Rare disease drug development : clinical, scientific, patient, and caregiver perspectives / Raymond A. Huml, editor.

Format:

Book

Contributor:

Huml, Raymond A., editor.

Language:

English

Subjects (All):

Drug development.

Rare diseases--Chemotherapy.

Rare diseases.

Chemotherapy.

Genre:

Electronic books.

Physical Description:

1 online resource

Place of Publication:

Cham, Switzerland : Springer, 2021.

System Details:

text file

Summary:

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&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.

Notes:

Includes index.

Online resource; title from PDF title page (SpringerLink, viewed November 23, 2021).

ISBN:

9783030786052

3030786056

OCLC:

1285274795

Access Restriction:

Restricted for use by site license.