This Brief discusses key statistical concepts that facilitate the inferential analysis of data collected from a group of individuals participating in a pharmaceutical clinical trial, the estimation of their clinical significance in the general population of individuals likely to be prescribed the drug if approved, and the related decision-making that occurs at both the public health level (by regulatory agencies when deciding whether or not to approve a new drug for marketing) and the individual patient level (by physicians and their patients when deciding whether or not the patient should be prescribed a drug that is on the market). These concepts include drug safety and efficacy, statistical significance, clinical significance, and benefit-risk balance.

This Brief defines competitive intelligence (CI) as a tool for making investment decisions within the pharmaceutical industry.  It provides an overview of processes that the regulatory affairs professional must take into account when evaluating data impacting product-based risk evaluations. These apply particularly to evaluations that focus on outputs such as regulatory approval, or the commercial impact of product labeling on the sales forecast over a limited timeframe. The Brief also provides an overview of intellectual property assessment that can impact a product’s lifespan on the market due to patent protection itself (or loss of patent protection) or via regulatory exclusivity. Case examples are discussed to illustrate the importance of keeping up with the ever-changing regulations, and how to interpret them in the context of CI. In addition, there is a section on virtual data rooms (VDRs) which currently function as the cornerstone of due diligence investigations. While aimed primarily at regulatory affairs professionals in the United States, this publication provides a useful adjunct for other pharmaceutical executives, especially those new to product-based investments, and regulatory affairs professionals in other regions. ​

Macromolecular (specifically peptide-based) drugs could potentially be highly effective medicines. However they have a relatively short duration of action and variable therapeutic index. An example of such a peptide is Glucagon-like Peptide I which could potentially be used as a revolutionary drug for diabetes. This is because it stimulates insulin only when the blood glucose level is high thereby reducing the risk of hypoglycemia (a significant disadvantage of using insulin is that an insulin overdose is the single most potent cause of life-threatening hypoglycemia). However it’s short duration of action (half-life of 2 minutes in plasma) precludes its therapeutic use. In this volume, the use of novel therapeutics like GLP1 as an alternative to tradition insulin-based drugs in diabetes is described. Application of Peptide-Based Prodrug Chemistry in Drug Development elucidates the traditional concept of prodrugs as “specialized non-toxic protective groups used in a transient manner to alter or to eliminate certain limiting properties in the parent small molecule” (IUPAC definition). It goes on to provide insight into how prodrugs of peptides (with GLP1 as an example) could be appropriately used to extend the biological half life, broaden the therapeutic index of macromolecules and improve the pharmacodynamics of such drugs.  Author explains the logic behind designing peptide prodrugs, synthetic procedures and bioassays to examine the conversion of the prodrug to the drug under therapeutic conditions. The prodrugs described slowly convert to the parent drug at physiological conditions of 37C and pH 7.2 driven by their inherent chemical instability without the need of any enzymatic cleavage. The diketopiperazine and diketomorpholine (DKP and DMP) strategies for prodrug conversion are demonstrated in detail with special emphasis on the chemical flexibility that it offers to develop prodrugs with variable time actions.This book will be of usefulto chemists, biochemists, medicinal chemists, biologists and people in the medical profession (doctors). It may be used in undergraduate classes but will certainly help post-graduate students and advanced professionals. The author is grateful to Prof. Richard DiMarchi (Standiford H. Cox Professor of Chemistry and the Linda & Jack Gill Chair in Biomolecular Sciences at Indiana University) for valuable suggestions. The foreword for the book has been written by Prof. Jean Martinez, (Legion d'Honneur awarded by the French Republic; Professor of Chemistry and Medicinal Chemistry of the University of Montpellier, France; and Chairman of European Peptide Society, 2002-2010).

Dr. Catalano has for the last ten years been doing consulting for the Pharmaceutical Industry. During his consulting he discovered that small businesses such as, generic, startups, and virtual companies do not have the budget or the resources to apply the computer software utilized in project management and therefore do not apply project management principles in their business model. This reduces their effectiveness and increases their operating cost. Application of Project Management Principles to the Management of Pharmaceutical R&D Projects is presented as a paper-based system for completing all the critical activities needed apply the project management system. This will allow these small business to take advantage of the project management principles and gain all the advantages of the system. This book will be beneficial for beginners to understand the concepts of project management and for small pharmaceutical companies to apply the principlesof project management to their business model.

