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Rare Disease Drug Development Clinical,
Scientific, Patient, and Caregiver Perspectives
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Rare Disease Drug Development Clinical,
Scientific, Patient, and Caregiver Perspectives
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Description
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.nbspAunique
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 accelera
tions, and market access strategies. The book
concludes with reflections on the authors' case
forreal-world data and evidence generation in
orphan medicinal drug development.nbspRar
Disease Drug Development is an expertly written
text optimized for biopharmaceutical
RampDexperts, commercial experts, third-party
capital providers, patient advocacy groups,
patients, and caregivers.