Pharmaceutical Medicine最新文献

Implementation science offers a systematic approach to bridging the gap between research and clinical practice by promoting the adoption, scale-up, and sustainment of evidence-based therapies. Historically, academic implementation science efforts in the USA have focused on later-stage post-market research through government-funded initiatives, leaving untapped opportunities to engage more proactively with life science partners, such as pharmaceutical sponsors, earlier in the development process. Limited integration with life science partners, especially during early-phase clinical trials, represents a missed opportunity to address implementation challenges that affect real-world uptake proactively. The objective of this current opinion is to explore how implementation scientists can more effectively position their expertise to support the development, evaluation, and delivery of new therapies throughout the entire clinical trial lifecycle with life science partners. This article proposes a conceptual framework for collaboration between implementation scientists and life science partners, emphasizing how early integration can help identify healthcare system constraints, clinician adoption barriers, and patient acceptability issues, factors that often shape a therapy's downstream impact. Even before efficacy or regulatory certainty is established, implementation science can strengthen trial design, inform site and stakeholder selection, and enhance the interpretability and readiness of findings for real-world uptake. By aligning implementation methodologies with the strategic priorities of pharmaceutical sponsors, academic researchers can provide structured actionable insights that increase the likelihood of clinical and commercial success. Stronger partnerships established earlier in the development process may improve return on investment, reduce time to impact, and accelerate the translation of effective therapies into routine care.

The role of medical affairs (MA) in the pharmaceutical industry is undergoing a profound transformation, driven by the increasing complexity of healthcare ecosystems, the rise of digital technologies, and the need for enhanced stakeholder engagement. In alignment with the MA 2030 vision, AstraZeneca Spain has implemented a forward-thinking transformation strategy, positioning MA as a strategic healthcare partner through a structured, data-driven, and patient-centered approach. Our transformation journey has been anchored in five strategic priorities: boosting MA leadership, integrating end-to-end data and analytics, refocusing resources using data-backed strategies, aligning evidence generation with stakeholder needs, and orchestrating omnichannel scientific engagement. To achieve this, we developed and implemented innovative methodologies such as regional archetyping, healthcare account characterization, and stakeholder mapping to systematically analyze and tailor engagement strategies across different healthcare settings. A core component of this strategy is the CARABELA initiative, a structured approach aimed at fostering the optimization clinical pathways, promoting public-private collaborations, and driving practice-changing interventions to improve healthcare efficiency. In addition, we have transitioned from descriptive to predictive analytics through advanced real-world evidence models, ensuring that MA-led initiatives remain proactive and impact-driven. This transformation serves as a scalable framework for MA evolution globally, reinforcing its role as a catalyst for healthcare innovation. By integrating real-world data, digital engagement, and strategic collaboration, MA departments should position themselves to navigate the evolving healthcare landscape while delivering tangible benefits for patients, healthcare professionals, and systems worldwide.

The development of health technologies involves complex, costly, and risky investments, with significant contributions from both public and private sectors. Recent EU pharmaceutical directives propose transparency on public funding to aid pricing negotiations and affordability. However, questions remain regarding how public investments should be measured and their influence on pricing and reimbursement (P&R) decisions. In this paper, we characterise public sector institutions as "payers," "R&D investors," and "regulators". Through a myriad of agencies and decisions, these institutions directly, indirectly or sometime unexpectedly influence risk and return on private research and development (R&D) through P&R, direct investments, and regulatory policy. P&R decisions by payers for innovative therapies influence risk and expected return of future R&D. Value-based pricing offers a more reliable signal of payers' priorities than cost-plus or (international) reference pricing. For greatest impact, public R&D investment should be directed to areas where markets are deficient, such as basic science, translational research, real-world studies, and towards emerging fields like AI and gene editing that will play an increasing role in healthcare and drug development. Applied R&D should be conducted on a financially sustainable basis. Licensing arrangements can be used to recover those investments, while promoting spillover benefits to wider society. Market access regulators are aware of the need for scrupulous transparency and neutrality, but other public sector actors (payers and R&D investors) also must recognise their policies affect the level playing field.