For many Doctor of Pharmacy (PharmD) candidates, post-graduate training is a highly prestigious and sought after experience that can further one’s pre-existing education. Some of these post-graduate training opportunities include but are not limited to pharmacy residencies, pharmaceutical industry fellowships, and PhD/Masters programs. There is a large gap in the current Doctor of Pharmacy curriculum about pharmaceutical industry fellowship programs and potential pharmaceutical industry opportunities. Through the eyes of current industry fellows and professionals, this book serves as a resource to learn more about fellowship opportunities and their respective application processes. In addition, this manual aims to provide a clear and concise roadmap to achieving a career in industry through a fellowship program. Whether you are a current student pharmacist, post-graduate pharmacy trainee (pharmacy resident), or industry professional, this manual addresses the current landscape of industry fellowship programs, an overview of industry opportunities, and a glimpse into what being a fellow entails. Readers of this book can expect to gain:  An overview of pharmaceutical industry fellowship programs and opportunities    Helpful advice and insight from current fellows and industry professionals    A streamlined pathway to navigating the fellowship application processAn understanding of the journey from being a student, to achieving a fellowship, to becoming an industry professionalThis book bridges the gap in education surrounding pharmaceutical industry fellowships and pharmacy careers in industry and increases access to first-hand information and real-world perspectives of current professionals to provide a firm foundation for those interested in pursuing a career in industry or learning more about fellowship programs.

Good Laboratory Practices for Forensic Chemistry acknowledges the limitations that often challenge the validity of data and resultant conclusions. Eight chapters examine current practices in analytical chemistry as well as business practices, guidelines and regulations in the pharmaceutical industry to offer improvements to current practices in forensic chemistry. It discusses topics ranging from good manufacturing practices (GMP), good laboratory practices (GLP), the International Conference on Harmonisation (ICH), quality assurance (QA), and quality risk management (QRM), among others. This book is a guide for scientists, professors, and students interested in expanding their knowledge of forensic chemistry.

​What are lipid nanoparticles?  How are they structured?  How are they formed? What techniques are best to characterize them?  How great is their potential as drug delivery systems?  These questions and more are answered in this comprehensive and highly readable work on lipid nanoparticles. This work sets out to provide the reader with a clear and understandable understanding of the current practices in formulation, characterization and drug delivery of lipid nanoparticles. A comprehensive description of the current understanding of synthesis, characterization, stability optimization and drug incorporation of solid lipid nanoparticles is provided. Nanoparticles have attracted great interest over the past few decades with almost exponential growth in their research and application. Their small particle size and subsequent high surface area make them ideal in many uses, but particularly as drug carrier systems. Nanoparticles made from lipids are especially attractive because of their enhanced biocompatibility imparted by the lipid. The work provides a detailed description of the types of lipid nanoparticles available (e.g. SLN, NLC, LDC, PLN) and how they range from imperfect crystalline to amorphous in structure. Current thoughts on where drugs are situated (e.g. in the core, or at the interface) and how this can be manipulated are discussed.  The many techniques for production, including the author’s own variant of microwave heating, are fully discussed. Techniques for measuring arguably the most important characteristics of particle size and polydispersity are discussed, along with techniques to measure crystallinity, shape and drug capacity.  Finally, a full chapter on techniques for measuring stability, both in the absence and presence of drugs, is discussed, along with suggestions on how to optimize that stability. This work appeals to students of colloid science, practitioners of research into drug delivery and academics alike.

This volume presents recent developments in the novel drug development and potential of extremophilic cyanobacteria. It discusses how these tiny organism originated, produce oxygen that leads to evolution of life on the earth, how their survival strategies in extreme climatic conditions lead to diverse metabolic pathways, and the opportunity to use them to develop novel drugs. The book is comprised of five chapters, starting with the origin of cyanobacteria, their survival strategies under extreme conditions, and their capabilities to change metabolic activities.The second chapter explores the different metabolic pathways found in cyanobacteria and examines advances in this field and recent techniques like MALDI-TOF imaging and metagenomics tools as well as in silico techniques for rapid screening of secondary metabolites. Further chapters cover the glycomics of cyanobacteria, anticancer drug development, and some issues and challenges of using cyanobacteria to develop drugs.Extremophilic Cyanobacteria For Novel Drug Development provides insight into future perspectives in drug development and is a key resource for students, researchers and professionals in pharmacy, medicinal chemistry, pharmacognosy biotechnology, biology, and academics.

