Pharmaceutical Medicine最新文献

{"title":"Integrated Evidence Generation Planning: Why Medical Affairs is a Critical Partner.","authors":"Ronaldo Fujii, Masoud Nouri, Mohammad Khan, Lucija Fleisinger, Jamie Pugh, Gary Male, Antonia Panayi","doi":"10.1007/s40290-026-00620-2","DOIUrl":"10.1007/s40290-026-00620-2","url":null,"abstract":"<p><p>In an era of rapidly evolving healthcare systems and escalating demands for value demonstration, pharmaceutical companies face unprecedented pressure to generate robust, relevant evidence for a diverse array of stakeholders. Traditional, siloed approaches to evidence generation are no longer sufficient. The development and use of integrated evidence generation plans (IEGPs) have emerged as strategic imperatives-reshaping how organisations align scientific, regulatory and patient-centred objectives across the product life cycle. The IEGPs bring together evidence-generation activities across functions and geographies, to ensure that robust evidence is delivered throughout the product life cycle. In this article, we present IEGP development as a collaborative and strategic process, which enables pharmaceutical companies to demonstrate the holistic value of a new therapy to a broader range of healthcare decision makers. We argue that Medical Affairs professionals are well positioned to lead this initiative, given their in-depth understanding of the evidence generation needs and priorities of patients and healthcare professionals; however, a close and strategic partnership with other key functions, including Market Access and Clinical Development, regions and countries, is vital to the holistic understanding of all key evidence needs and to establish the strategic differentiation required to bring new medicines to patients. Our companion article in this edition of Pharmaceutical Medicine expands on the role of specific subfunctions of Medical Affairs in the development of an IEGP; it also considers how the impact of Medical Affairs' role can be measured. Please note that in both articles we prefer to use the term 'IEGP', but the reader may also see the similar term 'IEP' (Integrated Evidence Plan) used in the literature-these terms are used interchangeably in the field.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"351-356"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13545106/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148707399","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Regulatory Considerations for Combined Drug-Device Studies in Europe: A Case Study Involving Bile Sampling.","authors":"Hans-Peter Podhaisky, Emiko Tsuruta, Isabel Piel, Christiane Otto, Sofia Busto Paez, Miriam Font-Nieves","doi":"10.1007/s40290-026-00619-9","DOIUrl":"10.1007/s40290-026-00619-9","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"327-333"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148382639","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Impact of Clinical Trial Participation on Quality of Life in Duchenne Muscular Dystrophy.","authors":"Aidan W Leffler, Graham J Leffler, Marielle G Contesse","doi":"10.1007/s40290-026-00624-y","DOIUrl":"10.1007/s40290-026-00624-y","url":null,"abstract":"<p><strong>Background and objectives: </strong>While there has been an increase in Duchenne muscular dystrophy (DMD) clinical trials over the last couple of decades, there is still no cure. Many individuals with DMD spend much of their lives participating in clinical trials. The impact of clinical trial participation on quality of life (QoL) is not well understood. This study aimed to understand the aspects of clinical trial participation that impact QoL for families affected by DMD.</p><p><strong>Methods: </strong>Qualitative interviews were conducted with patients aged ≥ 12 years and caregivers using a semi-structured interview guide. Participants described the clinical trial factors that impacted QoL and rated the impact of each factor using a 7-point Likert scale. Transcripts were single coded for themes. A Participant-Specific Total Impact Factor (PSTIF) score was derived using the mean score across factors identified as impacting QoL. Agreement was calculated using Cohen's Kappa between PSTIF score and the participant's reported net trial experience (positive, neutral, or negative).</p><p><strong>Results: </strong>A total of 24 caregivers and 7 patients participated. Participants mentioned 23 clinical trial factors with an impact on QoL. While most participants (82%) had a negative PSTIF score, the majority (65%) described a positive overall experience with clinical trials. There was poor agreement (Kappa = 0.12) between PSTIF score and net clinical trial experience.</p><p><strong>Conclusion: </strong>When patients and caregivers quantified the impact of specific clinical trial factors on QoL, the impact was primarily negative; however, the majority of participants described their overall trial experience as positive. The study's findings have implications for the collection and interpretation of QoL data in interventional studies.