Pharmaceutical Medicine最新文献

Alopecia is a multifactorial condition resulting from the convergence of systemic pathologies, pharmacologic exposures, genetic predispositions, and environmental factors. Among these, systemic diseases and medications play a prominent role by initiating biological cascades that disrupt follicular integrity. Systemic diseases and medications disrupt follicular homeostasis via mechanisms such as inflammation, oxidative stress, hormonal imbalance, and immune dysregulation. Endocrine disorders, metabolic syndromes, and nutritional deficiencies impair follicular function, while chemotherapeutics, antifungals, anticoagulants, and hormonal agents further compromise follicular cycling and stem cell viability. Understanding these mechanistic pathways is essential for predicting and mitigating drug-associated alopecia and informing targeted interventions. Precision medicine approaches that address systemic drivers while optimizing pharmacologic regimens are critical to preserving follicular integrity. Current treatments like minoxidil, platelet-rich plasma, and surgical restoration provide symptomatic relief but often fail to achieve lasting results. In contrast, emerging molecular therapies, including bioengineered exosomes, stem cell applications, and peptide-based formulations that target key signalling pathways and growth factors, hold promise for more regenerative and sustained hair restoration outcomes. Given the condition's complexity, a multidisciplinary framework integrating dermatology, endocrinology, immunology, and pharmacology is essential. Cross-specialty collaboration will be pivotal for advancing individualised, mechanism-driven care. Ultimately, accurate etiologic diagnosis remains the cornerstone of effective, tailored treatment strategies.

Medical information departments within pharmaceutical companies play a critical role in addressing unsolicited healthcare professionals' inquires with accurate, evidence-based, and balanced medical information through scientific response documents. Global response documents (GRDs) aim to harmonize information between global and local teams and the end product, the scientific response document. To address the challenges associated with the development and maintenance of global response documents and this harmonization, phactMI and Medical Information Leaders Europe (MILE) convened an international expert panel representing 11 major pharmaceutical companies. Over months of discussion and deliberation, the panel developed consensus-based best practices for the creation and management of global response documents. The experts contributed nuanced insights regarding optimal document structure and format that consider various stakeholders while maintaining medical accuracy. Consensus was achieved via a structured survey with a 75% approval threshold for each best practice. Overall, GRDs are essential tools for pharmaceutical companies, offering accurate evidence-based responses to healthcare professionals. While companies may respond to patient inquiries as well in different formats (verbal vs written), GRDs are exclusively intended for healthcare professionals currently. Best practices for GRD development include: being focused on a single topic, developed in a label-agnostic manner, utilize the most robust scientific information available, developed up to 12-15 months prior to launch, have regular feedback in a structured approach from local teams, follow internal guidelines and work instructions, and be peer reviewed internally whenever new data are added. In addition, local response documents or scientific response documents should be based on a GRD that is localized with appropriate documentation. Overall, GRD development is a collaborative and innovative approach (including interactive formats), supported by defined metrics (including response time) and ongoing updates to ensure global relevance and compliance.

The evolving role of Medical Affairs (MA) has positioned this department as a key element in the development and lifecycle management of pharmaceutical products. In response to internal and external assessments, AstraZeneca (AZ) Spain implemented a transformation of its MA central teams through a newly designed Working Model. This model ensures full engagement across the product lifecycle, spanning early access, pre-launch, launch, and post-launch, balancing global strategy with local execution, enhancing cross-functional collaboration, and timely evidence generation. Central to this transformation is the deployment of four coordinated teams: the Early Asset Strategy Team, the Launch Readiness Team, the Core Value Team, and the Brand Team. These teams follow a structured work methodology and coordination model underpinned by dedicated information management processes that streamline communication and knowledge transfer. Training and capability-building programmes were also introduced to strengthen critical functional and leadership competencies. These changes aim to improve product positioning, accelerate market access, and drive value-based healthcare outcomes. The initiative aligns with broader industry trends emphasising early stakeholder engagement, data integration, and patient-centric strategies. By fostering agility, strategic foresight, and internal consistency, the transformation of MA central teams at AZ Spain provides a replicable model for enhancing MA's contribution to product development and healthcare ecosystem innovation. This article builds on our previous publications about the national ecosystem assessment of stakeholder needs, the definition of our MA transformation framework (articulating the strategic roadmap), and the description of the evolution of in-field MA teams in AZ Spain. Here we detail the transformation of the MA central teams and their working model. Future MA evolution will integrate emerging technologies like artificial intelligence (AI) and digital tools, improving stakeholder engagement, evidence generation, and decision making. The transformative potential of AI in anticipating trends and optimising trials will enhance the role of MA in driving innovation and improving patient outcomes.

Background and objectives: Efficient and informed patient recruitment, followed by successful enrolment, is essential for the conduct of clinical trials. As major stakeholders in the research process, pharmaceutical companies have an important role in ensuring that information for patients is available and readable. Several global institutions recommend that patient-facing material be written at a literacy level suitable for 11-12-year-olds. This study assessed the availability and readability of clinical trial sections on UK, Canadian, Australian, and global pharmaceutical company websites.

Methods: The 30 largest global pharmaceutical companies (assessed by market capitalisation in April 2025) were selected. Clinical trial content was reviewed for availability and analysed using three validated readability metrics: the Flesch-Kincaid Grade (FKG), Flesch Reading Ease Score (FRES) and Simple Measure of Gobbledygook (SMOG) Index.