This book provides an up-to-date review of recently identified natural anti-tumor compounds from various natural origins including plants, fungi, endophytic fungi and marine organisms. It also includes discussion of new areas such as biotechnology and nanoparticles. Chapters explain the challenges and developments in anti-cancer drug discovery approaches, traditional remedies for prevention and treatment of cancer, marine-derived anti-cancer compounds, and antibiotics used as anti-cancer agents, as well as different classes of terpenoids and carbohydrates, which have been the subject of discussion in this field as efficient anti-cancer candidates. This book will be a concise guide for researchers in the field of pharmaceutical sciences, students and residents in pharmacy and medicine as well as those researching phytochemistry and natural products.

This book reviews a number of issues including: Why data generated from POC machines are considered as Big Data. What are the challenges in storing, managing, extracting knowledge from data from POC devices? Why is it inefficient to use traditional data analysis with big data?What are the solutions for the mentioned issues and challenges? What type of analytics skills are required in health care? What big data technologies and tools can be used efficiently with data generated from POC devices? This book shows how it is feasible to store vast numbers of anonymous data and ask highly specific questions that can be performed in real-time to give precise and meaningful evidence to guide public health policy.

The natural world with a large number of terrestrial and marine plants and lower organisms is a great source of bioactive compounds historically used as remedies in various diseases. Within the last decade, such compounds became more attractive targets for pharmacologists and the pharmaceutical industry in drug development projects. This volume presents the pharmacological potential of chemically defined natural compounds obtained from plants, fungi, algae and cyanobacteria with antiparasitic activity, that have been tested against various endo-parasitic protozoan and helminth species. Additionally, the advantages of combined therapy using antiparasitic drugs and natural compounds with selected specific activity are reviewed and explained in the context of host pathology and immunosuppression induced by the parasites. The conclusions of this new book give suggestions for further non-empirical drug development and discuss perspectives of alternative approaches to therapy of parasitic diseases.  ​

The US is currently well ahead of the rest of the world in the development and application of SB and its principles especially as they pertain to basic medical research and development. This lead is largely due to its earlier start in the academic arena. However, there is evidence of rapid development in both the UK/EU and Japan, and the gap is narrowing, particularly in the UK. From an industrial point of view, the Pharmaceutical Industry based in the US and UK can capitalize on these opportunities and gain the benefits of this technology. Many educational institutions (particularly their medical divisions) at present are heavily business-oriented, realize that in this particular industrial environment, that every dollar counts.

This Brief discusses key statistical concepts that facilitate the inferential analysis of data collected from a group of individuals participating in a pharmaceutical clinical trial, the estimation of their clinical significance in the general population of individuals likely to be prescribed the drug if approved, and the related decision-making that occurs at both the public health level (by regulatory agencies when deciding whether or not to approve a new drug for marketing) and the individual patient level (by physicians and their patients when deciding whether or not the patient should be prescribed a drug that is on the market). These concepts include drug safety and efficacy, statistical significance, clinical significance, and benefit-risk balance.

This Brief defines competitive intelligence (CI) as a tool for making investment decisions within the pharmaceutical industry.  It provides an overview of processes that the regulatory affairs professional must take into account when evaluating data impacting product-based risk evaluations. These apply particularly to evaluations that focus on outputs such as regulatory approval, or the commercial impact of product labeling on the sales forecast over a limited timeframe. The Brief also provides an overview of intellectual property assessment that can impact a product’s lifespan on the market due to patent protection itself (or loss of patent protection) or via regulatory exclusivity. Case examples are discussed to illustrate the importance of keeping up with the ever-changing regulations, and how to interpret them in the context of CI. In addition, there is a section on virtual data rooms (VDRs) which currently function as the cornerstone of due diligence investigations. While aimed primarily at regulatory affairs professionals in the United States, this publication provides a useful adjunct for other pharmaceutical executives, especially those new to product-based investments, and regulatory affairs professionals in other regions. ​

