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  • Book
    Raymond A. Huml, editor.
    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.

    Introduction to Rare Diseases and Market Overview
    The Patient Perspective
    Select Patient Narratives
    The Caregiver Perspective
    The Critical, Multidimensional Role of Patient Advocacy Groups in Rare Disease
    A Mental Health Perspective
    Investment Decisions Related to Rare Disease Drug Development
    Optimizing Rare Disease Registries and Natural History Studies
    Novel Approaches to Clinical Trials in Rare Diseases
    Patient Benefits from Innovative Designs in Rare Diseases
    Central Nervous System Rare Disease Drug Development
    Oncologic Rare Disease Drug Development
    Hematologic Rare Disease Drug Development
    Lessons From Rare Disease and Gene Therapy Clinical Studies in Ophthalmology
    Rare Diseases in the Pediatric Population
    Cell and Gene Therapy in Rare Diseases
    The Feasibility Assessment
    The Evolving Regulatory Space and the Advent of Patient-Focused Drug Development
    Operational Aspects of Rare Disease Drug Development
    Accelerating Rare Disease Drug Development
    Select Rare Disease Drug Approvals: Lessons Learned
    A Rapid Market Access Strategy for Orphan Medicinal Products (OMPs) with Highlights Regarding the Pricing and Reimbursement Process and Barriers to Patient Use
    Integrated Life Cycle Management for Rare and Orphan Products
    The Case for Real-World Data and Real-World Evidence Generation in Rare and Orphan Medicinal Drug Development
    Closing Remarks --
    Digital Access Springer 2021