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Biomarkers, diagnostics and precision medicine in the drug industry : critical challenges, limitations and roadmaps for the best practices
- Author / Creator
- Available as
- Online
Details
- Creator
- Abdel Halim
- Format
- Books
- Language
- English
- Publication
-
- London, England : Academic Press, [2019]
- ©2019
- Physical Details
-
- 1 online resource (296 pages)
- ISBNs
- 0128161213, 9780128161210, 9780128161227, 0128161221
- OCLC
- on1104533252
- Front Cover -- Biomarkers, Diagnostics, and Precision Medicine in the Drug Industry -- Copyright Page -- Dedication -- Contents -- Preface -- 1 Pharmaceutical crisis -- 1.1 Value of medicines -- 1.1.1 Examples of major achievements -- 1.1.2 Medications for the two leading causes of death -- 1.1.3 Advancement in orphan drug development -- 1.2 Drug development crisis -- 1.2.1 Cost of medicines -- 1.2.2 What factors determine a medication price? -- 1.2.3 Spend on pharmaceutical research and development -- 1.2.4 Cost-effectiveness of pharmaceuticals -- 1.2.5 Return on investment -- 1.2.6 Probability of success in drug development -- 1.2.7 Pharmaceutical crisis -- 1.2.8 Root causes of the pharmaceutical crisis and recommendations for improvement -- References -- 2 Applications of biomarkers for different purposes in drug development -- 2.1 Historical background -- 2.1.1 Biomarkers and diagnostics in ancient history -- 2.1.2 How revolution made a difference -- 2.2 What's a biomarker? -- 2.2.1 Published definitions -- 2.2.2 Matches and differences between published definitions -- 2.2.3 Author's suggested definition -- 2.3 Nature of biomarkers and matrix for testing -- 2.4 Categories of biomarkers from application perspectives -- 2.4.1 Diagnostic biomarker -- 2.4.2 Predictive biomarkers -- 2.4.2.1 Susceptibility biomarkers -- 2.4.2.2 Prognostic biomarkers -- 2.4.2.3 Predictive biomarkers -- 2.4.2.4 Pharmacogenetics and pharmacogenomics -- 2.4.3 Safety biomarkers -- 2.4.3.1 Examples of traditional safety biomarkers -- 2.4.3.2 Examples of specialized safety biomarkers -- 2.4.4 Surrogate endpoint biomarkers -- 2.4.4.1 Basis for a drug approval -- 2.4.4.2 Clinical trial endpoint -- 2.4.4.3 Value of surrogate endpoints -- 2.4.5 Monitoring biomarker -- 2.4.6 Pharmacodynamic biomarkers -- References -- Further reading -- 3 Pharmacogenetics of therapeutics
- 3.1 Pharmacogenetics and pharmacogenomics -- 3.2 Some key milestones in the road toward pharmacogenetics and pharmacogenomics -- 3.3 Pharmacogenetics and pharmacogenomics in drug development -- 3.4 Pharmacogenetics and pharmacogenomics on FDA-approved drug labels and package inserts -- 3.5 Classes of pharmacogenetics and pharmacogenomics biomarkers with some examples from each class -- 3.5.1 Drug transporters -- 3.5.1.1 P-glycoprotein -- 3.5.1.2 Cystic fibrosis transmembrane conductance regulator -- 3.5.2 Pharmacogenetics and pharmacogenomics of drug metabolism -- 3.5.2.1 Pharmacogenetics and pharmacogenomics of the oxidative reactions (phase I of drug metabolism) -- 3.5.2.1.1 Cytochrome-P-450 2D6 -- 3.5.2.1.2 CYP2C19 -- 3.5.2.1.3 CYP2C9 -- 3.5.2.1.4 Vitamin K epoxide reductase complex subunit 1 -- 3.5.2.2 Pharmacogenetics and pharmacogenomics of drug conjugation (phase II of drug metabolism) -- 3.5.2.2.1 Uridine diphosphate glucuronosyltransferase -- 3.5.2.2.1.1 