Macromolecular (specifically peptide-based) drugs could potentially be highly effective medicines. However they have a relatively short duration of action and variable therapeutic index. An example of such a peptide is Glucagon-like Peptide I which could potentially be used as a revolutionary drug for diabetes. This is because it stimulates insulin only when the blood glucose level is high thereby reducing the risk of hypoglycemia (a significant disadvantage of using insulin is that an insulin overdose is the single most potent cause of life-threatening hypoglycemia). However it’s short duration of action (half-life of 2 minutes in plasma) precludes its therapeutic use. In this volume, the use of novel therapeutics like GLP1 as an alternative to tradition insulin-based drugs in diabetes is described. Application of Peptide-Based Prodrug Chemistry in Drug Development elucidates the traditional concept of prodrugs as “specialized non-toxic protective groups used in a transient manner to alter or to eliminate certain limiting properties in the parent small molecule” (IUPAC definition). It goes on to provide insight into how prodrugs of peptides (with GLP1 as an example) could be appropriately used to extend the biological half life, broaden the therapeutic index of macromolecules and improve the pharmacodynamics of such drugs.  Author explains the logic behind designing peptide prodrugs, synthetic procedures and bioassays to examine the conversion of the prodrug to the drug under therapeutic conditions. The prodrugs described slowly convert to the parent drug at physiological conditions of 37C and pH 7.2 driven by their inherent chemical instability without the need of any enzymatic cleavage. The diketopiperazine and diketomorpholine (DKP and DMP) strategies for prodrug conversion are demonstrated in detail with special emphasis on the chemical flexibility that it offers to develop prodrugs with variable time actions.This book will be of usefulto chemists, biochemists, medicinal chemists, biologists and people in the medical profession (doctors). It may be used in undergraduate classes but will certainly help post-graduate students and advanced professionals. The author is grateful to Prof. Richard DiMarchi (Standiford H. Cox Professor of Chemistry and the Linda & Jack Gill Chair in Biomolecular Sciences at Indiana University) for valuable suggestions. The foreword for the book has been written by Prof. Jean Martinez, (Legion d'Honneur awarded by the French Republic; Professor of Chemistry and Medicinal Chemistry of the University of Montpellier, France; and Chairman of European Peptide Society, 2002-2010).

Dr. Catalano has for the last ten years been doing consulting for the Pharmaceutical Industry. During his consulting he discovered that small businesses such as, generic, startups, and virtual companies do not have the budget or the resources to apply the computer software utilized in project management and therefore do not apply project management principles in their business model. This reduces their effectiveness and increases their operating cost. Application of Project Management Principles to the Management of Pharmaceutical R&D Projects is presented as a paper-based system for completing all the critical activities needed apply the project management system. This will allow these small business to take advantage of the project management principles and gain all the advantages of the system. This book will be beneficial for beginners to understand the concepts of project management and for small pharmaceutical companies to apply the principlesof project management to their business model.

For many Doctor of Pharmacy (PharmD) candidates, post-graduate training is a highly prestigious and sought after experience that can further one’s pre-existing education. Some of these post-graduate training opportunities include but are not limited to pharmacy residencies, pharmaceutical industry fellowships, and PhD/Masters programs. There is a large gap in the current Doctor of Pharmacy curriculum about pharmaceutical industry fellowship programs and potential pharmaceutical industry opportunities. Through the eyes of current industry fellows and professionals, this book serves as a resource to learn more about fellowship opportunities and their respective application processes. In addition, this manual aims to provide a clear and concise roadmap to achieving a career in industry through a fellowship program. Whether you are a current student pharmacist, post-graduate pharmacy trainee (pharmacy resident), or industry professional, this manual addresses the current landscape of industry fellowship programs, an overview of industry opportunities, and a glimpse into what being a fellow entails. Readers of this book can expect to gain:  An overview of pharmaceutical industry fellowship programs and opportunities    Helpful advice and insight from current fellows and industry professionals    A streamlined pathway to navigating the fellowship application processAn understanding of the journey from being a student, to achieving a fellowship, to becoming an industry professionalThis book bridges the gap in education surrounding pharmaceutical industry fellowships and pharmacy careers in industry and increases access to first-hand information and real-world perspectives of current professionals to provide a firm foundation for those interested in pursuing a career in industry or learning more about fellowship programs.

Good Laboratory Practices for Forensic Chemistry acknowledges the limitations that often challenge the validity of data and resultant conclusions. Eight chapters examine current practices in analytical chemistry as well as business practices, guidelines and regulations in the pharmaceutical industry to offer improvements to current practices in forensic chemistry. It discusses topics ranging from good manufacturing practices (GMP), good laboratory practices (GLP), the International Conference on Harmonisation (ICH), quality assurance (QA), and quality risk management (QRM), among others. This book is a guide for scientists, professors, and students interested in expanding their knowledge of forensic chemistry.

