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  Rare Disease Drug Development : Clinical, Scientific, Patient, and Caregiver Perspectives

  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.
  
  Contents:
  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
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• Article

  [Experimental production of pemphigus-like lesions in guinea-pigs by sera of patients with pemphigus (author's transl)].

  Toshio, Orihara.

  Nihon Hifuka Gakkai Zasshi. 1978 Aug;88(9):561-78.

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  PubMed