Paediatric drug optimization for cancer medicines


Book Description

The goal of the Paediatric drug optimization for cancer exercise was to develop a PADO priority list of formulations to be prioritized with a time horizon of 3–5 years, and a PADO ‘watch list’ containing promising candidates for investigation and development for children with a time horizon of 5–10 years. The PADO–cancer exercise enables alignment between funders, procurers, market-coordination entities, researchers, innovators, generics manufacturers, product development partnerships and regulators on priority products to be investigated and developed, as well as increasing efforts to tackle challenges in access to cancer medicines in LMICs.




Childhood Cancer and Functional Impacts Across the Care Continuum


Book Description

Since the late 1960s, the survival rate in children and adolescents diagnosed with cancer has steadily improved, with a corresponding decline in the cancer-specific death rate. Although the improvements in survival are encouraging, they have come at the cost of acute, chronic, and late adverse effects precipitated by the toxicities associated with the individual or combined use of different types of treatment (e.g., surgery, radiation, chemotherapy). In some cases, the impairments resulting from cancer and its treatment are severe enough to qualify a child for U.S. Social Security Administration disability benefits. At the request of Social Security Administration, Childhood Cancer and Functional Impacts Across the Care Continuum provides current information and findings and conclusions regarding the diagnosis, treatment, and prognosis of selected childhood cancers, including different types of malignant solid tumors, and the effect of those cancers on childrenâ (TM)s health and functional capacity, including the relative levels of functional limitation typically associated with the cancers and their treatment. This report also provides a summary of selected treatments currently being studied in clinical trials and identifies any limitations on the availability of these treatments, such as whether treatments are available only in certain geographic areas.




Childhood Acute Lymphoblastic Leukemia


Book Description

This book provides a comprehensive and up-to-date review of all aspects of childhood Acute Lymphoblastic Leukemia, from basic biology to supportive care. It offers new insights into the genetic pre-disposition to the condition and discusses how response to early therapy and its basic biology are utilized to develop new prognostic stratification systems and target therapy. Readers will learn about current treatment and outcomes, such as immunotherapy and targeted therapy approaches. Supportive care and management of the condition in resource poor countries are also discussed in detail. This is an indispensable guide for research and laboratory scientists, pediatric hematologists as well as specialist nurses involved in the care of childhood leukemia.




Addressing the Barriers to Pediatric Drug Development


Book Description

Decades of research have demonstrated that children do not respond to medications in the same way as adults. Differences between children and adults in the overall response to medications are due to profound anatomical, physiological, and developmental differences. Although few would argue that children should receive medications that have not been adequately tested for safety and efficacy, the majority of drugs prescribed for children-50 to 75 percent-have not been tested in pediatric populations. Without adequate data from such testing, prescribing drugs appropriately becomes challenging for clinicians treating children, from infancy through adolescence. Addressing the Barriers to Pediatric Drug Development is the summary of a workshop, held in Washington, D.C. on June 13, 2006, that was organized to identify barriers to the development and testing of drugs for pediatric populations, as well as ways in which the system can be improved to facilitate better treatments for children.




The Drug Development Paradigm in Oncology


Book Description

Advances in cancer research have led to an improved understanding of the molecular mechanisms underpinning the development of cancer and how the immune system responds to cancer. This influx of research has led to an increasing number and variety of therapies in the drug development pipeline, including targeted therapies and associated biomarker tests that can select which patients are most likely to respond, and immunotherapies that harness the body's immune system to destroy cancer cells. Compared with standard chemotherapies, these new cancer therapies may demonstrate evidence of benefit and clearer distinctions between efficacy and toxicity at an earlier stage of development. However, there is a concern that the traditional processes for cancer drug development, evaluation, and regulatory approval could impede or delay the use of these promising cancer treatments in clinical practice. This has led to a number of effortsâ€"by patient advocates, the pharmaceutical industry, and the Food and Drug Administration (FDA)â€"to accelerate the review of promising new cancer therapies, especially for cancers that currently lack effective treatments. However, generating the necessary data to confirm safety and efficacy during expedited drug development programs can present a unique set of challenges and opportunities. To explore this new landscape in cancer drug development, the National Academies of Sciences, Engineering, and Medicine developed a workshop held in December 2016. This workshop convened cancer researchers, patient advocates, and representatives from industry, academia, and government to discuss challenges with traditional approaches to drug development, opportunities to improve the efficiency of drug development, and strategies to enhance the information available about a cancer therapy throughout its life cycle in order to improve its use in clinical practice. This publication summarizes the presentations and discussions from the workshop.