Examples of uridine diphosphate glucuronosyltransferase substrates -- 3.5.2.2.1.2 Examples of uridine diphosphate glucuronosyltransferase inhibitors -- 3.5.2.2.2 Sulfate (sulfonate) conjugation -- 3.5.3 Pharmacogenetics and pharmacogenomics of body susceptibility to a drug action -- 3.5.3.1 Ability of the body to accommodate an adverse reaction -- 3.5.3.2 Individual prognostic impact on a drug efficacy -- 3.5.4 Pharmacogenetics and pharmacogenomics of drug target -- 3.5.4.1 BRAF -- 3.5.4.1.1 Vemurafenib -- 3.5.4.1.2 Dabrafenib -- 3.5.4.1.3 Cobimetinib -- 3.5.4.2 Breakpoint cluster region-Abelson -- 3.5.4.2.1 Imatinib -- 3.5.4.2.2 Nilotinib -- 3.5.4.2.3 Dasatinib -- 3.5.4.2.4 Blinatumomab -- 3.5.4.3 Estrogen receptor/progesterone receptor and HER2 -- 3.5.4.3.1 Tamoxifen -- 3.5.4.3.2 Herceptin -- 3.5.4.3.3 Lapatinib -- 3.5.4.3.4 Everolimus
- 3.6 Expectations and requirements for pharmacogenetics and pharmacogenomics biomarker analysis -- References -- 4 Precision medicine and companion diagnostics in drug development -- 4.1 Conventional medicine -- 4.2 Personalization of medicine -- 4.3 Precision medicine -- 4.4 Companion diagnostics -- 4.4.1 FDA-approved companion diagnostic -- 4.4.2 Development of companion diagnostic -- 4.4.2.1 Biomarker hypothesis generation -- 4.4.2.2 Testing a biomarker hypothesis in a preclinical model -- 4.4.2.3 Clinical qualification -- 4.4.2.4 Application of a biomarker test in a pivotal trial -- 4.4.3 Regulatory approval of companion diagnostic -- 4.4.3.1 Contemporaneous drug and diagnostic developments -- 4.4.3.2 Noncontemporaneous development but contemporaneous marketing -- 4.4.3.2.1 Use of clinical trial assay different from to-be-marketed assay in the pivotal trial -- 4.4.3.2.2 Prospective-retrospective approach -- 4.4.3.3 Noncontemporaneous development, noncontemporaneous marketing -- 4.4.3.3.1 Approval of companion diagnostic using similar technique, for same drug in the same indication depending on its c... -- 4.4.3.3.2 Approval of companion diagnostic using a different technique, for multiple drugs in multiple indications dependin... -- 4.4.3.3.3 Approval of companion diagnostic through 510K path -- 4.4.3.3.4 Approval of companion diagnostic under humanitarian device exempt -- 4.4.3.4 Approval of a therapeutic product without an approved or cleared in vitro diagnostic companion diagnostic device -- 4.4.4 Types of assays used in clinical trials from regulatory perspectives -- 4.4.4.1 FDA-approved test -- 4.4.4.2 FDA-approved investigation-use-only test -- 4.4.4.3 Lab-developed test under Clinical Laboratory Improvement Amendment -- 4.5 Value of precision medicine and companion diagnostic in drug development -- 4.6 Complementary diagnostics
- 4.7 Economic impact of precision medicine and companion diagnostic -- References -- Further reading -- 5 Essential attributes of an acceptable in vitro diagnostic test -- 5.1 Classification of in vitro diagnostic systems -- 5.1.1 Regulatory classification -- 5.1.1.1 Regulatory classification of in vitro diagnostic by complexity -- 5.1.1.2 Classification according to level of regulatory control -- 5.1.1.3 Relationship between the two regulatory classifications -- 5.1.2 Classification according to assay readouts -- 5.1.2.1 Qualitative raw signal, qualitative reportable results -- 5.1.2.2 Quantitative signal, qualitative reportable