The transformation of China's healthcare system is increasingly driven by the development of real-world data (RWD) and real-world evidence (RWE). In the context of the "Healthy China 2030" initiative, which emphasizes addressing the growing burden of non-communicable diseases (NCDs), this article assesses the current state and potential of RWD/RWE development. Key areas of focus include the policy environment, guideline systems, data foundations and tools, and talent development. The article outlines a comprehensive roadmap for advancing high-quality RWD/RWE in China, emphasizing the construction of nationwide RWD sources and the harmonization of patient-centered RWD public-private partnerships. As a call to action, the article proposes specific recommendations for various healthcare stakeholders (government, regulatory authorities, industry, academia, clinical researchers and institutions, and data service providers) to collaboratively establish a robust and sustainable RWE ecosystem in China. By leveraging these efforts, the ultimate goal is to significantly improve healthcare outcomes, enhance the efficiency of healthcare delivery, and support evidence-based policymaking, thereby contributing to the overall health and wellbeing of the population.

Background: Australia has one of the highest rates of rheumatoid arthritis (RA) worldwide. Etanercept, a widely used biologic for severe RA, has had a publicly subsidised biosimilar available in Australia since 2017. However, real-world data on how biosimilar availability has affected treatment patterns remain limited.

Objective: This study aimed to assess treatment persistence-a surrogate measure of long-term treatment effectiveness-with etanercept before and after public subsidy of its biosimilar for RA, utilising a national sample.

Methods: This retrospective cohort study analysed national healthcare claims data from the Pharmaceutical Benefits Scheme (PBS) via the Australian Bureau of Statistics DataLab. Adults (age ≥ 18 years) initiating etanercept for severe RA were stratified into two cohorts: historical (before biosimilar PBS listing, comprising only originator users) and contemporary (after biosimilar PBS listing, comprising both biosimilar and originator users). Kaplan-Meier analysis and multivariate Cox regression were employed to assess treatment persistence. Subgroup analysis of older adults and sensitivity analysis limited to biologic-naïve individuals were also performed.

Results: A total of 10,234 individuals initiating etanercept for severe RA were included, with 4461 in the historical cohort and 5773 in the contemporary cohort. The median time to treatment discontinuation was 10.0 months (95% confidence interval (CI) 9.7-10.6) in the contemporary cohort and 10.6 months (95% CI 10.0-11.4) in the historical cohort (p = 0.08). At 12 and 24 months, treatment retention rates were similar between cohorts. The adjusted hazard ratio for treatment discontinuation in the contemporary cohort was 1.00 (95% CI 0.96-1.05), indicating no significant differences. Subgroup and sensitivity analyses yielded similar results.

Conclusion: This large, population-based study found no significant difference in treatment persistence following the introduction of the etanercept biosimilar in Australia. These findings support the real-world integration of biosimilars into routine care. Further research should include direct comparative analyses of originator and biosimilars to inform long-term treatment strategies, clinician confidence, and sustainable healthcare policy.

The role of Medical Affairs (MA) has shifted from traditional evidence dissemination to an integrated process leveraging real-world data (RWD) and diverse data sources. To adapt and lead this shift, AstraZeneca (AZ) Spain recognized the essential need to identify gaps in evidence-generation capabilities and establish targeted strategies aligned with both global and local priorities. The objective of this work was to assess AZ Spain's current evidence-generation capabilities and identify areas for improvement by using the so-called "Evidence Blueprint" framework. To do this, we conducted a systematic self-assessment following the Blueprint to evaluate performance across ten core areas. An approach to identify gaps and enhance evidence-generation processes was undertaken in four phases: defining capabilities, assessing maturity, developing a roadmap for improvement, and implementing changes. The self-assessment identified five priority areas, including two focused priorities-Innovative Value Strategies/Payer Evidence and RWD Vision and Strategy-as well as three improvement areas-Evidence Planning and Value Team Implementation, Research/Evidence Partnerships, and Patient-Centric Evidence. Tangible actions included the development of processes to assess outcome-based agreements, comprehensive mapping of existing national and regional databases to strengthen RWD strategies, creating a cross-functional strategy for evidence planning, establishing research partnerships leveraging European funding, and adopting patient-centric methodologies such as ethnographic studies and patient-authored publications. The initiatives undertaken by AZ Spain demonstrate the transformative potential of an Evidence Blueprint framework in addressing gaps and enhancing evidence-generation capabilities. By aligning local strategies with AZ's MA 2030 vision, these efforts ensure continuous innovation, improvement of decision-making, and a more substantial contribution to the healthcare ecosystem.