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"413-422"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148536515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction: Paediatric Phase I/II Clinical Trial Assessments Under the New EU Clinical Trial Regulation: A Descriptive Analysis.","authors":"Laura Fankhauser, Franca Ligas, Giovanni Lesa, Anette Solli Karlsen, Marianne Lunzer, Monique Al, Sabine Scherer, Sylvie Benchetrit, Claudia Riedel, Annette Kopp-Schneider, Ruth Witt, Olaf Witt, Dominik Karres, Cornelis M van Tilburg","doi":"10.1007/s40290-026-00616-y","DOIUrl":"10.1007/s40290-026-00616-y","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"325"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13332877/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148278264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Regulating the Irregular: Phage Therapy and the Case for a Regulatory Sandbox Approach in Australia.","authors":"Marco Rizzi, Stephen M Stick, David G Hancock, Anthony Kicic, Yuliya V Karpievitch","doi":"10.1007/s40290-026-00614-0","DOIUrl":"10.1007/s40290-026-00614-0","url":null,"abstract":"<p><p>Bacteriophages (phages) are viruses that selectively kill bacteria and offer a promising option to address the growing global pandemic of antimicrobial-resistant infections. However, phage therapy does not easily align with traditional regulatory pathways designed for fixed-composition chemical drugs or biologics with fixed non-evolving compositions. Globally, alternative models such as personalised pharmacy-based formulations (known as magistral preparations), compassionate use provisions and flexible licensing have emerged to accommodate the biological complexity and personalised nature of phage-based therapeutics. Australia is well positioned to take a leadership role and consider a regulatory sandbox approach to the therapeutic use of phages that balances innovation with safety. Being time limited, this controlled framework would allow regulators and innovators to test new approaches under provisional rules and inform potential reforms. A sandbox should include strict participation criteria, and lead to the development of regulatory-ready biobanks of well-characterised phages, accelerating clinical access and manufacturing capacity. This model is a pragmatic and proactive approach that would enable safe scalable phage therapy in response to rising antimicrobial resistance threats.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"265-273"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13332890/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148157493","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Language as an Eligibility Criterion in Pediatric Study Protocols Conducted in Europe (2007-2024) Reported in the ClinicalTrials.gov Database.","authors":"Begonya Nafria, Alexander Perera, Segolene Gaillard, Martine Dehlinger-Kremer, Pamela Dicks, Ricardo M Fernandes, Gilles Vassal, Pirkko Lepola, Gunter F Egger, Lucy Bray, Barbara E Bierer, Bob Phillips","doi":"10.1007/s40290-026-00613-1","DOIUrl":"10.1007/s40290-026-00613-1","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;One of the challenges in accessing cross-border clinical trials involving international participants in Europe is language diversity, with 32 official languages in the European continent. Some patients have reported being excluded from trials owing to their native language, and in certain studies, language has been used as an eligibility criterion for participation. Considering that pediatric studies in Europe are not conducted in all countries, cross-border access to clinical trials may represent the only therapeutic opportunity for children living with rare diseases for which no approved treatment exists.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;This study aimed to assess the use of language as an eligibility criterion in pediatric clinical trial protocols conducted in Europe (2007-2024) and published in the Clinicaltrials.gov database. It evaluated the frequency and context of language requirements and whether these were scientifically justified. The overall objective was to identify potential sources of language-based discrimination that may prevent cross-border access to pediatric clinical trials.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The 32 official languages of the European continent were used as keywords to search the eligibility criteria of 1,754 pediatric clinical trial protocols for studies conducted in Europe between 2007 and 2024 and registered in the largest clinical trial registry, ClinicalTrials.gov, via an Application Programming Interface. Acceptable scientific justifications to use language as an eligibility criterion were defined as being (1) related to specific therapeutic areas that required language or cognitive assessments in communication with the health professionals, who do not speak the patient's language or (2) related to the use of patient- or caregiver-reported outcome measures that had only been validated in specific languages.