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A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case forreal-world data and evidence generation in orphan medicinal drug development.
Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.
A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case forreal-world data and evidence generation in orphan medicinal drug development.
Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.
Erscheinungsjahr: | 2021 |
---|---|
Fachbereich: | Allgemeine Lexika |
Genre: | Mathematik, Medizin, Naturwissenschaften, Technik |
Rubrik: | Wissenschaften |
Medium: | Buch |
Inhalt: |
xxiv
409 S. 28 s/w Illustr. 8 farbige Illustr. 409 p. 36 illus. 8 illus. in color. |
ISBN-13: | 9783030786045 |
ISBN-10: | 3030786048 |
Sprache: | Englisch |
Herstellernummer: | 978-3-030-78604-5 |
Redaktion: | Huml, Raymond A. |
Herausgeber: | Raymond A Huml |
Auflage: | 1st ed. 2021 |
Hersteller: |
Springer
Springer, Berlin Springer International Publishing |
Verantwortliche Person für die EU: | Springer Verlag GmbH, Tiergartenstr. 17, D-69121 Heidelberg, juergen.hartmann@springer.com |
Abbildungen: | XXIV, 409 p. 36 illus., 8 illus. in color. |
Maße: | 241 x 160 x 23 mm |
Von/Mit: | Raymond A. Huml |
Erscheinungsdatum: | 09.11.2021 |
Gewicht: | 0,862 kg |
Erscheinungsjahr: | 2021 |
---|---|
Fachbereich: | Allgemeine Lexika |
Genre: | Mathematik, Medizin, Naturwissenschaften, Technik |
Rubrik: | Wissenschaften |
Medium: | Buch |
Inhalt: |
xxiv
409 S. 28 s/w Illustr. 8 farbige Illustr. 409 p. 36 illus. 8 illus. in color. |
ISBN-13: | 9783030786045 |
ISBN-10: | 3030786048 |
Sprache: | Englisch |
Herstellernummer: | 978-3-030-78604-5 |
Redaktion: | Huml, Raymond A. |
Herausgeber: | Raymond A Huml |
Auflage: | 1st ed. 2021 |
Hersteller: |
Springer
Springer, Berlin Springer International Publishing |
Verantwortliche Person für die EU: | Springer Verlag GmbH, Tiergartenstr. 17, D-69121 Heidelberg, juergen.hartmann@springer.com |
Abbildungen: | XXIV, 409 p. 36 illus., 8 illus. in color. |
Maße: | 241 x 160 x 23 mm |
Von/Mit: | Raymond A. Huml |
Erscheinungsdatum: | 09.11.2021 |
Gewicht: | 0,862 kg |