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Organotypic models in drug development / / Monika Schäfer-Korting, Silvya Stuchi Maria-Engler, Robert Landsiedel, editors
| Organotypic models in drug development / / Monika Schäfer-Korting, Silvya Stuchi Maria-Engler, Robert Landsiedel, editors |
| Pubbl/distr/stampa | Cham, Switzerland : , : Springer, , [2021] |
| Descrizione fisica | 1 online resource (viii, 323 pages) : illustrations |
| Disciplina | 615.19 |
| Collana | Handbook of experimental pharmacology |
| Soggetto topico |
Drug development
Drugs - Testing Desenvolupament de medicaments Cultiu cel·lular |
| Soggetto genere / forma | Llibres electrònics |
| ISBN | 3-030-70063-1 |
| Formato | Materiale a stampa  |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto |
Intro -- Preface -- Contents -- Part I: General Topics - Quality Assurance and Predictability -- Human-Derived In Vitro Models Used for Skin Toxicity Testing Under REACh -- 1 Introduction -- 2 Validation and Regulatory Acceptance -- 3 Regulatory-Accepted Human-Derived In Vitro Models -- 3.1 Skin Irritation and Corrosion -- 3.1.1 Testing Methods: Reconstructed Human Epidermis (RhE) Used in OECD Test Guidelines No. 431 and 439 -- 3.1.2 Combination of Methods to Assess Skin Irritation and Skin Corrosion -- 3.2 Phototoxicity -- 3.3 Eye Irritation -- 3.3.1 Testing Methods: Reconstructed Human Cornea-Like Epithelium Models (RhE) Used in OECD Test Guideline No. 492 -- 3.3.2 Testing Methods: Immortalized Corneal Epithelial Cells Used in OECD Test Guideline No. 494 -- 3.3.3 Defined Approaches: Combination of Methods to Assess Eye Irritation and Serious Eye Damage -- 3.4 Skin Sensitization -- 3.4.1 Testing Methods: Synthetic Peptides Used in OECD Test Guideline No. 442C -- 3.4.2 Testing Methods: Human-Derived Keratinocytes Used in OECD Test Guideline No. 442D -- 3.4.3 Testing Methods: Human-Derived Dendritic-Like Cells Used in OECD Test Guideline No. 442E -- 3.4.4 Defined Approaches: Combination of In Vitro Methods to Assess Skin Sensitization -- 3.5 Genotoxicity -- 3.6 Dermal Penetration and Absorption -- 4 Limitations -- 4.1 Technical Limitations -- 4.2 Predictive Limitations -- 4.2.1 Mechanistic Limitations -- Metabolic Capacity -- Water Solubility and Lipophilicity -- 4.2.2 Agrochemical Formulation in In Vitro Skin and Eye Irritation Tests -- 4.3 Uncertainty -- 4.3.1 Reference Data and Validation Sets -- 4.3.2 Borderline Range: Uncertainty Arising from Technical and Biological Variance -- References -- How Qualification of 3D Disease Models Cuts the Gordian Knot in Preclinical Drug Development -- 1 Current Efficiency in Preclinical Research.