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The majority of the study protocols (95.2%) did not include any reference to European official languages in the eligibility criteria. Of the 85 study protocols that did include language requirements, only 23 (27.1%) had a scientific justification for the use of this criterion. The most frequent European languages required as eligibility criteria were English (20%) and French (16.5%).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;A minority of European paediatric studies (4.8%) included language in eligibility criteria in the study protocols, but of those that did, most offered no reasonable explanation for the restriction. To prevent language-based discrimination within the European regulatory framework, we recommend that ethics committees should require justification for any language-based eligibility criteria. Additionally, including dedicated sections on cross-border access in clinical trial protocols will help ensure the provision of appropriate resources such as translations, interpreters, travel, and accommodation when recruiting international p","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"311-324"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13332970/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148295892","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimising Formulations for Paediatric Patients with Inherited Metabolic Disorders: The Case Study of Levocarnitine.","authors":"Stephen Tomlin, Chris Mühlhausen, Andriy Krendyukov, Anibh Martin Das","doi":"10.1007/s40290-026-00611-3","DOIUrl":"10.1007/s40290-026-00611-3","url":null,"abstract":"<p><p>Inherited metabolic disorders (IMDs) are rare genetic conditions that disrupt normal biochemical pathways, leading to potentially life-threatening metabolic imbalances. Early diagnosis, often through newborn screening, and treatment can significantly improve outcomes, but pharmacological management presents unique challenges that are amplified by developmental immaturity, altered pharmacokinetics, and heightened sensitivity to drug excipients. The selection of excipients becomes especially important in IMDs such as primary carnitine deficiency (PCD), where lifelong supplementation is required. Substances such as parabens, benzoates, saccharin sodium, propylene glycol, and ethanol are generally safe in adults, but may potentially contribute to metabolic crises or cumulative toxicity in neonates due to immature organ systems and altered metabolism. Current regulatory frameworks are addressing these concerns through guidelines, labelling revisions, and tools such as the STEP and SEEN databases, which compile paediatric safety data for excipients. Despite regulatory progress, significant gaps in excipient transparency, standardisation, and labelling persist, complicating clinical decision making. Healthcare providers often lack access to precise excipient concentrations and may unintentionally exceed safe thresholds in polypharmacy scenarios. This opinion paper underscores the critical need for age-appropriate, excipient-conscious drug formulations for children with IMDs. It advocates for improved regulatory oversight, increased formulation transparency, and interdisciplinary collaboration to minimise risk and enhance the safety of paediatric treatments, particularly for neonates requiring chronic therapy.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"275-285"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148164118","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Changes in Saccadic Hypometria over Time to Monitor Parkinson's Disease Progression.","authors":"Edmund Ben-Ami, Eitan Raveh, Eran Harpaz, Igal Riklin, Tali Band, Tanya Gurevich, Roy N Alcalay, Pablo Mir, Michelle H S Tosin, Christopher G Goetz, Olivier Rascol","doi":"10.1007/s40290-026-00612-2","DOIUrl":"10.1007/s40290-026-00612-2","url":null,"abstract":"<p><strong>Background: </strong>Eye movement biomarkers are emerging as promising tools for monitoring neurodegenerative diseases in clinical trials. Saccadic hypometria, the reduced saccade amplitude leading to undershooting visual targets, is a recognized feature of Parkinson's disease (PD), correlated with motor symptoms severity in cross-sectional studies. However, its use as a biomarker to monitor disease progression has not been studied.</p><p><strong>Objectives: </strong>The aim was to assess the sensitivity and reproducibility of saccadic hypometria as a biomarker of PD progression.</p><p><strong>Methods: </strong>The amplitude of saccadic hypometria (ASH) was measured in two cohorts: a single-center cohort (SCC) (30 PD patients, 50 healthy controls) followed by a multicenter cohort (MCC) (250 PD patients, 91 healthy controls across 4 sites). Assessments occurred every 3 months over 9 months using a software-based platform (NeuraLight). Motor symptoms were assessed with the Movement Disorders Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III. ASH and MDS-UPDRS changes were analyzed using individual slopes of change and compared between groups.</p><p><strong>Results: </strong>ASH significantly declined over time in PD patients compared to controls in both cohorts (SCC: -  1.96 ± 4.14% vs. 0.33 ± 1.75%, P < 0.01; MCC: - 1.05 ± 3.09% vs. 0.54 ± 1.85%, P < 0.001), and these changes were consistent across MCC sites. MDS-UPDRS III scores remained unchanged over the same period in the SCC, and changes were highly variable across MCC sites, resulting in a non-statistically significant change over time.