Medical Affairs (MA) is the pharmaceutical medicine discipline responsible for medically governed scientific exchange, post-approval evidence generation, and stakeholder engagement. As MA adopts artificial intelligence (AI), governance must keep pace. We propose that AI use be directed by MA accountability and named human medical judgement. The European Union (EU) AI Act’s transparency provisions generally apply from August 2026. Its high-risk requirements apply from 2 December 2027 for Annex III systems and 2 August 2028 for systems classified through Annex I. In this narrative review, we critically examine the emerging evidence base and current regulatory guidance to propose eight hallmarks characterising AI-enabled MA. Four are foundational governance capabilities: scientific and ethical integrity, evidence generation at scale, advanced scientific communications, and global alignment and regulatory adaptation. Four are emerging applications requiring validation: AI-empowered strategic leadership, AI-enhanced stakeholder engagement, field medical excellence, and digital innovation and agentic AI systems. Named human accountability underpins all eight hallmarks. We provide pharmaceutical leaders with author-proposed implementation tools including a maturity assessment model, a mapping of common MA AI use cases to EU AI Act obligations, a medical-legal-regulatory workflow for AI-generated content, and equity-by-design metrics. The framework proposes a governance-first, structured pathway for responsible AI adoption in MA that is designed to preserve scientific integrity.
Advances in analytical characterisation, functional assays, pharmacokinetic evaluation and manufacturing science have enabled regulatory agencies to increasingly rely on the totality of evidence, reducing the need for comparative clinical efficacy trials in scientifically justified circumstances. Consequently, the principal challenge surrounding biosimilars has shifted from demonstrating biosimilarity to ensuring their effective implementation in routine clinical practice. Despite this strong scientific consensus, variability in clinical uptake persists across healthcare systems, limited by professional uncertainty, patient concerns, organisational barriers and inadequate communication. Clinical pharmacists are uniquely positioned to bridge this gap between regulatory science and clinical implementation. This article discusses concrete strategies through which pharmacists translate regulatory confidence into clinical confidence, including interpretation of comparability evidence for prescribers, structured patient counselling to mitigate the nocebo effect and support shared decision-making, coordination of switching and traceability programmes, multidisciplinary education, and pharmacovigilance and real-world evidence generation. Their contributions across hospital, community and ambulatory care settings are explored, together with their strategic role in Pharmacy and Therapeutics Committees, procurement processes and health policy implementation. Beyond facilitating biosimilar adoption, pharmacists contribute to healthcare sustainability by promoting value-based medicine, resilient procurement strategies, responsible resource allocation and environmentally conscious pharmaceutical practices. Future opportunities, including digital pharmacovigilance, artificial intelligence and personalised implementation strategies, are also discussed. Clinical pharmacists should therefore be regarded not only as medicines experts but also as knowledge translators capable of transforming regulatory guidance into patient-centred clinical practice and sustainable healthcare delivery.
Ataxias are a heterogeneous group of rare neurological disorders characterized by progressive impairment of motor coordination and balance, caused by a broad range of genetic, immune-mediated, and degenerative mechanisms. Despite significant advances in molecular diagnostics and disease characterization, effective disease-targeted treatments remain limited, with omaveloxolone currently representing the only approved therapy for Friedreich’s ataxia. In this review, we provide a comprehensive and up-to-date overview of the clinical drug development pipeline for ataxias, based on a systematic analysis of international clinical trial registries up to April 2026. One hundred and fifty-four interventional pharmacological trials investigating 66 therapeutic agents across phases 1–3 were identified. The majority of ongoing clinical efforts focus on symptomatic and supportive therapies, which together constitute more than half of the pipeline and include the most developmentally advanced programs. Neuroprotective therapies represent a substantial portion of trials but have shown mixed efficacy to date. In contrast, disease-targeted approaches, including gene therapies, antisense oligonucleotides, cell-based therapies, and protein replacement strategies, account for a smaller share of the pipeline and remain largely confined to early-phase development. More than half of investigational agents are repurposed compounds, reflecting a pragmatic strategy to accelerate therapeutic progress in rare disorders. Overall, the ataxia therapeutic landscape is expanding but remains fragmented, underscoring the need for improved patient stratification, validated biomarkers, harmonized registries, and international collaboration to enable the successful translation of emerging therapies into clinical practice.