​What are lipid nanoparticles?  How are they structured?  How are they formed? What techniques are best to characterize them?  How great is their potential as drug delivery systems?  These questions and more are answered in this comprehensive and highly readable work on lipid nanoparticles. This work sets out to provide the reader with a clear and understandable understanding of the current practices in formulation, characterization and drug delivery of lipid nanoparticles. A comprehensive description of the current understanding of synthesis, characterization, stability optimization and drug incorporation of solid lipid nanoparticles is provided. Nanoparticles have attracted great interest over the past few decades with almost exponential growth in their research and application. Their small particle size and subsequent high surface area make them ideal in many uses, but particularly as drug carrier systems. Nanoparticles made from lipids are especially attractive because of their enhanced biocompatibility imparted by the lipid. The work provides a detailed description of the types of lipid nanoparticles available (e.g. SLN, NLC, LDC, PLN) and how they range from imperfect crystalline to amorphous in structure. Current thoughts on where drugs are situated (e.g. in the core, or at the interface) and how this can be manipulated are discussed.  The many techniques for production, including the author’s own variant of microwave heating, are fully discussed. Techniques for measuring arguably the most important characteristics of particle size and polydispersity are discussed, along with techniques to measure crystallinity, shape and drug capacity.  Finally, a full chapter on techniques for measuring stability, both in the absence and presence of drugs, is discussed, along with suggestions on how to optimize that stability. This work appeals to students of colloid science, practitioners of research into drug delivery and academics alike.

This volume presents recent developments in the novel drug development and potential of extremophilic cyanobacteria. It discusses how these tiny organism originated, produce oxygen that leads to evolution of life on the earth, how their survival strategies in extreme climatic conditions lead to diverse metabolic pathways, and the opportunity to use them to develop novel drugs. The book is comprised of five chapters, starting with the origin of cyanobacteria, their survival strategies under extreme conditions, and their capabilities to change metabolic activities.The second chapter explores the different metabolic pathways found in cyanobacteria and examines advances in this field and recent techniques like MALDI-TOF imaging and metagenomics tools as well as in silico techniques for rapid screening of secondary metabolites. Further chapters cover the glycomics of cyanobacteria, anticancer drug development, and some issues and challenges of using cyanobacteria to develop drugs.Extremophilic Cyanobacteria For Novel Drug Development provides insight into future perspectives in drug development and is a key resource for students, researchers and professionals in pharmacy, medicinal chemistry, pharmacognosy biotechnology, biology, and academics.

This book provides an up-to-date review of recently identified natural anti-tumor compounds from various natural origins including plants, fungi, endophytic fungi and marine organisms. It also includes discussion of new areas such as biotechnology and nanoparticles. Chapters explain the challenges and developments in anti-cancer drug discovery approaches, traditional remedies for prevention and treatment of cancer, marine-derived anti-cancer compounds, and antibiotics used as anti-cancer agents, as well as different classes of terpenoids and carbohydrates, which have been the subject of discussion in this field as efficient anti-cancer candidates. This book will be a concise guide for researchers in the field of pharmaceutical sciences, students and residents in pharmacy and medicine as well as those researching phytochemistry and natural products.

This book reviews a number of issues including: Why data generated from POC machines are considered as Big Data. What are the challenges in storing, managing, extracting knowledge from data from POC devices? Why is it inefficient to use traditional data analysis with big data?What are the solutions for the mentioned issues and challenges? What type of analytics skills are required in health care? What big data technologies and tools can be used efficiently with data generated from POC devices? This book shows how it is feasible to store vast numbers of anonymous data and ask highly specific questions that can be performed in real-time to give precise and meaningful evidence to guide public health policy.

The natural world with a large number of terrestrial and marine plants and lower organisms is a great source of bioactive compounds historically used as remedies in various diseases. Within the last decade, such compounds became more attractive targets for pharmacologists and the pharmaceutical industry in drug development projects. This volume presents the pharmacological potential of chemically defined natural compounds obtained from plants, fungi, algae and cyanobacteria with antiparasitic activity, that have been tested against various endo-parasitic protozoan and helminth species. Additionally, the advantages of combined therapy using antiparasitic drugs and natural compounds with selected specific activity are reviewed and explained in the context of host pathology and immunosuppression induced by the parasites. The conclusions of this new book give suggestions for further non-empirical drug development and discuss perspectives of alternative approaches to therapy of parasitic diseases.  ​

The US is currently well ahead of the rest of the world in the development and application of SB and its principles especially as they pertain to basic medical research and development. This lead is largely due to its earlier start in the academic arena. However, there is evidence of rapid development in both the UK/EU and Japan, and the gap is narrowing, particularly in the UK. From an industrial point of view, the Pharmaceutical Industry based in the US and UK can capitalize on these opportunities and gain the benefits of this technology. Many educational institutions (particularly their medical divisions) at present are heavily business-oriented, realize that in this particular industrial environment, that every dollar counts.