative disease population. However, risk mitigation strategies are needed to ensure the safety of the developing embryo in case of unintended pregnancy in female study participants or in female partners of male study participants. These risk mitigation strategies often include contraception requirements, however we observed that such requirements vary widely across sponsors and trials. Some sponsors and contract research organizations adhere to the strictest precautions by default, assuming a worst-case scenario for each new compound's risk assessment. In this review, we provide an overview of the risks addressed by and introduced by contraception requirements in clinical trials. We critically discuss the 'worst-case' approach, and we present a holistic, risk-based approach that takes into account all relevant data, the pros and cons of using hormonal contraceptives, and subject burden.