1.1 Phases of Preclinical Research -- 1.2 Models and Test Methods -- 2 Reasons for Poor Translational Success -- 3 From Validation to Qualification -- 3.1 Validation -- 3.2 Quality Function Deployment: Learning from Industry -- 3.3 Qualification -- 3.4 Qualification of 3D In Vitro Models -- 3.5 Qualification of Test Methods -- 3.6 Selection of Relevant Drug Doses -- 4 Current Strategies to Rethink Preclinical Drug Research -- 4.1 Strategy 1: Characterized Cell Lines -- 4.2 Strategy 2: Primary Cells to Recapitulate Human Heterogeneity -- 4.3 Strategy 3: Patient-Derived Cells -- 4.4 Strategy 4: New Technologies in Tissue Engineering -- 4.5 Strategy 5: Comparing New Test Methods to Current Standards -- 5 Phases of Innovative Preclinical Drug Research -- 5.1 Preclinical Phase I -- 5.2 Preclinical Phase II -- 5.3 Preclinical Phase III -- 6 The Price of Quality -- References -- Standardised Reconstructed Skin Models in Toxicology and Pharmacology: State of the Art and Future Development -- 1 Introduction -- 2 Industrial Production of the 3D Skin Models in Standardised Conditions -- 2.1 Barrier Properties -- 2.2 Histology and Immunohistochemistry -- 2.3 Lipid Profile Characterisation -- 2.4 Tissue Viability and the Effects of Transport -- 3 Industrial Production of 3D Disease Tissue Models -- 4 Safety and Ethical Concerns in the Industrial Production of Normal and Disease Models -- 5 Innovative Approaches to the Production of the Standardised 3D Skin Models -- 6 Outlook -- References -- Part II: Models For Drug Research -- In Vitro Models of the Blood-Brain Barrier -- 1 The Blood-Brain Barrier -- 2 In Vitro Methods to Study the Blood-Brain Barrier -- 3 Cell Sources for Cell Culture Models -- 3.1 Criteria for Blood-Brain Barrier In Vitro Models in Drug Development -- 3.2 Stem Cell-Derived Brain Capillary Endothelial Cells -- 4 Cell Culture Models. References -- Neural In Vitro Models for Studying Substances Acting on the Central Nervous System -- 1 Introduction into In Vitro Neurotoxicity Evaluation -- 1.1 Stem Cell-Based Human 2D Neuronal and Mixed Neuronal/Astrocyte Models -- 1.2 In Vitro Cultures of Microglia -- 1.3 Moving In Vitro Cultures into the Third Dimension with Brain Organoids -- 1.4 3D Bioprinted In Vitro Neural Models -- 1.5 CNS Disease Models -- 2 Summary and Conclusion -- References -- Engineered Heart Muscle Models in Phenotypic Drug Screens -- 1 Introduction -- 2 Macro-scale Heart Muscle Models for Drug Discovery -- 3 Pros and Cons of Tissue-Engineered Myocardium in Drug Screening Applications -- 4 Conclusion -- References -- Disease Models: Lung Models for Testing Drugs Against Inflammation and Infection -- 1 Introduction -- 2 Morpho-functional Characteristics of the Lung -- 3 Requirements to Model the Lung In Vitro -- 3.1 Technical Prerequisites -- 3.2 Sources of Cells -- 4 Modeling Pulmonary Diseases In Vitro -- 4.1 Asthma -- 4.2 Chronic Obstructive Pulmonary Disease (COPD) -- 4.3 Pulmonary Fibrosis -- 4.4 Bacterial Infections in the Context of Cystic Fibrosis and Pneumonia -- 4.4.1 Modeling the Treatment of Bacterial Infections on Cells In Vitro -- 4.4.2 Experimental Conditions to Build an In Vitro Model of CF -- 4.4.3 Testing Drug Efficacy on Infected Cell Lines -- 4.5 Tuberculosis -- 5 Outlook -- References -- Skin Disease Models In Vitro and Inflammatory Mechanisms: Predictability for Drug Development -- 1 Introduction -- 2 History -- 3 Monogenic Diseases -- 3.1 Monolayer Versus Three-Dimensional Cultures -- 3.2 Patient-Derived 3D Models -- 3.3 Knockdown and Knockout Models -- 4 Atopic Dermatitis -- 4.1 Barrier Function -- 4.2 Monolayer Cultures -- 4.3 Knockdown and Knockout Models -- 4.3.1 Filaggrin -- 4.3.2 TMEM45A -- 4.4 The Role of Cytokines -- 5 Psoriasis. 5.1 Barrier Function -- 5.2 Cell Signalling -- 5.3 Immune Cells -- 6 Infection and Immunity -- 7 Immunocompetent Skin Models -- 8 Drug Development and Testing -- 8.1 Retinoic Acid Metabolism Blocking Agents (RAMBA) -- 8.2 Antifungal Compounds -- 8.3 Protein Replacement -- 9 Future Directions -- References -- Immunocompetent Human Intestinal Models in Preclinical Drug Development -- 1 Introduction -- 2 Inflammatory Responses in the Intestine -- 3 Human In Vitro Models for Intestinal Inflammation -- 3.1 Coculture Models -- 3.2 Intestinal Organoids -- 3.3 Microfluidic Gut-on-a-Chip Models -- 4 Limitations of Current In Vitro Models of the Inflamed Intestine -- 5 Conclusions and Outlook -- References -- Tissue Engineering for