Results: Of 115 websites assessed, 54 were eligible for readability analysis. While 96% of global websites included clinical trial information, 55% of non-global websites lacked such content or contained only external links. FKG scores, which estimate the US school grade level needed for comprehension, averaged 10.9 (± 3.5) for global, 14.2 (± 2.0) for UK, 12.1 (± 2.7) for Canadian and 12.8 (± 1.9) for Australian websites-suggesting readability at high school to college level. FRES scores showed similar trends: 42.2 (± 8.0) global, 31.2 (± 9.4) UK, 38.8 (± 12.3) Canadian and 35.7 (± 12.3) Australian, indicating college-level complexity. SMOG scores suggested that 13-15 years of education were needed to understand the material.

Conclusions: These results indicate that clinical trial information on pharmaceutical company websites is often missing or difficult to read and exceeds recommended literacy levels, which may limit comprehension and engagement. Poor readability disproportionately affects individuals with lower literacy, limited English proficiency or disabilities, creating inequities in trial participation. Applying health literacy and plain-language principles-such as simplifying terminology, shortening sentences and using clear formatting-could improve accessibility and support informed decision-making.

Background and objectives: Medical Affairs (MA) plays a critical role in bridging the gap between research, clinical practice and business strategy. With the rapid growth in this field, it is essential to have a competency framework to support individuals' professional development. A well-defined competency framework will not only empower MA professionals to excel and develop in their roles but also contribute to better patient outcomes and improved stakeholder engagement. This paper discusses the development of a competency framework for Medical Affairs professionals in Australasia.

Methods: The MA competency framework was developed using an iterative method by a team of MA professionals across Australia through a series of workshops and surveys over 2 years with the cooperation of the local MA community. The core development team debated and finessed the final framework over this time via meetings and discussions to arrive at the draft framework. This was pilot tested by a local pharmaceutical organisation and feedback informed some minor changes to the final framework. This was then endorsed by the Medical Affairs of Australasia (MAPA) Executive Committee.

Results: The framework consists of six domains: Scientific/Technical Knowledge, Evidence Generation, Compliance, Governance and Ethics, Leadership/Professionalism, Communication and Collaboration and Business Acumen, each with specific competencies and across four clearly defined levels from novice to expert.

Conclusion: This framework has been endorsed by the Medical Affairs Professionals of Australasia Executive Committee and provides a clear framework for the professional development of medical affairs professionals across our region. It is also applicable to MA professionals more broadly.

The Joint Clinical Assessment is a crucial development under the European Union Health Technology Assessment Regulation (EU HTAR, 2021/2282), aimed at standardizing clinical assessments across the Member States. This review explores the opportunities, challenges, and future actions for Joint Clinical Assessment implementation, particularly emphasizing methodological, regulatory, and stakeholder considerations. With the evolution of the European healthcare landscape from decentralized health technology assessment processes to a more streamlined framework, the Joint Clinical Assessment aims to enhance efficiency, reduce duplication, and expedite patient access to novel therapies while maintaining national reimbursement autonomy. Central to the Joint Clinical Assessment is the Patients, Interventions, Comparator, Outcomes (PICO) framework, which guides evidence generation but varies across countries, creating uncertainty and requiring early transparent agreement. Challenges to Joint Clinical Assessment implementation include tight timelines, misalignment with national processes, autonomy of national health technology assessment agencies over reimbursement, and the uncertainty about real-world evidence, specifically for oncology and rare diseases. Orphan drugs, gene therapies, and vaccines present unique concerns owing to smaller patient populations, limited data from clinical trials, or ethical restrictions in conducting randomized controlled trials. These concerns can be overcome with scientifically robust alternatives, such as adaptive assessment approaches enabling staged assessments and updates with evolving data. Early patient involvement through advisory panels, surveys, and feedback loops in processes, such as scoping, assessment, and evidence-generation phases can improve transparency and integration of patient preferences while reflecting real-world treatment needs in clinical evaluations. The long-term success of the Joint Clinical Assessment will depend on both standardisation and flexibility, supported by stakeholder collaboration and robust methodological guidance.

Background and objectives: The biopharmaceutical industry requires fast role adaptation and comprehensive competency development to thrive in a rapidly evolving and challenging environment. Whilst competency frameworks exist for some roles, lack of a function-wide framework remains a challenge. The objective of this pilot study was to evaluate the applicability and potential utility of the Medical Affairs Professionals of Australasia (MAPA) competency framework within Amgen's Medical Affairs to support role-specific competency development.

Methods: This competency framework encompasses six domains: scientific and technical knowledge; evidence generation; compliance, governance and ethics; leadership, strategic vision and professionalism; communication and collaboration; and business knowledge/acumen; each with specific sub-domains with four clearly defined levels from novice to expert. Implementation involved self-assessment followed by managerial ratification for competency level. Overall team and affiliate results were made available on a PowerBI dashboard. The pilot included a diverse range of roles from Amgen Medical Affairs, such as Medical Science Liaisons (MSLs), MSL Managers, Medical Advisors, Medical Capability & Operations, Medical Communications, and Therapy Area Leads.

Results: Our pilot consisted of 42 staff and revealed relatively general levels of consistency for benchmarks across our three affiliates. Most participants met or exceeded the benchmark set for their roles. We observed some variance between self and manager competency level assessment and relevance/applicability of all capabilities for all roles but this was addressed by self and manager discussions to agree on a consensus value.

Conclusion: This pilot confirmed the MAPA competency framework's feasibility and relevance across diverse Medical Affairs roles and affiliates. While benchmarks were generally consistent, minor differences were observed across Australia, China, and Japan. The successful pilot outcomes highlight the potential for this framework's adoption across the biopharmaceutical industry's medical affairs for developmental discussions and planning.