Main 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.

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