results -- 5.1.2.3 Quantitative signal, quantitative reportable results -- 5.1.2.4 Relative quantitative signal, quantitative reportable results -- 5.1.3 Classification of in vitro diagnostic according to disease area, panel of tests, technology used or sample matrix -- 5.2 Fundamental characteristics of a reliable in vitro diagnostic -- 5.2.1 Setting specifications of analytical methods for clinical applications -- 5.2.2 Universal specifications of analytical methods with associated statistical terms -- 5.2.2.1 Precision -- 5.2.2.2 Trueness and accuracy -- 5.2.2.3 Total error, total analytical error, and total allowable error of an assay -- 5.2.2.3.1 Total analytical error -- 5.2.2.3.2 Total allowable error -- 5.2.2.3.2.1 Medical requirements -- 5.2.2.3.2.2 Biological variation -- 5.2.2.3.2.3 Regulatory requirements -- 5.2.2.3.2.4 Peer group survey results from proficiency testing -- 5.2.2.4 Assay measurement range and clinical reportable range -- 5.2.2.4.1 Assay analytical sensitivity -- 5.2.2.4.2 What range of assay linearity can be enough? -- 5.2.2.5 Additional parameters for qualitative assays -- 5.2.2.5.1 Clinical sensitivity and specificity -- 5.2.2.5.2 Diagnostic power of a biomarker or assay
- 5.2.2.5.3 Positive predictive value and negative predictive value -- 5.2.2.5.4 Percent agreement -- 5.2.2.6 Sigma metrics and quality goal -- 5.2.2.6.1 Sigma metric for quantitative assays -- 5.2.2.6.2 Sigma metric for qualitative assays -- 5.2.2.6.3 Cost of quality -- References -- 6 Fundamentals of assay development and validation -- 6.1 What is an assay? -- 6.2 Assay development -- 6.2.1 Identify the purpose -- 6.2.2 Build a biological concept -- 6.2.3 Select analyte form and technology -- 6.2.4 Select sample matrix -- 6.2.5 Generate an analytical concept -- 6.2.6 Select or design reagents -- 6.2.7 Preliminary test of the analytical concept -- 6.2.8 Assay optimization -- 6.3 Assay validation -- 6.3.1 Determination of the assay measurement range -- 6.3.1.1 Preliminary/exploratory step -- 6.3.1.2 Confirmatory step -- 6.3.1.3 Assay measurement range for immunohistochemistry and similar cell-based technologies -- 6.3.2 Sample dilutability -- 6.3.2.1 Obligatory dilution -- 6.3.2.2 Optional dilution -- 6.3.2.3 Matrix effect -- 6.3.2.3.1 Specific -- 6.3.2.3.2 Nonspecific -- 6.3.3 Types of assays that can be candidates for sample dilution -- 6.3.4 Assessment of matrix interference -- 6.3.4.1 Interferent spike -- 6.3.4.2 Analyte spike recovery -- 6.3.5 Testing of sample dilutability -- 6.3.6 Can dilution eliminate matrix effect? -- 6.3.7 Matrix interference in immunohistochemistry -- 6.4 Precision and accuracy -- 6.4.1 Precision and accuracy requirements in the FDA bioanalytical guidance -- 6.4.2 Precision and accuracy requirements in laboratory guidelines -- 6.4.2.1 Clinical and Laboratory Standards Institute EP05 -- 6.4.2.2 Clinical and Laboratory Standards Institute EP15 -- 6.4.2.3 Suggested acceptance criteria -- 6.4.2.4 Illustrative example -- 6.4.3 Precision and accuracy of qualitative assays -- 6.5 Method comparison
- 6.5.1 Study setup for quantitative method comparison
Subjects
- Biochemical markers.
- Precision medicine.
- Drug development.
- Drug Discovery.
Medical Subject Heading
- Biomarkers.
Medical Subject Heading
- Drug Development.
Medical Subject Heading
- Precision Medicine.
Medical Subject Heading