Antimicrobial resistance is a pressing global health threat fueled by a complex interplay of biological, social, and economic factors. Despite widespread recognition of its impact, the antimicrobial resistance crisis continues to deepen because of inadequate innovation, poor access to effective treatments, and irrational antimicrobial use. Effectively combating antimicrobial resistance requires a multisectoral, multistakeholder, and multidimensional approach, with the pharmaceutical industry playing a pivotal role in new antimicrobial discovery along with diagnostic and other stakeholders. This review critically examines the central role of the pharmaceutical industry in addressing antimicrobial resistance, focusing on drug discovery, manufacturing practices, and stewardship efforts. While the industry has made notable contributions through the development of new antimicrobials and alternative approaches such as drug repurposing, artificial intelligence-driven discovery, and improved diagnostics, major challenges persist-including a declining antibiotic pipeline, limited access in low- and middle-income countries, antimicrobial pollution, irrational fixed-dose combinations, and the prevalence of substandard or falsified drugs. To overcome these barriers, this review explores strategic directions, including public-private partnerships, delinked incentive models, small-molecule innovation, ethical marketing, and equitable access strategies. It also underscores the industry's responsibility in promoting antimicrobial stewardship, participating in global surveillance systems, and educating prescribers and the public on responsible use. Future directions highlight the need for diversified funding, global collaboration, and the adoption of the "triple shield" approach-integrating infection prevention and control, antimicrobial stewardship, and robust surveillance to combat antimicrobial resistance. This review presents an integrated analysis of pharmaceutical accountability, highlighting actionable pathways that align innovation with equitable access, environmental safety, and ethical governance. By bridging gaps between discovery and delivery, the pharmaceutical sector can become a driving force in the global response to antimicrobial resistance.

Background: Safety advisories provide critical information to clinicians and patients on the harms of medicines. Previous research has shown that national regulators vary in their decisions to issue safety warnings. However, it is not known whether clinicians receive similar information when regulators communicate about the same medicines' harms.

Aim: Our aim was to assess whether content provided to clinicians in safety advisories on risk, fatal outcomes, evidence and clinician advice was comparable.

Methods: This retrospective content analysis examines safety advisories issued by the Australian Therapeutic Goods Administration, Health Canada, the United Kingdom Medicines and Healthcare products Regulatory Agency, and the US Food and Drug Administration between 2007 and 2016. Content was extracted from advisories issued on the same medicine and harm (n = 40), including evidence, risk quantification, fatal outcomes and clinician advice. A case study on pioglitazone and bladder cancer illustrates differences in regulatory communications.

Results: Variation was seen in the detail and presentation of information on evidence, deaths, risk quantification and advice to clinicians. Specific advice to clinicians was provided in 70% (96/155) of advisories with no significant differences between regulators (p = 0.19). Evidence of harm was presented in 81% (130/160) of advisories and risk quantification in 61% (98/160). The type of evidence presented and directness of information differed however. In the pioglitazone case study, for example, regulators differed in how bladder cancer risks were characterised and advice provided.

Conclusions: Our analysis of safety advisories on the same harms of medicines indicates that while regulators provide similar content elements in safety advisories, risk messages to clinicians vary. This may lead to differences in knowledge and awareness between countries and potentially impact public health outcomes. Further transparency around regulatory decisions on safety advisories is needed.

The global pharmaceutical landscape is undergoing a profound shift with the rise of biosimilars-biologic medical products that are highly similar to original biologics but manufactured by different companies. Biological therapies encompass a range of therapies, including hormones such as insulin and growth hormone, vaccines, erythropoietin (EPO), monoclonal antibodies (mAbs), antibody-drug conjugates (ADCs), and bispecific antibodies (BsAbs), etc. As patents for many blockbuster biologic drugs expire, biosimilars provide an opportunity to improve patient access to life-saving treatments while potentially lowering healthcare costs, especially in regions with high unmet medical needs. However, the development and integration of biosimilars into mainstream healthcare comes with various challenges, including regulatory approval processes, ensuring quality, potency, efficacy, immunogenicity, safety, and overcoming market resistance. In this article, we will explore the development of biosimilars in-depth, their approval pathways, benefits, and the hurdles in achieving widespread adoption.