Pizzo and Poplack's Pediatric Oncology


Book Description

"This authoritative reference is a comprehensive resource on the biology and genetics of childhood cancer as well as its diagnosis, multimodal treatment, as well as long-term management of young patients with cancer. Also addressed are a broad array of topics on the supportive and psychosocial aspects of care of children and families. Covering virtually every aspect of the breadth and depth of childhood cancer, this reference provides expert guidance on state-of-the-art, multidisciplinary care for children and families"--




Advanced Drug Delivery Systems in the Management of Cancer


Book Description

Advanced Drug Delivery Systems in the Management of Cancer discusses recent developments in nanomedicine and nano-based drug delivery systems used in the treatment of cancers affecting the blood, lungs, brain, and kidneys. The research presented in this book includes international collaborations in the area of novel drug delivery for the treatment of cancer. Cancer therapy remains one of the greatest challenges in modern medicine, as successful treatment requires the elimination of malignant cells that are closely related to normal cells within the body. Advanced drug delivery systems are carriers for a wide range of pharmacotherapies used in many applications, including cancer treatment. The use of such carrier systems in cancer treatment is growing rapidly as they help overcome the limitations associated with conventional drug delivery systems. Some of the conventional limitations that these advanced drug delivery systems help overcome include nonspecific targeting, systemic toxicity, poor oral bioavailability, reduced efficacy, and low therapeutic index. This book begins with a brief introduction to cancer biology. This is followed by an overview of the current landscape in pharmacotherapy for the cancer management. The need for advanced drug delivery systems in oncology and cancer treatment is established, and the systems that can be used for several specific cancers are discussed. Several chapters of the book are devoted to discussing the latest technologies and advances in nanotechnology. These include practical solutions on how to design a more effective nanocarrier for the drugs used in cancer therapeutics. Each chapter is written with the goal of informing readers about the latest advancements in drug delivery system technologies while reinforcing understanding through various detailed tables, figures, and illustrations. Advanced Drug Delivery Systems in the Management of Cancer is a valuable resource for anyone working in the fields of cancer biology and drug delivery, whether in academia, research, or industry. The book will be especially useful for researchers in drug formulation and drug delivery as well as for biological and translational researchers working in the field of cancer. - Presents an overview of the recent perspectives and challenges within the management and diagnosis of cancer - Provides insights into how advanced drug delivery systems can effectively be used in the management of a wide range of cancers - Includes up-to-date information on diagnostic methods and treatment strategies using controlled drug delivery systems




Optimization in Medicine and Biology


Book Description

Thanks to recent advancements, optimization is now recognized as a crucial component in research and decision-making across a number of fields. Through optimization, scientists have made tremendous advances in cancer treatment planning, disease control, and drug development, as well as in sequencing DNA, and identifying protein structures. Op




Animal Models in Cancer Drug Discovery


Book Description

Animal Models in Cancer Drug Discovery brings forward the most cutting-edge developments in tumor model systems for translational cancer research. The reader can find under this one volume virtually all types of existing and emerging tumor models in use by the research community. This book provides a deeper insight on how these newer models could de-risk modern drug discovery. Areas covered include up to date information on latest organoid derived models and newer genetic models. Additionally, the book discusses humanized animal tumor models for cancer immunotherapy and how they leverage personalized therapies. The chapter on larger animal, canine models and their use in and their use in pre-investigational new drug (pre-IND) development makes the volume unique. Unlike before, the incorporation of several simplified protocols, breeding methodologies, handling and assessment procedures to study drug intervention makes this book a must read. Animal Models in Cancer Drug Discovery is a valuable resource for basic and translational cancer researchers, drug discovery researchers, contract research organizations, and knowledge seekers at all levels in the biomedical field.




Pediatric Formulations


Book Description

Until the 1990s, it was generally accepted that medicines were first developed for adults and their use in children was investigated later, if at all. One of the main tasks of hospital pharmacies was the manufacturing of child-appropriate formulations in a more or less makeshift way. The first change came in 1997 with U.S. legislation that rewarded manufacturers to do voluntary pediatric research. Ten years later, the European Union passed legislation that required manufacturers to discuss all pediatric aspects, including formulations, with the regulatory authorities as a condition of starting the registration procedure. In consequence, manufacturers must now cover all age groups, including the youngest ones. So far, pediatric formulations were more a focus for academic researchers. Through the changed regulatory environment, there is now a sudden high commercial demand for age-appropriate formulations. This book begins by highlighting the anatomical, physiological and developmental differences between adults and children of different ages. It goes on to review the existing technologies and attempts to draw a roadmap to better, innovative formulations, in particular for oral administration. The regulatory, clinical, ethical and pharmaceutical framework is also addressed.