Musculoskeletal Regeneration and Disease Modeling -- 1 Introduction -- 2 Key Elements of Musculoskeletal Tissue Models -- 2.1 Biomaterials and Scaffolds -- 2.2 Cells -- 2.3 Signaling Molecules -- 3 Musculoskeletal Microphysiological Systems -- 4 Summary and Future Perspectives -- References -- Tumor Models and Cancer Systems Biology for the Investigation of Anticancer Drugs and Resistance Development -- 1 Introduction -- 2 Methods for Modeling and Overcoming Resistant Tumors -- 2.1 2D Models -- 2.2 3D Models -- 2.3 Microfluidic Devices -- 2.4 Bioinformatics/System Biology -- 2.4.1 Sequencing Technologies -- RNA Sequencing -- Single-Cell Sequencing -- ChIP Sequencing -- Reverse-Phase Protein Array -- 2.4.2 Public Genomic Databases -- The Cancer Genome Atlas -- The International Cancer Genomics Consortium -- The Gene Expression Omnibus -- Drug Response Databases -- 3 Challenges and Future Directions -- References -- The CAM Assay as an Alternative In Vivo Model for Drug Testing -- 1 General Remarks: Development and Structure of the Chick Chorioallantoic Membrane (CAM) -- 2 CAM Model: History and Regularities. 3 CAM Model in Cancer Research -- 4 The CAM Model Compared to the Mouse Xenograft Model -- 5 Analysis of CAM Xenografts -- 6 The CAM and Study of Hallmarks of Cancer -- 6.1 Angiogenesis -- 6.2 Tumor Invasion and Metastasis -- 6.3 Visualization and Detection of Tumor Cells (Table 4) -- 7 The CAM Model and Molecular Pathways -- 7.1 Pharmacological Inhibition of Angiogenesis -- 7.2 Pharmacological Inhibition of Tumor Growth -- 8 CAM Use in Drug Delivery Studies -- 9 The CAM Model and Personalized Medicine -- 10 Outlook -- References. |
| Record Nr. | UNINA-9910483160603321 |
| Cham, Switzerland : , : Springer, , [2021] | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Quantitative decisions in drug development / / Christy Chuang-Stein, Simon Kirby
| Quantitative decisions in drug development / / Christy Chuang-Stein, Simon Kirby |
| Autore | Chuang-Stein Christy |
| Edizione | [Second edition.] |
| Pubbl/distr/stampa | Cham, Switzerland : , : Springer, , [2021] |
| Descrizione fisica | 1 online resource (354 pages) |
| Disciplina | 615.19 |
| Collana | Springer Series in Pharmaceutical Statistics |
| Soggetto topico |
Drug development - Decision making
Drug development - Economic aspects Desenvolupament de medicaments Presa de decisions |
| Soggetto genere / forma | Llibres electrònics |
| ISBN | 3-030-79731-7 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Record Nr. | UNINA-9910497090203321 |
Chuang-Stein Christy
|
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| Cham, Switzerland : , : Springer, , [2021] | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Quantitative decisions in drug development / / Christy Chuang-Stein, Simon Kirby
| Quantitative decisions in drug development / / Christy Chuang-Stein, Simon Kirby |
| Autore | Chuang-Stein Christy |
| Edizione | [Second edition.] |
| Pubbl/distr/stampa | Cham, Switzerland : , : Springer, , [2021] |
| Descrizione fisica | 1 online resource (354 pages) |
| Disciplina | 615.19 |
| Collana | Springer Series in Pharmaceutical Statistics |
| Soggetto topico |
Drug development - Decision making
Drug development - Economic aspects Desenvolupament de medicaments Presa de decisions |
| Soggetto genere / forma | Llibres electrònics |
| ISBN | 3-030-79731-7 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Record Nr. | UNISA-996466409503316 |
|
Chuang-Stein Christy
|
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| Cham, Switzerland : , : Springer, , [2021] | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. di Salerno | ||
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Rare Disease Drug Development : Clinical, Scientific, Patient, and Caregiver Perspectives / / edited by Raymond A. Huml
| Rare Disease Drug Development : Clinical, Scientific, Patient, and Caregiver Perspectives / / edited by Raymond A. Huml |
| Edizione | [1st ed. 2021.] |
| Pubbl/distr/stampa | Cham : , : Springer International Publishing : , : Imprint : Springer, , 2021 |
| Descrizione fisica | 1 online resource (417 pages) |
| Disciplina | 615.19 |
| Soggetto topico |
Medicine
Clinical medicine - Research Pharmacology Immunology Therapeutics Pharmacovigilance Clinical Medicine Clinical Research Drug Safety and Pharmacovigilance Malalties rares Desenvolupament de medicaments Quimioteràpia |
| Soggetto genere / forma | Llibres electrònics |