The Medical Affairs (MA) 2030 vision positions the medical division as a strategic, patient impact-oriented function designed to translate scientific leadership, stakeholder partnerships, and evidence generation into measurable improvements in clinical practice and patient care. To align with this vision, AstraZeneca (AZ) is implementing a global transformation strategy based on common principles that can be adapted to diverse organizational and healthcare-system contexts. This transformation was first operationalized in AZ Spain through a structured MA transformation into an evolved medical operating model (OM) with data-driven, patient-centered workstreams, embedding transforming care (TC) as a working method focused on improving care pathways and health-system resilience. Scaling this approach requires maintaining a stable scientific and compliance core while allowing local configurations across diverse healthcare contexts, care pathways, and data readiness. We describe how this framework is being implemented in Latin America (LATAM) and outline the design elements intended to support phased implementation and comparability across settings. Latin American countries were organized into five clusters: Mexico, Brazil, Central America and the Caribbean (CAMCAR), the Andean region, and the Southern Cone. This structure supports phased implementation and the adaptation of priorities within a shared framework. The process comprises a four-step pathway starting with a baseline assessment, OM design and configuration, governance-led implementation, and monitoring of implementation progress, operational maturity, and longer-term impact through a focused set of indicators, an implementation roadmap, validation gates, and initial milestones. The approach combines a consistent, locally configurable transformation model and methodological assets, including CARABELA, to support implementation under different healthcare-system contexts. Key design elements include: (i) a baseline maturity assessment and cluster adaptation logic to define each setting’s starting point and priority pathway gaps; (ii) a consistent set of roles, responsibilities and capability enablers to support coordinated execution; (iii) defined governance forums to align evidence planning, launch activities, and TC initiatives through standardized decision-making processes; and (iv) a monitoring approach based on leading and lagging indicators that supports implementation adjustment, assessment of maturity progression, and cross-cluster comparability despite differences in data availability. Early evidence, based on more than 25 TC active projects across clusters and all therapeutic areas reached and potentially impacted 2.5 million patients, with more than 190,000 individuals at risk of selected diseases identified through screening and case-finding initiatives. These aggregate findings indicate implementation scale and uptake, even though they cannot establish causal effectiveness. Subsequent publications will provide detailed cluster-level implementation results, key performance indicator trajectories, changes in maturity assessment, operational lessons, and practical guidance intended to facilitate implementation in other regions and healthcare systems. Medical Affairs (MA) teams in pharmaceutical companies help connect scientific evidence with the needs of healthcare professionals, healthcare systems, and patients. However, countries differ in how healthcare is organized, how patients access care, and what data and resources are available. This means that the same way of working cannot be applied identically in every country. This article describes how a MA operating model developed by AstraZeneca in Spain is being adapted for use in Latin America. The countries were organized into five clusters: Mexico, Brazil, the Andean region, the South Cone, and Central America and the Caribbean. A shared framework was developed and implemented across these clusters, while allowing activities to be adapted to local healthcare needs and capabilities. The framework has four steps. First, each cluster assesses its healthcare environment, care pathways, available data, and MA capabilities. Second, the recommended operating model is adapted to the local setting. Third, implementation is coordinated through defined responsibilities and regular decision-making forums. Fourth, progress and impact are monitored using agreed indicators. The operating model is intended to help MA teams work more consistently across the region while adapting their activities to local needs. It provides a structured approach for using scientific evidence, collaborating with healthcare stakeholders, and identifying opportunities to improve care pathways and address gaps in patient care. This article describes the framework and its implementation across Latin America. Future publications will report the experience gained, lessons learned, and results from individual countries as sufficiently mature data become available.
