Rare Disease Drug Development

Rare Disease Drug Development

罕见病药物开发：临床、科学、患者与护理人员展望
药理学

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￥ 443.75

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作者

Raymond A. Huml

出版社

Springer Berlin Heidelberg

出版时间

2021年11月09日

装帧

精装

ＩＳＢＮ

9783030786045

复制

页码

410

语种

英文

版次

2021

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暂无评分

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图书简介

This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. 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 for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R

1. Introduction to Rare Diseases and Market Overview – Raymond A. Huml, MS, DVM, RAC2. The Patient Perspective - Meredith Huml3. The Caregiver Perspective - Pat Furlong, RN4. The Critical Role of Patient Advocacy Groups - Keri McDonough, MA5. A Mental Health Perspective - Michelle Bailey, MD, MPH6. Investment Decisions Related to Rare Disease Drug Development - Jonathan Tunnicliffe, MS, MBA and Devin Rosenthal, PhD, MBA7. Optimizing Rare Disease Registries and Natural History Studies – Sharon Hesterlee, PhD8. Innovative Clinical Trial Design - Zoran Antonijevic, MS, and Jonathan R. Huml9. CNS Rare Disease Drug Development – Jane Williams, MD, MPH10. Oncologic Rare Disease Drug Development – Laura Vidal, MD and Jozsef Palatka, MD11. Hematologic Rare Disease Drug Development – Daniel Mazzolenis, MD and Liat Vidal, MD12. Ophthalmic Rare Disease Drug Development – Nick Spittal, MBA13. Pediatric Challenges and Issues –Alexander Cvetkovic Muntañola, MD14. Cell and Gene Therapy - Peter Robinson, MBA15. The Feasibility Assessment – Gloria Alley, RN and Marie Emms16. The Evolving Regulatory Space – And the Advent of Patient Focused Drug Development - Annie Kennedy and James Valentine, JD, MHS17. Operational Aspects Related to Rare Disease Drug Development – Cinzia Dorigo, PharmD18. Accelerating Rare Disease Drug Development in Light of COVID-19 – Zizi Imatorbhebhe, MS, MBA, PMP19. Rare Disease Drug Approvals: Lessons Learned – Raymond A. Huml, MS, DVM, RAC20. Commercial and Reimbursement Challenges – Maryna Kolochavina, PharmD21. Integrated Life Cycle Management – Maryna Kolochavina, PharmaD22. The Case for Real World Evidence in Orphan Drug Development – David Thompson, PhD and Maryna Kolochavina, PharmD23. Summary

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