| ISBN | 3-030-78605-6 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto | Introduction to Rare Diseases and Market Overview -- The Patient Perspective -- Select Patient Narratives -- The Caregiver Perspective -- The Critical, Multidimensional Role of Patient Advocacy Groups in Rare Disease -- A Mental Health Perspective -- Investment Decisions Related to Rare Disease Drug Development -- Optimizing Rare Disease Registries and Natural History Studies -- Novel Approaches to Clinical Trials in Rare Diseases -- Patient Benefits from Innovative Designs in Rare Diseases -- Central Nervous System Rare Disease Drug Development -- Oncologic Rare Disease Drug Development -- Hematologic Rare Disease Drug Development -- Lessons From Rare Disease and Gene Therapy Clinical Studies in Ophthalmology -- Rare Diseases in the Pediatric Population -- Cell and Gene Therapy in Rare Diseases -- The Feasibility Assessment -- The Evolving Regulatory Space and the Advent of Patient-Focused Drug Development -- Operational Aspects of Rare Disease Drug Development -- Accelerating Rare Disease Drug Development -- Select Rare Disease Drug Approvals: Lessons Learned -- A Rapid Market Access Strategy for Orphan Medicinal Products (OMPs) with Highlights Regarding the Pricing and Reimbursement Process and Barriers to Patient Use -- Integrated Life Cycle Management for Rare and Orphan Products -- The Case for Real-World Data and Real-World Evidence Generation in Rare and Orphan Medicinal Drug Development -- Closing Remarks -- . |
| Record Nr. | UNINA-9910508447303321 |
| Cham : , : Springer International Publishing : , : Imprint : Springer, , 2021 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Real-World Evidence in Medical Product Development / / edited by Weili He, Yixin Fang, Hongwei Wang
| Real-World Evidence in Medical Product Development / / edited by Weili He, Yixin Fang, Hongwei Wang |
| Edizione | [1st ed. 2023.] |
| Pubbl/distr/stampa | Cham : , : Springer International Publishing : , : Imprint : Springer, , 2023 |
| Descrizione fisica | 1 online resource (431 pages) |
| Disciplina | 615.19 |
| Soggetto topico |
Statistics
Artificial intelligence—Data processing Computer science—Mathematics Data Science Mathematics of Computing Desenvolupament de medicaments Assaigs clínics |
| Soggetto genere / forma | Llibres electrònics |
| Soggetto non controllato |
Therapeutics
Medical |
| ISBN | 3-031-26328-6 |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto | Preface -- Part I. Real-World Data and Evidence to Accelerate Medical Product Development -- The need for real world data/evidence in clinical development and life cycle management, and future directions -- Overview of current RWE/RWD landscape -- Key considerations in forming research questions -- Part II. Fit-for-use RWD Assessment and Data Standards -- Assessment of fit-for-use real-world data sources and applications -- Key variables ascertainment and validation in real-world setting -- Data standards and platform interoperability -- Privacy-preserving data linkage for real-world datasets -- Part III. Causal Inference Framework and Methodologies in RWE Research -- Causal Inference with Targeted Learning for Producing and Evaluating Real-World Evidence -- Framework and Examples of Estimands in Real-World Studies -- Clinical Studies Leveraging Real-World Data Using Propensity Score-Based Methods -- Recent statistical development for comparative effectiveness research beyond propensity-score methods -- Innovative Hybrid Designs and Analytical Approaches leveraging Real-Word Data and Clinical Trial data -- Statistical challenges for causal inference using time-to-event real-world data -- Sensitivity Analyses for Unmeasured Confounding: This is the way -- Sensitivity analysis in the analysis of real-world data -- Personalized medicine with advanced analytics -- Use of Real-World Evidence in Health Technology Assessment Submissions -- Part IV. Application and Case studies -- Examples of applying causal-inference roadmap to real-world studies -- Applications using real-world evidence to accelerate medical product development -- The use of real-world data to support the assessment of the benefit and risk of a medicine to treat spinal muscular atrophy -- Index. |
| Record Nr. | UNINA-9910726291303321 |
| Cham : , : Springer International Publishing : , : Imprint : Springer, , 2023 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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