</p><p><strong>Conclusion: </strong>ASH progressively worsened in PD patients over 9 months, even when standard clinical scales showed no significant change. These findings support ASH as a sensitive, objective, and quantitative biomarker for monitoring PD progression.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"299-309"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148101834","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Framework for Standardising Toxicity Management Guidelines in Oncology Clinical Trials.","authors":"Sanjana Singarayer, Heidi Lloyd-Williams, Abigail Morrison, Ken Twomey","doi":"10.1007/s40290-026-00615-z","DOIUrl":"10.1007/s40290-026-00615-z","url":null,"abstract":"<p><p>For new investigational medicinal products (IMPs) entering clinical development, toxicity management guidelines (TMGs) can be developed as an addition to the clinical study protocol. These guidelines inform best clinical practice for the treatment of potential product-related toxicities including dose modifications and symptom management recommendations. TMGs ensure that early identification and intervention strategies are in place, helping to minimise risks and improve patient outcomes. Antibody-drug conjugates (ADCs) are a rapidly evolving drug class within oncology, and their accelerated development should be accompanied by high quality TMGs to support clinical trial success. Having a pre-prepared, 'off-the-shelf' set of TMGs for ADCs ensures consistency of care across several clinical trials investigating this drug class. The authors present a framework for developing these standardised TMGs across AstraZeneca's ADC portfolio. It incorporates information gathering from internal documents and published labels or guidelines, cross-product comparison, and standardisation decisions based on biological rationale and study/patient context. Although patient- and context-specific modifications remain necessary, a semi-standardised framework prioritises safety while allowing clinical judgment. Considerations for specific circumstances when the TMGs could be deviated from and how the approach can be extended to additional modalities and therapy areas are discussed.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"287-298"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13332982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148295809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Expert Consensus-Based Review of Considerations for Developing a Quality Management System Framework for Studies Utilising Secondary Data for Real-World Evidence Generation.","authors":"Deborah Layton, Antonella Porta, Dony Patel, Darmendra Ramcharran, Yola Moride, Andrea Splendiani, Tzuyung Douglas Kou, Efe Eworuke, Judit Riera-Arnau, Smitha Sreedharan, Susan A Oliveria","doi":"10.1007/s40290-026-00609-x","DOIUrl":"10.1007/s40290-026-00609-x","url":null,"abstract":"<p><p>High-quality pharmacoepidemiological research is essential for credible real-world evidence (RWE). This expert consensus-based review explores how pharmaceutical Quality Management Systems (QMS) developed for studies using primary data collection (e.g., clinical trials, registries) can be adapted to support studies utilising secondary data to generate RWE, in line with regulatory expectations. The aim was to identify fit-for-purpose considerations for proportional oversight, data integrity, and regulatory-grade evidence generation. An international working group (WG) comprising RWE experts from industry, academia, and independent consultancy (including QMS specialist) assessed which elements of QMS frameworks developed for primary data collection are applicable to RWE studies utilising secondary data sources and those elements where adaptation would be required. Using the Nominal Group Technique, the WG identified and prioritised relevant quality systems and adaptation needs. In parallel, a targeted literature review of guidance and reports from International Coalition of Medicines Regulatory Authorities (ICMRA) member sites (focusing on the EU, USA and UK) assessed guidance against prioritized quality systems. Of 21 quality systems identified, 12 were considered most relevant to RWE studies utilising secondary data, with ten requiring contextual adaptations. The literature review identified 26 publications referencing one or more of these systems; quality manuals (69.2% (n = 18)) and quality control (34.6% (n = 9)) were most frequently addressed, whilst other systems were inconsistently covered; vendor management was not explicitly addressed. Overall, this review highlights the need for fit-for-purpose adaptation of existing QMS frameworks to support regulatory-relevant, scientifically robust RWE generated from secondary use of existing data sources.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"249-263"},"PeriodicalIF":4.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148176553","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}